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ClinicalTrials.gov 5 recruiting now official registry

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
Phase 21Phase 37

Active & Recent Trials

15 total, page 1 of 1

RECRUITING 2,000 participants

The International PNH Interest Group PNH Registry

International PNH Interest Group

NCT06524726

RECRUITING 500 participants

Global PNH Patient Registry

Aplastic Anemia and MDS International Foundation

NCT05755867

RECRUITING Phase 3 190 participants

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

RECRUITING 128 participants

Home Reported Outcomes in PNH

Novartis Pharmaceuticals

NCT06411626

RECRUITING Phase 2 60 participants

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

ACTIVE NOT RECRUITING Phase 3 208 participants

Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria

Novartis Pharmaceuticals

NCT04747613

ACTIVE NOT RECRUITING Phase 3 190 participants

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

ACTIVE NOT RECRUITING Phase 3 80 participants

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

ACTIVE NOT RECRUITING 50 participants

Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data

Alexion Pharmaceuticals

NCT07413250

ACTIVE NOT RECRUITING 50 participants

Long-term Safety of Danicopan: IPIG Registry-based Cohort Study

Alexion Pharmaceuticals

NCT07413679

ACTIVE NOT RECRUITING NA 50 participants

Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF

Children's Hospital of Philadelphia

NCT03047746

COMPLETED Phase 3 139 participants

Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab

Alexion Pharmaceuticals

NCT03748823

COMPLETED Phase 3 42 participants

A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH

Amgen

NCT03818607

COMPLETED Phase 3 13 participants

A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria

Alexion Pharmaceuticals

NCT03406507

COMPLETED 6 participants

Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease

Oregon Health and Science University

NCT01294891

Phase Distribution

PhaseTrial count
Phase 2 1
Phase 3 7

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.

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.

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.

Verify with NIH →

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.