Metabolism, Inborn Errors
18
2
2
11
Key Insights
Highlights
Success Rate
92% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 27/100
5.6%
1 terminated out of 18 trials
91.7%
+5.2% vs benchmark
22%
4 trials in Phase 3/4
45%
5 of 11 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 11 completed trials
Clinical Trials (18)
COVID-19 and Hereditary Metabolic Diseases
A Phase 3 Study of NTLA-2001 in ATTRv-PN
Effect of Large Neutral Amino Acids in Adults With Classical Phenylketonuria
Cardiac Manifestation and Inherited Metabolic Diseases
Biomarker for Hurler Disease (BioHurler)
Genetic Etiology in Patients With Cerebral Palsy
Natural History Study of Children With Metachromatic Leukodystrophy
Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia
North Carolina Newborn Exome Sequencing for Universal Screening
Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy
Measure Liver Fat Content After ISIS 301012 (Mipomersen) Administration
Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia
Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia
An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia
Study of Total Body Irradiation and Fludarabine Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation in Combination With Cyclosporine and Mycophenolate Mofetil in Patients With Inherited Disorders
Higher-Dose Ezetimibe to Treat Homozygous Sitosterolemia
Study of Phosphatidylcholine in a Patient With Methionine Adenosyltransferase Deficiency
Diagnostic and Screening Study of Genetic Disorders