🧪 UPDATED DAILY FROM CLINICALTRIALS.GOVSynced Apr 1

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24+ recruiting trials across all rare diseases. Filter by phase, location, and condition.

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Showing 20 of 24 recruiting trials for “Paroxysmal nocturnal hemoglobinuria”

Phase 3RecruitingNCT07154745 ↗

A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently

👨‍⚕️ Clinical Trial Management, Regeneron Pharmaceuticals📍 5 sites📅 Started Mar 2026View details ↗
Phase 1, PHASE2RecruitingNCT07470762 ↗

Study of Safety and Efficacy of HS-10542 in Patients With Paroxysmal Nocturnal Hemoglobinuria

🏥 Jiangsu Hansoh Pharmaceutical Co., Ltd.📍 1 site📅 Started Feb 2026View details ↗
Phase 1, PHASE2RecruitingNCT07187401 ↗

A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor

👨‍⚕️ Clinical Trial Management, Regeneron Pharmaceuticals📍 7 sites📅 Started Feb 2026View details ↗
Phase 3RecruitingNCT06934967 ↗

Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients

👨‍⚕️ Novartis Pharmaceuticals, Novartis Pharmaceuticals📍 12 sites📅 Started Oct 2025View details ↗
Phase 3RecruitingNCT06449001 ↗

Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis

🏥 Alexion Pharmaceuticals, Inc.📍 4 sites📅 Started Aug 2025View details ↗
RecruitingNCT07036718 ↗

NIS PDC the Efficacy and Safety of Iptacopan in Adults in Routine Clinical Practice

👨‍⚕️ Novartis Pharmaceuticals, Novartis Pharmaceuticals📍 8 sites📅 Started Jul 2025View details ↗
RecruitingNCT06931691 ↗

A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China

🏥 Novartis Pharmaceuticals📍 9 sites📅 Started Jun 2025View details ↗
Phase 1RecruitingNCT07152288 ↗

Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic Impairment

🏥 Haisco Pharmaceutical Group Co., Ltd.📍 1 site📅 Started May 2025View details ↗
Phase 2RecruitingNCT06978699 ↗

A Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

🏥 S-INFINITY Pharmaceuticals Co., Ltd📍 1 site📅 Started Apr 2025View details ↗
Phase 2RecruitingNCT06745622 ↗

Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria

🏥 Haisco Pharmaceutical Group Co., Ltd.📍 1 site📅 Started Jan 2025View details ↗
RecruitingNCT06312644 ↗

Study of Ultomiris® (Ravulizumab) Safety in Pregnancy

👨‍⚕️ Sydney Williams, North American Coordinating Center (NACC)📍 8 sites📅 Started Dec 2024View details ↗
Phase 2, PHASE3RecruitingNCT06933914 ↗

Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria

🏥 Wuhan Createrna Science and Technology Co., Ltd📍 1 site📅 Started Nov 2024View details ↗
RecruitingNCT06606314 ↗

Specified Drug-use Surveillance of Fabhalta Capsules

👨‍⚕️ Novartis Pharmaceuticals, Novartis Pharmaceuticals📍 91 sites📅 Started Sep 2024View details ↗
Phase 3RecruitingNCT06932744 ↗

Study of Safety and Efficacy of MY008211A in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy

🏥 Wuhan Createrna Science and Technology Co., Ltd📍 2 sites📅 Started Aug 2024View details ↗
Phase 3RecruitingNCT06932471 ↗

Study of Safety and Efficacy of MY008211A in Patients With Residual Anemia Despite Anti-C5 Antibody Treatment

🏥 Wuhan Createrna Science and Technology Co., Ltd📍 1 site📅 Started Aug 2024View details ↗
RecruitingNCT06411626 ↗

Home Reported Outcomes in PNH

👨‍⚕️ Novartis Pharmaceuticals, Novartis Pharmaceuticals📍 1 site📅 Started Jun 2024View details ↗
RecruitingNCT06524726 ↗

The International PNH Interest Group PNH Registry

👨‍⚕️ Richard Kelly, MBChB PhD, International PNH Interest Group📍 1 site📅 Started May 2024View details ↗
Phase 2RecruitingNCT06298955 ↗

Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria

👨‍⚕️ William Pullman, Omeros Corporation📍 5 sites📅 Started Feb 2024View details ↗
Phase 1RecruitingNCT07256301 ↗

A Phase Ib, Multicenter, Open-Label Study of Multiple-Dose EA5 in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH)

👨‍⚕️ Fengkui Zhang, Dr., The Institute of Hematology & Blood Diseases Hospital (Chinese Academy of Medical Sciences)📍 2 sites📅 Started Jan 2024View details ↗
NARecruitingNCT05776472 ↗

A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

👨‍⚕️ Study Physician +46 08-697-20 00, medical.info@sobi.com, Swedish Orphan Biovitrum📍 20 sites📅 Started Jun 2023View details ↗
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