Rescue of Infants With MCT8 Deficiency
NCT04143295 · Status: AVAILABLE · Type: EXPANDED_ACCESS
Last updated 2025-12-11
Summary
Monocarboxylate Transporter 8 (MCT8) deficiency (that is also known as Allan-Herndon-Dudley syndrome) is a rare X-linked inherited disorder of brain development that causes severe intellectual disability and problems with movement. This condition, which occurs almost exclusively in males, disrupts development from before birth.
Conditions
- Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency
Interventions
- DRUG
-
Diiodothyropropionic acid (DITPA)
Drug Administration
Sponsors & Collaborators
-
Roy E. Weiss, M.D.
lead OTHER
Principal Investigators
-
Roy E Weiss, M.D. · University of Miami
Eligibility
- Max Age
- 18 Years
- Sex
- MALE
- Healthy Volunteers
- No
Timeline & Regulatory
Countries
- United States
Study Locations
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