Methylmalonic Acidemia
17
4
5
3
Key Insights
Highlights
Success Rate
33% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 75/100
35.3%
6 terminated out of 17 trials
33.3%
-53.2% vs benchmark
6%
1 trials in Phase 3/4
100%
3 of 3 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 3 completed trials
Clinical Trials (17)
A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia
Clinical and Laboratory Study of Methylmalonic Acidemia
Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy
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
An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
MyRareDiet A Novel Diet Tracking Tool
Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia
Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia
Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia
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
"The MaP Study": Mapping the Patient Journey in MMA and PA
Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia
Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia
Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia
Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate
Combined Malonic and Methylmalonic Aciduria (CMAMMA): Gene Identification and Outcome Study