Methylmalonic Acidemia Clinical Trials

22 clinical trials for Methylmalonic Acidemia across international registries, including 6 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• Clinical and Laboratory Study of Methylmalonic Acidemia — RECRUITING
• A Prospective Study of Pediatric Participants up to 16 Years of Age With Methylmalonic Acidemia (MMA) Due to Mutations in the MMUT Gene — RECRUITING
• An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705 — 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 GLOBAL, PHASE 1/2, OPEN-LABEL, DOSE OPTIMIZATION STUDY TO EVALUATE THE SAFETY, TOLERABILITY, PHARMACODYNAMICS, AND PHARMACOKINETICS OF MRNA-3705 IN PARTICIPANTS WITH ISOLATED METHYLMALONIC ACIDEMIA DUE TO METHYLMALONYL-COA MUTASE DEFICIENCY — RECRUITING · PHASE1
• A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia — TERMINATED · PHASE1
• Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy — ACTIVE_NOT_RECRUITING
• A Study to Evaluate the Effects and Safety of Hydroxocobalamin in Participants With Combined Methylmalonic Academia (cblC Type) — NOT_YET_RECRUITING · PHASE3
• MyRareDiet A Novel Diet Tracking Tool — NOT_YET_RECRUITING · NA
• A Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of SEL-302 in Pediatric Subjects With MMA — WITHDRAWN · PHASE1
• Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia — TERMINATED · PHASE1
• 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
• Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia — COMPLETED · PHASE2
• Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia — WITHDRAWN · PHASE1
• 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
• Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate — WITHDRAWN · PHASE2
• Combined Malonic and Methylmalonic Aciduria (CMAMMA): Gene Identification and Outcome Study — OTHER

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