Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Paroxysmal Nocturnal Hemoglobinuria: later-stage trial registry
5 of 15 indexed Paroxysmal Nocturnal Hemoglobinuria trials are currently recruiting, with more weight in Phase 3-4.
Corpus placement: #1,140 of 28,707 conditions by registered trial count.
15 US clinical trials · 5 currently recruiting · #1,140 of 28,707 conditions by registered trial count
Paroxysmal Nocturnal Hemoglobinuria: 15 US clinical trials tracked, 5 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.
Enrollment posture brief
Registry condition paroxysmal-nocturnal-hemoglobinuria
OPEN 5 | RECR 33% | LATE 7 | EARLY 1 | RANK #1140 | SPON 5
Volume-matched condition peers
Nearest open-share peer: Adherence, Treatment (33% recruiting · 6 trials)
Paroxysmal Nocturnal Hemoglobinuria lists 5 open slots (33% of 15 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Paroxysmal Nocturnal Hemoglobinuria on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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Paroxysmal Nocturnal Hemoglobinuria is recruiting below its size-band peers.
5 of 15 indexed trials (33.3%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
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One sponsor concentrates Paroxysmal Nocturnal Hemoglobinuria research.
Alexion Pharmaceuticals accounts for 5 of 15 trials (33.3%). 10 distinct sponsors appear on at least one study for this condition label.
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Typical Paroxysmal Nocturnal Hemoglobinuria enrolment sits around 80 participants.
Median target enrolment is 80 among the 15 trials that report a genuine target (sentinel values excluded). 8 of those (53.3%) plan for fewer than 100 participants.
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Paroxysmal Nocturnal Hemoglobinuria splits between interventional and observational designs.
9 interventional and 6 observational studies make up this index (60% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and 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
Named interventions
Most-linked intervention names on Paroxysmal Nocturnal Hemoglobinuria records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Paroxysmal Nocturnal Hemoglobinuria trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Paroxysmal Nocturnal Hemoglobinuria page
15 registered trials, 5 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 5 Paroxysmal Nocturnal Hemoglobinuria trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Alexion Pharmaceuticals sponsors the most Paroxysmal Nocturnal Hemoglobinuria trials on record, review their full trial history before evaluating a specific study. See Alexion Pharmaceuticals's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Paroxysmal Nocturnal Hemoglobinuria condition card totals 15 registered trials; most-linked intervention Danicopan; top US site state California. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.