🧪 UPDATED DAILY FROM CLINICALTRIALS.GOVSynced Apr 1

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

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Showing 4 of 4 recruiting trials for “congenital-erythropoietic-porphyria”

Phase 1RecruitingNCT07471516 ↗

Zoledronic Acid Treatment in Patients With Congenital Dyserythropoietic Anemia

👨‍⚕️ Xin Zhao, MD📍 1 site📅 Started Feb 2026View details ↗
Phase 2, PHASE3Enrolling by InvitationNCT05883748 ↗

HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP

👨‍⚕️ Will Savage, MD PhD, Disc Medicine📍 13 sites📅 Started Aug 2023View details ↗
RecruitingNCT07206095 ↗

Integrative Diagnosis for SCD and Other RADs

🏥 Hospital Universitari Vall d'Hebron Research Institute📍 9 sites📅 Started Nov 2020View details ↗
RecruitingNCT02964494 ↗

The Congenital Dyserythropoietic Anemia Registry (CDAR)

👨‍⚕️ Theodosia Kalfa, MD, PhD, Children's Hospital Medical Center, Cincinnati📍 1 site📅 Started Aug 2016View details ↗

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