Trials / Conditions / Methylmalonic Acidemia

Methylmalonic Acidemia

17 registered clinical trials studyying Methylmalonic Acidemia — 5 currently recruiting.

Status	Trial	Sponsor	Phase
Not Yet Recruiting	MyRareDiet A Novel Diet Tracking Tool NCT06664840	Oregon Health and Science University	N/A
Active Not Recruiting	Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy NCT05506254	Alexion Pharmaceuticals, Inc.	—
Recruiting	An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics NCT05040178	RECORDATI GROUP	—
Recruiting	An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previou NCT05295433	ModernaTX, Inc.	Phase 1 / Phase 2
Terminated	Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia NCT05438485	HemoShear Therapeutics	—
Active Not Recruiting	A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolate NCT04899310	ModernaTX, Inc.	Phase 1 / Phase 2
Terminated	Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia NCT04581785	LogicBio Therapeutics, Inc	Phase 1 / Phase 2
Terminated	A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Voluntee NCT04836494	CoA Therapeutics, Inc., a BridgeBio company	Phase 1
Terminated	Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia NCT04732429	HemoShear Therapeutics	Phase 2
Recruiting	Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach NCT04176523	RECORDATI GROUP	—
Completed	"The MaP Study": Mapping the Patient Journey in MMA and PA NCT03484767	ModernaTX, Inc.	—
Completed	Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia NCT02426775	King Abdullah International Medical Research Center	Phase 3
Completed	Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia NCT01599286	Mendel Tuchman	Phase 2
Terminated	Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia NCT01597440	Mendel Tuchman	Phase 2
Unknown	Combined Malonic and Methylmalonic Aciduria (CMAMMA): Gene Identification and Outcome Study NCT01289158	McGill University Health Centre/Research Institute of the McGill University Health Centre	—
Withdrawn	Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate NCT01341379	Children's Hospital of Philadelphia	Phase 2
Recruiting	Clinical and Laboratory Study of Methylmalonic Acidemia NCT00078078	National Human Genome Research Institute (NHGRI)	—