Homocystinuria
15
1
4
8
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 40/100
0.0%
0 terminated out of 15 trials
100.0%
+13.5% vs benchmark
13%
2 trials in Phase 3/4
13%
1 of 8 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 8 completed trials
Clinical Trials (15)
Evaluation of the Express Plus Range
Proof of Concept Creatine Supplementation for Homocystinuria Study
Baby Detect : Genomic Newborn Screening
A Phase 3 Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)
Health Related Quality of Life (HrQoL) in Classical Homocystinuria (CBS Deficiency)
A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment
Early Check: Expanded Screening in Newborns
Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE)
Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan
Safety, Tolerability and Pharmacodynamics of SYNB1353 in Healthy Adult Volunteers
Betaine METABOLISM OF PATIENTS With Homocystinuria
Effects of Exercise on Metabolic Parameters in Classical Homocystinuria
Oxidative Stress Markers In Inherited Homocystinuria And The Impact Of Taurine
Homocystinuria: Treatment With N-Acetylcysteine
Study of Homocysteine Metabolism in Homocystinuria