Propionic Acidemia Clinical Trials

22 clinical trials for Propionic Acidemia across international registries, including 10 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia — RECRUITING
• A Global, Phase 1/2, Open-label, Dose Optimization Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants with Propionic Acidemia — RECRUITING · PHASE2
• AAVrh10-PCCA Gene Therapy for Propionic Acidemia — RECRUITING · PHASE1
• A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study — RECRUITING · PHASE1
• A Retrospective Study to Characterize Participants With Propionic Acidemia — RECRUITING
• Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia — RECRUITING · PHASE1
• An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics — RECRUITING
• Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach — RECRUITING
• A PHASE 1/2, GLOBAL, OPEN-LABEL, EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY AND CLINICAL ACTIVITY OF MRNA-3927 IN PARTICIPANTS PREVIOUSLY ENROLLED IN THE MRNA-3927-P101 STUDY — RECRUITING · PHASE1
• A GLOBAL, PHASE 1/2, OPEN-LABEL, DOSE OPTIMIZATION STUDY TO EVALUATE THE SAFETY, PHARMACODYNAMICS, AND PHARMACOKINETICS OF MRNA-3927 IN PARTICIPANTS WITH PROPIONIC ACIDEMIA — RECRUITING · PHASE1
• A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study — TERMINATED · PHASE2
• Early Check: Expanded Screening in Newborns — ACTIVE_NOT_RECRUITING
• Review of Charts From Amish/Mennonite Variant PA Patients — COMPLETED
• Baby Detect : Genomic Newborn Screening — COMPLETED
• Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia — TERMINATED · PHASE2
• Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia — TERMINATED
• A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia — TERMINATED · PHASE1
• "The MaP Study": Mapping the Patient Journey in MMA and PA — COMPLETED
• Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia — COMPLETED · PHASE3
• Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia — TERMINATED · PHASE2
• Anaplerotic Therapy in Propionic Acidemia — COMPLETED · PHASE1
• Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate — WITHDRAWN · PHASE2

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