NCT05330039
Characterization of Intestinal Microbiota in Children With Inborn Errors of Metabolism (IEM) No drug interventions Not Available Not Available completed NCT05778877
A Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of SEL-302 in Pediatric Subjects With MMA treatment 1 / 2 withdrawn NCT03810690
Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia No drug interventions treatment 1 / 2 withdrawn NCT04248062
Patient and Observer Reported Outcome Measurements in Inborn Errors of Metabolism No drug interventions Not Available Not Available completed NCT00078078
Clinical and Laboratory Study of Methylmalonic Acidemia No drug interventions Not Available Not Available recruiting NCT05769621
A Retrospective Study to Characterize Participants With Propionic Acidemia and Methylmalonic Acidemia No drug interventions Not Available Not Available active_not_recruiting NCT05295433
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 treatment 1 / 2 recruiting NCT05506254
Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy No drug interventions Not Available Not Available active_not_recruiting NCT05040178
An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics Not Available Not Available recruiting NCT04581785
Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia No drug interventions treatment 1 / 2 terminated NCT02426775
Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia treatment 3 completed NCT01289158
Combined Malonic and Methylmalonic Aciduria (CMAMMA): Gene Identification and Outcome Study No drug interventions Not Available Not Available unknown_status NCT04836494
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 treatment 1 terminated NCT04732429
Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia No drug interventions treatment 2 terminated NCT01597440
Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia treatment 2 terminated NCT04899310
A Study to Assess Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia treatment 1 / 2 recruiting NCT01341379
Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate treatment 2 withdrawn NCT05438485
Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia No drug interventions Not Available Not Available terminated NCT01599286
Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia treatment 2 completed NCT03484767
"The MaP Study": Mapping the Patient Journey in MMA and PA No drug interventions Not Available Not Available completed NCT04176523
Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach Not Available Not Available recruiting NCT05842837
A Non-Interventional Post-Authorization Study of Carbaglu for the Treatment of Hyperammonemia Due to MMA and PA Not Available Not Available enrolling_by_invitation