Inborn Errors of Metabolism
39
6
6
24
Key Insights
Highlights
Success Rate
96% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
2.6%
1 terminated out of 39 trials
96.0%
+9.5% vs benchmark
3%
1 trials in Phase 3/4
13%
3 of 24 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 24 completed trials
Clinical Trials (39)
Maternal Inborn Errors of Metabolism in Pregnancy: A Pregnancy Registry Protocol
Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT
Clinical and Laboratory Study of Methylmalonic Acidemia
Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC
Identification and Characterization of Novel Non-Coding Variants That Contribute to Genetic Disorders
Institutional Registry of Rare Diseases
Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases
North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2
ORCHARD- Optimising Home Assessment of Rural Patients
Orphan Drugs for Inherited Metabolic Diseases
Growth and Development of Children With Inborn Errors of Metabolism in Assiut Governorate
Targeted Interventions for Successful Transition and Transfer of Adolescents With Inborn Errors of Metabolism to Adult Services
Blood Spot and Urine Metabolomic Screening Applied to Rare Diseases
Characterization of Intestinal Microbiota in Children With Inborn Errors of Metabolism (IEM)
Evaluation of PKU Explore
Evaluation of PKU Start
Trial of Erythrocyte Encapsulated Thymidine Phosphorylase In Mitochondrial Neurogastrointestinal Encephalomyopathy
Induced Pluripotent Stem Cells for the Development of Novel Drug Therapies for Inborn Errors of Metabolism (iPSC-IEM)
Utilizing Augmented Artificial Intelligence for Aminoacidopathies
Biomarkers for Inborn Errors of Metabolism