Allan-Herndon-Dudley Syndrome
9
3
4
4
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 9 trials
100.0%
+13.5% vs benchmark
11%
1 trials in Phase 3/4
25%
1 of 4 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 4 completed trials
Clinical Trials (9)
Expanded Access Program for Tiratricol in Patients With Monocarboxylate Transporter 8 Deficiency
Withdrawal of Tiratricol Treatment in Males With Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency)
Triac Trial II in MCT8 Deficiency Patients
LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
The Myelin Disorders Biorepository Project
Register for Patients With Thyroid Hormone Resistance.
MCT8 Deficiency Caregiver Study
GROWing Up With Rare GENEtic Syndromes
Thyroid Hormone Analog Therapy in MCT8 Deficiency: Triac Trial Patients