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"
12 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2018-003956-19-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
2NCT03225287
(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
3EUCTR2016-003522-16-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
4EUCTR2016-003523-34-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
5EUCTR2016-003522-16-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT03030183
(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
7EUCTR2016-003523-34-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
8EUCTR2016-003522-16-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
9EUCTR2016-003523-34-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
10NCT03078582
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2016-003523-34-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
12EUCTR2016-003522-16-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