Monocarboxylate Transporter 8 Deficiency
3
0
0
1
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Treatment Research
2 treatments under study, led by Tiratricol
Research Leaders
6 sponsors active, led by Rare Thyroid Therapeutics International AB
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 30/100
0.0%
0 terminated out of 3 trials
100.0%
+13.4% vs benchmark
33%
1 trials in Phase 3/4
0%
0 of 1 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 1 completed trials
Clinical Trials (3)
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)
Glycerol-Phenylbutyrate Treatment in Children With MCT Mutation (Allan-Herndon- Dudley Syndrome)
Research Network
Common Treatments
- Tiratricol2
- Glycerol Phenylbutyrate1
Active Sponsors
- Rare Thyroid Therapeutics International AB2
- Egetis Therapeutics AB (publ)2
- AnovoRx1
- Kaplan Medical Center1
- Premier Research1
Top Investigators
Kaplan Medical Center, affiliated with the Hebrew University of Jerusalem, Israel
Erasmus Medical Center
Children's Hospital of Philadelphia
Related Conditions
- Allanherndondudley Syndrome2
- Monocarboxylate Transporter 8 Mct8 Deficiency1