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

Last updated: May 7, 2025
Sponsor: Rare Thyroid Therapeutics International AB
Overall Status: Planned

Phase

N/A

Condition

Muscular Dystrophy

Martin-bell Syndrome

Myasthenia Gravis (Chronic Weakness)

Treatment

Tiratricol

Clinical Study ID

NCT05911399
Tiratricol-EAP-Pro MCT8-2022-4
  • All Genders

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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

Each patient must meet all of the following criteria to be eligible:

  1. Diagnosis of MCT8 deficiency confirmed with a genetic test.

  2. Either tiratricol treatment naïve, or patients who may be on a stable dose oftiratricol having transferred from the Phase 3 MCT8-2021-3 (ReTRIACt) study (NCT05579327) or prior individual investigational new drug (IND).

  3. In the Treating Physician's medical opinion, the potential benefits of treatmentwith tiratricol outweigh the potential risks for the patient.

  4. Patient or legal representative provided signed and dated informed consent to betreated with tiratricol, through this EAP.

  5. Given the severity of the disease, sexual activity in these patients is deemedunlikely. However, where, at the discretion of the Treating Physician sexualactivity is possible for the patient, patients must followprotocol-specified-contraception guidance.

  6. Patient is approved for enrolment by the sponsor RTT.

Exclusion

Exclusion Criteria:

Patients who meet any of the following criteria will be excluded from the EAP:

  1. Parents, legal representative or, if applicable, patients unwilling or unable tocomply with the Treating Physician's treatment plan related to this EAP for anyreason.

  2. Major illness or recent major surgery unrelated to MCT8 deficiency (in the TreatingPhysician's judgement), defined as:

  • Conditions requiring repeated hospitalizations that are likely to confoundability to participate in the program.

  • Major illness in the 3 months before the screening visit that is likely toconfound the ability of the patient to participate fully within the programand/or confound the assessment of serum total T3 and/or safety.

  • Major surgery within the 3 months before the screening visit, or planned totake place during the program, including but not limited to majorabdominal/thoracic/neurosurgical procedures.

  • Major/minor abdominal and/or maxillofacial surgery that may inhibit theadministration and/or absorption of tiratricol.

  1. Patients with any contra-indication for treatment with tiratricol or any excipientsin the program treatment.

  2. 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 havestabilized after the cessation of these medications. For patients currently usingthese medications the switch to tiratricol should be made following the above andunder the guidance of an endocrinologist with knowledge of MCT8 deficiency, ifneeded, after consultation with pharmacologist.

  3. Known hypersensitivity to tiratricol including any ingredient in the pharmaceuticalformulation.

  4. Although very unlikely, as this is a severe X-linked disease: Women who are nursingor pregnant (or women who are planning to become pregnant during treatment withtiratricol).

  5. Patients eligible for clinical trials with tiratricol.

Study Design

Treatment Group(s): 1
Primary Treatment: Tiratricol
Phase:
Study Start date:
Estimated Completion Date:

Study 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.

Connect with a study center

  • University of Alabama at Birmingham Hospital

    Birmingham, Alabama 35249
    United States

    Planned

  • Childrens Valley Hospital in

    Madera, California 93636-8762
    United States

    Planned

  • Valley Children's Hospital

    Madera, California 93636-8762
    United States

    Planned

  • Rady Children's Hospital

    San Diego, California 92123
    United States

    Planned

  • Nemour's Children Hospital

    Jacksonville, Florida 32207
    United States

    Planned

  • Children's Healthcare of Atlanta

    Atlanta, Georgia 30329
    United States

    Planned

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois 60611
    United States

    Planned

  • Louisiana State University Health Sciences Center and Children's Hospital

    New Orleans, Louisiana 70118
    United States

    Planned

  • Baystate Medical Center

    Springfield, Massachusetts 01199
    United States

    Planned

  • Gillette's Children's Specialty Healthcare

    Saint Paul, Minnesota 55101
    United States

    Planned

  • Oregon Health and Science University

    Portland, Oregon 97239
    United States

    Planned

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania 19104
    United States

    Planned

  • Le Bonheur Children's Hospital Foundation

    Memphis, Tennessee 38105
    United States

    Planned

  • Cook Children's Health Care System

    Fort Worth, Texas 76106
    United States

    Planned

  • MultiCare Mary Bridge Children's Hospital

    Tacoma, Washington 98403
    United States

    Planned

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