🧪 UPDATED DAILY FROM CLINICALTRIALS.GOVSynced Apr 1

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

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Showing 6 of 6 recruiting trials for “disorder-of-branched-chain-amino-acid-metabolism”

NAEnrolling by InvitationNCT06953505 ↗

At Home Ammonia Monitoring of Inborn Errors of Ammonia Metabolism

👨‍⚕️ Marylaura L Thomas, Ph.D., Sequitur Health Corp.📍 1 site📅 Started Sep 2025View details ↗
RecruitingNCT05040178 ↗

An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics

👨‍⚕️ William Ludlum, MD, Recordati Rare Diseases Inc📍 5 sites📅 Started Jun 2022View details ↗
Phase 1, PHASE2RecruitingNCT05130437 ↗

A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study

🏥 ModernaTX, Inc.📍 22 sites📅 Started Nov 2021View details ↗
Phase 1, PHASE2RecruitingNCT04159103 ↗

Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia

🏥 ModernaTX, Inc.📍 35 sites📅 Started Apr 2021View details ↗
RecruitingNCT04176523 ↗

Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach

👨‍⚕️ Sufin Yap, MD, Sheffield Children's NHS Trust📍 32 sites📅 Started Jan 2019View details ↗
RecruitingNCT02890342 ↗

Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia

👨‍⚕️ Charles P Venditti, M.D., National Human Genome Research Institute (NHGRI)📍 2 sites📅 Started Nov 2016View details ↗

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