62. 発作性夜間ヘモグロビン尿症
[臨床試験数:202,薬物数:120(DrugBank:25),標的遺伝子数:15,標的パスウェイ数:90

Searched query = "Paroxysmal nocturnal hemoglobinuria", "PNH"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04654468
(ClinicalTrials.gov)
March 10, 202130/11/2020A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement InhibitionA Phase III, Multicenter, Single Arm Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement InhibitionParoxysmal Nocturnal HemoglobinuriaDrug: CrovalimabHoffmann-La RocheNULLNot yet recruiting12 YearsN/AAll50Phase 3China
2NCT04320602
(ClinicalTrials.gov)
January 202123/3/2020Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose EculizumabPhase 4, Single-Arm Study of Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose EculizumabParoxysmal Nocturnal HemoglobinuriaBiological: Eculizumab;Biological: RavulizumabAlexion PharmaceuticalsNULLNot yet recruiting18 YearsN/AAll20Phase 4NULL
3NCT04558918
(ClinicalTrials.gov)
December 15, 202027/8/2020Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody TreatmentA Randomized, Multicenter, Active-comparator Controlled, Open-label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily LNP023 in Adult Patients With PNH and Residual Anemia, Despite Treatment With an Intravenous Anti-C5 Antibody.Paroxysmal Nocturnal Hemoglobinuria (PNH)Drug: LNP023;Drug: Eculizumab;Drug: RavulizumabNovartis PharmaceuticalsNULLNot yet recruiting18 YearsN/AAll91Phase 3NULL
4EUCTR2020-000597-26-GR
(EUCTR)
04/12/202016/10/2020A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated with Complement Inhibitors.A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) CURRENTLY TREATED WITH COMPLEMENT INHIBITORS PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Crovalimab
Product Code: RO7112689
INN or Proposed INN: CROVALIMAB
Trade Name: Eculizumab
Product Name: Eculizumab
INN or Proposed INN: ECULIZUMAB
F.Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
250Phase 3United States;Portugal;Hong Kong;Taiwan;Greece;Spain;Thailand;Israel;Colombia;Italy;France;Australia;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Brazil;Belgium;Poland;Singapore;Germany;Japan;Sweden
5EUCTR2019-004931-21-GR
(EUCTR)
04/12/202016/10/2020A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not previously Treated with Complement InhibitorsA PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) NOT PREVIOUSLY TREATED WITHCOMPLEMENT INHIBITORS Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: Crovalimab
Product Code: RO7112689
INN or Proposed INN: CROVALIMAB
Trade Name: Soliris
Product Name: Eculizumab
INN or Proposed INN: ECULIZUMAB
F. Hoffman-La Roche Ltd.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
200Phase 3United States;Portugal;Taiwan;Hong Kong;Greece;Thailand;Spain;Israel;Colombia;Italy;France;Malaysia;Australia;Peru;South Africa;Netherlands;China;Korea, Republic of;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Romania;Germany;Japan;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT04469465
(ClinicalTrials.gov)
December 20209/7/2020Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)Paroxysmal Nocturnal HemoglobinuriaDrug: Danicopan;Drug: Placebo;Drug: C5 InhibitorAlexion PharmaceuticalsNULLNot yet recruiting18 YearsN/AAll84Phase 3NULL
7EUCTR2020-000501-93-AT
(EUCTR)
27/11/202008/09/2020A long term study of BCX9930 in patients with PNHA Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: BCX9930
Product Code: BCX9930
INN or Proposed INN: BCX9930
Other descriptive name: BCX9930 hydrochloride
Product Name: BCX9930
Product Code: BCX9930
INN or Proposed INN: BCX9930
Other descriptive name: BCX9930 hydrochloride
Product Name: BCX9930
Product Code: BCX9930
INN or Proposed INN: BCX9930
Other descriptive name: BCX9930 hydrochloride
BioCryst Pharmaceuticals IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
200Phase 2European Union;Austria;South Africa;United Kingdom
8EUCTR2019-004665-40-NL
(EUCTR)
20/11/202009/11/2020Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia despite anti-C5 antibody treatmentA randomized, multicenter, active-comparator controlled, open-label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult patients with PNH and residual anemia, despite treatment with an intravenous anti-C5 antibody Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: LNP023
Product Code: LNP023
INN or Proposed INN: not yet established
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Product Name: LNP023
Product Code: LNP023
INN or Proposed INN: not yet established
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Trade Name: Soliris
Product Name: Eculizumab
INN or Proposed INN: ECULIZUMAB
Trade Name: Ultomiris
Product Name: Ravulizumab
INN or Proposed INN: RAVULIZUMAB
Other descriptive name: ravulizumab
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
91Phase 3United States;Czechia;Taiwan;Spain;Italy;United Kingdom;France;Hungary;Canada;Brazil;Netherlands;Germany;Japan;Korea, Republic of
9EUCTR2019-004665-40-HU
(EUCTR)
17/11/202023/09/2020Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia despite anti-C5 antibody treatmentA randomized, multicenter, active-comparator controlled, open-label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult patients with PNH and residual anemia, despite treatment with an intravenous anti-C5 antibody Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: LNP023
Product Code: LNP023
INN or Proposed INN: not yet established
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Product Name: LNP023
Product Code: LNP023
INN or Proposed INN: not yet established
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Trade Name: Soliris
Product Name: Eculizumab
INN or Proposed INN: ECULIZUMAB
Trade Name: Ultomiris
Product Name: Ravulizumab
INN or Proposed INN: RAVULIZUMAB
Other descriptive name: ravulizumab
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
91Phase 3United States;Taiwan;Spain;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Brazil;Germany;Netherlands;Japan;Korea, Republic of
10EUCTR2019-004931-21-HU
(EUCTR)
05/11/202008/09/2020A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not previously Treated with Complement InhibitorsA PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) NOT PREVIOUSLY TREATED WITHCOMPLEMENT INHIBITORS Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: Crovalimab
Product Code: RO7112689
INN or Proposed INN: CROVALIMAB
Trade Name: Soliris
Product Name: Eculizumab
INN or Proposed INN: ECULIZUMAB
F. Hoffman-La Roche Ltd.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
10Phase 3Hungary;United States;Portugal;Taiwan;Hong Kong;Greece;Thailand;Spain;Israel;Colombia;Italy;France;Malaysia;Australia;Peru;South Africa;Netherlands;China;Korea, Republic of;Turkey;United Kingdom;Czech Republic;Mexico;Argentina;Brazil;Belgium;Poland;Germany;Japan;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2019-004931-21-SE
(EUCTR)
26/10/202003/06/2020A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not previously Treated with Complement InhibitorsA PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) NOT PREVIOUSLY TREATED WITHCOMPLEMENT INHIBITORS Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: Crovalimab
Product Code: RO7112689
INN or Proposed INN: CROVALIMAB
Trade Name: Soliris
Product Name: Eculizumab
INN or Proposed INN: ECULIZUMAB
F. Hoffman-La Roche Ltd.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
200Phase 3United States;Portugal;Taiwan;Hong Kong;Greece;Thailand;Spain;Israel;Colombia;Italy;France;Malaysia;Australia;Peru;South Africa;Netherlands;China;Korea, Republic of;Turkey;United Kingdom;Hungary;Czech Republic;Mexico;Argentina;Brazil;Belgium;Poland;Germany;Japan;Sweden
12NCT04434092
(ClinicalTrials.gov)
October 8, 20203/6/2020A Phase III Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Adult And Adolescent Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors.A Phase III, Randomized, Open-Label, Active-Controlled, Multicenter Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Adult And Adolescent Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors.Paroxysmal Nocturnal HemoglobinuriaDrug: Crovalimab;Drug: EculizumabHoffmann-La RocheChugai PharmaceuticalRecruiting12 YearsN/AAll200Phase 3United States;Argentina;Australia;Belgium;Brazil;Canada;China;Colombia;Czechia;France;Germany;Greece;Hong Kong;Hungary;Italy;Japan;Korea, Republic of;Malaysia;Mexico;Netherlands;Peru;Philippines;Poland;Portugal;Romania;Singapore;South Africa;Spain;Sweden;Taiwan;Thailand;Turkey;United Kingdom;Israel
13JPRN-JapicCTI-205425
08/10/202021/08/2020A Phase III Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors. ( COMMODORE 2 )A Phase III, Randomized, Open-Label, Active-Controlled, Multicenter Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors. Paroxysmal Nocturnal HemoglobinuriaIntervention name : Crovalimab
INN of the intervention : crovalimab
Dosage And administration of the intervention : Participants will receive an initial intravenous (IV) loading dose on Week 1 Day 1, followed by 4 weekly crovalimab subcutaneous (SC) doses on Week 1 Day 2, then on Weeks 2, 3 and 4. Maintenance dosing will begin at Week 5 and will continue Q4W (every 4 weeks) thereafter, for a total of at least 24 weeks of study treatment. Crovalimab will be administered at a dose of 1000 mg IV (for participants with body weight between 40 and 100kg) or 1500 mg IV (for participants with body weight >=100kg) on Week 1 Day 1. On Week 1 Day 2 and on Weeks 2, 3 and 4, it will be administered at a dose of 340 mg SC. For Week 5 and Q4W thereafter, it will be administered at a dose of 680 mg SC (for participants with body weight between 40 and 100kg) or 1020 mg SC (for participants with body weight >=100kg).
Control intervention name : Eculizumab
INN of the control intervention : eculizumab
Dosage And administration of the control intervention : Participants will receive initial IV weekly doses for 4 weeks which will be followed by Q2W (every 2 weeks) IV administrations starting on Week 5. Eculizumab will be administered at a dose of 600 mg for the first 4 weeks, followed by maintenance doses of 900 mg starting on Week 5.
Chugai Pharmaceutical Co., Ltd.NULLrecruitingBOTH200Phase 3Japan, Asia except Japan, North America, South America, Europe, Oceania, Africa
14NCT04079257
(ClinicalTrials.gov)
October 202011/7/2019The Pharmacokinetics and Pharmacodynamics of Eculizumab in Patients With Paroxysmal Nocturnal HemoglobinuriaThe Pharmacokinetics and Pharmacodynamics of Eculizumab in Patients With Paroxysmal Nocturnal HemoglobinuriaParoxysmal Nocturnal Hemoglobinuria;Eculizumab;PK-PDOther: Blood collection for measurement of eculizumab peak concentrationsRadboud UniversityNULLNot yet recruiting18 YearsN/AAll60NULL
15NCT04432584
(ClinicalTrials.gov)
September 30, 20203/6/2020A Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Adult And Adolescent Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors.A Phase III, Randomized, Open-Label, Active-Controlled, Multicenter Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Adult And Adolescent Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors.Paroxysmal Nocturnal HemoglobinuriaDrug: Crovalimab;Drug: EculizumabHoffmann-La RocheChugai PharmaceuticalRecruiting12 YearsN/AAll250Phase 3United States;Australia;Belgium;Brazil;Canada;Colombia;Czechia;France;Germany;Greece;Hong Kong;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Singapore;Spain;Sweden;Taiwan;Turkey;United Kingdom;Israel
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16JPRN-JapicCTI-205430
29/9/202024/08/2020A Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors. (COMMODORE 1)A Phase III, Randomized, Open-Label, Active-Controlled, Multicenter Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors. Paroxysmal Nocturnal HemoglobinuriaIntervention name : Crovalimab
INN of the intervention : crovalimab
Dosage And administration of the intervention : Participants will receive a loading series of crovalimab comprised of an intravenous (IV) dose on Day 1, followed by weekly crovalimab subcutaneous (SC) doses for 4 weeks on Week 1 Day 2, then on Weeks 2, 3, and 4. Maintenance SC dosing will begin at Week 5 and will continue Q4W (every 4 weeks) thereafter for a total of at least 24 weeks of study treatment. Crovalimab will be administered at a dose of 1000 mg IV (for participants with body weight between 40 and 100kg) or 1500 mg IV (for participants with body weight >=100kg) on Day 1. On Week 1 Day 2 and on Weeks 2, 3 and 4, it will be administered at a dose of 340 mg SC. For Week 5 and Q4W thereafter, it will be administered at a dose of 680 mg SC (for participants with body weight between 40 and 100kg) or 1020 mg SC (for participants with body weight >=100kg).
Control intervention name : Eculizumab
INN of the control intervention : eculizumab
Dosage And administration of the control intervention : Participants will receive an approved maintenance dose of eculizumab starting on Day 1 and Q2W (every 2 weeks) thereafter for a total of 24 weeks of study treatment. After 24 weeks of study eculizumab treatment, participants will have the option to switch to crovalimab or to discontinue from the study after completion of 10 weeks of safety follow-up. Eculizumab will be administered at a dose of 900 mg every 2 weeks.
Chugai Pharmaceutical Co., Ltd.NULLrecruitingBOTH250Phase 3Japan, Asia except Japan, North America, South America, Europe, Oceania
17EUCTR2020-000501-93-GB
(EUCTR)
29/09/202009/07/2020A long term study of BCX9930 in patients with PNHA Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: BCX9930
Product Code: BCX9930
INN or Proposed INN: BCX9930
Other descriptive name: BCX9930 hydrochloride
BioCryst Pharmaceuticals IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
200Phase 2European Union;South Africa;United Kingdom
18EUCTR2019-003829-18-GB
(EUCTR)
18/09/202005/02/2020Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)
MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: Danicopan (ALXN2040)
Product Code: ACH-0144471
INN or Proposed INN: DANICOPAN
Other descriptive name: DANICOPAN
Product Name: Danicopan (ALXN2040)
Product Code: ACH-0144471
INN or Proposed INN: DANICOPAN
Other descriptive name: DANICOPAN
Alexion Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
84Phase 3United States;Taiwan;Finland;Thailand;Spain;Austria;Switzerland;United Kingdom;Italy;France;Canada;Poland;Brazil;Malaysia;Australia;Denmark;Netherlands;Germany;Japan;Sweden;Korea, Republic of
19EUCTR2019-003830-17-GB
(EUCTR)
16/09/202016/06/2020Study of the Oral Factor D (fD) Inhibitor ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy and with background use of an Approved C5 InhibitorA Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of the Oral Factor D (FD) Inhibitor ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy and with an Approved C5 Inhibitor as Background Therapy Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: ALXN2050
Product Code: ACH-0145228
INN or Proposed INN: N/A
Other descriptive name: ACH-0145228
Product Name: ALXN2050
Product Code: ACH-0145228
INN or Proposed INN: N/A
Other descriptive name: ACH-0145228
Achillion Pharmaceuticals, Inc., a wholly owned subsidiary of Alexion Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
26Phase 2United States;New Zealand;Italy;United Kingdom;Korea, Republic of
20EUCTR2018-002734-20-CZ
(EUCTR)
07/08/202015/03/2019A trial with REGN3918 in patients Paroxysmal Nocturnal Hemoglobinuria (PNH) who have not been treated previously with a Complement Inhibitor or Have Not Recently Received Complement Inhibitor TherapyAn Open Label, Single Arm Study to Evaluate the Efficacy and Safety of REGN3918 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor Naive or Have Not Recently Received Complement Inhibitor Therapy Paroxysmal nocturnal hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: REGN3918
Product Code: REGN3918
INN or Proposed INN: REGN3918
Other descriptive name: REGN3918
Product Name: REGN3918
Product Code: REGN3918
INN or Proposed INN: REGN3918
Other descriptive name: REGN3918
Regeneron Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
42Phase 2United States;Taiwan;Hong Kong;Korea, Democratic People's Republic of;United Kingdom;Italy;Czech Republic;Hungary;Canada;Poland;Malaysia;Singapore;Romania;South Africa;Germany;Netherlands
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21EUCTR2019-003440-74-GB
(EUCTR)
14/05/202017/03/2020Phase 4, Single-Arm Study of Ravulizumab in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with High-Dose EculizumabPhase 4, Single-Arm Study of Ravulizumab in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with High-Dose Eculizumab - ALXN1210-PNH-401 Paroxysmal nocturnal hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Trade Name: ULTOMIRIS
Product Name: ULTOMIRIS
Product Code: ALXN1210
INN or Proposed INN: RAVULIZUMAB
Alexion Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 4United Kingdom
22EUCTR2019-000130-20-GB
(EUCTR)
23/04/202019/11/2019REGN3918 in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) to evaluate its long term safety, efficacy and tolerability.An open-label extension study to evaluate the long-term safety, tolerability, and efficacy of REGN3918 in patients with paroxysmal nocturnal hemoglobinuria Paroxysmal nocturnal hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: REGN3918
Product Code: REGN3918
INN or Proposed INN: REGN3918
Other descriptive name: REGN3918
Product Name: REGN3918
Product Code: REGN3918
INN or Proposed INN: REGN3918
Other descriptive name: REGN3918
Regeneron Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
147Phase 3United States;Taiwan;Hong Kong;Korea, Democratic People's Republic of;United Kingdom;Italy;Hungary;Canada;Malaysia;Poland;Singapore;Romania;South Africa;Germany;Netherlands
23EUCTR2017-001418-27-FI
(EUCTR)
01/04/202008/10/2019This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on theinvestigational medicine after you have been given it, and if this iscomparable to what is seen for the licensed medicine, eculizumab, inpatients with Paroxysmal Nocturnal Hemoglobinuria (PNH).This study will assess if the investigational medicine is safe and effective intreating PNH compared to the licensed medicine.A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - Not applicable Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: ABP 959
Product Code: ABP 959
INN or Proposed INN: ECULIZUMAB
Other descriptive name: ABP 959 - biosimilar to eculizumab
Trade Name: Soliris
Product Name: Soliris
INN or Proposed INN: ECULIZUMAB
Trade Name: Soliris
Product Name: Soliris
INN or Proposed INN: ECULIZUMAB
Amgen Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
40Phase 3United States;Portugal;Czechia;Taiwan;Slovenia;Finland;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Brazil;Germany;Netherlands;Norway;Sweden
24EUCTR2019-000130-20-NL
(EUCTR)
01/04/202016/10/2019A trial with REGN3918 in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) to evaluate its long term safety, efficacy and tolerability.An open-label extension study to evaluate the long-term safety, tolerability, and efficacy of REGN3918 in patients with paroxysmal nocturnal hemoglobinuria Paroxysmal nocturnal hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: REGN3918
Product Code: REGN3918
INN or Proposed INN: REGN3918
Other descriptive name: REGN3918
Regeneron Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
147Phase 3United States;Hong Kong;Taiwan;Korea, Democratic People's Republic of;United Kingdom;Italy;Hungary;Canada;Malaysia;Poland;Singapore;Romania;South Africa;Netherlands;Germany
25EUCTR2017-001418-27-PT
(EUCTR)
04/03/202028/11/2019 This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on the investigational medicine after you have been given it, and if this is comparable to what is seen for the licensed medicine, eculizumab, in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). This study will assess if the investigational medicine is safe and effective in treating PNH compared to the licensed medicine.A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - Not applicable Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Amgen Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
40Phase 3France;United States;Portugal;Czech Republic;Slovenia;Spain;Ireland;Norway;Netherlands;Germany;Italy;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26NCT04330534
(ClinicalTrials.gov)
March 3, 202023/3/2020First-in-Human Study of BCX9930 in Healthy Volunteers and Patients With PNHA Phase 1 Dose-ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of BCX9930 in Healthy Subjects and in Subjects With Paroxysmal Nocturnal HemoglobinuriaParoxysmal Nocturnal HemoglobinuriaDrug: BCX9930;Drug: PlaceboBioCryst PharmaceuticalsNULLRecruiting18 YearsN/AAll148Phase 1;Phase 2South Africa
27EUCTR2017-001418-27-SE
(EUCTR)
20/02/202008/11/2019This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on theinvestigational medicine after you have been given it, and if this iscomparable to what is seen for the licensed medicine, eculizumab, inpatients with Paroxysmal Nocturnal Hemoglobinuria (PNH).This study will assess if the investigational medicine is safe and effective intreating PNH compared to the licensed medicine.A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - Not applicable Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: ABP 959
Product Code: ABP 959
INN or Proposed INN: ECULIZUMAB
Other descriptive name: ABP 959 - proposed biosimilar to eculizumab
Trade Name: Soliris
Product Name: Soliris
INN or Proposed INN: ECULIZUMAB
Trade Name: Soliris
Product Name: Soliris
Amgen Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
40Phase 3United States;Portugal;Taiwan;Slovenia;Finland;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Brazil;Germany;Netherlands;Norway;Sweden
28EUCTR2019-000130-20-CZ
(EUCTR)
12/02/202014/11/2019REGN3918 in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) to evaluate its long term safety, efficacy and tolerability.An open-label extension study to evaluate the long-term safety, tolerability, and efficacy of REGN3918 in patients with paroxysmal nocturnal hemoglobinuria Paroxysmal nocturnal hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: REGN3918
Product Code: REGN3918
INN or Proposed INN: REGN3918
Other descriptive name: REGN3918
Regeneron Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
147Phase 3United States;Taiwan;Hong Kong;Korea, Democratic People's Republic of;United Kingdom;Italy;Czech Republic;Hungary;Canada;Poland;Malaysia;Singapore;Romania;South Africa;Germany;Netherlands
29EUCTR2019-001106-23-FR
(EUCTR)
04/02/202015/10/2019An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH).An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: APL-2
Product Code: APL-2
INN or Proposed INN: pegcetacoplan
Other descriptive name: APL-2
Apellis Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
119Phase 3United States;Serbia;Hong Kong;Thailand;Spain;Russian Federation;United Kingdom;France;Canada;Belgium;Malaysia;Poland;Australia;Bulgaria;Germany;Japan;Korea, Republic of
30EUCTR2019-000130-20-HU
(EUCTR)
27/01/202025/11/2019REGN3918 in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) to evaluate its long term safety, efficacy and tolerability.An open-label extension study to evaluate the long-term safety, tolerability, and efficacy of REGN3918 in patients with paroxysmal nocturnal hemoglobinuria Paroxysmal nocturnal hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: REGN3918
Product Code: REGN3918
INN or Proposed INN: REGN3918
Other descriptive name: REGN3918
Regeneron Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
147Phase 3United States;Hong Kong;Taiwan;Korea, Democratic People's Republic of;United Kingdom;Italy;Hungary;Canada;Malaysia;Poland;Singapore;Romania;South Africa;Germany;Netherlands
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
31EUCTR2019-001106-23-ES
(EUCTR)
22/01/202014/01/2020An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH).An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Apellis Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
119Phase 3Serbia;United States;Hong Kong;Spain;Thailand;Russian Federation;United Kingdom;France;Canada;Poland;Malaysia;Belgium;Australia;Bulgaria;Germany;Japan;Korea, Republic of
32EUCTR2017-001418-27-IE
(EUCTR)
15/01/202002/10/2019This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on theinvestigational medicine after you have been given it, and if this iscomparable to what is seen for the licensed medicine, eculizumab, inpatients with Paroxysmal Nocturnal Hemoglobinuria (PNH).This study will assess if the investigational medicine is safe and effective intreating PNH compared to the licensed medicine.A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - Not applicable Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: ABP 959
Product Code: ABP 959
INN or Proposed INN: ECULIZUMAB
Other descriptive name: ABP 959 - biosimilar to eculizumab
Trade Name: Soliris
Product Name: Soliris
INN or Proposed INN: ECULIZUMAB
Trade Name: Soliris
Product Name: Soliris
INN or Proposed INN: ECULIZUMAB
Amgen Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
40Phase 3United States;Portugal;Czechia;Taiwan;Slovenia;Finland;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Brazil;Germany;Netherlands;Norway;Sweden
33EUCTR2019-001106-23-GB
(EUCTR)
19/12/201930/09/2019An Open Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long Term Safety and Efficacy of Pegcetacoplan in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH).An Open Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long Term Safety and Efficacy of Pegcetacoplan in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Pegcetacoplan (APL-2)
Product Code: Pegcetacoplan (APL-2)
INN or Proposed INN: pegcetacoplan
Other descriptive name: Pegcetacoplan (APL-2)
Apellis Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
160Phase 3Serbia;United States;Hong Kong;Spain;Thailand;Russian Federation;United Kingdom;France;Canada;Malaysia;Australia;Bulgaria;Japan;Korea, Republic of
34NCT04170023
(ClinicalTrials.gov)
December 16, 20196/11/2019Proof of Concept Study of ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH)A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of the Oral Factor D (FD) Inhibitor ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy and With an Approved C5 Inhibitor as Background TherapyParoxysmal Nocturnal Hemoglobinuria (PNH)Drug: ALXN2050;Drug: C5 inhibitor background therapyAlexion PharmaceuticalsNULLRecruiting18 YearsN/AAll26Phase 2United States;Italy;Korea, Republic of;New Zealand;United Kingdom
35NCT04162470
(ClinicalTrials.gov)
December 3, 201911/11/2019REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate Its Long Term Safety, Efficacy and Tolerability.An Open-label Extension Study to Evaluate the Long-term Safety, Tolerability, and Efficacy of REGN3918 in Patients With Paroxysmal Nocturnal HemoglobinuriaParoxysmal Nocturnal HemoglobinuriaDrug: REGN3918Regeneron PharmaceuticalsNULLEnrolling by invitation18 YearsN/AAll147Phase 3Hong Kong;Korea, Republic of;Malaysia;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
36EUCTR2017-001418-27-CZ
(EUCTR)
22/11/201905/08/2019This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on theinvestigational medicine after you have been given it, and if this iscomparable to what is seen for the licensed medicine, eculizumab, inpatients with Paroxysmal Nocturnal Hemoglobinuria (PNH).This study will assess if the investigational medicine is safe and effective intreating PNH compared to the licensed medicine.A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - Not applicable Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: ABP 959
Product Code: ABP 959
INN or Proposed INN: ECULIZUMAB
Other descriptive name: ABP 959 - biosimilar to eculizumab
Trade Name: Soliris
Product Name: Soliris
INN or Proposed INN: ECULIZUMAB
Trade Name: Soliris
Product Name: Soliris
INN or Proposed INN: ECULIZUMAB
Amgen Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
40Phase 3United States;Portugal;Czechia;Taiwan;Slovenia;Finland;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Brazil;Germany;Netherlands;Norway;Sweden
37EUCTR2017-001418-27-SI
(EUCTR)
23/10/201910/09/2019This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on theinvestigational medicine after you have been given it, and if this iscomparable to what is seen for the licensed medicine, eculizumab, inpatients with Paroxysmal Nocturnal Hemoglobinuria (PNH).This study will assess if the investigational medicine is safe and effective intreating PNH compared to the licensed medicine.A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - Not applicable Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: ABP 959
Product Code: ABP 959
INN or Proposed INN: ECULIZUMAB
Other descriptive name: ABP 959 - biosimilar to eculizumab
Trade Name: Soliris
Product Name: Soliris
INN or Proposed INN: ECULIZUMAB
Trade Name: Soliris
Product Name: Soliris
INN or Proposed INN: ECULIZUMAB
Amgen Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
40Phase 3United States;Portugal;Czechia;Taiwan;Slovenia;Finland;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Brazil;Germany;Netherlands;Norway;Sweden
38EUCTR2017-001418-27-NO
(EUCTR)
18/10/201922/08/2019This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on theinvestigational medicine after you have been given it, and if this iscomparable to what is seen for the licensed medicine, eculizumab, inpatients with Paroxysmal Nocturnal Hemoglobinuria (PNH).This study will assess if the investigational medicine is safe and effective intreating PNH compared to the licensed medicine.A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - Not applicable Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: ABP 959
Product Code: ABP 959
INN or Proposed INN: ECULIZUMAB
Other descriptive name: ABP 959 - biosimilar to eculizumab
Trade Name: Soliris
Product Name: Soliris
INN or Proposed INN: ECULIZUMAB
Trade Name: Soliris
Product Name: Soliris
INN or Proposed INN: ECULIZUMAB
Amgen Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
40Phase 3United States;Portugal;Czechia;Taiwan;Slovenia;Finland;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Brazil;Norway;Germany;Netherlands;Sweden
39EUCTR2019-001453-10-ES
(EUCTR)
16/09/201909/08/2019A Low-Interventional Study Documenting the Efficacy, Health-Related Quality of Life, and Safety of Standard-Of-Care Treatment with Eculizumab or Treatment with Ravulizumab in Patients with Paroxysmal Nocturnal HemoglobinuriaA MULTICENTER, LOW-INTERVENTIONAL STUDY DOCUMENTING THE EFFICACY, HEALTH-RELATED QUALITY OF LIFE, AND SAFETY OF STANDARD-OF-CARE TREATMENT WITH ECULIZUMAB OR TREATMENT WITH RAVULIZUMAB IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA Paroxysmal nocturnal hemoglobinuria (PNH) that is treated with either eculizumab or ravulizumab as per local label
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Trade Name: Soliris 300
Product Name: ECULIZUMAB
INN or Proposed INN: ECULIZUMAB
Trade Name: ULTOMIRIS
Product Name: Ultomiris
INN or Proposed INN: RAVULIZUMAB
Other descriptive name: Ultomiris
Roche Farma S. A. U. que realiza el ensayo en España y que actua como representante F.Hoffmann-La Roche LtdNULLNot RecruitingFemale: yes
Male: yes
200Phase 4United States;Taiwan;Greece;Spain;Turkey;Israel;Colombia;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Argentina;Poland;Brazil;Romania;Netherlands;Germany;Japan;Sweden;Korea, Republic of
40NCT04085601
(ClinicalTrials.gov)
August 15, 20199/9/2019A Study to Evaluate the Efficacy and Safety of APL-2 in Patients With PNHA Phase 3, Randomized, Multicenter, Open-Label, Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)Paroxysmal Nocturnal HemoglobinuriaDrug: APL-2Apellis Pharmaceuticals, Inc.NULLActive, not recruiting18 YearsN/AAll53Phase 3Colombia;Hong Kong;Malaysia;Mexico;Peru;Philippines;Poland;Serbia;Singapore;Thailand
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
41EUCTR2019-001106-23-BG
(EUCTR)
09/08/201909/05/2019An Open-label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)An Open-label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: pegcetacoplan
Product Code: pegcetacoplan
INN or Proposed INN: pegcetacoplan
Other descriptive name: pegcetacoplan
Apellis Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
160Phase 3United States;Serbia;Hong Kong;Thailand;Spain;Ireland;Russian Federation;United Kingdom;Italy;France;Canada;Malaysia;Poland;Australia;Bulgaria;Germany;Netherlands;Japan;Korea, Republic of
42NCT04058158
(ClinicalTrials.gov)
August 7, 201912/8/2019A Study to Compare SB12 (Proposed Eculizumab Biosimilar) to Soliris in Subjects With Paroxysmal Nocturnal HaemoglobinuriaA Phase III Randomised, Double-blind, Multicentre Study to Compare the Efficacy, Safety, Pharmacokinetics, and Immunogenicity Between SB12 (Proposed Eculizumab Biosimilar) and Soliris® in Subjects With Paroxysmal Nocturnal HaemoglobinuriaParoxysmal Nocturnal HemoglobinuriaDrug: SB12 (proposed eculizumab biosimilar);Drug: Soliris (eculizumab)Samsung Bioepis Co., Ltd.NULLActive, not recruiting18 YearsN/AAll50Phase 3India;Korea, Republic of;Malaysia;Mexico;Romania;Taiwan;Thailand;Ukraine
43EUCTR2018-002734-20-NL
(EUCTR)
29/07/201902/04/2019A trial with REGN3918 in patients Paroxysmal Nocturnal Hemoglobinuria (PNH) who have not been treated previously with a Complement Inhibitor or Have Not Recently Received Complement Inhibitor TherapyAn Open Label, Single Arm Study to Evaluate the Efficacy and Safety of REGN3918 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor Naive or Have Not Recently Received Complement Inhibitor Therapy Paroxysmal nocturnal hemoglobinuria
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Regeneron Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
42Phase 2United States;Taiwan;Hong Kong;Korea, Democratic People's Republic of;United Kingdom;Italy;Hungary;Canada;Malaysia;Poland;Singapore;Romania;South Africa;Netherlands;Germany
44EUCTR2018-002734-20-GB
(EUCTR)
24/07/201922/03/2019A trial with REGN3918 in patients Paroxysmal Nocturnal Hemoglobinuria (PNH) who have not been treated previously with a Complement Inhibitor or Have Not Recently Received Complement Inhibitor TherapyAn Open Label, Single Arm Study to Evaluate the Efficacy and Safety of REGN3918 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor Naive or Have Not Recently Received Complement Inhibitor Therapy Paroxysmal nocturnal hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: REGN3918
Product Code: REGN3918
INN or Proposed INN: REGN3918
Other descriptive name: REGN3918
Product Name: REGN3918
Product Code: REGN3918
INN or Proposed INN: REGN3918
Other descriptive name: REGN3918
Regeneron Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
42Phase 2United States;Taiwan;Hong Kong;Korea, Democratic People's Republic of;United Kingdom;Italy;Hungary;Canada;Malaysia;Poland;Singapore;Romania;South Africa;Germany;Netherlands
45EUCTR2018-004220-11-PL
(EUCTR)
23/07/201913/05/2019A Research Study to Gather Scientific Information About the Efficacy and Safety of the Investigational Drug APL-2 In Treating Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH), a Disease Associated with Anemia, In a Randomly Assigned Comparison with the Current Standard of Care Treatment Approved for PNHA Phase 3, Randomized, Multicenter, Open-Label, Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - PRINCE Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: APL-2
Product Code: APL-2
INN or Proposed INN: pegcetacoplan
Other descriptive name: APL-2
Apellis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
54Phase 3Serbia;Philippines;Hungary;Hong Kong;Mexico;Argentina;Thailand;Malaysia;Brazil;Poland;Peru;Colombia
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
46EUCTR2017-002370-39-DE
(EUCTR)
02/07/201915/11/2018This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Alexion Pharmaceuticals, Inc.NULLNot Recruiting Female: yes
Male: yes
105Phase 3United States;Finland;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Netherlands;Germany;Korea, Republic of;Sweden
47EUCTR2017-002370-39-NL
(EUCTR)
01/07/201927/11/2018This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Alexion Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
105Phase 3United States;Finland;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Germany;Netherlands;Korea, Republic of;Sweden
48EUCTR2017-004268-36-NL
(EUCTR)
23/05/201909/08/2018A Research Study to Gather Scientific Information About the Efficacy, Safety, and Tolerability of the Investigational Drug APL-2 In Treating Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH), a Disease Associated With Anemia, In A Randomly Assigned Comparison With the Current Standard of Care Treatment Approved for PNH At Multiple Research Centres.A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - PEGASUS PNH is an acquired, rare, clonal, non-malignant hematologic disease characterized by complement-mediated RBC hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes, and/or some degree of bone marrow dysfunction. PNH is caused by complement-mediated lysis of erythrocyte clones lacking functional CD55 and CD59 on their surface. These RBCs are particularly susceptible to the MAC and have been shown to lyse readily in the presence of complement activation.
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Apellis Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
70Phase 3United States;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Australia;Netherlands;Germany;Japan;Korea, Republic of
49NCT03946748
(ClinicalTrials.gov)
May 16, 20198/5/2019Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)An Open-Label, Single Arm Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor-Naive or Have Not Recently Received Complement Inhibitor TherapyParoxysmal Nocturnal Hemoglobinuria (PNH)Drug: REGN3918Regeneron PharmaceuticalsNULLActive, not recruiting18 YearsN/AAll42Phase 2Hong Kong;Hungary;Korea, Republic of;Malaysia;United Kingdom
50EUCTR2018-002734-20-HU
(EUCTR)
10/05/201925/03/2019A trial with REGN3918 in patients Paroxysmal Nocturnal Hemoglobinuria (PNH) who have not been treated previously with a Complement Inhibitor or Have Not Recently Received Complement Inhibitor TherapyAn Open Label, Single Arm Study to Evaluate the Efficacy and Safety of REGN3918 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor Naive or Have Not Recently Received Complement Inhibitor Therapy Paroxysmal nocturnal hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: REGN3918
Product Code: REGN3918
INN or Proposed INN: REGN3918
Other descriptive name: REGN3918
Product Name: REGN3918
Product Code: REGN3918
INN or Proposed INN: REGN3918
Other descriptive name: REGN3918
Regeneron Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
42Phase 2United States;Hong Kong;Taiwan;Korea, Democratic People's Republic of;United Kingdom;Italy;Hungary;Canada;Malaysia;Poland;Singapore;Romania;South Africa;Germany;Netherlands
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
51NCT03818607
(ClinicalTrials.gov)
April 24, 201917/1/2019A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNHA Randomized, Double-Blind, Active-Controlled Phase 3 Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)Paroxysmal Nocturnal HemoglobinuriaDrug: ABP 959;Drug: EculizumabAmgenNULLRecruiting18 YearsN/AAll40Phase 3United States;Czechia;Finland;France;Ireland;Italy;Netherlands;Norway;Portugal;Slovenia;Spain;Sweden;Taiwan;Turkey;United Kingdom;Germany
52EUCTR2017-002370-39-CZ
(EUCTR)
09/04/201902/01/2019This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Alexion Pharmaceuticals, Inc.NULLNot Recruiting Female: yes
Male: yes
105Phase 3United States;Finland;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Netherlands;Germany;Korea, Republic of;Sweden
53NCT03896152
(ClinicalTrials.gov)
April 5, 201928/3/2019Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal HemoglobinuriaA Multi-center, Randomized, Open-label, Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria and Active HemolysisParoxysmal Nocturnal HemoglobinuriaDrug: LNP023Novartis PharmaceuticalsNULLActive, not recruiting18 YearsN/AAll13Phase 2Korea, Republic of;Malaysia;Singapore;Taiwan;Russian Federation
54NCT04060264
(ClinicalTrials.gov)
April 4, 201913/8/2019Clinical Trial of BCD-148 and Soliris® for the Treatment of Patients With Paroxysmal Nocturnal HemoglobinuriaRandomized, Open-Label, International, Multi-center, Comparative Study of Efficacy and Safety of BCD-148 (JSC BIOCAD, Russia) and Soliris® in Patients With Paroxysmal Nocturnal HemoglobinuriaParoxysmal Nocturnal HemoglobinuriaBiological: BCD-148;Biological: SolirisBiocadNULLActive, not recruiting18 Years65 YearsAll28Phase 3Russian Federation
55NCT03866681
(ClinicalTrials.gov)
April 1, 201928/2/2019Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory PNHSirolimus Combined With Low-dose Warfarin for the Treatment of Refractory Classic Paroxysmal Nocturnal Hemoglobinuria ,a Prospective StudyParoxysmal Nocturnal HemoglobinuriaDrug: sirolimusPeking Union Medical College HospitalNULLNot yet recruiting18 Years70 YearsAll40Phase 4China
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
56EUCTR2017-002370-39-AT
(EUCTR)
01/04/201924/01/2019This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With EculizumabA Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label,Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Ravulizumab
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Ravulizumab
Product Name: Ravulizumab
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Ravulizumab
Alexion Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
105Phase 3United States;Finland;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Netherlands;Germany;Korea, Republic of;Sweden
57EUCTR2017-002370-39-SE
(EUCTR)
27/03/201930/11/2018This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With EculizumabA Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label,Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Ravulizumab
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Ravulizumab
Trade Name: ULTOMIRIS
Product Name: Ravulizumab
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Ravulizumab
Alexion Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
105Phase 3United States;Finland;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Brazil;Australia;Germany;Netherlands;Sweden;Korea, Republic of
58EUCTR2016-004129-18-LT
(EUCTR)
15/03/201929/01/2019An extension study on rVA576 (Coversin) for Patients with PNH and aHUS who received benefit from their initial studyCONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study Paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS)
MedDRA version: 21.1;Level: PT;Classification code 10018932;Term: Haemolytic uraemic syndrome;System Organ Class: 10005329 - Blood and lymphatic system disorders
MedDRA version: 21.1;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Code: rVA576
INN or Proposed INN: rVA576
Other descriptive name: COVERSIN
Akari Therapeutics PlcNULLNot RecruitingFemale: yes
Male: yes
50Phase 3France;Argentina;Poland;Lithuania;Kazakhstan;Netherlands;Germany;Sri Lanka;United Kingdom;India
59EUCTR2017-002370-39-BE
(EUCTR)
13/03/201923/11/2018This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With EculizumabA Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label,Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Ravulizumab
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Ravulizumab
Product Name: Ravulizumab
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Ravulizumab
Alexion Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
105Phase 3United States;Finland;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Netherlands;Germany;Korea, Republic of;Sweden
60EUCTR2017-004268-36-IT
(EUCTR)
04/03/201926/09/2018A Research Study to Gather Scientific Information About the Efficacy, Safety, and Tolerability of the Investigational Drug APL-2 In Treating Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH), a Disease Associated With Anemia, In A Randomly Assigned Comparison With the Current Standard of Care Treatment Approved for PNH At Multiple Research Centres.A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - PEGASUS PNH is an acquired, rare, clonal, non-malignant hematologic disease characterized by complement-mediated RBC hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes, and/or some degree of bone marrow dysfunction.PNH is caused by complement-mediated lysis of erythrocyte clones lacking functional CD55 and CD59 on their surface. These RBCs are particularly susceptible to the MAC and have been shown to lyse readily in the presence of complement activation.
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: APL-2
Product Code: APL-2
INN or Proposed INN: n/a
Other descriptive name: n/a
Trade Name: Soliris
Product Name: Soliris
INN or Proposed INN: ECULIZUMAB
Apellis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
70Phase 3United States;Spain;Russian Federation;Italy;United Kingdom;France;Canada;Belgium;Australia;Germany;Netherlands;Japan;Korea, Republic of
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
61EUCTR2017-002370-39-GB
(EUCTR)
19/02/201926/11/2018This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Alexion Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
105Phase 3United States;Finland;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Netherlands;Germany;Korea, Republic of;Sweden
62EUCTR2018-003956-19-FI
(EUCTR)
13/02/201928/01/2019Study of Zilucoplan in Treatment-Naïve Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH)Phase 3, Multicenter, Open-Label, Single-Arm Study to Confirm the Safety and Efficacy of Zilucoplan in Treatment-Naïve Subjects with Paroxysmal Nocturnal Hemoglobinuria - Phase 3 Study in Treatment-Naive Paroxysmal Nocturnal Hemoglobinuria Patients Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Zilucoplan
Product Code: RA101495
INN or Proposed INN: ZILUCOPLAN
Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a polyethyleneglycol palmitoylated linker
Ra Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
40Phase 3United States;Hong Kong;Taiwan;Finland;Spain;Turkey;Russian Federation;Italy;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Malaysia;Belgium;Brazil;Australia;Georgia;Germany;New Zealand;Korea, Republic of
63EUCTR2017-002370-39-ES
(EUCTR)
08/02/201918/01/2019This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Alexion Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
105Phase 3United States;Finland;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Netherlands;Germany;Korea, Republic of;Sweden
64EUCTR2017-001418-27-DE
(EUCTR)
29/01/201927/07/2017 This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on the investigational medicine after you have been given it, and if this is comparable to what is seen for the licensed medicine, eculizumab, in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). This study will assess if the investigational medicine is safe and effective in treating PNH compared to the licensed medicine.A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - Not applicable Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Amgen Inc.NULLNot Recruiting Female: yes
Male: yes
40Phase 3France;United States;Czech Republic;Slovenia;Spain;Ireland;Norway;Netherlands;Germany;Italy;United Kingdom
65EUCTR2017-002370-39-FI
(EUCTR)
29/01/201921/12/2018This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Alexion Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
105Phase 3United States;Finland;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Netherlands;Germany;Korea, Republic of;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
66EUCTR2017-001418-27-NL
(EUCTR)
22/01/201902/11/2017 This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on the investigational medicine after you have been given it, and if this is comparable to what is seen for the licensed medicine, eculizumab, in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). This study will assess if the investigational medicine is safe and effective in treating PNH compared to the licensed medicine.A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - Not applicable Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Amgen Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
40Phase 3Spain;Norway;Germany;Netherlands;Italy;United Kingdom;Korea, Republic of
67EUCTR2017-004268-36-GB
(EUCTR)
08/01/201921/08/2018A Research Study to Gather Scientific Information About the Efficacy, Safety, and Tolerability of the Investigational Drug Pegcetacoplan In Treating Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH), a Disease Associated With Anemia, In A Randomly Assigned Comparison With the Current Standard of Care Treatment Approved for PNH At Multiple Research Centres.A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - PEGASUS PNH is an acquired, rare, clonal, non-malignant hematologic disease characterized by complement-mediated RBC hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes, and/or some degree of bone marrow dysfunction.PNH is caused by complement-mediated lysis of erythrocyte clones lacking functional CD55 and CD59 on their surface. These RBCs are particularly susceptible to the MAC and have been shown to lyse readily in the presence of complement activation.
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Pegcetacoplan (APL-2)
Product Code: Pegcetacoplan (APL-2)
INN or Proposed INN: n/a
Other descriptive name: n/a
Trade Name: Soliris
Product Name: Soliris
INN or Proposed INN: ECULIZUMAB
Trade Name: Soliris
Product Name: Soliris
INN or Proposed INN: ECULIZUMAB
Apellis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
70Phase 3United States;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Netherlands;Japan;Korea, Republic of
68EUCTR2017-002370-39-FR
(EUCTR)
28/12/201820/11/2018This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Alexion Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
105Phase 3United States;Finland;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Netherlands;Germany;Korea, Republic of;Sweden
69EUCTR2017-001418-27-GB
(EUCTR)
14/12/201811/12/2018 This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on the investigational medicine after you have been given it, and if this is comparable to what is seen for the licensed medicine, eculizumab, in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). This study will assess if the investigational medicine is safe and effective in treating PNH compared to the licensed medicine.A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - ABP 959 in PNH Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Amgen Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
40Phase 3Spain;Norway;Netherlands;Germany;Italy;United Kingdom
70EUCTR2017-004268-36-BE
(EUCTR)
25/10/201823/07/2018A Research Study to Gather Scientific Information About the Efficacy, Safety, and Tolerability of the Investigational Drug APL-2 In Treating Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH), a Disease Associated With Anemia, In A Randomly Assigned Comparison With the Current Standard of Care Treatment Approved for PNH At Multiple Research Centres.A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - PEGASUS PNH is an acquired, rare, clonal, non-malignant hematologic disease characterized by complement-mediated RBC hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes, and/or some degree of bone marrow dysfunction.PNH is caused by complement-mediated lysis of erythrocyte clones lacking functional CD55 and CD59 on their surface. These RBCs are particularly susceptible to the MAC and have been shown to lyse readily in the presence of complement activation.
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: APL-2
Product Code: APL-2
INN or Proposed INN: n/a
Other descriptive name: n/a
Trade Name: Soliris
Product Name: Soliris
INN or Proposed INN: ECULIZUMAB
Trade Name: Soliris
Product Name: Soliris
INN or Proposed INN: ECULIZUMAB
Apellis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
70Phase 3United States;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Netherlands;Japan;Korea, Republic of
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
71EUCTR2017-004268-36-FR
(EUCTR)
05/10/201820/08/2018A Research Study to Gather Scientific Information About the Efficacy, Safety, and Tolerability of the Investigational Drug APL-2 In Treating Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH), a Disease Associated With Anemia, In A Randomly Assigned Comparison With the Current Standard of Care Treatment Approved for PNH At Multiple Research Centres.A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - PEGASUS PNH is an acquired, rare, clonal, non-malignant hematologic disease characterized by complement-mediated RBC hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes, and/or some degree of bone marrow dysfunction.PNH is caused by complement-mediated lysis of erythrocyte clones lacking functional CD55 and CD59 on their surface. These RBCs are particularly susceptible to the MAC and have been shown to lyse readily in the presence of complement activation.
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: APL-2
Product Code: APL-2
INN or Proposed INN: n/a
Other descriptive name: n/a
Trade Name: Soliris
Product Name: Soliris
INN or Proposed INN: ECULIZUMAB
Apellis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
70Phase 3United States;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Netherlands;Japan;Korea, Republic of
72EUCTR2017-004268-36-DE
(EUCTR)
01/10/201814/06/2018A Research Study to Gather Scientific Information About the Efficacy, Safety, and Tolerability of the Investigational Drug APL-2 In Treating Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH), a Disease Associated With Anemia, In A Randomly Assigned Comparison With the Current Standard of Care Treatment Approved for PNH At Multiple Research Centres.A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - PEGASUS PNH is an acquired, rare, clonal, non-malignant hematologic disease characterized by complement-mediated RBC hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes, and/or some degree of bone marrow dysfunction.PNH is caused by complement-mediated lysis of erythrocyte clones lacking functional CD55 and CD59 on their surface. These RBCs are particularly susceptible to the MAC and have been shown to lyse readily in the presence of complement activation.
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: APL-2
Product Code: APL-2
INN or Proposed INN: n/a
Other descriptive name: n/a
Trade Name: Soliris
Product Name: Soliris
INN or Proposed INN: ECULIZUMAB
Trade Name: Soliris
Product Name: Soliris
INN or Proposed INN: ECULIZUMAB
Apellis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
70Phase 3United States;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Netherlands;Japan;Korea, Republic of
73EUCTR2017-004268-36-ES
(EUCTR)
19/09/201807/08/2018A Research Study to Gather Scientific Information About the Efficacy, Safety, and Tolerability of the Investigational Drug APL-2 In Treating Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH), a Disease Associated With Anemia, In A Randomly Assigned Comparison With the Current Standard of Care Treatment Approved for PNH At Multiple Research Centres.A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - PEGASUS PNH is an acquired, rare, clonal, non-malignant hematologic disease characterized by complement-mediated RBC hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes, and/or some degree of bone marrow dysfunction.PNH is caused by complement-mediated lysis of erythrocyte clones lacking functional CD55 and CD59 on their surface. These RBCs are particularly susceptible to the MAC and have been shown to lyse readily in the presence of complement activation.
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: APL-2
Product Code: APL-2
INN or Proposed INN: n/a
Other descriptive name: n/a
Trade Name: Soliris
Product Name: Soliris
INN or Proposed INN: ECULIZUMAB
Apellis Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
70Phase 3United States;Canada;Spain;Australia;Russian Federation;Japan;Korea, Republic of
74NCT03531255
(ClinicalTrials.gov)
August 27, 20189/5/2018Pegcetacoplan Long Term Safety and Efficacy Extension StudyAn Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegcetacoplan in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)PNHDrug: PegcetacoplanApellis Pharmaceuticals, Inc.NULLEnrolling by invitation18 YearsN/AAll160Phase 3United States;Australia;Belgium;Bulgaria;Canada;France;Germany;Hong Kong;Japan;Malaysia;Russian Federation;Serbia;Singapore;Spain;Thailand;United Kingdom
75NCT03593200
(ClinicalTrials.gov)
August 16, 201828/6/2018A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects With PNHPhase IIa, Open Label, Multiple Dose Study to Assess the Safety, Efficacy and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH).PNHDrug: APL-2Apellis Pharmaceuticals, Inc.NULLCompleted18 YearsN/AAll4Phase 2Bulgaria;Serbia;Greece;Poland;Romania
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
76EUCTR2017-000665-79-GB
(EUCTR)
23/07/201827/04/2018A treatment study of ACH-0144471 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who Completed Clinical Study ACH471-100An Open-Label Study to Evaluate Efficacy and Safety of Long-term Treatment with ACH-0144471 in Patients with PNH who Completed Clinical Study ACH471-100 Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Achillion Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
12Phase 2New Zealand;Italy;United Kingdom;Korea, Republic of
77EUCTR2016-003526-16-GB
(EUCTR)
20/07/201823/04/2018A treatment study of ACH-0144471 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have poor Response to Eculizumab MonotherapyA Phase 2 Open-label Study of ACH-0144471 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab Monotherapy Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Danicopan
Product Code: ALXN2040 (ACH-0144471)
INN or Proposed INN: Not available
Other descriptive name: ACH-0144471
Product Name: Danicopan
Product Code: ALXN2040 (ACH-0144471)
INN or Proposed INN: Not available
Other descriptive name: ACH-0144471
Product Name: Danicopan
Product Code: ALXN2040 (ACH-0144471)
INN or Proposed INN: Not available
Other descriptive name: ACH-0144471
Alexion Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
14Phase 2United States;Italy;United Kingdom
78EUCTR2017-005140-16-BG
(EUCTR)
18/07/201826/03/2018Phase IIa study of APL-2 in patients with PNHA Phase IIa, Open Label, Multiple Dose Study to Assess the Safety, Efficacy and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: APL-2
INN or Proposed INN: Not yet assigned
Apellis PharmaceuticalsNULLNot RecruitingFemale: yes
Male: yes
20Phase 2Serbia;Greece;Poland;Romania;Bulgaria
79NCT03500549
(ClinicalTrials.gov)
June 14, 201810/4/2018Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)Paroxysmal Nocturnal HemoglobinuriaDrug: APL-2;Drug: SolirisApellis Pharmaceuticals, Inc.NULLCompleted18 YearsN/AAll80Phase 3United States;Australia;Belgium;Canada;France;Germany;Japan;Korea, Republic of;Russian Federation;Spain;United Kingdom
80NCT03588026
(ClinicalTrials.gov)
June 7, 20181/6/2018Treating Paroxysmal Nocturnal Haemoglobinuria Patients With rVA576Investigational Product ; Coversin. Phase III Safety and Efficacy in Three-Part, Two-Arm, Randomised Open Label Evaluation in Patients With Paroxysmal Nocturnal Haemoglobinuria (PNH)Paroxysmal Nocturnal Hemoglobinuria (PNH)Drug: rVA576;Other: Standard of care (SOC)AKARI TherapeuticsNULLCompleted18 YearsN/AAll9Phase 3Kazakhstan;Lithuania;Sri Lanka
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
81NCT03427060
(ClinicalTrials.gov)
May 14, 201824/1/2018Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 PolymorphismsCONSENT II: Coversin in Paroxysmal Nocturnal Hemoglobinuria (PNH) in Patients With Resistance to Eculizumab Due to Complement C5 PolymorphismsParoxysmal Nocturnal Hemoglobinuria (PNH)Drug: CoversinAKARI TherapeuticsNULLEnrolling by invitation18 YearsN/AAll6Phase 2United States;Argentina
82NCT03472885
(ClinicalTrials.gov)
May 8, 201815/3/2018A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) With Inadequate Response to EculizumabA Phase 2 Open-label Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab MonotherapyParoxysmal Nocturnal Hemoglobinuria (PNH)Drug: ACH-0144471;Drug: EculizumabAlexion PharmaceuticalsAchillion, a wholly owned subsidiary of AlexionActive, not recruiting18 Years65 YearsAll12Phase 2United States
83EUCTR2017-005140-16-GR
(EUCTR)
04/05/201802/04/2018Phase IIa study of APL-2 in patients with PNHA Phase IIa, Open Label, Multiple Dose Study to Assess the Safety, Efficacy and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Apellis PharmaceuticalsNULLNot Recruiting Female: yes
Male: yes
20Phase 2Serbia;Greece;Poland;Romania;Bulgaria
84NCT03439839
(ClinicalTrials.gov)
April 9, 201814/2/2018Study of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)An Open Label, Single Arm, Multiple Dose Study to Assess Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of LNP023 When Administered in Addition to Standard of Care (SoC) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) With Signs of Active HemolysisPatients With Paroxysmal Nocturnal Hemoglobinuria (PNH)Drug: LNP023Novartis PharmaceuticalsNULLRecruiting18 Years80 YearsAll15Phase 2France;Germany;Italy
85EUCTR2017-002820-26-NL
(EUCTR)
21/03/201810/01/2018A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH)A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 20.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: ALXN1210
INN or Proposed INN: ALXN1210
Other descriptive name: ALXN1210
Alexion Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
12Phase 3France;United States;Netherlands;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
86EUCTR2017-003847-39-LT
(EUCTR)
12/03/201823/01/2018A study to assess the safety and efficacy of rVA576 in patients with Paroxysmal Nocturnal Haemoglobinuria (PNH)CAPSTONE: Phase III Confirmatory Assessment Protocol: rVA576 Safety and Efficacy in Three-Part, Two-Arm, Randomised Open Label Evaluation in patients with Paroxysmal Nocturnal Haemoglobinuria (PNH) - CAPSTONE Paroxysmal Nocturnal Haemoglobinuria (PNH)
MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Code: rVA576
INN or Proposed INN: NA
Akari Therapeutics Plc.NULLNot RecruitingFemale: yes
Male: yes
30Phase 3Ecuador;Turkey;Romania;Lithuania;Peru;Kazakhstan;Sri Lanka
87NCT03406507
(ClinicalTrials.gov)
February 22, 20185/1/2018A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria (PNH)A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria (PNH)Paroxysmal Nocturnal Hemoglobinuria (PNH)Biological: ALXN1210Alexion PharmaceuticalsNULLRecruitingN/A18 YearsAll13Phase 3United States;France;Netherlands;Norway;Russian Federation;United Kingdom
88EUCTR2016-004129-18-NL
(EUCTR)
14/02/201816/11/2017An extension study on Coversin for Patients with PNH and aHUS who received benefit from their initial studyCONSERVE: Coversin Long Term Safety and Efficacy Surveillance Study Paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS)
MedDRA version: 21.1;Level: PT;Classification code 10018932;Term: Haemolytic uraemic syndrome;System Organ Class: 10005329 - Blood and lymphatic system disorders
MedDRA version: 21.1;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: Coversin
Product Code: rVA576
INN or Proposed INN: COVERSIN
Other descriptive name: COVERSIN
Akari Therapeutics PlcNULLNot RecruitingFemale: yes
Male: yes
50Phase 3Argentina;Poland;Lithuania;Netherlands;United Kingdom
89EUCTR2017-000888-33-DE
(EUCTR)
05/02/201817/10/2017A Study evaluating the safety and efficacy of LNP023 in patients with a blood disorder called paroxysmal nocturnal hemoglobinuria (PNH)An open label, single arm, multiple dose study to assess efficacy, safety, pharmacokinetics and pharmacodynamics of LNP023 when administered in addition to Standard of Care (SoC) in patients with paroxysmal nocturnal hemoglobinuria (PNH) with signs of active hemolysis Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 20.0;Level: HLGT;Classification code 10018911;Term: Haemolyses and related conditions;System Organ Class: 10005329 - Blood and lymphatic system disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Code: LNP023
INN or Proposed INN: not yet established
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Product Code: LNP023
INN or Proposed INN: not yet established
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Product Code: LNP023
INN or Proposed INN: not yet established
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
15Phase 2France;Germany;Italy
90EUCTR2016-002652-25-GB
(EUCTR)
02/02/201827/10/2017A treatment study of ACH-0144471 in Untreated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH-0144471 in Untreated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Untreated Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Achillion Pharmaceuticals, Inc.NULLNot Recruiting Female: yes
Male: yes
12Phase 2New Zealand;Italy;United Kingdom;Korea, Republic of
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
91EUCTR2017-000888-33-FR
(EUCTR)
15/01/201822/01/2018 A Study evaluating the safety and efficacy of LNP023 in patients with a blood disorder called paroxysmal nocturnal hemoglobinuria (PNH)An open label, single arm, multiple dose study to assess efficacy, safety, pharmacokinetics and pharmacodynamics of LNP023 when administered in addition to Standard of Care (SoC) in patients with paroxysmal nocturnal hemoglobinuria (PNH) with signs of active hemolysis Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 20.0;Level: HLGT;Classification code 10018911;Term: Haemolyses and related conditions;System Organ Class: 10005329 - Blood and lymphatic system disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
10Phase 2France;Germany;Italy
92NCT03333486
(ClinicalTrials.gov)
December 7, 20172/11/2017Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood CancerA Phase II Trial of Haploidentical Allogeneic Stem Cell Transplantation Utilizing Mobilized Peripheral Blood Stem CellsAccelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Acute Leukemia in Remission;Acute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Acute Myeloid Leukemia With FLT3/ITD Mutation;Acute Myeloid Leukemia With Gene Mutations;Aplastic Anemia;B-Cell Non-Hodgkin Lymphoma;CD40 Ligand Deficiency;Chronic Granulomatous Disease;Chronic Leukemia in Remission;Chronic Lymphocytic Leukemia;Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Congenital Amegakaryocytic Thrombocytopenia;Congenital Neutropenia;Congenital Pure Red Cell Aplasia;Glanzmann Thrombasthenia;Immunodeficiency Syndrome;Myelodysplastic Syndrome;Myelofibrosis;Myeloproliferative Neoplasm;Paroxysmal Nocturnal Hemoglobinuria;Plasma Cell Myeloma;Polycythemia Vera;Recurrent Non-Hodgkin Lymphoma;Refractory Non-Hodgkin Lymphoma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndrome;Severe Aplastic Anemia;Shwachman-Diamond Syndrome;Sickle Cell Disease;T-Cell Non-Hodgkin Lymphoma;Thalassemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich SyndromeDrug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Procedure: Peripheral Blood Stem Cell Transplantation;Radiation: Total-Body IrradiationRoswell Park Cancer InstituteNational Cancer Institute (NCI)Recruiting1 Year75 YearsAll58Phase 2United States
93NCT04463056
(ClinicalTrials.gov)
November 29, 20173/7/2020Efficacy and Safety of Elizaria® vs. Soliris® in Patients With PNHA Multicenter, Open Label, Randomized, Parallel-group Study of Efficacy and Safety of Eculizumab (JSC GENERIUM, Russia) vs. Soliris® (Alexion Pharma GmbH, Switzerland) in Patients With Paroxysmal Nocturnal HemoglobinuriaParoxysmal Nocturnal Hemoglobinuria;Marchiafava-Micheli Syndrome;Paroxysmal HemoglobinuriaBiological: Elizaria®;Biological: Soliris®AO GENERIUMNULLCompleted18 Years75 YearsAll32Phase 3Russian Federation
94EUCTR2017-002820-26-GB
(EUCTR)
24/11/201726/10/2017A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH)A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Trade Name: Ultomiris
Product Name: Ravulizumab
Product Code: ALXN1210
INN or Proposed INN: Ravulizumab
Other descriptive name: ALXN1210
Alexion Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
13Phase 3United States;France;Russian Federation;Norway;Netherlands;United Kingdom
95JPRN-JapicCTI-173517
17/10/201717/02/2017An adaptive Phase I/II study of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH)An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH) Paroxysmal Nocturnal HemoglobinuriaIntervention name : RO7112689
INN of the intervention : Crovalimab
Dosage And administration of the intervention : Intravenous and Subcutaneous
Control intervention name : -
INN of the control intervention : -
Dosage And administration of the control intervention : -
CHUGAI PHARMACEUTICAL CO., LTD.NULLrecruiting1875BOTH24Phase 1;Phase 2Japan, Asia except Japan, Europe
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
96EUCTR2016-002128-10-IT
(EUCTR)
19/09/201706/02/2018Studio volto a valutare la sicurezza, l’efficacia, la farmacocinetica e la farmacodinamica di RO7112689 in volontari sani e in pazienti con Emoglobinuria Parossistica NotturnaAn adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH) - An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO Paroxysmal nocturnal hemoglobinuria (PNH)
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: RO7112689/F01
Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230
F. HOFFMANN - LA ROCHE LTD.NULLNot RecruitingFemale: yes
Male: yes
49Phase 1;Phase 2France;Hungary;Brazil;Netherlands;Germany;New Zealand;Japan;Italy;Korea, Republic of
97EUCTR2017-001418-27-ES
(EUCTR)
28/08/201731/07/2017This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on theinvestigational medicine after you have been given it, and if this iscomparable to what is seen for the licensed medicine, eculizumab, inpatients with Paroxysmal Nocturnal Hemoglobinuria (PNH).This study will assess if the investigational medicine is safe and effective intreating PNH compared to the licensed medicine.A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - Not applicable Paroxysmal Nocturnal Hemoglobinuria (PNH);Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]Product Name: ABP 959
Product Code: ABP 959
INN or Proposed INN: ECULIZUMAB
Other descriptive name: ABP 959 - biosimilar to eculizumab
Trade Name: Soliris
Product Name: Soliris
INN or Proposed INN: ECULIZUMAB
Amgen Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
40Phase 3France;Spain;Russian Federation;Netherlands;Germany;Japan;Italy;United Kingdom;Korea, Republic of
98EUCTR2016-002128-10-FR
(EUCTR)
17/08/201708/09/2017A Study to Assess Safety, Effectiveness, Pharmacokinetics, and Pharmacodynamics of RO7112689 in Healthy Volunteers and Patients With Paroxysmal Nocturnal HemoglobinuriaAn adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH) Paroxysmal nocturnal hemoglobinuria (PNH)
MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: RO7112689/F01
INN or Proposed INN: Not available
Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230
F. Hoffmann-La Roche LtdNULLNot RecruitingFemale: yes
Male: yes
49Phase 1;Phase 2France;Hungary;Netherlands;Germany;Japan;Italy;Korea, Republic of
99EUCTR2016-002026-36-NL
(EUCTR)
27/07/201727/03/2017ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) currently Treated With EculizumabA Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5
Trade Name: Soliris
INN or Proposed INN: ECULIZUMAB
Alexion Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
197Phase 3United States;Spain;Germany;Netherlands;United Kingdom
100NCT03225287
(ClinicalTrials.gov)
July 17, 201717/7/2017Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical StudyA Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical StudyParoxysmal Nocturnal Hemoglobinuria (PNH)Drug: Zilucoplan (RA101495)Ra PharmaceuticalsNULLActive, not recruiting18 YearsN/AAll28Phase 2United States;Australia;Denmark;Finland;Germany;Hungary;New Zealand;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
101NCT03181633
(ClinicalTrials.gov)
June 22, 20175/6/2017A Long-term Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)An Open-Label Study to Evaluate Efficacy and Safety of Long-term Treatment With ACH-0144471 in Patients Who Completed Clinical Study ACH471-100Paroxysmal Nocturnal HemoglobinuriaDrug: ACH-0144471Alexion PharmaceuticalsNULLActive, not recruiting18 YearsN/AAll8Phase 2New Zealand
102EUCTR2016-003522-16-FI
(EUCTR)
20/06/201727/12/2016Study of RA101495 in Paroxysmal Nocturnal Hemoglobinuria (PNH)A PHASE 2 MULTICENTER,OPEN-LABEL, UNCONTROLLED STUDY TO EVALUATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RA101495 IN SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: RA101495
Product Code: RA101495
INN or Proposed INN: RA101495 sodium
Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide
Ra Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
20Phase 2Hungary;Canada;Finland;Denmark;Australia;Germany;New Zealand;United Kingdom
103EUCTR2016-002026-36-DE
(EUCTR)
14/06/201723/02/2017ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) currently Treated With EculizumabA Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5
Trade Name: Soliris
INN or Proposed INN: ECULIZUMAB
Alexion Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
192Phase 3France;United States;Canada;Spain;Australia;Netherlands;Germany;Japan;Italy;United Kingdom;Korea, Republic of
104NCT03056040
(ClinicalTrials.gov)
June 5, 201714/2/2017ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With EculizumabA Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With EculizumabParoxysmal Nocturnal Hemoglobinuria (PNH)Biological: Ravulizumab;Biological: EculizumabAlexion PharmaceuticalsNULLActive, not recruiting18 YearsN/AAll195Phase 3United States;Australia;Canada;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Spain;United Kingdom
105EUCTR2016-002026-36-ES
(EUCTR)
31/05/201722/03/2017ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) currently Treated With EculizumabA Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 19.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5
Trade Name: Soliris
INN or Proposed INN: ECULIZUMAB
Alexion Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
192Phase 3United States;Spain;Germany
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
106EUCTR2016-002026-36-GB
(EUCTR)
30/05/201714/02/2017ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) currently Treated With EculizumabA Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Alexion Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
192Phase 3United States;Spain;Netherlands;Germany;United Kingdom
107EUCTR2016-003522-16-DK
(EUCTR)
01/05/201714/02/2017Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (PNH)A PHASE 2 MULTICENTER, OPEN-LABEL, UNCONTROLLED STUDY TO EVALUATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RA101495 IN SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA Paroxysmal Nocturnal Haemoglobinuria (PNH)
MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: RA101495
Product Code: RA101495
INN or Proposed INN: RA101495 sodium
Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24
Ra Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
20Phase 2Hungary;Canada;Finland;Australia;Denmark;Germany;New Zealand;United Kingdom
108EUCTR2016-003523-34-DK
(EUCTR)
01/05/201714/02/2017Extension Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (PNH)A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects with Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study Paroxysmal Nocturnal Haemoglobinuria (PNH)
MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: RA101495
Product Code: RA101495
INN or Proposed INN: RA101495 sodium
Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24
Ra Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
28Phase 2United States;Hungary;Canada;Finland;Australia;Denmark;Germany;New Zealand;United Kingdom
109EUCTR2016-002025-11-FI
(EUCTR)
28/04/201701/11/2016Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor.A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5
Trade Name: Soliris
INN or Proposed INN: ECULIZUMAB
Alexion Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
214Phase 3United States;Portugal;Estonia;Finland;Spain;Austria;United Kingdom;Czech Republic;Poland;Belgium;Denmark;Germany;Netherlands;Korea, Republic of;Sweden
110NCT03030183
(ClinicalTrials.gov)
April 17, 201720/1/2017Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to EculizumabA Phase 2 Multicenter, Open-Label, Uncontrolled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Have an Inadequate Response to EculizumabParoxysmal Nocturnal Hemoglobinuria (PNH)Drug: Zilucoplan (RA101495)Ra PharmaceuticalsNULLCompleted18 YearsN/AAll3Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
111EUCTR2016-003523-34-FI
(EUCTR)
06/04/201727/12/2016Extension Study of RA101495 in Paroxysmal Nocturnal Hemoglobinuria (PNH)A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects with Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Ra Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
28Phase 2United States;Hungary;Finland;Denmark;Australia;Germany;New Zealand;United Kingdom
112NCT03053102
(ClinicalTrials.gov)
March 31, 20171/2/2017A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH-0144471 in Untreated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)Paroxysmal Nocturnal Hemoglobinuria (PNH)Drug: ACH-0144471Alexion PharmaceuticalsNULLCompleted18 YearsN/AAll10Phase 2Italy;Korea, Republic of;New Zealand;United Kingdom
113EUCTR2016-002025-11-NL
(EUCTR)
27/03/201708/11/2016Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor.A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5
Trade Name: Soliris
INN or Proposed INN: ECULIZUMAB
Alexion Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
214Phase 3United States;Portugal;Estonia;Finland;Spain;Austria;United Kingdom;Belgium;Poland;Denmark;Netherlands;Germany;Korea, Republic of
114EUCTR2016-003523-34-HU
(EUCTR)
17/03/201731/01/2017Extension Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (PNH)A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects with Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study Paroxysmal Nocturnal Haemoglobinuria (PNH)
MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: RA101495
Product Code: RA101495
INN or Proposed INN: RA101495 sodium
Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24
Ra Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
28Phase 2United States;Hungary;Finland;Denmark;Australia;Germany;New Zealand;United Kingdom
115EUCTR2016-004129-18-GB
(EUCTR)
17/03/201721/12/2016An extension study on rVA576 (Coversin) for Patients with PNH and aHUS who received benefit from their initial studyCONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study Paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS)
MedDRA version: 21.1;Level: PT;Classification code 10018932;Term: Haemolytic uraemic syndrome;System Organ Class: 10005329 - Blood and lymphatic system disorders
MedDRA version: 21.1;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Code: rVA576
INN or Proposed INN: rVA576
Other descriptive name: rVA576
Akari Therapeutics PlcNULLNot RecruitingFemale: yes
Male: yes
50Phase 3France;Argentina;Poland;Lithuania;Kazakhstan;Netherlands;Germany;Sri Lanka;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
116EUCTR2016-003522-16-HU
(EUCTR)
17/03/201731/01/2017Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (PNH)A PHASE 2 MULTICENTER,OPEN-LABEL, UNCONTROLLED STUDY TO EVALUATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RA101495 IN SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA Paroxysmal Nocturnal Haemoglobinuria (PNH)
MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: RA101495
Product Code: RA101495
INN or Proposed INN: RA101495 sodium
Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24
Ra Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
20Phase 2Hungary;Finland;Denmark;Australia;Germany;New Zealand;United Kingdom
117EUCTR2016-002025-11-PL
(EUCTR)
16/03/201720/03/2017Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor.A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5
Trade Name: Soliris
INN or Proposed INN: ECULIZUMAB
Alexion Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
214Phase 3United States;Portugal;Estonia;Finland;Spain;Austria;United Kingdom;Czech Republic;Poland;Belgium;Denmark;Germany;Netherlands;Korea, Republic of;Sweden
118EUCTR2016-002025-11-BE
(EUCTR)
14/03/201717/10/2016Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor.A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5
Trade Name: Soliris
INN or Proposed INN: ECULIZUMAB
Alexion Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
214Phase 3United States;Portugal;Estonia;Finland;Spain;Austria;United Kingdom;Czech Republic;Poland;Belgium;Denmark;Germany;Netherlands;Korea, Republic of;Sweden
119EUCTR2016-002128-10-DE
(EUCTR)
14/03/201724/10/2016A Study to Assess Safety, Effectiveness, Pharmacokinetics, and Pharmacodynamics of crovalimab in Healthy Volunteers and Patients With Paroxysmal Nocturnal HemoglobinuriaAn adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of crovalimab in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH) Paroxysmal nocturnal hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: RO7112689/F01
INN or Proposed INN: Not available
Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230
Product Code: RO7112689/F03-01
INN or Proposed INN: Not available
Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230
Product Code: RO7112689/F03-03
INN or Proposed INN: Not available
Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
49Phase 1;Phase 2France;Hungary;Netherlands;Germany;Italy;Japan;Korea, Republic of
120NCT03829449
(ClinicalTrials.gov)
March 13, 201720/12/2018rVA576 (Coversin) Long Term Safety and Efficacy Surveillance StudyCONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance StudyParoxysmal Nocturnal Hemoglobinuria;Atypical Hemolytic Uremic SyndromeDrug: rVA576 (Coversin)AKARI TherapeuticsNULLRecruiting18 YearsN/AAll50Phase 3Poland
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
121NCT03078582
(ClinicalTrials.gov)
March 8, 20178/3/2017Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH PatientsPhase 2 Multicenter, Open-Label, Uncontrolled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of RA101495 in Subjects With Paroxysmal Nocturnal HemoglobinuriaParoxysmal Nocturnal Hemoglobinuria (PNH)Drug: Zilucoplan (RA101495)Ra PharmaceuticalsNULLCompleted18 YearsN/AAll26Phase 2Australia;Canada;Denmark;Finland;Germany;Hungary;New Zealand;United Kingdom
122EUCTR2016-004129-18-PL
(EUCTR)
07/03/201718/01/2017An extension study on Coversin for Patients with PNH and aHUS who received benefit from their initial studyCONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study Paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS)
MedDRA version: 21.1;Level: PT;Classification code 10018932;Term: Haemolytic uraemic syndrome;System Organ Class: 10005329 - Blood and lymphatic system disorders
MedDRA version: 21.1;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: rVA576 (Coversin)
Product Code: rVA576
INN or Proposed INN: rVA576 (COVERSIN)
Other descriptive name: rVA576 (COVERSIN)
Akari Therapeutics PlcNULLNot RecruitingFemale: yes
Male: yes
50Phase 3France;Argentina;Poland;Lithuania;Kazakhstan;Netherlands;Germany;Sri Lanka;United Kingdom
123EUCTR2016-002025-11-EE
(EUCTR)
20/02/201723/01/2017Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor.A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Alexion Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
214Phase 3Portugal;United States;Taiwan;Estonia;Thailand;Spain;Russian Federation;Colombia;Italy;France;Malaysia;Denmark;Australia;Netherlands;Korea, Republic of;Finland;Turkey;Austria;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Singapore;Germany;Japan;Sweden
124EUCTR2016-003523-34-GB
(EUCTR)
20/02/201726/09/2017Extension Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (PNH)A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects with Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study Paroxysmal Nocturnal Haemoglobinuria (PNH)
MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Ra Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
28Phase 2United States;Hungary;Canada;Finland;Denmark;Australia;Germany;New Zealand;United Kingdom
125EUCTR2016-003522-16-GB
(EUCTR)
15/02/201703/01/2017Study of RA101495 in Paroxysmal Nocturnal Hemoglobinuria (PNH)A PHASE 2 MULTICENTER,OPEN-LABEL, UNCONTROLLED STUDY TO EVALUATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RA101495 IN SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: RA101495
Product Code: RA101495
INN or Proposed INN: RA101495 sodium
Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24
Ra Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
20Phase 2Hungary;Canada;Finland;Denmark;Australia;Germany;New Zealand;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
126EUCTR2016-002025-11-CZ
(EUCTR)
02/02/201717/10/2016Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor.A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Alexion Pharmaceuticals IncorporatedNULLNot Recruiting Female: yes
Male: yes
214Phase 3United States;Portugal;Estonia;Finland;Spain;Austria;United Kingdom;Czech Republic;Poland;Belgium;Denmark;Germany;Netherlands;Korea, Republic of;Sweden
127EUCTR2016-002025-11-PT
(EUCTR)
30/01/201721/10/2016Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor.A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 19.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5
Trade Name: Soliris
INN or Proposed INN: ECULIZUMAB
Alexion Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
214Phase 3United States;Portugal;Estonia;Poland;Spain;Austria;Denmark;Germany;United Kingdom;Korea, Republic of
128EUCTR2016-002128-10-HU
(EUCTR)
24/01/201714/11/2016A Study to Assess Safety, Effectiveness, Pharmacokinetics, and Pharmacodynamics of RO7112689 in Healthy Volunteers and Patients With Paroxysmal Nocturnal HemoglobinuriaAn adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH) Paroxysmal nocturnal hemoglobinuria (PNH)
MedDRA version: 19.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: RO7112689/F01
INN or Proposed INN: Not available
Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
49Phase 1;Phase 2France;Hungary;Brazil;Netherlands;Germany;New Zealand;Japan;Italy;Korea, Republic of
129EUCTR2016-002025-11-ES
(EUCTR)
16/01/201704/11/2016Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor.A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 19.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5
Trade Name: Soliris
INN or Proposed INN: ECULIZUMAB
Alexion Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
214Phase 3United States;Spain;Austria;Denmark;Germany;Korea, Republic of
130EUCTR2016-002025-11-AT
(EUCTR)
11/01/201714/12/2016Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor.A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5
Trade Name: Soliris
INN or Proposed INN: ECULIZUMAB
Alexion Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
214Phase 3Portugal;United States;Estonia;Taiwan;Spain;Thailand;Russian Federation;Colombia;Italy;France;Malaysia;Denmark;Australia;Netherlands;Korea, Republic of;Finland;Turkey;Austria;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Singapore;Germany;Japan;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
131EUCTR2016-002025-11-DK
(EUCTR)
23/12/201620/10/2016Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor.A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5
Trade Name: Soliris
INN or Proposed INN: ECULIZUMAB
Alexion Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
214Phase 3United States;Portugal;Estonia;Finland;Spain;Austria;United Kingdom;Belgium;Poland;Denmark;Germany;Netherlands;Korea, Republic of
132EUCTR2016-002025-11-DE
(EUCTR)
20/12/201607/10/2016Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor.A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5
Trade Name: Soliris
INN or Proposed INN: ECULIZUMAB
Alexion Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
214Phase 3Portugal;United States;Taiwan;Estonia;Thailand;Spain;Russian Federation;Colombia;Italy;France;Malaysia;Denmark;Australia;Netherlands;Korea, Republic of;Finland;Turkey;Austria;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Singapore;Germany;Japan;Sweden
133NCT02946463
(ClinicalTrials.gov)
December 20, 201625/10/2016ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)Paroxysmal Nocturnal Hemoglobinuria (PNH)Biological: Ravulizumab;Biological: EculizumabAlexion PharmaceuticalsNULLActive, not recruiting18 YearsN/AAll246Phase 3United States;Argentina;Australia;Austria;Belgium;Brazil;Canada;Czechia;Estonia;France;Germany;Italy;Japan;Korea, Republic of;Malaysia;Mexico;Poland;Russian Federation;Singapore;Spain;Sweden;Taiwan;Thailand;Turkey;United Kingdom;Colombia
134EUCTR2016-002025-11-GB
(EUCTR)
02/12/201607/10/2016Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor.A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5
Trade Name: Soliris
INN or Proposed INN: ECULIZUMAB
Alexion Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
214Phase 3United States;Portugal;Estonia;Finland;Spain;Austria;United Kingdom;Czech Republic;Poland;Belgium;Denmark;Germany;Netherlands;Korea, Republic of;Sweden
135EUCTR2016-002943-40-ES
(EUCTR)
21/11/201630/09/2016A Clinical Study with Investigational Drug, ALN-CC5, in patients with Paroxysmal Nocturnal Hemoglobinuria who are Inadequate Responders to EculizumabA Phase 2, Open-label, Single Dose, Study of Subcutaneously Administered ALN-CC5 in Patients with Paroxysmal Nocturnal Hemoglobinuria who are Inadequate Responders to Eculizumab Paroxysmal nocturnal hemoglobinuria (PNH)
MedDRA version: 19.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: ALN-CC5
INN or Proposed INN: ALN-62643
Alnylam Pharmaceuticals IncNULLNot RecruitingFemale: yes
Male: yes
15Phase 2Taiwan;Finland;Spain;Turkey;Austria;Russian Federation;Israel;United Kingdom;France;Czech Republic;Canada;Belgium;Australia;Denmark;Bulgaria;Norway;Netherlands;Germany;Korea, Republic of;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
136NCT03157635
(ClinicalTrials.gov)
November 14, 20161/11/2016Study to Assess Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Healthy Volunteers and Participants With Paroxysmal Nocturnal HemoglobinuriaAn Adaptive Phase I/II Study to Assess Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Crovalimab in Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)Paroxysmal Hemoglobinuria, NocturnalDrug: Crovalimab;Drug: PlaceboHoffmann-La RocheNULLActive, not recruiting18 Years75 YearsAll59Phase 1;Phase 2France;Germany;Hungary;Italy;Japan;Korea, Republic of;Netherlands
137EUCTR2016-002128-10-NL
(EUCTR)
14/09/201605/09/2016A Study to Assess Safety, Effectiveness, Pharmacokinetics, and Pharmacodynamics of RO7112689 in Healthy Volunteers and Patients With Paroxysmal Nocturnal HemoglobinuriaAn adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH) Paroxysmal nocturnal hemoglobinuria (PNH)
MedDRA version: 19.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: RO7112689/F01
INN or Proposed INN: Not available
Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230
F. Hoffmann-La Roche LtdNULLNot RecruitingFemale: no
Male: yes
74Phase 1;Phase 2France;Hungary;Czech Republic;Brazil;Germany;Netherlands;Japan;Italy;United Kingdom
138EUCTR2016-002067-33-GB
(EUCTR)
18/07/201616/08/2016An Open Clincal Trial of Coversin in Patients with PNHCOBALT: Coversin Global Study: An Open-Label, Safety and Efficacy Trial in PNH Patients Paroxysmal nocturnal haemoglobinuria (PNH)
MedDRA version: 20.0;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Akari Therapeutics PlcNULLNot Recruiting Female: yes
Male: yes
10Phase 2United Kingdom
139NCT02591862
(ClinicalTrials.gov)
February 201610/9/2015Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH)Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) in Patients With Resistance to Eculizumab Due to Complement C5 PolymorphismsParoxysmal Nocturnal Haemoglobinuria (PNH)Drug: CoversinAKARI TherapeuticsRadboud UniversityCompleted18 Years80 YearsAll6Phase 2Netherlands
140EUCTR2015-003778-34-NL
(EUCTR)
13/01/201610/12/2015Coversin in Paroxysmal Nocturnal Haemoglobulinuria (PNH) in patients with resistance to Eculizumab due to complement C5 PolymorphismsCoversin in Paroxysmal Nocturnal Haemoglobulinuria (PNH) in patients with resistance to Eculizumab due to complement C5 Polymorphisms - Coversin in PNH patients with resistance to Eculizumab Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) in patients with resistance to Eculizumab due to complement C5 polymorphisms.
MedDRA version: 18.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: Coversin
Product Code: rVA576
INN or Proposed INN: Not applicable
Other descriptive name: coversin
Volution Immuno Pharmaceuticals (UK) LtdNULLNot RecruitingFemale: yes
Male: yes
6Phase 2Netherlands
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
141NCT02605993
(ClinicalTrials.gov)
January 4, 201611/11/2015Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)A Phase 2, Open-label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients With Paroxysmal Nocturnal HemoglobinuriaParoxysmal Nocturnal Hemoglobinuria;PNHBiological: RavulizumabAlexion PharmaceuticalsNULLActive, not recruiting18 YearsN/AAll26Phase 2Canada;France;Germany;Korea, Republic of;Spain;Taiwan;United Kingdom;Sweden
142EUCTR2015-002674-20-DE
(EUCTR)
04/12/201511/08/2015A Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients with Paroxysmal Nocturnal HemoglobinuriaA Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients with Paroxysmal Nocturnal Hemoglobinuria Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: ALXN1210
INN or Proposed INN: ALXN1210
Product Code: ALXN1210
INN or Proposed INN: ALXN1210
Alexion Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
26Phase 2Taiwan;Canada;Finland;Spain;Russian Federation;Germany;United Kingdom;Sweden;Korea, Republic of;France;United States
143EUCTR2015-002674-20-SE
(EUCTR)
30/11/201502/10/2015A Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients with Paroxysmal Nocturnal HemoglobinuriaA Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients with Paroxysmal Nocturnal Hemoglobinuria Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 18.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: ALXN1210
INN or Proposed INN: ALXN1210
Alexion Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
18Phase 2France;Taiwan;Canada;Finland;Spain;Russian Federation;Germany;United Kingdom;Sweden
144EUCTR2015-002674-20-ES
(EUCTR)
27/11/201505/10/2015A Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients with Paroxysmal Nocturnal HemoglobinuriaA Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients with Paroxysmal Nocturnal Hemoglobinuria Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 18.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: ALXN1210
INN or Proposed INN: ALXN1210
Alexion Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
18Phase 2France;Taiwan;Canada;Finland;Spain;Russian Federation;Germany;United Kingdom;Sweden
145NCT02598583
(ClinicalTrials.gov)
November 20152/11/2015Dose-Escalation Study of ALXN1210 IV in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)An Open-Label, Intrapatient, Dose-Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients With Paroxysmal Nocturnal HemoglobinuriaPNHBiological: ALXN1210Alexion PharmaceuticalsNULLActive, not recruiting18 YearsN/AAll13Phase 1;Phase 2Australia;Korea, Republic of
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
146NCT02588833
(ClinicalTrials.gov)
November 201527/10/2015Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. APL-2 in PNH SubjectsA Phase Ib, Open Label, Multiple Ascending Dose, Pilot Study to Assess the Safety, Preliminary Efficacy and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)Paroxysmal Nocturnal HemoglobinuriaDrug: APL-2Apellis Pharmaceuticals, Inc.NULLCompleted18 YearsN/AAll20Phase 1Hong Kong;Malaysia;New Zealand;Thailand
147EUCTR2015-002674-20-GB
(EUCTR)
28/10/201501/10/2015A Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients with Paroxysmal Nocturnal HemoglobinuriaA Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients with Paroxysmal Nocturnal Hemoglobinuria Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Alexion Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
26Phase 2France;United States;Taiwan;Canada;Finland;Spain;Russian Federation;Germany;United Kingdom;Korea, Republic of;Sweden
148NCT02534909
(ClinicalTrials.gov)
September 9, 20157/8/2015Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients With Paroxysmal Nocturnal HemoglobinuriaAn Open-label Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316, an Anti-C5 Monoclonal Antibody in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)Paroxysmal Nocturnal Hemoglobinuria PNHBiological: LFG316Novartis PharmaceuticalsNULLActive, not recruiting18 YearsN/AAll10Phase 2Czechia;Japan;Lithuania;Czech Republic;Moldova, Republic of
149EUCTR2014-005338-74-LT
(EUCTR)
07/07/201509/06/2015To find out whether LFG316 is able to reduce the destruction of red blood cells in patients with PNHAn open-label proof of concept study to assess the efficacy, safety and pharmacokinetics of LFG316, an anti-C5 monoclonal antibody in patients with paroxysmal nocturnal hemoglobinuria (PNH) - CLFG316X2201 Paroxysmal nocturnal hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Code: LFG316
INN or Proposed INN: No
Other descriptive name: human monoclonal antibody directed against complement 5 protein
Product Code: LFG316
INN or Proposed INN: No
Other descriptive name: human monoclonal antibody directed against complement 5 protein
Novartis Pharma Services AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
10Phase 2Czech Republic;Lithuania;Japan
150EUCTR2014-005338-74-CZ
(EUCTR)
17/06/201523/04/2015To find out whether LFG316 is able to reduce the destruction of red blood cells in patients with PNHAn open-label proof of concept study to assess the efficacy, safety and pharmacokinetics of LFG316, an anti-C5 monoclonal antibody in patients with paroxysmal nocturnal hemoglobinuria (PNH) - CLFG316X2201 Paroxysmal nocturnal hemoglobinuria
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Novartis Pharma Services AGNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
10Phase 2Czech Republic;Lithuania;Japan
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
151NCT02264639
(ClinicalTrials.gov)
February 23, 20158/10/2014A Phase I Study to Assess the Safety APL-2 as an Add-On to Standard of Care in Subjects With PNHAn Open Label, Single and Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of APL-2 as an Add-On to Standard of Care in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH).Paroxysmal Nocturnal Hemoglobinuria (PNH)Drug: APL-2Apellis Pharmaceuticals, Inc.NULLCompleted18 YearsN/AAll9Phase 1United States
152NCT02352493
(ClinicalTrials.gov)
January 201523/1/2015A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNHA Phase 1/2 Single-ascending and Multiple-ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-CC5 in Healthy Adult Volunteers and Patients With Paroxysmal Nocturnal HemoglobinuriaParoxysmal Nocturnal Hemoglobinuria (PNH)Drug: ALN-CC5;Drug: Sterile Normal Saline (0.9% NaCl)Alnylam PharmaceuticalsNULLCompleted18 Years45 YearsAll62Phase 1;Phase 2Spain;United Kingdom
153NCT02179359
(ClinicalTrials.gov)
September 2, 201427/6/2014Hematopoietic Stem Cell Transplant for High Risk HemoglobinopathiesMT2014-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent DisordersSickle Cell Disease;Transfusion Dependent Alpha- or Beta- Thalassemia;Diamond Blackfan Anemia;Paroxysmal Nocturnal Hemoglobinuria;Glanzmann Thrombasthenia;Severe Congenital Neutropenia;Shwachman-Diamond Syndrome;Non-Malignant Hematologic DisordersDrug: Reduced Toxicity Ablative Regimen;Drug: Reduced Intensity Preparative Regimen;Drug: Myeloablative Preparative RegimenMasonic Cancer Center, University of MinnesotaNULLRecruitingN/A55 YearsAll25N/AUnited States
154ChiCTR-TRC-14004219
2014-01-242014-01-24A prospective, randomized, open label, single-center trial of comparing intravenous iron sucrose versus iron dextran tablet in the treatment of iron deficiency anemia and classic paroxysmal nocturnal hemoglobinuriaA prospective, randomized, open label, single-center trial of comparing intravenous iron sucrose versus iron dextran tablet in the treatment of iron deficiency anemia and classic paroxysmal nocturnal hemoglobinuria iron deficiency anemia and classic paroxysmal nocturnal hemoglobinuriaintravenous iron sucrose:intravenous iron sucrose;iron dextran tablet:Oral iron dextran tablet;Institute of Hematology and Blood Diseases Hospital,Chinese Academy of Medical SciencesNULLRecruiting1865Bothintravenous iron sucrose:24;iron dextran tablet:24;China
155ChiCTR-ONC-13003994
2014-01-012013-11-06Chemotherapy plus hematopoietic growth factors (DAG) for refractory paroxysmal nocturnal hemoglobinuria: diminishing PNH clone and stimulating hematopoisisChemotherapy plus hematopoietic growth factors (DAG) for refractory paroxysmal nocturnal hemoglobinuria: diminishing PNH clone and stimulating hematopoisis paroxymal nocturnal hemoglobinuriaA1:DA chemotherapy regimen plus G-CSF ;Tianjin Medical University General HospitalNULLRecruiting1675BothA1:50;China
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
156NCT01760096
(ClinicalTrials.gov)
January 20131/1/2013Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013)Phase II Study of the Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013)Paroxysmal Nocturnal Hemoglobinuria;Hemoglobinuria;Hemoglobinuria, Paroxysmal;Proteinuria;Urination Disorders;Thrombosis;Bone Marrow Failure;Aplastic Anemia,;Anemia, HemolyticDrug: Levamisole+cyclosporin A+Glucocorticoids;Drug: cyclosporin A+Glucocorticoids;Drug: GlucocorticoidsInstitute of Hematology & Blood Diseases HospitalNULLActive, not recruiting18 YearsN/ABoth50Phase 2China
157NCT01642979
(ClinicalTrials.gov)
July 201214/7/2012Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal HemoglobinuriaPhase II Study of the Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal HemoglobinuriaParoxysmal Nocturnal Hemoglobinuria;Hemoglobinuria;Hemoglobinuria, Paroxysmal;Proteinuria;Urination Disorders;Thrombosis;Bone Marrow Failure;Aplastic Anemia,;Anemia, HemolyticDrug: Levamisole+cyclosporin A+Glucocorticoids;Drug: cyclosporin A+Glucocorticoids;Drug: GlucocorticoidsInstitute of Hematology & Blood Diseases HospitalNULLActive, not recruiting18 YearsN/ABoth120Phase 2China
158NCT01529827
(ClinicalTrials.gov)
February 28, 20126/2/2012Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic MalignanciesA Phase II Trial of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan and Low Dose Total Body IrradiationAccelerated Phase Chronic Myelogenous Leukemia;Adult Acute Lymphoblastic Leukemia in Remission;Adult Acute Myeloid Leukemia in Remission;Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities;Adult Acute Myeloid Leukemia With Del(5q);Adult Acute Myeloid Leukemia With Inv(16)(p13;q22);Adult Acute Myeloid Leukemia With t(15;17)(q22;q12);Adult Acute Myeloid Leukemia With t(16;16)(p13;Adult Acute Myeloid Leukemia With t(8;21)(q22;Adult Grade III Lymphomatoid Granulomatosis;Adult Nasal Type Extranodal NK/T-cell Lymphoma;Anaplastic Large Cell Lymphoma;Angioimmunoblastic T-cell Lymphoma;Aplastic Anemia;Burkitt Lymphoma;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Chronic Myelogenous Leukemia;Childhood Diffuse Large Cell Lymphoma;Childhood Grade III Lymphomatoid Granulomatosis;Childhood Immunoblastic Large Cell Lymphoma;Childhood Myelodysplastic Syndromes;Childhood Nasal Type Extranodal NK/T-cell Lymphoma;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia;Congenital Amegakaryocytic Thrombocytopenia;Diamond-Blackfan Anemia;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Hepatosplenic T-cell Lymphoma;Juvenile Myelomonocytic Leukemia;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Nodal Marginal Zone B-cell Lymphoma;Paroxysmal Nocturnal Hemoglobinuria;Peripheral T-cell Lymphoma;Polycythemia Vera;Post-transplant Lymphoproliferative Disorder;Previously Treated Myelodysplastic Syndromes;Primary Myelofibrosis;Recurrent Adult Acute Lymphoblastic Leukemia;Recurrent Adult Acute Myeloid Leukemia;Recurrent Adult Burkitt Lymphoma;Recurrent Adult Diffuse Large Cell Lymphoma;Recurrent Adult Diffuse Mixed Cell Lymphoma;Recurrent Adult Diffuse Small Cleaved Cell Lymphoma;Recurrent Adult Grade III Lymphomatoid Granulomatosis;Recurrent Adult Hodgkin Lymphoma;Recurrent Adult Immunoblastic Large Cell Lymphoma;Recurrent Adult Lymphoblastic Lymphoma;Recurrent Adult T-cell Leukemia/Lymphoma;Recurrent Childhood Acute Lymphoblastic Leukemia;Recurrent Childhood Acute Myeloid Leukemia;Recurrent Childhood Anaplastic Large Cell Lymphoma;Recurrent Childhood Grade III Lymphomatoid Granulomatosis;Recurrent Childhood Large Cell Lymphoma;Recurrent Childhood Lymphoblastic Lymphoma;Recurrent Childhood Small Noncleaved Cell Lymphoma;Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma;Recurrent Grade 1 Follicular Lymphoma;Recurrent Grade 2 Follicular Lymphoma;Recurrent Grade 3 Follicular Lymphoma;Recurrent Mantle Cell Lymphoma;Recurrent Marginal Zone Lymphoma;Recurrent Mycosis Fungoides/Sezary Syndrome;Recurrent Small Lymphocytic Lymphoma;Recurrent/Refractory Childhood Hodgkin Lymphoma;Refractory Chronic Lymphocytic Leukemia;Refractory Hairy Cell Leukemia;Refractory Multiple Myeloma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndromes;Secondary Myelofibrosis;Severe Combined Immunodeficiency;Severe Congenital Neutropenia;Shwachman-Diamond Syndrome;Splenic Marginal Zone Lymphoma;T-cell Large Granular Lymphocyte Leukemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich SyndromeDrug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiation;Drug: tacrolimus;Drug: mycophenolate mofetil;Drug: methotrexate;Other: laboratory biomarker analysis;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantationRoswell Park Cancer InstituteNULLCompleted3 Years75 YearsAll94Phase 2United States
159NCT01335165
(ClinicalTrials.gov)
April 201112/4/2011Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)A Phase 1, Single Ascending Dose Study of the Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)Paroxysmal Nocturnal Hemoglobinuria (PNH)Drug: ALXN1102;Drug: ALXN1103Alexion PharmaceuticalsNULLTerminated18 YearsN/ABoth10Phase 1United States;Czech Republic;Italy;Poland;United Kingdom
160NCT00867932
(ClinicalTrials.gov)
October 2, 200923/3/2009Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria (PNH)An Open-Label Multi-Center Study of Eculizumab in Children and Adolescents With a Diagnosis of Paroxysmal Nocturnal HemoglobinuriaHemoglobinuria, ParoxysmalDrug: EculizumabAlexion PharmaceuticalsNULLCompleted2 Years17 YearsAll7Phase 4United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
161NCT00856388
(ClinicalTrials.gov)
January 14, 20094/3/2009Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure DisordersA Pilot Trial Of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan, And Low Dose Total Body IrradiationAccelerated Phase Chronic Myelogenous Leukemia;Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome;Adult Acute Lymphoblastic Leukemia in Remission;Adult Acute Myeloid Leukemia in Remission;Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities;Adult Acute Myeloid Leukemia With Del(5q);Adult Acute Myeloid Leukemia With Inv(16)(p13;q22);Adult Acute Myeloid Leukemia With t(15;17)(q22;q12);Adult Acute Myeloid Leukemia With t(16;16)(p13;Adult Acute Myeloid Leukemia With t(8;21)(q22;Adult Nasal Type Extranodal NK/T-cell Lymphoma;Anaplastic Large Cell Lymphoma;Angioimmunoblastic T-cell Lymphoma;Aplastic Anemia;Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Chronic Myelogenous Leukemia;Childhood Diffuse Large Cell Lymphoma;Childhood Immunoblastic Large Cell Lymphoma;Childhood Myelodysplastic Syndromes;Childhood Nasal Type Extranodal NK/T-cell Lymphoma;Chronic Eosinophilic Leukemia;Chronic Myelomonocytic Leukemia;Chronic Neutrophilic Leukemia;Chronic Phase Chronic Myelogenous Leukemia;de Novo Myelodysplastic Syndromes;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Fanconi Anemia;Juvenile Myelomonocytic Leukemia;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Nodal Marginal Zone B-cell Lymphoma;Noncontiguous Stage II Adult Burkitt Lymphoma;Noncontiguous Stage II Adult Diffuse Large Cell Lymphoma;Noncontiguous Stage II Adult Diffuse Mixed Cell Lymphoma;Noncontiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma;Noncontiguous Stage II Adult Immunoblastic Large Cell Lymphoma;Noncontiguous Stage II Adult Lymphoblastic Lymphoma;Noncontiguous Stage II Grade 1 Follicular Lymphoma;Noncontiguous Stage II Grade 2 Follicular Lymphoma;Noncontiguous Stage II Grade 3 Follicular Lymphoma;Noncontiguous Stage II Mantle Cell Lymphoma;Noncontiguous Stage II Marginal Zone Lymphoma;Noncontiguous Stage II Small Lymphocytic Lymphoma;Paroxysmal Nocturnal Hemoglobinuria;Previously Treated Myelodysplastic Syndromes;Primary Myelofibrosis;Recurrent Adult Acute Lymphoblastic Leukemia;Recurrent Adult Acute Myeloid Leukemia;Recurrent Adult Burkitt Lymphoma;Recurrent Adult Diffuse Large Cell Lymphoma;Recurrent Adult Diffuse Mixed Cell Lymphoma;Recurrent Adult Diffuse Small Cleaved Cell Lymphoma;Recurrent Adult Grade III Lymphomatoid Granulomatosis;Recurrent Adult Hodgkin Lymphoma;Recurrent Adult Immunoblastic Large Cell Lymphoma;Recurrent Adult Lymphoblastic Lymphoma;Recurrent Adult T-cell Leukemia/Lymphoma;Recurrent Childhood Acute Lymphoblastic Leukemia;Recurrent Childhood Acute Myeloid Leukemia;Recurrent Childhood Anaplastic Large Cell Lymphoma;Recurrent Childhood Grade III Lymphomatoid Granulomatosis;Recurrent Childhood Large Cell Lymphoma;Recurrent Childhood Lymphoblastic Lymphoma;Recurrent Childhood Small Noncleaved Cell Lymphoma;Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma;Recurrent Grade 1 Follicular Lymphoma;Recurrent Grade 2 Follicular Lymphoma;Recurrent Grade 3 Follicular Lymphoma;Recurrent Mantle Cell Lymphoma;Recurrent Marginal Zone Lymphoma;Recurrent Mycosis Fungoides/Sezary Syndrome;Recurrent Small Lymphocytic Lymphoma;Recurrent/Refractory Childhood Hodgkin Lymphoma;Refractory Chronic Lymphocytic Leukemia;Refractory Multiple Myeloma;Relapsing Chronic Myelogenous Leukemia;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndromes;Splenic Marginal Zone Lymphoma;Stage III Adult Diffuse Small Cleaved Cell Lymphoma;Stage III Adult Immunoblastic Large Cell Lymphoma;Stage III Adult Lymphoblastic Lymphoma;Stage III Grade 1 Follicular Lymphoma;Stage III Grade 2 Follicular Lymphoma;Stage III Grade 3 Follicular Lymphoma;Stage III Mantle Cell Lymphoma;Stage III Marginal Zone Lymphoma;Stage III Small Lymphocytic Lymphoma;Stage IV Adult Burkitt Lymphoma;Stage IV Adult Diffuse Small Cleaved Cell Lymphoma;Stage IV Adult Immunoblastic Large Cell Lymphoma;Stage IV Adult Lymphoblastic Lymphoma;Stage IV Grade 1 Follicular Lymphoma;Stage IV Grade 2 Follicular Lymphoma;Stage IV Grade 3 Follicular Lymphoma;Stage IV Mantle Cell Lymphoma;Stage IV Marginal Zone Lymphoma;Stage IV Small Lymphocytic Lymphoma;Waldenström MacroglobulinemiaDrug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiation;Procedure: allogeneic hematopoietic stem cell transplantation;Biological: anti-thymocyte globulinRoswell Park Cancer InstituteNational Cancer Institute (NCI)Completed3 Years75 YearsAll62N/AUnited States
162NCT01194804
(ClinicalTrials.gov)
April 200830/8/2010E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria PatientsPhase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients: Extension Study to C07-001 ProtocolParoxysmal Nocturnal HemoglobinuriaDrug: EculizumabAlexion PharmaceuticalsCMIC Co, Ltd. JapanCompleted12 YearsN/AAll27Phase 2NULL
163NCT01192399
(ClinicalTrials.gov)
November 200730/8/2010Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria PatientsPhase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) PatientsParoxysmal Nocturnal HemoglobinuriaBiological: EculizumabAlexion PharmaceuticalsNULLCompleted12 YearsN/AAll29Phase 2NULL
164NCT00438789
(ClinicalTrials.gov)
February 200720/2/2007The Paroxysmal Nocturnal Hemoglobinuria Early Access Treatment ProtocolHemoglobinuria, ParoxysmalDrug: eculizumabAlexion PharmaceuticalsNULLApproved for marketing18 YearsN/ABothPhase 3United States
165EUCTR2005-000043-28-ES
(EUCTR)
07/04/200621/10/2005A Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 Studies. - E05 ExtensionA Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 Studies. - E05 Extension paroxysmal nocturnal hemoglobinuria
MedDRA version: 7.0;Level: PT;Classification code 10034042
Product Name: eculizumab
INN or Proposed INN: eculizumab
Other descriptive name: Anti-C5 antibody
ALEXION PHARMACEUTICALS INCORPORATEDNULLNot RecruitingFemale: yes
Male: yes
170Phase 3Germany;United Kingdom;Ireland;Spain;Italy;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
166NCT00397813
(ClinicalTrials.gov)
January 20069/11/2006Fludarabine Phosphate and Total Body Irradiation Followed by a Donor Peripheral Stem Cell Transplant in Treating Patients With Myelodysplastic Syndromes or Myeloproliferative DisordersLow-Dose TBI Dose Escalation to Decrease Risks of Progression and Graft Rejection After Hematopoietic Cell Transplantation With Nonmyeloablative Conditioning as Treatment for Untreated Myelodysplastic Syndrome or Myeloproliferative Disorders - A Multi-Center TrialAtypical Chronic Myeloid Leukemia, BCR-ABL1 Negative;Chronic Myelomonocytic Leukemia;de Novo Myelodysplastic Syndrome;Essential Thrombocythemia;Myeloproliferative Neoplasm;Paroxysmal Nocturnal Hemoglobinuria;Polycythemia Vera;Polycythemia Vera, Post-Polycythemic Myelofibrosis Phase;Primary Myelofibrosis;Refractory Anemia;Refractory Anemia With Excess Blasts;Refractory Anemia With Ring Sideroblasts;Refractory Cytopenia With Multilineage Dysplasia;Refractory Cytopenia With Multilineage Dysplasia and Ring SideroblastsDrug: Cyclosporine;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Procedure: Peripheral Blood Stem Cell Transplantation;Radiation: Total-Body IrradiationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI)Completed50 Years75 YearsAll77Phase 2United States;Denmark;Germany;Italy
167EUCTR2005-000043-28-IT
(EUCTR)
01/09/200506/02/2007A Phase III Open label extension study of Eculizumab in patients with transfusion dependent hemolytic paroxysmal nocturnal hemoglobinuria PNH who have participated in Triumph C04-001 , Shepherd C04-002 or X03-001 studiesA Phase III Open label extension study of Eculizumab in patients with transfusion dependent hemolytic paroxysmal nocturnal hemoglobinuria PNH who have participated in Triumph C04-001 , Shepherd C04-002 or X03-001 studies paroxymal nocturnal hemoglobinuria
MedDRA version: 6.1;Level: PT;Classification code 10034042
Product Name: Eculizumab
Product Code: h5G1.1-mAb
Product Name: Eculizumab
Product Code: h5G1.1-mAb
ALEXION PHARMACEUTICALS INCNULLNot RecruitingFemale: yes
Male: yes
170Phase 3Germany;United Kingdom;Spain;Ireland;Italy;Sweden
168NCT00130000
(ClinicalTrials.gov)
August 9, 200511/8/2005Eculizumab to Treat Paroxysmal Nocturnal HemoglobinuriaSHEPHERD: Safety in Hemolytic PNH Patients Treated With Eculizumab: A Multi-Center Open-Label Research DesignParoxysmal Hemoglobinuria, NocturnalDrug: EculizumabNational Heart, Lung, and Blood Institute (NHLBI)NULLCompleted18 YearsN/AAll87Phase 3United States
169NCT00143559
(ClinicalTrials.gov)
August 20051/9/2005Stem Cell Transplantation as Immunotherapy for Hematologic MalignanciesHaploidentical Hematopoietic Stem Cell Transplantation Utilizing Partial T-Cell Depletion as Immunotherapy for Hematologic MalignanciesLeukemia;Acute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Chronic Myeloid Leukemia;Juvenile Myelomonocytic Leukemia;Myelodysplastic Syndrome;Paroxysmal Nocturnal Hemoglobinuria;Hodgkin's Lymphoma;Non-Hodgkin LymphomaDrug: Systematic chemotherapy and antibodies;Procedure: Allogeneic stem cell transplantation;Device: Miltenyi CliniMACSSt. Jude Children's Research HospitalNULLCompleted2 Years21 YearsBoth17Phase 2United States
170EUCTR2005-000043-28-DE
(EUCTR)
06/07/200508/04/2005A Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 Studies. - E05-001 ExtensionA Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 Studies. - E05-001 Extension paroxysmal nocturnal hemoglobinuria
MedDRA version: 7.0;Level: PT;Classification code 10034042
MedDRA version: 9.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria
Product Name: eculizumab
Product Code: L04AA25
INN or Proposed INN: eculizumab
Other descriptive name: Anti-C5 antibody
ALEXION PHARMACEUTICALS INCORPORATEDNULLNot RecruitingFemale: yes
Male: yes
170Phase 3United Kingdom;Germany;Spain;Ireland;Italy;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
171EUCTR2005-000043-28-IE
(EUCTR)
27/05/200506/04/2005A Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 Studies. - E05 ExtensionA Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 Studies. - E05 Extension paroxysmal nocturnal hemoglobinuria
MedDRA version: 7.0;Level: PT;Classification code 10034042
Product Name: eculizumab
INN or Proposed INN: eculizumab
Other descriptive name: Anti-C5 antibody
ALEXION PHARMACEUTICALS INCORPORATEDNULLNot RecruitingFemale: yes
Male: yes
170Phase 3Spain;Ireland;Germany;Italy;United Kingdom;Sweden
172EUCTR2005-000043-28-SE
(EUCTR)
24/05/200507/04/2005A Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 Studies. - E05-001 ExtensionA Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 Studies. - E05-001 Extension paroxysmal nocturnal hemoglobinuria
MedDRA version: 7.0;Level: PT;Classification code 10034042
MedDRA version: 9.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria
Product Name: eculizumab
INN or Proposed INN: eculizumab
Other descriptive name: Anti-C5 antibody
ALEXION PHARMACEUTICALS INCORPORATEDNULLNot RecruitingFemale: yes
Male: yes
170Phase 3Germany;United Kingdom;Spain;Ireland;Italy;Sweden
173EUCTR2004-002795-42-ES
(EUCTR)
24/05/200522/02/2006Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERDSafety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERD paroxysmal nocturnal hemoglobinuria
MedDRA version: 7.0;Level: PT;Classification code 10034042
Product Name: eculizumab
INN or Proposed INN: eculizumab
Other descriptive name: Anti-C5 antibody
ALEXION PHARMACEUTICALS INCORPORATEDNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
85United Kingdom;Germany;Ireland;Spain;Italy;Sweden
174NCT00122317
(ClinicalTrials.gov)
May 200520/7/2005Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)Open Label Extension Study of Eculizumab in Patients With Transfusion Dependent PNHParoxysmal Hemoglobinuria, NocturnalDrug: eculizumabAlexion PharmaceuticalsNULLCompleted18 YearsN/AAll187Phase 3United States;Australia;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Spain;Sweden;United Kingdom
175EUCTR2004-002795-42-IT
(EUCTR)
29/04/200506/07/2005SHEPHERD: Safety in hemolytic PNH patients treated with Eculizumab: a multi-center open label research design studySHEPHERD: Safety in hemolytic PNH patients treated with Eculizumab: a multi-center open label research design study paroxysmal nocturnal hemoglobinuria
MedDRA version: 6.1;Level: PT;Classification code 10034042
Product Name: EculizumabALEXION PHARMACEUTICALS INCNULLNot RecruitingFemale: yes
Male: yes
85United Kingdom;Germany;Spain;Ireland;Italy;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
176EUCTR2004-002795-42-GB
(EUCTR)
20/04/200523/02/2005Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERDSafety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERD paroxysmal nocturnal hemoglobinuria
MedDRA version: 7.0;Level: PT;Classification code 10034042
Product Name: eculizumab
INN or Proposed INN: eculizumab
Other descriptive name: Anti-C5 antibody
ALEXION PHARMACEUTICALS INCORPORATEDNULLNot RecruitingFemale: yes
Male: yes
85Phase 3Spain;Ireland;Germany;Italy;United Kingdom;Sweden
177EUCTR2005-000043-28-GB
(EUCTR)
19/04/200516/03/2005A Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 Studies. - E05 ExtensionA Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 Studies. - E05 Extension paroxysmal nocturnal hemoglobinuria
MedDRA version: 7.0;Level: PT;Classification code 10034042
ALEXION PHARMACEUTICALS INCORPORATEDNULLNot Recruiting Female: yes
Male: yes
170Phase 3Spain;Ireland;Germany;Italy;United Kingdom;Sweden
178EUCTR2004-002795-42-DE
(EUCTR)
24/03/200523/12/2004Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERDSafety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERD paroxysmal nocturnal hemoglobinuria
MedDRA version: 7.0;Level: PT;Classification code 10034042
Product Name: eculizumab
INN or Proposed INN: eculizumab
Other descriptive name: Anti-C5 antibody
ALEXION PHARMACEUTICALS INCORPORATEDNULLNot RecruitingFemale: yes
Male: yes
85United Kingdom;Germany;Spain;Ireland;Italy;Sweden
179EUCTR2004-002795-42-SE
(EUCTR)
16/02/200509/12/2004Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERDSafety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERD paroxysmal nocturnal hemoglobinuria
MedDRA version: 7.0;Level: PT;Classification code 10034042
Product Name: eculizumab
INN or Proposed INN: eculizumab
Other descriptive name: Anti-C5 antibody
ALEXION PHARMACEUTICALS INCORPORATEDNULLNot RecruitingFemale: yes
Male: yes
85United Kingdom;Germany;Spain;Ireland;Italy;Sweden
180EUCTR2004-002795-42-IE
(EUCTR)
03/02/200509/12/2004Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERDSafety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERD paroxysmal nocturnal hemoglobinuria
MedDRA version: 7.0;Level: PT;Classification code 10034042
Product Name: eculizumab
INN or Proposed INN: eculizumab
Other descriptive name: Anti-C5 antibody
ALEXION PHARMACEUTICALS INCORPORATEDNULLNot RecruitingFemale: yes
Male: yes
85Phase 3Spain;Ireland;Germany;Italy;United Kingdom;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
181EUCTR2004-000646-20-GB
(EUCTR)
02/12/200414/02/2005 A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients - TRIUMPH A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients - TRIUMPH paroxysmal nocturnal hemoglobinuria
MedDRA version: 7.0;Level: PT;Classification code 10034042
ALEXION PHARMACEUTICALS INCORPORATEDNULLNot Recruiting Female: yes
Male: yes
75Phase 3Ireland;Italy;United Kingdom;Sweden
182NCT00122304
(ClinicalTrials.gov)
December 200419/7/2005Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With EculizumabSafety in Hemolytic PNH Patients Treated With Eculizumab: a Multi-Center Open-Label Research Design StudyHemoglobinuria, ParoxysmalDrug: eculizumabAlexion PharmaceuticalsNULLCompleted18 YearsN/ABoth85Phase 3United States;Australia;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Spain;Sweden;Switzerland;United Kingdom
183NCT00098280
(ClinicalTrials.gov)
December 20043/12/2004Eculizumab to Treat Paroxysmal Nocturnal HemoglobinuriaA Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria PatientsHemoglobinuria, ParoxysmalDrug: EculizumabNational Heart, Lung, and Blood Institute (NHLBI)NULLCompletedN/AN/ABoth75Phase 3United States
184EUCTR2004-000646-20-IT
(EUCTR)
01/11/200423/12/2005TRIUMPH A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria PatientsTRIUMPH A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients paaroxysmal nocturnal hemoglobinuria
MedDRA version: 6.1;Level: PT;Classification code 10034042
Product Name: Eculizumab
INN or Proposed INN: VARI
ALEXION PHARMACEUTICALS INCNULLNot RecruitingFemale: yes
Male: yes
75Ireland;Italy;Sweden
185NCT00112983
(ClinicalTrials.gov)
November 20042/6/2005Eculizumab in Treating Patients With Paroxysmal Nocturnal HemoglobinuriaTRIUMPH: A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria PatientsLeukemiaBiological: eculizumabJonsson Comprehensive Cancer CenterNULLCompleted18 YearsN/ABothPhase 3United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
186EUCTR2004-000646-20-IE
(EUCTR)
26/10/200416/08/2004A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients - TRIUMPHA Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients - TRIUMPH paroxysmal nocturnal hemoglobinuria
MedDRA version: 7.0;Level: PT;Classification code 10034042
Product Name: eculizumab
INN or Proposed INN: eculizumab
Other descriptive name: Anti-C5 antibody
ALEXION PHARMACEUTICALS INCORPORATEDNULLNot RecruitingFemale: yes
Male: yes
75Phase 3Ireland;Italy;United Kingdom;Sweden
187NCT00122330
(ClinicalTrials.gov)
October 200418/7/2005Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) PatientsRandomized, Double-Blind, and Placebo-Controlled Study Using Eculizumab in Transfusion Dependent PNH PatientsHemoglobinuria, ParoxysmalDrug: eculizumabAlexion PharmaceuticalsNULLCompleted18 YearsN/ABoth75Phase 3United States;Australia;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Sweden;Switzerland;United Kingdom
188EUCTR2004-000646-20-SE
(EUCTR)
21/09/200419/07/2004A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients - TRIUMPHA Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients - TRIUMPH paroxysmal nocturnal hemoglobinuria
MedDRA version: 7.0;Level: PT;Classification code 10034042
Product Name: eculizumab
INN or Proposed INN: eculizumab
Other descriptive name: Anti-C5 antibody
ALEXION PHARMACEUTICALS INCORPORATEDNULLNot RecruitingFemale: yes
Male: yes
75Ireland;Italy;Sweden
189NCT00145613
(ClinicalTrials.gov)
June 20031/9/2005Haploidentical Stem Cell Transplant for Treatment Refractory Hematological MalignanciesHaploidentical Stem Cell Transplantation Utilizing T-Cell Depletion as Therapy for Patients With Refractory Hematological MalignanciesAcute Lymphoblastic Leukemia (ALL);Acute Myeloid Leukemia (AML);Secondary AML;Myelodysplastic Syndrome (MDS);Secondary MDS;Chronic Myeloid Leukemia;Juvenile Myelomonocytic Leukemia (JMML);Paroxysmal Nocturnal Hemoglobinuria (PNH);Lymphoma, Non-Hodgkin;Hodgkin DiseaseProcedure: Stem Cell Transplantation;Device: Miltenyi Biotec CliniMACS;Drug: Systemic chemotherapy and antibodiesSt. Jude Children's Research HospitalNULLCompleted2 Years21 YearsBoth25Phase 2United States
190NCT00587054
(ClinicalTrials.gov)
June 200121/12/2007Trial of Allogeneic Stem Cell Transplants From HLA Compatible, Related and Unrelated Donors After a Myeloablative Preparative Regimen With Hyperfractionated TBI, Thiotepa and Fludarabine For Adult Patients With Lymphohematopoietic DisordersPhase II Trial of Allogeneic T-Cell Depleted Hematopoietic Stem Cell Transplants From HLA Compatible, Related and Unrelated Donors After a Myeloablative Preparative Regimen With Hyperfractionated TBI, Thiotepa and Fludarabine For Treatment of Adult Patients (>18 Years) With Lymphohematopoietic DisordersAllogeneic Stem Cell Transplant;Leukemia;Non-Hodgkins;Lymphoblastic Lymphoma;Myelodysplastic Syndrome;Paroxysmal Nocturnal Hemoglobinuria (PNH)Drug: cytoreductive regimen followed by a CD34+E- selected allogeneic stem cell transplantMemorial Sloan Kettering Cancer CenterNational Cancer Institute (NCI)Completed18 Years55 YearsAll129Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
191NCT00012376
(ClinicalTrials.gov)
March 20013/3/2001Chemotherapy Plus Sargramostim in Treating Patients With Refractory Myeloid CancerDose Finding Study of Bryostatin-1 and GM-CSF in Refractory Myeloid MalignanciesAccelerated Phase Chronic Myelogenous Leukemia;Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities;Adult Acute Myeloid Leukemia With Del(5q);Adult Acute Myeloid Leukemia With Inv(16)(p13;q22);Adult Acute Myeloid Leukemia With t(15;17)(q22;q12);Adult Acute Myeloid Leukemia With t(16;16)(p13;Adult Acute Myeloid Leukemia With t(8;21)(q22;Blastic Phase Chronic Myelogenous Leukemia;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia;Paroxysmal Nocturnal Hemoglobinuria;Previously Treated Myelodysplastic Syndromes;Recurrent Adult Acute Myeloid Leukemia;Refractory Anemia;Refractory Anemia With Ringed Sideroblasts;Relapsing Chronic Myelogenous Leukemia;Thrombocytopenia;Untreated Adult Acute Myeloid LeukemiaDrug: bryostatin 1;Biological: sargramostim;Other: laboratory biomarker analysis;Other: pharmacological studyNational Cancer Institute (NCI)NULLCompleted18 YearsN/ABoth35Phase 1United States
192NCT00004143
(ClinicalTrials.gov)
September 199910/12/1999Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure SyndromesAllogeneic Mixed Chimerism Stem Cell Transplantation Utilizing In Vivo and In Vitro Campath for Hemoglobinopathies and Bone Marrow Failure SyndromesSickle Cell Anemia;Severe Aplastic Anemia;Paroxysmal Nocturnal Hemoglobinuria (PNH);Pure Red Cell AplasiaDrug: Campath, Chemo and/or TBI Allo SCTDavid Rizzieri, MDNULLCompleted18 YearsN/AAll2Phase 2United States
193NCT00004464
(ClinicalTrials.gov)
February 199618/10/1999Study of High Dose Cyclophosphamide in Patients With Severe Aplastic Anemia and Paroxysmal Nocturnal HemoglobinuriaHigh Dose Cyclophosphamide for the Treatment of Severe Aplastic Anemia and Paroxysmal Nocturnal HemoglobinuriaAplastic Anemia;Paroxysmal Hemoglobinuria, NocturnalDrug: cyclophosphamide;Drug: filgrastimSidney Kimmel Comprehensive Cancer Center at Johns HopkinsNULLCompletedN/A70 YearsAll69Phase 2United States
194EUCTR2019-004931-21-DE
(EUCTR)
14/07/2020A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not previously Treated with Complement InhibitorsA PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) NOT PREVIOUSLY TREATED WITHCOMPLEMENT INHIBITORS Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: Crovalimab
Product Code: RO7112689
INN or Proposed INN: CROVALIMAB
Trade Name: Soliris
Product Name: Eculizumab
INN or Proposed INN: ECULIZUMAB
F. Hoffman-La Roche Ltd.NULLNAFemale: yes
Male: yes
200Phase 3United States;Portugal;Taiwan;Hong Kong;Greece;Thailand;Spain;Israel;Colombia;Italy;France;Malaysia;Australia;Peru;South Africa;Netherlands;China;Korea, Republic of;Turkey;United Kingdom;Hungary;Czech Republic;Mexico;Argentina;Brazil;Belgium;Poland;Germany;Japan;Sweden
195EUCTR2020-000597-26-SE
(EUCTR)
02/06/2020A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated with Complement Inhibitors.A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) CURRENTLY TREATED WITH COMPLEMENT INHIBITORS PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Crovalimab
Product Code: RO7112689
INN or Proposed INN: CROVALIMAB
Trade Name: Eculizumab
Product Name: Eculizumab
INN or Proposed INN: ECULIZUMAB
F.Hoffmann-La Roche LtdNULLNAFemale: yes
Male: yes
250Phase 3Portugal;United States;Hong Kong;Taiwan;Greece;Spain;Thailand;Israel;Colombia;Italy;France;Australia;Netherlands;Korea, Republic of;Turkey;United Kingdom;Hungary;Czech Republic;Mexico;Brazil;Belgium;Poland;Germany;Japan;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
196EUCTR2020-001350-21-CZ
(EUCTR)
08/12/2020A single arm study to evaluate the safety and biological activity of pegcetacoplan in patients 12- 17 years old with paroxysmal nocturnal hemoglobinuriaAn Open-Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients with Paroxysmal Nocturnal Hemoglobinuria Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: pegcetacoplan
Product Code: pegcetacoplan
INN or Proposed INN: Pegcetacoplan
Other descriptive name: pegcetacoplan
Apellis Pharmaceuticals, Inc.NULLNAFemale: yes
Male: yes
12Phase 2United States;Serbia;Czechia;Philippines;Thailand;Spain;Ireland;United Kingdom;Italy;France;Czech Republic;Hungary;Malaysia;Poland;Romania;Australia;Germany
197EUCTR2020-000597-26-PL
(EUCTR)
16/09/2020A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated with Complement Inhibitors.A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) CURRENTLY TREATED WITH COMPLEMENT INHIBITORS PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Crovalimab
Product Code: RO7112689
INN or Proposed INN: CROVALIMAB
Trade Name: Eculizumab
Product Name: Eculizumab
INN or Proposed INN: ECULIZUMAB
F.Hoffmann-La Roche LtdNULLNAFemale: yes
Male: yes
250Phase 3Portugal;United States;Hong Kong;Taiwan;Greece;Spain;Thailand;Israel;Colombia;Italy;France;Australia;Netherlands;Korea, Republic of;Turkey;United Kingdom;Hungary;Czech Republic;Mexico;Brazil;Belgium;Poland;Germany;Japan;Sweden
198EUCTR2019-001106-23-DE
(EUCTR)
30/09/2019An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH).An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: APL-2
Product Code: APL-2
INN or Proposed INN: pegcetacoplan
Other descriptive name: APL-2
Apellis Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
160Phase 3Serbia;United States;Hong Kong;Spain;Thailand;Russian Federation;United Kingdom;France;Canada;Malaysia;Australia;Bulgaria;Germany;Japan;Korea, Republic of
199EUCTR2017-002820-26-FR
(EUCTR)
12/01/2018A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH)A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 20.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: ALXN1210
INN or Proposed INN: ALXN1210
Other descriptive name: ALXN1210
Alexion Pharmaceuticals IncorporatedNULLNAFemale: yes
Male: yes
12Phase 3United States;France;Netherlands
200EUCTR2009-010402-11-Outside-EU/EEA
(EUCTR)
30/04/2012Not ApplicableAN OPEN-LABEL MULTI-CENTER STUDY OF ECULIZUMAB IN CHILDREN AND ADOLESCENTS WITH A DIAGNOSIS OF PAROXYSMAL NOCTURNAL HEMOGLOBINURIA PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
MedDRA version: 14.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Body processes [G] - Immune system processes [G12]
Trade Name: SOLIRIS
INN or Proposed INN: ECULIZUMAB
Other descriptive name: Anti-C5 antibody
ALEXION PHARMACEUTICALS, INC.NULLNAFemale: yes
Male: yes
6United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
201EUCTR2019-004931-21-PL
(EUCTR)
18/09/2020A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not previously Treated with Complement InhibitorsA PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) NOT PREVIOUSLY TREATED WITHCOMPLEMENT INHIBITORS Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: Crovalimab
Product Code: RO7112689
INN or Proposed INN: CROVALIMAB
Trade Name: Soliris
Product Name: Eculizumab
INN or Proposed INN: ECULIZUMAB
F. Hoffman-La Roche Ltd.NULLNAFemale: yes
Male: yes
200Phase 3United States;Portugal;Taiwan;Hong Kong;Greece;Thailand;Spain;Israel;Colombia;Italy;France;Malaysia;Australia;Peru;South Africa;Netherlands;China;Korea, Republic of;Turkey;United Kingdom;Hungary;Czech Republic;Mexico;Argentina;Brazil;Belgium;Poland;Germany;Japan;Sweden
202EUCTR2020-000597-26-DE
(EUCTR)
14/07/2020A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated with Complement Inhibitors.A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) CURRENTLY TREATED WITH COMPLEMENT INHIBITORS PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Crovalimab
Product Code: RO7112689
INN or Proposed INN: CROVALIMAB
Trade Name: Eculizumab
Product Name: Eculizumab
INN or Proposed INN: ECULIZUMAB
F.Hoffmann-La Roche LtdNULLNAFemale: yes
Male: yes
250Phase 3Portugal;United States;Hong Kong;Taiwan;Greece;Spain;Thailand;Israel;Colombia;Italy;France;Australia;Netherlands;Korea, Republic of;Turkey;United Kingdom;Hungary;Czech Republic;Mexico;Brazil;Belgium;Poland;Germany;Japan;Sweden