Clinical trials: Hemoglobinuria, Paroxysmal
Paroxysmal Nocturnal Hemoglobinuria
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors
A study designed to evaluate the safety of crovalimab with eculizumab in participants with PNH currently treated with complement inhibitors. This study will enroll approximately 190 participants.
- Trial name
- COMMODORE 1 (NCT04432584)
- Testing
- Crovalimab, Eculizumab
- Where
- 80 sites in United States, Belgium and 22 more
- Volunteers
- 190 people
- Sponsor
- Hoffmann-La Roche
Paroxysmal Nocturnal Hemoglobinuria
A Safety and Immunogenicity Study in Long-term Treatment of Eculizumab (JSC "GENERIUM", Russian Federation)
A safety and immunogenicity study in long-term treatment of Eculizumab (JSC "GENERIUM", Russian Federation) in patients with paroxysmal nocturnal hemoglobinuria, who have been involved earlier in the clinical trials of Eculizumab…
- Trial name
- NAP (NCT04679103)
- Testing
- Eculizumab (JSC GENERIUM, Russia)
- Where
- 9 sites in Russia
- Volunteers
- 50 people
- Sponsor
- AO GENERIUM
Paroxysmal Nocturnal Hemoglobinuria
A Study to Compare SB12 (Proposed Eculizumab Biosimilar) to Soliris in Subjects With Paroxysmal Nocturnal Haemoglobinuria
This is a randomised Phase III, double-blind, multicentre, cross-over study to compare the efficacy, safety, pharmacokinetics, and immunogenicity between SB12 and Soliris® in subjects with PNH.
- Trial name
- NCT04058158
- Testing
- SB12 (proposed eculizumab biosimilar), Soliris (eculizumab)
- Where
- 27 sites in India, Malaysia and 6 more
- Volunteers
- 50 people
- Sponsor
- Samsung Bioepis Co., Ltd.
Paroxysmal Nocturnal Hemoglobinuria
Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
The primary objective of the study is: To evaluate the effect of pozelimab and cemdisiran combination therapy on hemolysis, as assessed by lactate dehydrogenase (LDH), after 36 weeks of treatment, in patients with PNH who switch…
- Trial name
- ACCESS 2 (NCT05131204)
- Testing
- Cemdisiran, Eculizumab, Pozelimab
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Regeneron Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The purpose of this study is to evaluate the long-term safety and efficacy of ACH-0144471 in participants with paroxysmal nocturnal hemoglobinuria (PNH) who have demonstrated clinical benefit from ACH-0144471 in Study ACH471-100.
- Trial name
- NCT03181633
- Testing
- ACH-0144471
- Where
- 4 sites in Italy, New Zealand and 1 more
- Volunteers
- 8 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
PNH - Paroxysmal Nocturnal Hemoglobinuria
Study of SGB-9768 in Patients With Paroxysmal Nocturnal Hemoglobinuria
This is a multicenter, randomized, open-Label Phase II study to evaluate the efficacy and safety of multiple doses of SGB-9768 in adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
- Trial name
- NCT07796256
- Testing
- SGB-9768
- Where
- 2 sites in China
- Volunteers
- 24 people
- Sponsor
- Suzhou Sanegene Bio Inc.
Paroxysmal Nocturnal Hemoglobinuria
Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria®
This is prospective observational study of long-term pathogenic treatment of Elizaria® in patients with paroxysmal nocturnal hemoglobinuria.
- Trial name
- NAP (NCT04671810)
- Testing
- Elizaria®
- Where
- 8 sites in Russia
- Volunteers
- 44 people
- Sponsor
- AO GENERIUM
Paroxysmal Nocturnal Haemoglobinuria
Real-life Treatment Outcomes of Ravulizumab in PNH
The PNH-RECORD study, a Polish multicenter observational (non-interventional), open-label, retrospective with prospective follow-up.
- Trial name
- PNH-RECORD (NCT06633536)
- Testing
- Ravulizumab
- Where
- 11 sites in Poland
- Volunteers
- 64 people
- Sponsor
- AstraZeneca
Paroxysmal Nocturnal Hemoglobinuria, PNH
Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The primary objective of this study is to evaluate the efficacy of ravulizumab in adult participants with PNH.
- Trial name
- NCT06578949
- Testing
- Ravulizumab
- Where
- 8 sites in China
- Volunteers
- 18 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
IgA Nephropathy, Paroxysmal Nocturnal Hemoglobinuria
A Clinical Study to Evaluate the Effect of Food on the Pharmacokinetic Profiles of HRS-5965 Capsules in Healthy Subjects
The study is being conducted to evaluate the effect of food on pharmacokinetics of HRS-5965 capsules after oral administration.
- Trial name
- NCT07035665
- Testing
- HRS-5965 Capsules
- Where
- China
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Chengdu Suncadia Medicine Co., Ltd.
Paroxysmal Nocturnal Hemoglobinuria
A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria
The purpose of this study was to assess the pharmacokinetics (PK), pharmacodynamics (PD), safety, and efficacy of ravulizumab in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH).
- Trial name
- NCT03406507
- Testing
- Ravulizumab
- Where
- 9 sites in United States, France and 4 more
- Volunteers
- 13 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy
The study will evaluate the efficacy and safety of the oral Factor D (FD) inhibitor ALXN2050 (ACH-0145228) monotherapy in patients with PNH that are treatment naïve, or patients currently treated with eculizumab who still…
- Trial name
- NCT04170023
- Testing
- ALXN2050
- Where
- 11 sites in Canada, Italy and 5 more
- Volunteers
- 29 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
Effect of Food on Pharmacokinetics of MY008211A Tablets in Healthy Adult Subjects
This was a single-center, single-dose, open-label clinical study. 12 subjects were randomly assigned in a 1:1 ratio to one of the following dosing sequences (sequence 1: AB; Sequence 2: BA).
- Trial name
- NCT05828485
- Testing
- MY008211A tablets
- Where
- China
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Wuhan Createrna Science and Technology Co., Ltd
Paroxysmal Nocturnal Hemoglobinuria
Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
This study will collect clinical response data on participants who were already treated with eculizumab for at least 26 weeks and who started ravulizumab treatment as a specific therapeutic strategy as per ordinary clinical…
- Trial name
- NCT05274633
- Testing
- Ravulizumab
- Where
- 6 sites in Italy
- Volunteers
- 120 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The primary objective of the study is to demonstrate a reduction in intravascular hemolysis by REGN3918 over 26 weeks of treatment in patients with active PNH who are treatment-naive to complement inhibitor therapy or have not…
- Trial name
- NCT03946748
- Testing
- REGN3918
- Where
- 12 sites in Hong Kong, Hungary and 3 more
- Volunteers
- 24 people
- Sponsor
- Regeneron Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
Study of NM8074 in Adult PNH Patients With Inadequate Response to Soliris
This is a Phase II, open-label study designed to evaluate the safety, efficacy, and immunogenicity of NM8074 in PNH patients undergoing complement-inhibitor therapy with Soliris.
- Trial name
- NCT05646563
- Testing
- NM8074, Soliris
- Volunteers
- 12 people
- Sponsor
- NovelMed Therapeutics
Hemoglobinuria, Paroxysmal
The Paroxysmal Nocturnal Hemoglobinuria Early Access Treatment Protocol
The primary objective is to provide access to eculizumab for PNH patient pending commercial availability.
- Trial name
- EMBRACE (NCT00438789)
- Testing
- eculizumab
- Where
- United States
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This is a 36-month, long-term, multicenter, observational study designed to describe the real world effectiveness of pegcetacoplan in patients with PNH.
- Trial name
- COMPLETE (NCT05776472)
- Testing
- Pegcetacoplan
- Where
- 87 sites in Australia, Belgium and 13 more
- Volunteers
- 165 people
- Sponsor
- Swedish Orphan Biovitrum
PNH
A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered Pegcetacoplan (APL-2) in Subjects With PNH
This is a Phase IIa, open-label, multiple dose, study in patients with PNH who have not received eculizumab (Soliris ®) in the past. A single cohort of subjects is planned for evaluation.
- Trial name
- NCT03593200
- Testing
- Pegcetacoplan
- Where
- 3 sites in Bulgaria, Serbia
- Volunteers
- 4 people
- Sponsor
- Apellis Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria, PNH
To Evaluate Safety, Tolerability, PK/PD & Food Effect of Single/Multiple Oral TUL321 Capsules in Healthy Participants.
Phase I Study Evaluating Safety, Tolerability, PK and PD of Single and Multiple Doses of TUL321 Capsule in Healthy Participants and Food Effect on PK
- Trial name
- NCT07760467
- Testing
- Part1: TUL321, Part2: TUL321
- Where
- China
- Volunteers
- 94 people, healthy volunteers welcome
- Sponsor
- Zhuhai United Laboratories Co., Ltd.