Paroxysmal Nocturnal Hemoglobinuria Clinical Trials

182 clinical trials for Paroxysmal Nocturnal Hemoglobinuria across international registries, including 38 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

Danicopan PMS in Korea — RECRUITING
Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients — RECRUITING · PHASE3
A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently — RECRUITING · PHASE3
An Open-Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients with Paroxysmal Nocturnal Hemoglobinuria — RECRUITING · PHASE2
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works — RECRUITING · PHASE3
A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor — RECRUITING · PHASE1
An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria — RECRUITING · PHASE3
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis — RECRUITING · PHASE2
A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) — RECRUITING
A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy — RECRUITING
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) — RECRUITING · PHASE2
REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria — RECRUITING
A Multicenter, Open-label Study Evaluating the Long-term Safety and Efficacy of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria — RECRUITING · NA
Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients — RECRUITING · PHASE1
MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure. — RECRUITING · PHASE2
A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China — RECRUITING
Study of Ultomiris® (Ravulizumab) Safety in Pregnancy — RECRUITING
Study of Safety and Efficacy of HS-10542 in Patients With Paroxysmal Nocturnal Hemoglobinuria — RECRUITING · PHASE1
An open-label, single-arm, multicenter, phase 3 study to assess pharmacokinetics, safety and tolerability of iptacopan in pediatric PNH patients aged 2 to <18 years of age — RECRUITING · PHASE3
Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis — RECRUITING · PHASE3
Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic Impairment — RECRUITING · PHASE1
Home Reported Outcomes in PNH — RECRUITING
A SINGLE ARM STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WITH INADEQUATE CONTROL OF INTRAVASCULAR HEMOLYSIS ON CURRENTLY AVAILABLE C5 INHIBITOR THERAPY — RECRUITING
A PHASE 3 OPEN-LABEL STUDY OF DANICOPAN AS ADD-ON TREATMENT TO RAVULIZUMAB OR ECULIZUMAB IN PEDIATRIC PARTICIPANTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO HAVE CLINICALLY SIGNIFICANT EXTRAVASCULAR HEMOLYSIS — RECRUITING · PHASE3
Study of Safety and Efficacy of MY008211A in Patients With Residual Anemia Despite Anti-C5 Antibody Treatment — RECRUITING · PHASE3
A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, FIRST-IN-HUMAN STUDY OF THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PRELIMINARY EFFICACY OF ALN-CFB, AN INVESTIGATIONAL SIRNA THERAPEUTIC AGAINST COMPLEMENT FACTOR B, IN PARTICIPANTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA AND PERSISTENT ANEMIA ON APPROVED C5-INHIBITOR THERAPY — RECRUITING
Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria — RECRUITING · PHASE2
Global PNH Patient Registry — RECRUITING
Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria — RECRUITING · PHASE2
The International PNH Interest Group PNH Registry — RECRUITING
LONG-TERM EXTENSION (LTE) STUDY TO CHARACTERIZE THE SAFETY AND EFFICACY OF DANICOPAN AS AN ADD-ON THERAPY TO A COMPLEMENT COMPONENT 5 INHIBITOR (C5I) IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) PREVIOUSLY TREATED WITH DANICOPAN IN AN ALEXION-SPONSORED CLINICAL STUDY — RECRUITING
AN OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY, TOLERABILITY, AND EFFICACY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA — RECRUITING
A RANDOMIZED, OPEN-LABEL, RAVULIZUMAB-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE COMPLEMENT INHIBITOR TREATMENT-NAIVE OR HAVE NOT RECENTLY RECEIVED COMPLEMENT INHIBITOR THERAPY — RECRUITING
AN 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) — RECRUITING
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 — RECRUITING · PHASE2
A MULTICENTER, OPEN-LABEL, UNCONTROLLED, EXTENSION STUDY OF RA101495 IN SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO HAVE COMPLETED A RA101495 CLINICAL STUDY — RECRUITING
A PHASE 3, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED STUDY OF ALXN1210 VERSUS ECULIZUMAB IN COMPLEMENT INHIBITOR-NAIVE ADULT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) — RECRUITING · PHASE3
"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 HEMOGLOBINURIA," — RECRUITING · PHASE2
Not Applicable — OTHER · PHASE2
Phase IIa study of APL-2 in patients with PNH — COMPLETED · PHASE2
Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (PNH) — TERMINATED · PHASE2
A Phase III, Randomized, Open-Label, Active controlled, Multicenter Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not Previously Treated with Complement Inhibitors — TERMINATED · PHASE3
Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria — ACTIVE_NOT_RECRUITING · PHASE3
A Study of the Efficacy and Safety of FXS6837 Capsules Compared to Eculizumab for 24 Weeks in Patients With PNH. — NOT_YET_RECRUITING · PHASE3
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors — ACTIVE_NOT_RECRUITING · PHASE3
NIS PDC the Efficacy and Safety of Iptacopan in Adults in Routine Clinical Practice — TERMINATED
Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria — ACTIVE_NOT_RECRUITING · PHASE2
A Phase 3 Open-Label Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Significant Extravascular Hemolysis — TERMINATED · PHASE3
A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH — ACTIVE_NOT_RECRUITING · PHASE3
Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF — ACTIVE_NOT_RECRUITING · NA

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