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Expanded Access Program for Tiratricol in Patients With Monocarboxylate Transporter 8 Deficiency

NCT05911399 · Status: AVAILABLE · Type: EXPANDED_ACCESS

Last updated 2026-05-05

No results posted yet for this study

Summary

The goal of this program is to provide expanded access (i.e., before marketing authorization) to tiratricol as treatment for patients with monocarboxylate transporter 8 deficiency (MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome \[AHDS\]), who in their Treating Physician's opinion, could benefit from tiratricol and meet the eligibility criteria.

Conditions

  • Monocarboxylate Transporter 8 Deficiency
  • Allan-Herndon-Dudley Syndrome

Interventions

DRUG

Tiratricol

Tiratricol (3,3',5-triiodothyroacetic acid) is available as 350 µg tablets for oral administration (suspended in water and, if needed, mixed with food) or by percutaneous endoscopic gastrostomy (PEG), nasogastric or jejunal tube.

Sponsors & Collaborators

  • AnovoRx

    collaborator UNKNOWN

  • Egetis Therapeutics

    collaborator INDUSTRY

  • Rare Thyroid Therapeutics International AB

    lead INDUSTRY

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05911399 on ClinicalTrials.gov