62. Paroxysmal nocturnal hemoglobinuria
202 clinical trials,   120 drugs   (DrugBank: 25 drugs),   15 drug target genes,   90 drug target pathways

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"
8 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2019-001106-23-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
2EUCTR2019-001106-23-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
3EUCTR2019-001106-23-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
4EUCTR2018-004220-11-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
5EUCTR2017-004268-36-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT03531255
(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
7EUCTR2020-001350-21-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
8EUCTR2019-001106-23-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