Early research (Phase 1)Looking for participantsNCT07643844What this trial is testingAAVrh10-PCCA Gene Therapy for Propionic AcidemiaWho this might be right forPropionic Acidemia Mayo Clinic 9
Testing effectiveness (Phase 2)WithdrawnNCT01341379What this trial is testingIncreasing Ureagenesis in Inborn Errors of Metabolism With N-CarbamylglutamateWho this might be right forUrea Cycle Disorders, InbornInborn Errors of MetabolismPropionic Acidemia+2 more Children's Hospital of Philadelphia
Early research (Phase 1)Ended earlyNCT04836494What this trial is testingA 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 AcidemiaWho this might be right forHealthy VolunteersPropionic AcidemiaMethylmalonic Acidemia+1 more CoA Therapeutics, Inc., a BridgeBio company 79
Not applicableLooking for participantsNCT04176523What this trial is testingUnderstanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods ApproachWho this might be right forMethylmalonic AcidemiaPropionic Acidemia RECORDATI GROUP 95
Not applicableLooking for participantsNCT01793168What this trial is testingRare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at SanfordWho this might be right forRare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more Sanford Health 20,000
Testing effectiveness (Phase 2)Ended earlyNCT01597440What this trial is testingLong-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic AcidemiaWho this might be right forPropionic AcidemiaMethylmalonic Acidemia Mendel Tuchman 1