NCT05911399

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

Expanded Access Program for Tiratricol in Patients With Monocarboxylate Transporter 8 Deficiency Also Known as Allan-Herndon-Dudley Syndrome (AHDS)

• Status: Available
• Phase: —
• Study type: Expanded Access
• Enrollment: —
• Sponsor: Rare Thyroid Therapeutics International AB · Industry
• Sex: All
• Age: —
• Healthy volunteers: —

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.

Detailed description

This will be an open-label, single-arm, multi-center EAP, designed to provide treatment access in the US to tiratricol for eligible patients with MCT8 deficiency, also known as AHDS. Patients will undergo clinical and safety assessments before tiratricol treatment initiation, during the titration phase and then at approximately 3-6 monthly intervals or more frequently if clinically indicated, following the initiation of tiratricol treatment and until treatment is completed or discontinued. Adverse events (AEs) and serious adverse events (SAEs) will be recorded and reported as per FDA regulations.

Conditions

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

Interventions

Type	Name	Description
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.

Timeline

• First posted: 2023-06-22
• Last updated: 2026-02-25

Locations

16 sites across 1 country: United States