Expanded Access Program for Tiratricol in Patients With Monocarboxylate Transporter 8 Deficiency
Trial statusAvailable
Trial phaseNot listed
Trial typeExpanded Access
Biological sexAll
AgeNot listed
SponsorRare Thyroid Therapeutics International AB
About this trial
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.
Eligibility criteria
Qualifiers
Diagnosis of MCT8 deficiency confirmed with a genetic test.
Either tiratricol treatment naïve, or patients who may be on a stable dose of tiratricol having transferred from the Phase 3 MCT8-2021-3 (ReTRIACt) study (NCT05579327) or prior individual investigational new drug (IND).
In the Treating Physician's medical opinion, the potential benefits of treatment with tiratricol outweigh the potential risks for the patient.
Patient or legal representative provided signed and dated informed consent to be treated with tiratricol, through this EAP.
Disqualifiers
Parents, legal representative or, if applicable, patients unwilling or unable to comply with the Treating Physician's treatment plan related to this EAP for any reason.
Conditions requiring repeated hospitalizations that are likely to confound ability to participate in the program.
Major illness in the 3 months before the screening visit that is likely to confound the ability of the patient to participate fully within the program and/or confound the assessment of serum total T3 and/or safety.
Major surgery within the 3 months before the screening visit, or planned to take place during the program, including but not limited to major abdominal/thoracic/neurosurgical procedures.
Trial design
Treatments tested in this trial
- Tiratricol
Treatment groups
No treatment groups listed
Sponsors and collaborators
Rare Thyroid Therapeutics International AB
Lead sponsor
AnovoRx
Collaborator
Egetis Therapeutics
Collaborator
Source ClinicalTrials.gov