Status: Available
Expanded Access Program for Tiratricol in Patients With Monocarboxylate Transporter 8 Deficiency
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.
Trial details
Biological sex: AllType: Expanded AccessSponsor: Rare Thyroid Therapeutics International ABUpdated: May 5, 2026Locations: 17
Eligibility criteria
Diagnosis of MCT8 deficiency confirmed with a genetic test. [+5]
Parents, legal representative or, if applicable, patients unwilling or unable to... [+9]