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 62. 発作性夜間ヘモグロビン尿症 [臨床試験数:80,薬物数:68(DrugBank:26),標的遺伝子数:14,標的パスウェイ数:83] 

Searched query = "Paroxysmal nocturnal hemoglobinuria"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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Inclusion_
agemin
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Inclusion_
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PhaseCountries
1NCT04079257October 201916 September 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 UniversityNot recruiting18 YearsN/AAll60N/A
2NCT04085601August 14, 201930 September 2019A Phase III 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.Recruiting18 YearsN/AAll54Phase 3Malaysia;Poland
3NCT04058158August 7, 201914 October 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.Recruiting18 YearsN/AAll50Phase 3India;Korea, Republic of;Taiwan;Ukraine
4NCT04060264June 17, 20192 September 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: SolirisBiocadRecruiting18 Years65 YearsAll28Phase 3Russian Federation
5NCT03946748May 16, 201924 June 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 PharmaceuticalsRecruiting18 YearsN/AAll42Phase 2Korea, Republic of
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
Recruitment_
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Inclusion_
agemin
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PhaseCountries
6NCT03818607April 24, 20194 November 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: EculizumabAmgenRecruiting18 YearsN/AAll40Phase 3United States;France;Germany;Italy;Netherlands;Spain;United Kingdom
7NCT03896152April 5, 20194 November 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 PharmaceuticalsRecruiting18 YearsN/AAll10Phase 2Korea, Republic of;Malaysia;Russian Federation;Singapore;Taiwan
8NCT03866681April 1, 201918 March 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 HospitalNot recruiting18 Years70 YearsAll40Phase 4China
9EUCTR2017-002370-39-ES08/02/201928 February 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 EculizumabParoxysmal 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
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Current Sponsor code: ALXN1210
Other descriptive name: Ravulizumab
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 70-
Product Name: Ravulizumab
Product Code: ALXN1210
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Current Sponsor code: ALXN1210
Other descriptive name: Ravulizumab
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-
Alexion Pharmaceuticals, Inc.Authorised 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
10EUCTR2017-001418-27-NL22/01/201928 February 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 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 applicableParoxysmal 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 Name: ABP 959
Product Code: ABP 959
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: ECULIZUMAB
CAS Number: 219685-50-4
Current Sponsor code: ABP 959
Other descriptive name: ABP 959 - biosimilar to eculizumab
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 900-
Trade Name: Soliris
Product Name: Soliris
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: ECULIZUMAB
CAS Number: 219685-50-4
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 900-
Amgen Inc.Authorised Female: yes
Male: yes
40Phase 3Spain;Norway;Germany;Netherlands;Italy;United Kingdom;Korea, Republic of
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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Inclusion_
agemin
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size
PhaseCountries
11EUCTR2017-002370-39-FR28/12/201830 April 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 EculizumabParoxysmal 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
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Current Sponsor code: ALXN1210
Other descriptive name: Ravulizumab
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 70-
Product Name: Ravulizumab
Product Code: ALXN1210
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Current Sponsor code: ALXN1210
Other descriptive name: Ravulizumab
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-
Alexion Pharmaceuticals, Inc.Authorised 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
12EUCTR2017-004268-36-ES19/09/20181 October 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) - PEGASUSPNH 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
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: n/a
CAS Number: n/a
Current Sponsor code: APL-2
Other descriptive name: n/a
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 54-
Trade Name: Soliris
Product Name: Soliris
Pharmaceutical Form: Infusion
INN or Proposed INN: ECULIZUMAB
CAS Number: 219685-50-4
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-
Apellis Pharmaceuticals, Inc.AuthorisedFemale: yes
Male: yes
70Phase 3United States;Canada;Spain;Australia;Russian Federation;Japan;Korea, Republic of
13NCT03531255August 27, 201817 September 2018Multi-center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)A Phase III, 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)PNHDrug: APL-2Apellis Pharmaceuticals, Inc.Recruiting18 YearsN/AAll109Phase 3United States
14NCT03593200August 16, 20184 February 2019A 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.Not recruiting18 YearsN/AAll4Phase 2Bulgaria;Serbia;Greece;Poland;Romania
15EUCTR2017-005140-16-BG18/07/201830 April 2019Phase 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
Pharmaceutical Form: Solution for injection/infusion
INN or Proposed INN: Not yet assigned
CAS Number: 2019171-69-6
Current Sponsor code: APL-2
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 40-
Apellis PharmaceuticalsAuthorised Female: yes
Male: yes
20Phase 2Serbia;Greece;Poland;Romania;Bulgaria
No.TrialIDDate_
enrollement
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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PhaseCountries
16NCT03588026July 9, 20184 November 2019Treating 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 TherapeuticsRecruiting18 YearsN/AAll30Phase 3Kazakhstan;Lithuania;Sri Lanka
17NCT03500549June 14, 201827 May 2019Study to Evaluate the Efficacy and Safety of APL-2 in Patients With PNHA 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.Recruiting18 YearsN/AAll70Phase 3United States;Australia;Canada;Germany;Japan
18NCT03427060May 14, 201826 August 2019Coversin 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 TherapeuticsRecruiting18 YearsN/AAll6Phase 2United States;Argentina
19NCT03472885May 8, 20182 September 2019A 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: EculizumabAchillion PharmaceuticalsNot recruiting18 Years65 YearsAll12Phase 2United States
20EUCTR2017-002820-26-NL21/03/201828 February 2019A 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
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: ALXN1210
Other descriptive name: ALXN1210
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-
Alexion Pharmaceuticals IncorporatedAuthorisedFemale: yes
Male: yes
12Phase 3France;United States;Netherlands;United Kingdom
No.TrialIDDate_
enrollement
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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PhaseCountries
21EUCTR2017-003847-39-LT12/03/201826 March 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) - CAPSTONEParoxysmal Nocturnal Haemoglobinuria (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] - Immune System Diseases [C20]
Product Code: rVA576
Pharmaceutical Form: Powder for solution for injection
INN or Proposed INN: NA
Current Sponsor code: rVA576
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 18-
Akari Therapeutics Plc.AuthorisedFemale: yes
Male: yes
30Phase 3Ecuador;Romania;Turkey;Lithuania;Peru;Kazakhstan;Sri Lanka
22NCT03406507February 22, 201828 October 2019A 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 PharmaceuticalsRecruitingN/A18 YearsAll12Phase 3United States;France;Netherlands;Norway;Russian Federation;United Kingdom
23EUCTR2017-000888-33-DE05/02/201810 October 2018A 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 hemolysisParoxysmal 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
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: not yet established
Current Sponsor code: LNP023
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
Novartis Pharma AGAuthorisedFemale: yes
Male: yes
10Phase 2France;Germany;Italy
24EUCTR2017-000888-33-FR15/01/201828 February 2019A 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 hemolysisParoxysmal 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
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: not yet established
Current Sponsor code: LNP023
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
Novartis Pharma AGAuthorised Female: yes
Male: yes
10Phase 2France;Germany;Italy
25NCT03333486December 7, 201725 June 2018Fludarabine 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
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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agemin
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PhaseCountries
26EUCTR2017-001418-27-ES28/08/201716 October 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 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 applicableParoxysmal Nocturnal Hemoglobinuria (PNH);Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]Product Name: ABP 959
Product Code: ABP 959
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: ECULIZUMAB
CAS Number: 219685-50-4
Current Sponsor code: ABP 959
Other descriptive name: ABP 959 - biosimilar to eculizumab
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 900-
Trade Name: Soliris
Product Name: Soliris
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: ECULIZUMAB
CAS Number: 219685-50-4
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 900-
Amgen Inc.AuthorisedFemale: yes
Male: yes
40Phase 3France;Spain;Russian Federation;Netherlands;Germany;Japan;Italy;United Kingdom;Korea, Republic of
27NCT03225287July 17, 201715 July 2019Extension 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 PharmaceuticalsNot recruiting18 YearsN/AAll28Phase 2United States;Australia;Denmark;Finland;Germany;Hungary;New Zealand;United Kingdom
28NCT03181633June 22, 20172 September 2019A 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-0144471Achillion PharmaceuticalsNot recruiting18 YearsN/AAll8Phase 2New Zealand
29EUCTR2016-003522-16-FI20/06/20177 May 2018Study 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 HEMOGLOBINURIAParoxysmal 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
Pharmaceutical Form: Solution for injection
INN or Proposed INN: RA101495 sodium
CAS Number: 1841136-73-9
Current Sponsor code: RA101495
Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 40-
Ra Pharmaceuticals, Inc.Not RecruitingFemale: yes
Male: yes
20Phase 2Hungary;Canada;Finland;Denmark;Australia;Germany;New Zealand;United Kingdom
30NCT03056040June 5, 20171 April 2019ALXN1210 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 PharmaceuticalsNot recruiting18 YearsN/AAll195Phase 3United States;Australia;Canada;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Spain;United Kingdom
No.TrialIDDate_
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31EUCTR2016-002026-36-GB30/05/201723 July 2018ALXN1210 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 EculizumabParoxysmal 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
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Current Sponsor code: ALXN1210
Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-
Trade Name: Soliris
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: ECULIZUMAB
CAS Number: 219685-50-4
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-
Alexion Pharmaceuticals IncorporatedAuthorisedFemale: yes
Male: yes
192Phase 3United States;Spain;Netherlands;Germany;United Kingdom
32NCT03030183April 17, 201715 July 2019Phase 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 PharmaceuticalsNot recruiting18 YearsN/AAll3Phase 2United States
33EUCTR2016-003523-34-FI06/04/20179 October 2017Extension 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 StudyParoxysmal 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
Pharmaceutical Form: Solution for injection
INN or Proposed INN: RA101495 sodium
CAS Number: 1841136-73-9
Current Sponsor code: RA101495
Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24 palmitoylated linker
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 40-
Ra Pharmaceuticals, Inc.AuthorisedFemale: yes
Male: yes
28Phase 2United States;Hungary;Canada;Finland;Denmark;Australia;Germany;New Zealand;United Kingdom
34NCT03053102March 31, 20172 September 2019A 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-0144471Achillion PharmaceuticalsNot recruiting18 YearsN/AAll10Phase 2Italy;Korea, Republic of;New Zealand;United Kingdom
35EUCTR2016-004129-18-GB17/03/20175 June 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 StudyParoxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS)
MedDRA version: 20.0 Level: PT Classification code 10018932 Term: Haemolytic uraemic syndrome System Organ Class: 10005329 - Blood and lymphatic system disorders
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]
Product Name: Coversin
Product Code: rVA576
Pharmaceutical Form: Powder for solution for injection
INN or Proposed INN: COVERSIN
Current Sponsor code: rVA576
Other descriptive name: COVERSIN
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 18-
Akari Therapeutics PlcAuthorisedFemale: yes
Male: yes
50Phase 3Argentina;Poland;Netherlands;United Kingdom
No.TrialIDDate_
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36NCT03829449March 13, 201711 March 2019rVA576 (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 TherapeuticsRecruiting18 YearsN/AAll50Phase 3Poland
37NCT03078582March 8, 201717 September 2018Phase 2 Safety and Efficacy Study of 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: RA101495Ra PharmaceuticalsNot recruiting18 YearsN/AAll26Phase 2Australia;Canada;Denmark;Finland;Germany;Hungary;New Zealand;United Kingdom
38EUCTR2016-002025-11-DE20/12/20163 April 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: 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
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Current Sponsor code: ALXN1210
Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-
Trade Name: Soliris
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: ECULIZUMAB
CAS Number: 219685-50-4
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-
Alexion Pharmaceuticals IncorporatedAuthorisedFemale: yes
Male: yes
214Phase 3Portugal;United States;Estonia;Poland;Spain;Austria;Denmark;Germany;United Kingdom;Korea, Republic of
39EUCTR2016-002943-40-ES21/11/20169 January 2017A 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 EculizumabParoxysmal 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
Pharmaceutical Form: Solution for injection
INN or Proposed INN: ALN-62643
Current Sponsor code: ALN-62643
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 200-
Alnylam Pharmaceuticals IncNot 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
40NCT03157635November 14, 201622 October 2019Study to Assess Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of RO7112689 in Healthy Volunteers and Participants 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 Hemoglobinuria, NocturnalDrug: RO7112689;Drug: PlaceboHoffmann-La RocheRecruiting18 Years75 YearsAll49Phase 1/Phase 2France;Germany;Hungary;Italy;Japan;Korea, Republic of;Netherlands
No.TrialIDDate_
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PhaseCountries
41EUCTR2016-002128-10-NL14/09/20162 October 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: 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
Pharmaceutical Form: Solution for injection/infusion
INN or Proposed INN: Not available
CAS Number: 1917321-26-6
Current Sponsor code: RO7112689
Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 170-
Pharmaceutical form of the placebo: Solution for injection/infusion
Route of administration of the placebo: Intravenous use
Pharmaceutical form of the placebo: Solution for injection/infusion
Route of administration of the placebo: Subcutaneous use
F. Hoffmann-La Roche LtdNot RecruitingFemale: no
Male: yes
74Phase 1;Phase 2France;Hungary;Czech Republic;Brazil;Germany;Netherlands;Japan;Italy;United Kingdom
42EUCTR2016-002067-33-GB18/07/201628 February 2019An Open Clincal Trial of Coversin in Patients with PNHCOBALT: Coversin Global Study: An Open-Label, Safety and Efficacy Trial in PNH PatientsParoxysmal 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]
Product Name: Coversin
Product Code: rVA576
Pharmaceutical Form: Powder for solution for injection
INN or Proposed INN: COVERSIN
Current Sponsor code: rVA576
Other descriptive name: COVERSIN
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 18-
Akari Therapeutics PlcNot Recruiting Female: yes
Male: yes
10Phase 2United Kingdom
43NCT02591862February 201618 June 2018Coversin 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 UniversityNot recruiting18 Years80 YearsAll6Phase 2Netherlands
44EUCTR2015-003778-34-NL13/01/20165 June 2018Coversin 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 EculizumabCoversin 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
Pharmaceutical Form: Solution for injection/infusion
INN or Proposed INN: Not applicable
Current Sponsor code: rVA576
Other descriptive name: coversin
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 7.2-
Volution Immuno Pharmaceuticals (UK) LtdNot RecruitingFemale: yes
Male: yes
6Phase 2Netherlands
45NCT02605993January 4, 20164 March 2019Open-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 PharmaceuticalsNot recruiting18 YearsN/AAll26Phase 2Canada;France;Germany;Korea, Republic of;Spain;Taiwan;United Kingdom;Sweden
No.TrialIDDate_
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46EUCTR2015-002674-20-DE04/12/201520 August 2018A 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 HemoglobinuriaParoxysmal 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
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: ALXN1210
Current Sponsor code: ALXN1210
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-
Product Code: ALXN1210
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: ALXN1210
Current Sponsor code: ALXN1210
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
Alexion Pharmaceuticals, Inc.AuthorisedFemale: yes
Male: yes
26Phase 2France;United States;Taiwan;Canada;Finland;Spain;Russian Federation;Germany;United Kingdom;Korea, Republic of;Sweden
47NCT02588833November 20154 February 2019Pilot 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.Not recruiting18 YearsN/AAll20Phase 1Hong Kong;Malaysia;New Zealand;Thailand
48NCT02598583November 201516 December 2017An Open-Label, Intrapatient, Dose-Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients With Paroxysmal Nocturnal HemoglobinuriaAn Open-Label, Intrapatient, Dose-Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients With Paroxysmal Nocturnal HemoglobinuriaParoxysmal Nocturnal Hemoglobinuria (PNH)Biological: Study Drug - ALXN1210Alexion PharmaceuticalsNot recruiting18 YearsN/AAll13Phase 1/Phase 2Australia;Korea, Republic of
49NCT02534909September 9, 201511 November 2019Proof 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 PharmaceuticalsNot recruiting18 YearsN/AAll10Phase 2Czechia;Japan;Lithuania;Czech Republic;Moldova, Republic of
50EUCTR2014-005338-74-CZ17/06/201514 January 2019To 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) - CLFG316X2201Paroxysmal 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]
Product Code: LFG316
Pharmaceutical Form: Lyophilisate for solution for infusion
INN or Proposed INN: No
Current Sponsor code: LFG316
Other descriptive name: human monoclonal antibody directed against complement 5 protein
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
Product Code: LFG316
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: No
Current Sponsor code: LFG316
Other descriptive name: human monoclonal antibody directed against complement 5 protein
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 50-
Novartis Pharma Services AGAuthorisedFemale: yes
Male: yes
15Phase 2Czech Republic;Lithuania;Japan
No.TrialIDDate_
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51NCT02352493January 201516 December 2017A 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 PharmaceuticalsNot recruiting18 Years45 YearsAll62Phase 1/Phase 2Spain;United Kingdom
52NCT02264639November 20149 July 2018A 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.Not recruiting18 YearsN/AAll15Phase 1United States
53NCT02179359September 2, 20147 October 2019Hematopoietic 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 MinnesotaRecruitingN/A55 YearsAll25N/AUnited States
54ChiCTR-TRC-140042192014-01-2418 April 2017A 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 hemoglobinuriairon 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 SciencesRecruiting1865Bothintravenous iron sucrose:24;iron dextran tablet:24;OtherChina
55NCT01760096January 201319 February 2015Safety 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 HospitalNot recruiting18 YearsN/ABoth50Phase 2China
No.TrialIDDate_
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56NCT01642979July 201219 February 2015Safety 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 HospitalNot recruiting18 YearsN/ABoth120Phase 2China
57NCT01529827February 28, 201230 September 2019Fludarabine 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 IrradiationPrimary 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 Syndrome;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;Accelerated 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 LeukemiaDrug: 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 InstituteNot recruiting3 Years75 YearsAll94Phase 2United States
58NCT01335165April 201119 February 2015Safety 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 PharmaceuticalsNot recruiting18 YearsN/ABoth10Phase 1United States;Czech Republic;Italy;Poland;United Kingdom
59NCT00867932May 200916 December 2017Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent PNH SubjectsAn Open Label Multi Center Study of Eculizumab in Children and Adolescents With Diagnosis of Paroxysmal Nocturnal HemoglobinuriaHemoglobinuria, ParoxysmalDrug: EculizumabAlexion PharmaceuticalsNot recruiting2 Years17 YearsAll7Phase 4United States
60NCT00856388January 14, 200918 December 2018Fludarabine 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 MacroglobulinemiaProcedure: allogeneic hematopoietic stem cell transplantation;Biological: anti-thymocyte globulin;Drug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiationRoswell Park Cancer InstituteNational Cancer Institute (NCI)Not recruiting3 Years75 YearsAll62N/AUnited States
No.TrialIDDate_
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61EUCTR2009-010402-11-Outside-EU/EEA8 May 2012Not ApplicableAN OPEN-LABEL MULTI-CENTER STUDY OF ECULIZUMAB IN CHILDREN AND ADOLESCENTS WITH A DIAGNOSIS OF PAROXYSMAL NOCTURNAL HEMOGLOBINURIAPAROXYSMAL 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
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: ECULIZUMAB
CAS Number: 219685-50-4
Current Sponsor code: h5G1.1-mAb
Other descriptive name: Anti-C5 antibody
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-
ALEXION PHARMACEUTICALS, INC.Not AvailableFemale: yes
Male: yes
6United States
62NCT01194804April 200830 September 2019E07-001: 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. JapanNot recruiting12 YearsN/AAll27Phase 2
63NCT01192399November 200716 December 2017C07-001: Safety 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 PharmaceuticalsNot recruiting12 YearsN/AAll29Phase 2
64NCT00438789February 200719 February 2015The Paroxysmal Nocturnal Hemoglobinuria Early Access Treatment ProtocolHemoglobinuria, ParoxysmalDrug: eculizumabAlexion PharmaceuticalsNot recruiting18 YearsN/ABothPhase 3United States
65NCT00397813January 20063 June 2019Fludarabine 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)Not recruiting50 Years75 YearsAll77Phase 2United States;Denmark;Germany;Italy
No.TrialIDDate_
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66NCT00130000August 9, 200516 December 2017Eculizumab 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)Not recruiting18 YearsN/AAll87Phase 3United States
67NCT00143559August 200519 February 2015Stem 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 HospitalNot recruiting2 Years21 YearsBoth17Phase 2United States
68NCT00122317May 200516 December 2017Extension 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 PharmaceuticalsNot recruiting18 YearsN/AAll187Phase 3United States;Australia;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Spain;Sweden;United Kingdom
69EUCTR2005-000043-28-GB19/04/200518 April 2012A 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 Extensionparoxysmal nocturnal hemoglobinuria
MedDRA version: 7.0 Level: PT Classification code 10034042
Product Name: eculizumab
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: eculizumab
Current Sponsor code: h5G1.1-mAb
Other descriptive name: Anti-C5 antibody
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-
Pharmaceutical form of the placebo: Solution for infusion
Route of administration of the placebo: Intravenous use
ALEXION PHARMACEUTICALS INCORPORATEDAuthorisedFemale: yes
Male: yes
170Phase 3Germany;United Kingdom;Spain;Ireland;Italy;Sweden
70EUCTR2004-002795-42-SE16/02/20058 May 2012Safety 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 - SHEPHERDparoxysmal nocturnal hemoglobinuria
MedDRA version: 7.0 Level: PT Classification code 10034042
Product Name: eculizumab
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: eculizumab
Current Sponsor code: h5G1.1-mAb
Other descriptive name: Anti-C5 antibody
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-
ALEXION PHARMACEUTICALS INCORPORATEDNot RecruitingFemale: yes
Male: yes
85United Kingdom;Germany;Spain;Ireland;Italy;Sweden
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
71NCT00098280December 200419 February 2015Eculizumab 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)Not recruitingN/AN/ABoth75Phase 3United States
72NCT00122304December 200419 February 2015Study 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 PharmaceuticalsNot recruiting18 YearsN/ABoth85Phase 3United States;Australia;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Spain;Sweden;Switzerland;United Kingdom
73NCT00112983November 200419 February 2015Eculizumab 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 CenterNot recruiting18 YearsN/ABothPhase 3United States
74NCT00122330October 200419 February 2015Study 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 PharmaceuticalsNot recruiting18 YearsN/ABoth75Phase 3United States;Australia;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Sweden;Switzerland;United Kingdom
75EUCTR2004-000646-20-SE21/09/200419 March 2012A 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 - TRIUMPHparoxysmal nocturnal hemoglobinuria
MedDRA version: 7.0 Level: PT Classification code 10034042
Product Name: eculizumab
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: eculizumab
Current Sponsor code: h5G1.1-mAb
Other descriptive name: Anti-C5 antibody
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-
Pharmaceutical form of the placebo: Intravenous infusion
Route of administration of the placebo: Intravenous use
ALEXION PHARMACEUTICALS INCORPORATEDNot RecruitingFemale: yes
Male: yes
75Ireland;Italy;Sweden
No.TrialIDDate_
enrollement
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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agemax
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gender
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size
PhaseCountries
76NCT00145613June 200319 February 2015Haploidentical 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 HospitalNot recruiting2 Years21 YearsBoth25Phase 2United States
77NCT00587054June 200116 December 2017Trial 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)Not recruiting18 Years55 YearsAll129Phase 2United States
78NCT00012376March 200119 February 2015Chemotherapy 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)Not recruiting18 YearsN/ABoth35Phase 1United States
79NCT00004143September 199919 October 2017Allogeneic 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, MDNot recruiting18 YearsN/AAll2Phase 2United States
80NCT00004464February 199628 January 2019Study 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 HopkinsNot recruitingN/A70 YearsAll69Phase 2United States

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