Hemoglobinuria, Paroxysmal clinical trials
The registry lists 191 studies for Hemoglobinuria, Paroxysmal across more than 10 countries. 49 are recruiting or about to start. 156 of them are mainly about Hemoglobinuria, Paroxysmal (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-10.
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Status
Who runs them
Alexion Pharmaceuticals, Inc. (32) · Novartis Pharmaceuticals (16) · Regeneron Pharmaceuticals (10) · Wuhan Createrna Science and Technology Co., Ltd (9) · BioCryst Pharmaceuticals (6) · Apellis Pharmaceuticals, Inc. (6) · National Heart, Lung, and Blood Institute (NHLBI) (6) · Chengdu Suncadia Medicine Co., Ltd. (6)
What is being tested here
Drugs under test in Hemoglobinuria, Paroxysmal studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Lanoracopan | 5 | 3 | 9 |
| Ruxoprubart | 3 | 0 | 3 |
| Cyclophosphamide | 2 | 0 | 6 |
| Pozelimab | 2 | 3 | 6 |
| Cemdisiran | 2 | 2 | 5 |
| HSK39297 | 2 | 1 | 4 |
| NTQ5082 | 2 | 2 | 3 |
| FXS6837 | 2 | 2 | 2 |
| Iptacopan | 1 | 5 | 7 |
| Danicopan | 1 | 3 | 6 |
Drug classes tested here Alkylating agents 2 open · Antimetabolites 6
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
The 10 nearest, phase 3 first
- Nov 2026A Phase III Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients · Phase 3 · Nanjing Chia-tai Tianqing Pharmaceutical
- Dec 2026Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria · Phase 3 · Wuhan Createrna Science and Technology Co., Ltd
- Apr 2027A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors · Phase 3 · Hoffmann-La Roche
- May 2027Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis · Phase 3 · Alexion Pharmaceuticals, Inc.
- Sep 2027A Multicenter, Randomized, Open-Label, Positive Controlled Phase III Study to Evaluate the Efficacy and Safety of VSA012 Injection in Participants With Paroxysmal Nocturnal Hemoglobinuria Who Are Naive to Complement Inhibitor Therapy · Phase 3 · Bisirna Therapeutics (Suzhou) Co., Ltd.
- Oct 2027Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria · Phase 3 · Novartis Pharmaceuticals
- Nov 2026A Phase I Clinical Trial to Evaluate CMS-D017 Following Single and Multiple Doses in Healthy Participants · Shenzhen Kangzhe Biotechnology Co., Ltd.
- Dec 2026Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria · Omeros Corporation
- Dec 2026Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic Data · Istituto Giannina Gaslini
- Feb 2027Phase Ib Clinical Study to Evaluate the Safety and Tolerability of VSA012 Injection in Paroxysmal Nocturnal Hemoglobinuria · Bisirna Therapeutics Pte. Ltd.
Who these studies take
Of the 30 Hemoglobinuria, Paroxysmal studies recruiting now, 14 (47%) set no upper age limit and 12 (40%) stop at 75 or younger. 12 (40%) take children under 18. 1 (3%) are for women only and 0 (0%) for men only; 4 (13%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 10 (33%) | 9 (30%) |
| 18 | 28 (93%) | 27 (90%) |
| 30 | 27 (90%) | 26 (87%) |
| 40 | 27 (90%) | 26 (87%) |
| 50 | 26 (87%) | 25 (83%) |
| 65 | 25 (83%) | 24 (80%) |
| 75 | 23 (77%) | 22 (73%) |
| 85 | 16 (53%) | 15 (50%) |
Across all 191 studies, recruiting or not: 32% stop at 75 or younger and 18% at 65; 25% take children; 1% are for women only and 1% for men only; 11% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
How long Hemoglobinuria, Paroxysmal studies take
Of 35 phase 3 interventional studies under Hemoglobinuria, Paroxysmal, half reached primary completion within 23 months of starting, counting those still running.
| Start to primary completion | Every sponsor | Industry | Non-industry |
|---|---|---|---|
| Phase 1 | 13 mo5–34 · 19 studies | — | — |
| Phase 2 | 24 mo12–80 · 34 studies | 16 mo11–25 · 26 studies | — |
| Phase 3 | 23 mo15–41 · 35 studies | 23 mo15–41 · 35 studies | — |
Each figure is the month by which half the studies had reached primary completion, with the range in which the middle half did. Studies still running count until their last registry update; studies that stopped early are left out. Phase 1 includes early phase 1; observational means prospective or not stated, as retrospective and cross-sectional studies date a window of data rather than a study. How these figures are worked out
What changed recently
From the registry's own updates since Oct 13, 2026.
Newly registered2
Studies
Recruiting first, then most recently updated. Showing 50 of 191. Browse all 191 → or search them.
- Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cellsRecruitingNCT01174108Phase 21 country
- 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 WorksRecruitingNCT05744921Phase 321 countries
- A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.Not yet recruitingNCT07914894Phase 31 country
- Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD ProphylaxisRecruitingNCT03520647Phase 21 country
- Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic DataRecruitingNCT078777271 country
- Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research StudyEnrolling by invitationNCT071028491 country
- Danicopan PMS in KoreaRecruitingNCT074571511 country
- A Multicenter, Open-label Study Evaluating the Long-term Safety and Efficacy of XH-S003 Capsules in Patients With Paroxysmal Nocturnal HemoglobinuriaEnrolling by invitationNCT07657052Not applicable1 country
- A Multicenter, Randomized, Open-Label, Positive Controlled Phase III Study to Evaluate the Efficacy and Safety of VSA012 Injection in Participants With Paroxysmal Nocturnal Hemoglobinuria Who Are Naive to Complement Inhibitor TherapyNot yet recruitingNCT07816237Phase 31 country
- Study of SGB-9768 in Patients With Paroxysmal Nocturnal HemoglobinuriaNot yet recruitingNCT07796256Phase 21 country
- To Evaluate Safety, Tolerability, PK/PD & Food Effect of Single/Multiple Oral TUL321 Capsules in Healthy Participants.RecruitingNCT07760467Phase 11 country
- Efficacy and Safety of Lanoracopan in the Treatment of PNHNot yet recruitingNCT07788105
- Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH PatientsRecruitingNCT06934967Phase 36 countries
- A Study of the Efficacy and Safety of FXS6837 Capsules Compared to Eculizumab for 24 Weeks in Patients With PNH.Not yet recruitingNCT07747090Phase 31 country
- A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working EfficientlyRecruitingNCT07154745Phase 37 countries
- 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 InhibitorRecruitingNCT07187401Phase 1/23 countries
- A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 GlomerulopathyRecruitingNCT074161621 country
- A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)RecruitingNCT04901936Phase 29 countries
- REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal HemoglobinuriaRecruitingNCT072292351 country
- Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) PatientsRecruitingNCT05876312Phase 1/22 countries
- Danicopan Early Access ProgramAvailableNCT059829381 country
- 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.RecruitingNCT06412497Phase 21 country
- A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in ChinaRecruitingNCT069316911 country
- A Phase Ib, Multicenter, Open-Label Study of Multiple-Dose EA5 in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH)RecruitingNCT07256301Phase 11 country
- Study of Ultomiris® (Ravulizumab) Safety in PregnancyRecruitingNCT063126447 countries
- Study of NM8074 in Adult PNH Patients With Inadequate Response to SolirisNot yet recruitingNCT05646563Phase 2
- Study of Safety and Efficacy of HS-10542 in Patients With Paroxysmal Nocturnal HemoglobinuriaRecruitingNCT07470762Phase 1/21 country
- A Phase I Clinical Trial to Evaluate CMS-D017 Following Single and Multiple Doses in Healthy ParticipantsRecruitingNCT07462780Phase 11 country
- National Longitudinal Cohort of Hematological DiseasesRecruitingNCT046451991 country
- SLN12140 in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in ChinaNot yet recruitingNCT07387302Phase 2
- Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular HemolysisRecruitingNCT06449001Phase 33 countries
- Managed Access Programs for LNP023, IptacopanAvailableNCT05222412
- Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)Not yet recruitingNCT07266155Phase 21 country
- A Phase III Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria PatientsNot yet recruitingNCT07177859Phase 31 country
- A Long-term Efficacy and Safety of NTQ5082 CapsulesNot yet recruitingNCT07177872Phase 31 country
- Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic ImpairmentRecruitingNCT07152288Phase 11 country
- Home Reported Outcomes in PNHRecruitingNCT064116261 country
- Hematological Disorders in EHPVO PatientsNot yet recruitingNCT07108023
- Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)Not yet recruitingNCT06134414Phase 2
- 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
- BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants With Inadequate Response to C5 Inhibitor TherapyTerminatedNCT05116774Phase 25 countries