Browse recruiting and upcoming clinical trials for paroxysmal nocturnal hemoglobinuria.
Patient-friendly, AI-assisted summaries based on official ClinicalTrials.gov ↗ records. Last updated: Oct 10, 2026
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ClinicalTrials.gov is the complete and most up-to-date source. Trials may be missing, and trial information may have changed since our last check. Always verify important information in the official registry.
Who can join AIAdults (≥18) with paroxysmal nocturnal hemoglobinuria (PNH) confirmed by flow cytometry and evidence of active disease (e.g., LDH ≥1.5×ULN or recent PNH symptoms).
What is being studied AITests safety, pharmacokinetics, and pharmacodynamics of multiple IV doses of EA5 in adults with PNH.
Who can join AIAdults (18+) with paroxysmal nocturnal hemoglobinuria (PNH) who completed a Phase 2 or 3 MY008211A study and were judged to have benefited.
What is being studied AITests long-term safety, tolerability, and effects of MY008211A tablets in PNH.
Who can join AIChildren aged 12–17 with paroxysmal nocturnal hemoglobinuria (PNH) and clinically significant extravascular hemolysis on ravulizumab or eculizumab.
What is being studied AITests danicopan added to ravulizumab or eculizumab for blood and symptom measures in pediatric PNH.
Who can join AIPeople aged 0–75 with severe aplastic anemia, paroxysmal nocturnal hemoglobinuria, acquired pure red cell aplasia, or acquired amegakaryocytic thrombocytopenia.
What is being studied AITests a reduced-intensity allogeneic HCT regimen that uses post-transplant cyclophosphamide.
Who can join AIAdults (18+) with paroxysmal nocturnal hemoglobinuria (PNH), including parent-study completers and people with C5 polymorphisms refractory to eculizumab/ravulizumab.
What is being studied AITests long-term safety and efficacy of pozelimab plus cemdisiran in PNH.
Who can join AIPeople age 4–80 with severe aplastic anemia, certain myelodysplastic syndromes, PNH, or pure red cell aplasia who are candidates for allogeneic stem-cell transplant.
What is being studied AITests a modified donor stem-cell transplant using CD34+ selected cells with reduced non-mobilized T-cells.
Who can join AIAdults (≥18) with paroxysmal nocturnal hemoglobinuria who completed prior NTQ5082 treatment or are in a control group receiving eculizumab.
What is being studied AITests long-term safety and effects of NTQ5082 capsules in people with PNH.
Who can join AIHealthy adult volunteers (this early-phase study is developing a treatment for paroxysmal nocturnal hemoglobinuria, but does not enroll people with PNH).
What is being studied AITests safety, tolerability, pharmacokinetics, pharmacodynamics and food effect of oral TUL321 capsules.
Who can join AIAdults 18–99 with Paroxysmal Nocturnal Hemoglobinuria (PNH) or C3 Glomerulopathy (C3G) who will be treated with iptacopan and received required vaccination before starting.
What is being studied AICollects safety and lab data on iptacopan use in people with PNH or C3G.
Who can join AIPeople diagnosed with certain hematological (blood) diseases or who received a bone marrow transplant at a participating hospital on or after January 1, 2020.
What is being studied AICollects clinical, treatment, outcome, and cost data for people with hematological diseases.
Trials that may include paroxysmal nocturnal hemoglobinuria (1)
ClinicalTrials.gov returned these when we searched for this condition, but we could not confirm from the registry records that they are intended for people with it. Check each trial’s eligibility criteria, and ask the study team if you are unsure.
Who can join AIHealthy males and females aged 18–55 years who can volunteer as healthy participants; study lists PNH and complement‑mediated kidney disease.
What is being studied AITests safety, tolerability, and PK/PD of single and multiple oral doses of CMS-D017.