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About
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.
Full 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.
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Inclusion criteria
Each patient must meet all of the following criteria to be eligible:
Exclusion criteria
Patients who meet any of the following criteria will be excluded from the EAP:
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.
Major illness or recent major surgery unrelated to MCT8 deficiency (in the Treating Physician's judgement), defined as:
Patients with any contra-indication for treatment with tiratricol or any excipients in the program treatment.
Patients using thyroid hormone analogues- such as levothyroxine -or thionamides, such as propylthiouracil. Prior use of these drugs is not an exclusion criterion, provided the use of the medication has subsided and the thyroid hormone levels have stabilized after the cessation of these medications. For patients currently using these medications the switch to tiratricol should be made following the above and under the guidance of an endocrinologist with knowledge of MCT8 deficiency, if needed, after consultation with pharmacologist.
Known hypersensitivity to tiratricol including any ingredient in the pharmaceutical formulation.
Although very unlikely, as this is a severe X-linked disease: Women who are nursing or pregnant (or women who are planning to become pregnant during treatment with tiratricol).
Patients eligible for clinical trials with tiratricol.
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Central trial contact
AnovoRx; Egetis Therapeutics
Data sourced from clinicaltrials.gov
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