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Paroxysmal Nocturnal Hemoglobinuria clinical trials
5 of 15 indexed Paroxysmal Nocturnal Hemoglobinuria trials are currently recruiting.
15 US clinical trials · 5 currently recruiting
Paroxysmal Nocturnal Hemoglobinuria is the subject of 15 registered US clinical trials on ClinicalTrials.gov, 5 of them currently open to new participants. 7 are in Phase 3-4 (later-stage) and 1 in Phase 1-2 (earlier-stage). The most active sponsor is Alexion Pharmaceuticals, running 5 of these trials.
The research picture
Paroxysmal Nocturnal Hemoglobinuria has 15 registered US clinical trials, 5 of them open to new participants right now, about 33% of the total.
- 5
- recruiting participants now
- 33%
- of trials open to enrollment
- 7
- in Phase 3–4 (later-stage)
- 5
- top sponsor: Alexion Pharmaceuticals
Active & Recent Trials
15 total, page 1 of 1
The International PNH Interest Group PNH Registry
International PNH Interest Group
NCT06524726
Global PNH Patient Registry
Aplastic Anemia and MDS International Foundation
NCT05755867
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
Regeneron Pharmaceuticals
NCT05133531
Home Reported Outcomes in PNH
Novartis Pharmaceuticals
NCT06411626
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.
Masonic Cancer Center, University of Minnesota
NCT06412497
Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
Novartis Pharmaceuticals
NCT04747613
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors
Hoffmann-La Roche
NCT04432584
A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH
Alexion Pharmaceuticals
NCT05389449
Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data
Alexion Pharmaceuticals
NCT07413250
Long-term Safety of Danicopan: IPIG Registry-based Cohort Study
Alexion Pharmaceuticals
NCT07413679
Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF
Children's Hospital of Philadelphia
NCT03047746
Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
Alexion Pharmaceuticals
NCT03748823
A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH
Amgen
NCT03818607
A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria
Alexion Pharmaceuticals
NCT03406507
Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease
Oregon Health and Science University
NCT01294891
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 2 | 1 |
| Phase 3 | 7 |
Top Sponsors
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Reading the Paroxysmal Nocturnal Hemoglobinuria Trial Landscape
15 indexed Paroxysmal Nocturnal Hemoglobinuria studies · 5 recruiting (33%).
Phase mix: 7 late (III/IV) vs 1 early (I/II).
Top sponsor: Alexion Pharmaceuticals (5 trials), 9 others listed. 15 shown below, recruiting first.
How to read this condition's trial landscape
That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.
Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.
All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation, eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.
Frequently Asked Questions
How many clinical trials are there for Paroxysmal Nocturnal Hemoglobinuria?
PlainTrial tracks 15 US clinical trials for Paroxysmal Nocturnal Hemoglobinuria, of which 5 are currently recruiting participants. Data sourced from ClinicalTrials.gov.
How do I find a recruiting trial for Paroxysmal Nocturnal Hemoglobinuria?
Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.
Is this data current?
Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.
Related
Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.
Read our methodology - how this data is sourced, computed, and verified.
Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2026 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial for this condition, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.