Hemoglobinuria clinical trials
The registry lists 107 studies for Hemoglobinuria across more than 10 countries. 10 are recruiting or about to start. Per ClinicalTrials.gov, last updated 2026-06-24.
Status
- Completed
- 81
- Terminated
- 10
- Recruiting
- 7
- Status unknown
- 5
- Not yet recruiting
- 3
- Approved for marketing
- 1
Phase
- Phase 2
- 43
- Phase 3
- 30
- Phase 1
- 14
- Phase 4
- 3
Where
- United States
- 49
- United Kingdom
- 27
- Korea, Republic of
- 19
- France
- 17
- Italy
- 17
- China
- 15
Who runs them
Alexion Pharmaceuticals, Inc. (21) · Wuhan Createrna Science and Technology Co., Ltd (6) · BioCryst Pharmaceuticals (5) · Novartis Pharmaceuticals (5) · Apellis Pharmaceuticals, Inc. (5) · St. Jude Children's Research Hospital (5) · Regeneron Pharmaceuticals (4) · AKARI Therapeutics (4)
Studies
Recruiting first, then most recently updated. Showing 50 of 107 — search for more.
- A Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)RecruitingNCT06978699Phase 21 country
- Study of Safety and Efficacy of MY008211A in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor TherapyRecruitingNCT06932744Phase 31 country
- Study of Safety and Efficacy of MY008211A in Patients With Residual Anemia Despite Anti-C5 Antibody TreatmentRecruitingNCT06932471Phase 31 country
- Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal HemoglobinuriaRecruitingNCT06933914Phase 2/31 country
- Study of NM8074 in Soliris-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)Not yet recruitingNCT05731050Phase 2
- Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor TherapyNot yet recruitingNCT05646524Phase 2
- Global PNH Patient RegistryRecruitingNCT057558671 country
- Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal HemoglobinuriaRecruitingNCT06745622Phase 21 country
- To Evaluate the Effect of Single Oral Dose of MY008211A Tablets on QTc Interval in Healthy SubjectsNot yet recruitingNCT06543459Phase 11 country
- The International PNH Interest Group PNH RegistryRecruitingNCT065247261 country
- Paroxysmal Nocturnal Hemoglobinuria (PNH), Level of CD59 on Red and White Blood Cells in Bone Marrow Failure SyndromesCompletedNCT01192425
- An Extension Clinical Study of BCD-148 for the Treatment of Patients With PNH Previously Treated in Clinical Study No. BCD-148-2/NOCTURNCompletedNCT06987864Phase 31 country
- Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients With Paroxysmal Nocturnal HemoglobinuriaCompletedNCT02534909Phase 23 countries
- Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 PolymorphismsCompletedNCT03427060Phase 21 country
- Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)CompletedNCT04469465Phase 318 countries
- A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)TerminatedNCT04702568Phase 23 countries
- rVA576 (Coversin) Long Term Safety and Efficacy Surveillance StudyTerminatedNCT03829449Phase 31 country
- Treating Paroxysmal Nocturnal Haemoglobinuria Patients With rVA576CompletedNCT03588026Phase 33 countries
- Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal HemoglobinuriaTerminatedNCT05131204Phase 31 country
- Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab MonotherapyCompletedNCT04811716Phase 26 countries
- ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With EculizumabCompletedNCT03056040Phase 311 countries
- BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants With Inadequate Response to C5 Inhibitor TherapyTerminatedNCT05116774Phase 25 countries
- Hematopoietic Stem Cell Transplant for High Risk HemoglobinopathiesTerminatedNCT02179359Not applicable1 country
- Study on Mass Balance of [14C]HSK39297 in Healthy Chinese Adult Male SubjectsCompletedNCT06852092Phase 11 country
- Dose Escalation of BCX10013 in Participants with Paroxysmal Nocturnal Hemoglobinuria (PNH)CompletedNCT06100900Phase 12 countries
- BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants Not Receiving Other Complement Inhibitor TherapyTerminatedNCT05116787Phase 23 countries
- Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as MonotherapyTerminatedNCT04170023Phase 27 countries
- Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal HemoglobinuriaCompletedNCT06051357Phase 21 country
- Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody TreatmentCompletedNCT04558918Phase 312 countries
- Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor TherapyCompletedNCT04820530Phase 38 countries
- Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With EculizumabCompletedNCT03748823Phase 314 countries
- Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose EculizumabCompletedNCT04320602Phase 41 country
- Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal HemoglobinuriaCompletedNCT03896152Phase 24 countries
- Study of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)CompletedNCT03439839Phase 23 countries
- ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)CompletedNCT02946463Phase 326 countries
- A Study to Compare SB12 (Proposed Eculizumab Biosimilar) to Soliris in Subjects With Paroxysmal Nocturnal HaemoglobinuriaCompletedNCT04058158Phase 38 countries
- A Study to Test if SAR443809 is Tolerated and Safe When Taken as a Single Dose in Healthy AdultsCompletedNCT06326814Phase 11 country
- Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical StudyTerminatedNCT03225287Phase 28 countries
- Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria®CompletedNCT046718101 country
- A Study of Single-dose MY008211A in Healthy AdultsCompletedNCT05642585Phase 11 country
- A Study of Multiple Ascending Doses MY008211A in Healthy AdultsCompletedNCT05828472Phase 11 country
- Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH)CompletedNCT02591862Phase 21 country
- Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)CompletedNCT03946748Phase 25 countries
- REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate Its Long Term Safety, Efficacy and Tolerability.TerminatedNCT04162470Phase 36 countries
- A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNHCompletedNCT03818607Phase 314 countries
- Hematological Response in Patients With Paroxysmal Nocturnal Hemoglobinuria Treated With Anti-C5 Antibody: an External Control Arm Study for Iptacopan Use in Anti-C5 naïve PatientsCompletedNCT058424861 country
- A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal HemoglobinuriaCompletedNCT03406507Phase 36 countries
- A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)CompletedNCT03181633Phase 23 countries
- To Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of KP104CompletedNCT05490017Phase 11 country
- Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)CompletedNCT02605993Phase 27 countries