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Clinical trialNCT04143295AvailableExpanded Access

Rescue of Infants With MCT8 Deficiency

SponsorRoy E. Weiss, M.D.Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency
View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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.
clinicaltrials.gov ↗

Investigators

The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

1 registered facility, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

United States
  • University of Miami, Miller School of Medicine

    Miami, Florida, United States

    Roy E Weiss, M.D.
    Available

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

Diiodothyropropionic acid
Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start
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Primary completion
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Completion
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Last update posted
11 Dec 2025
Registered enrollment
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Registry id
NCT04143295
clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.