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Results for “Paroxysmal Nocturnal Haemoglobinuria (PNH)”

184 trials

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Showing 20 of 184 results

Testing effectiveness (Phase 2)Study completedNCT03078582
What this trial is testing

Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients

Who this might be right for
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Ra Pharmaceuticals 26
Testing effectiveness (Phase 2)Study completedNCT04888507
What this trial is testing

Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy

Who this might be right for
Paroxysmal Nocturnal Hemoglobinuria
Regeneron Pharmaceuticals 6
Testing effectiveness (Phase 2)Study completedNCT03946748
What this trial is testing

Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Who this might be right for
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Regeneron Pharmaceuticals 24
Testing effectiveness (Phase 2)Study completedNCT01192399
What this trial is testing

Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients

Who this might be right for
Paroxysmal Nocturnal Hemoglobinuria
Alexion Pharmaceuticals, Inc. 29
Testing effectiveness (Phase 2)Study completedNCT03896152
What this trial is testing

Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria

Who this might be right for
Paroxysmal Nocturnal Hemoglobinuria
Novartis Pharmaceuticals 13
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07266155
What this trial is testing

Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Who this might be right for
Paroxysmal Nocturnal Hemoglobinuria
Longbio Pharma 30
Testing effectiveness (Phase 2)Ended earlyNCT05116787
What this trial is testing

BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants Not Receiving Other Complement Inhibitor Therapy

Who this might be right for
Paroxysmal Nocturnal Hemoglobinuria (PNH)
BioCryst Pharmaceuticals 12
Testing effectiveness (Phase 2)Study completedNCT02605993
What this trial is testing

Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Who this might be right for
Paroxysmal Nocturnal HemoglobinuriaPNH
Alexion Pharmaceuticals, Inc. 26
Large-scale testing (Phase 3)Study completedNCT03500549
What this trial is testing

Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Who this might be right for
Paroxysmal Nocturnal Hemoglobinuria
Apellis Pharmaceuticals, Inc. 80
Testing effectiveness (Phase 2)Study completedNCT00004143
What this trial is testing

Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes

Who this might be right for
Sickle Cell AnemiaSevere Aplastic AnemiaParoxysmal Nocturnal Hemoglobinuria (PNH)+1 more
David Rizzieri, MD 2
Large-scale testing (Phase 3)Looking for participantsNCT07154745
What this trial is testing

Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently

Who this might be right for
Paroxysmal Nocturnal Hemoglobinuria
Regeneron Pharmaceuticals 35
Large-scale testing (Phase 3)Study completedNCT04463056
What this trial is testing

Efficacy and Safety of Elizaria® vs. Soliris® in Patients With PNH

Who this might be right for
Paroxysmal Nocturnal HemoglobinuriaMarchiafava-Micheli SyndromeParoxysmal Hemoglobinuria
AO GENERIUM 32
Testing effectiveness (Phase 2)Ended earlyNCT04702568
What this trial is testing

A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Who this might be right for
Paroxysmal Nocturnal HemoglobinuriaPNH
BioCryst Pharmaceuticals 19
Not applicableStudy completedNCT01412047
What this trial is testing

Paroxysmal Nocturnal Hemoglobinuria Human Anti-Human Antibodies Study

Who this might be right for
Paroxysmal Nocturnal HemoglobinuriaPNH
Alexion Pharmaceuticals, Inc. 75
Not applicableEnrolling By InvitationNCT07102849
What this trial is testing

Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research Study

Who this might be right for
Bone Marrow Failure DisordersVEXAS SyndromeHemoglobinurea, Paroxysmal+2 more
National Heart, Lung, and Blood Institute (NHLBI) 1,400
Not applicableActive Not RecruitingNCT06633536
What this trial is testing

Real-life Treatment Outcomes of Ravulizumab in PNH

Who this might be right for
Paroxysmal Nocturnal Haemoglobinuria
AstraZeneca 64
Testing effectiveness (Phase 2)Study completedNCT03439839
What this trial is testing

Study of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Who this might be right for
Paroxysmal Nocturnal Hemoglobinuria (PNH) With Signs of Active Hemolysis
Novartis Pharmaceuticals 16
Not applicableUnknownNCT00315419
What this trial is testing

Identifying Characteristics of Bone Marrow Failure Syndromes

Who this might be right for
Bone Marrow Failure SyndromesAnemia, AplasticMyelodysplastic Syndromes+4 more
Office of Rare Diseases (ORD) 450
Testing effectiveness (Phase 2)Study completedNCT02598583
What this trial is testing

Dose-Escalation Study of ALXN1210 IV in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Who this might be right for
PNH
Alexion Pharmaceuticals, Inc. 13
Early research (Phase 1)Study completedNCT06326814
What this trial is testing

Test if SAR443809 is Tolerated and Safe When Taken as a Single Dose in Healthy Adults

Who this might be right for
Paroxysmal Nocturnal Hemoglobinuria
Sanofi 54
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