Propionic Acidemia
18
7
8
5
Key Insights
Highlights
Success Rate
56% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 100/100
22.2%
4 terminated out of 18 trials
55.6%
-31.0% vs benchmark
6%
1 trials in Phase 3/4
20%
1 of 5 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 5 completed trials
Clinical Trials (18)
Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia
Early Check: Expanded Screening in Newborns
AAVrh10-PCCA Gene Therapy for Propionic Acidemia
A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study
A Retrospective Study to Characterize Participants With Propionic Acidemia
Review of Charts From Amish/Mennonite Variant PA Patients
Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
Baby Detect : Genomic Newborn Screening
An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
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
Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia
Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia
Anaplerotic Therapy in Propionic Acidemia
Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate