Clinical trials: Paroxysmal Hemoglobinuria, Nocturnal
Paroxysmal Nocturnal Hemoglobinuria
Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy
The primary objective of the study is to evaluate the safety and tolerability of 2 dosing regimens of pozelimab and cemdisiran combination therapy during the open-label treatment period (OLTP) The secondary objectives of the…
- Trial name
- NCT04811716
- Testing
- Pozelimab, Cemdisiran
- Where
- 13 sites in Hong Kong, Hungary and 4 more
- Volunteers
- 24 people
- Sponsor
- Regeneron Pharmaceuticals
Leukemia, Acute Lymphoblastic Leukemia
Stem Cell Transplantation as Immunotherapy for Hematologic Malignancies
Blood and marrow stem cell transplant has improved the outcome for patients with high-risk hematologic malignancies. However, most patients do not have an appropriate HLA (immune type) matched sibling donor available and/or are…
- Conditions
- Leukemia, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Chronic Myeloid Leukemia
- Trial name
- NCT00143559
- Testing
- Systematic chemotherapy and antibodies, Allogeneic stem cell transplantation, Miltenyi CliniMACS
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- St. Jude Children's Research Hospital
PNH
Dose-Escalation Study of ALXN1210 IV in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This study evaluated the safety, tolerability, efficacy, pharmacokinetics, pharmacodynamics, and immunogenicity of multiple intravenous (IV) doses of ALXN1210 administered to participants with PNH who have not previously been…
- Trial name
- NCT02598583
- Testing
- ALXN1210
- Where
- 8 sites in Australia, South Korea
- Volunteers
- 13 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment
This study is researching a clinical treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH).
- Trial name
- ACCESS-1 (NCT05133531)
- Testing
- Ravulizumab, Pozelimab, Cemdisiran
- Where
- 68 sites in United States, Brazil and 22 more
- Volunteers
- 202 people
- Sponsor
- Regeneron Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate Its Long Term Safety, Efficacy and Tolerability.
The primary objective of the study is to evaluate the long-term safety, tolerability, and effect on intravascular hemolysis of REGN3918 in patients with paroxysmal nocturnal hemoglobinuria (PNH).
- Trial name
- NCT04162470
- Testing
- REGN3918
- Where
- 14 sites in Hong Kong, Hungary and 4 more
- Volunteers
- 24 people
- Sponsor
- Regeneron Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors
A study designed to evaluate the non-inferiority of crovalimab compared with eculizumab in participants with PNH who have not been previously treated with complement inhibitor therapy.
- Trial name
- COMMODORE 2 (NCT04434092)
- Testing
- Crovalimab, Eculizumab
- Where
- 63 sites in Argentina, Brazil and 21 more
- Volunteers
- 210 people
- Sponsor
- Hoffmann-La Roche
Paroxysmal Nocturnal Hemoglobinuria
A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH
Evaluation of the Efficacy and Safety of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria .
- Trial name
- NCT04085601
- Testing
- APL-2
- Where
- 30 sites in Colombia, Hong Kong and 8 more
- Volunteers
- 53 people
- Sponsor
- Apellis Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
This study evaluates iptacopan effectiveness and safety in routine clinical practice, with a focus on hematological response, transfusion avoidance, and patient-reported outcomes.
- Trial name
- REAL-CARE (NCT07229235)
- Where
- 12 sites in Italy
- Volunteers
- 50 people
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
A Study of Single-dose MY008211A in Healthy Adults
The trial is the first human trial. The safety, tolerability, PK and PD of MY008211A Tablets will be evaluated in healthy subjects.
- Trial name
- NCT05642585
- Testing
- MY008211A tablets
- Where
- China
- Volunteers
- 63 people, healthy volunteers welcome
- Sponsor
- Wuhan Createrna Science and Technology Co., Ltd
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.
Paroxysmal Hemoglobinuria, Nocturnal
Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria
This study will evaluate the safety and effectiveness of the experimental drug eculizumab for treating paroxysmal nocturnal hemoglobinuria (PNH), a disorder that can cause premature destruction of red blood cells.
- Trial name
- NCT00130000
- Testing
- Eculizumab
- Where
- United States
- Volunteers
- 87 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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，IgA Nephropathy
Comparison of HRS-5965 Capsules and Tablets in Healthy Subjects
The study is designed as a single-center, randomized, open-label, cross-over phase I clinical trial with the intention of recruiting 16 healthy subjects.
- Trial name
- NCT06490991
- Testing
- HRS-5965 tablets；HRS-5965 capsules
- Where
- China
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Chengdu Suncadia Medicine Co., Ltd.