Trials / Available

AvailableNCT04143295

Rescue of Infants With MCT8 Deficiency

Status: Available
Phase: —
Study type: Expanded Access
Enrollment: —

Sponsor: Roy E. Weiss, M.D. · Academic / Other
Sex: Male
Age: 18 Years
Healthy volunteers: Not accepted

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

Type	Name	Description
DRUG	Diiodothyropropionic acid (DITPA)	Drug Administration

Timeline

First posted: 2019-10-29
Last updated: 2025-12-11

Locations

1 site across 1 country: United States

Source: ClinicalTrials.gov record NCT04143295. Inclusion in this directory is not an endorsement.