← Browse by Condition
Medical Condition

paroxysmal nocturnal hemoglobinuria

Total Trials
4
Recruiting Now
4
Trial Phases
Phase 2, Phase 3

ClinicalMetric tracks all active clinical trials for paroxysmal nocturnal hemoglobinuria sourced from ClinicalTrials.gov. Research in this area spans early-phase safety investigations through large Phase 3 confirmatory trials, conducted by NIH-funded academic centers, independent investigators, and pharmaceutical sponsors worldwide. Each listing is updated daily as new studies open enrollment and existing ones reach capacity or complete follow-up.

Trial listings include the NCT identifier, current recruitment status, phase classification, primary and secondary endpoints, estimated enrollment size, participating countries, and direct links to the full protocol on ClinicalTrials.gov. Eligibility criteria — including age range, disease stage, and prior treatment requirements — are documented for every study to help patients and clinicians assess suitability before contacting a trial site.

Frequently Asked Questions — paroxysmal nocturnal hemoglobinuria Clinical Trials

How many clinical trials are currently recruiting for paroxysmal nocturnal hemoglobinuria?
ClinicalMetric currently tracks 4 actively recruiting clinical trials for paroxysmal nocturnal hemoglobinuria, sourced in real time from ClinicalTrials.gov. The total number of registered studies—including those not yet enrolling or in active follow-up—is 4. Trial availability changes daily as new studies open enrollment and existing ones reach capacity.
What trial phases are available for paroxysmal nocturnal hemoglobinuria?
paroxysmal nocturnal hemoglobinuria research spans Phase 2 (1 trial), Phase 3 (1 trial). Phase 1 studies evaluate safety and dosing in small groups, Phase 2 studies assess preliminary efficacy in 100–300 participants, and Phase 3 trials compare the new treatment against the standard of care in 300–3,000+ patients. Phase 4 post-approval studies monitor long-term outcomes in real-world populations.
How do I find out if I qualify for a paroxysmal nocturnal hemoglobinuria clinical trial?
Eligibility criteria for paroxysmal nocturnal hemoglobinuria trials vary by study and typically specify age range, disease stage or severity, prior treatment history, and specific diagnostic or laboratory parameters. Each listing on ClinicalMetric links to the full protocol on ClinicalTrials.gov, where inclusion and exclusion criteria are documented. Contact the sponsoring site's research coordinator directly to confirm your eligibility—your treating physician or specialist can also help identify the most appropriate trial based on your medical history and current treatment status.
Trial Phases
Phase 2
1
Phase 3
1
Top Sponsors
AstraZeneca 1 trial
Masonic Cancer Center, University of Minnesota 1 trial
Novartis Pharmaceuticals 1 trial
Regeneron Pharmaceuticals 1 trial

Recruiting Clinical Trials

NCT07457151
Recruiting
Danicopan PMS in Korea
Enrollment
27 pts
Location
South Korea
Sponsor
AstraZeneca
View Trial →
NCT06412497 Phase 2
Recruiting
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.
Enrollment
60 pts
Location
United States
Sponsor
Masonic Cancer Center, Univers...
View Trial →
NCT06931691
Recruiting
A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China
Enrollment
80 pts
Location
China
Sponsor
Novartis Pharmaceuticals
View Trial →
NCT05744921 Phase 3
Recruiting
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 Works
Enrollment
202 pts
Location
Canada, Colombia, Hu...
Sponsor
Regeneron Pharmaceuticals
View Trial →

Related Conditions

severe aplastic anemia (1) acquired amegakaryocytic thrombocytopenia (1) acquired pure red cell aplasia (1)
ClinicalMetric — Independent clinical trial intelligence platform. Not affiliated with NIH, ClinicalTrials.gov, the U.S. FDA, or any pharmaceutical company, hospital, or clinical research organization. Trial data is sourced from ClinicalTrials.gov for informational purposes only and does not constitute medical advice. Do not make any treatment, enrollment, or health decisions based solely on information found here — always consult a qualified healthcare professional. Full Disclaimer  ·  Last Reviewed: September 2026  ·  Data Methodology