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New hope for rare brain disorder: early drug access program opens

NCT ID NCT05911399

Disease control Sponsor: Rare Thyroid Therapeutics International AB Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access This study
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This program offers early access to tiratricol, a thyroid hormone-like drug, for people with Allan-Herndon-Dudley syndrome (AHDS), a rare genetic condition that affects brain development and movement. Patients must have a confirmed genetic diagnosis and be considered likely to benefit by their doctor. The goal is to provide treatment before the drug is fully approved.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Tiratricol (a thyroid hormone-like drug taken by mouth or feeding tube)
What this could lead to
If it works, this could provide a treatment option for a rare, severe neurological condition with no approved therapies.
What could go wrong
This is an expanded access program, not a formal trial, so data on effectiveness and safety is still limited. The drug may not work for all patients and could have side effects.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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 of tiratricol 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 treatment with tiratricol outweigh the potential risks for the patient. 4. Patient or legal representative provided signed and dated informed consent to be treated with tiratricol, through this EAP. 5. Given the severity of the disease, sexual activity in these patients is deemed unlikely. However, where, at the discretion of the Treating Physician sexual activity is possible for the patient, patients must follow protocol-specified-contraception guidance. 6. Patient is approved for enrolment by the sponsor RTT. 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 to comply with the Treating Physician's treatment plan related to this EAP for any reason. 2. Major illness or recent major surgery unrelated to MCT8 deficiency (in the Treating Physician's judgement), defined as: * 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. * Major/minor abdominal and/or maxillofacial surgery that may inhibit the administration and/or absorption of tiratricol. 3. Patients with any contra-indication for treatment with tiratricol or any excipients in the program treatment. 4. 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. 5. Known hypersensitivity to tiratricol including any ingredient in the pharmaceutical formulation. 6. 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). 7. Patients eligible for clinical trials with tiratricol.

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Conditions

The condition(s) this trial relates to.

Allan-Herndon-Dudley syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Allan-herndon-dudley syndrome Monocarboxylate transporter 8 deficiency

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    17 sites. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

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

    AVAILABLE

    Chicago, Illinois, 60611, United States

  • Baystate Medical Center

    AVAILABLE

    Springfield, Massachusetts, 01199, United States

  • Children's Healthcare of Atlanta

    AVAILABLE

    Atlanta, Georgia, 30329, United States

  • Children's Hospital of Philadelphia

    AVAILABLE

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Wisconsin

    AVAILABLE

    Milwaukee, Wisconsin, 53326, United States

  • Columbia University Irving Medical Center

    AVAILABLE

    New York, New York, 10032, United States

  • Cook Children's Health Care System

    AVAILABLE

    Fort Worth, Texas, 76106, United States

  • Gillette's Children's Specialty Healthcare

    AVAILABLE

    Saint Paul, Minnesota, 55101, United States

  • Icahn School of Medicine at Mount Sinai

    AVAILABLE

    New York, New York, 10029, United States

  • Le Bonheur Children's Hospital Foundation

    AVAILABLE

    Memphis, Tennessee, 38105, United States

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

    AVAILABLE

    New Orleans, Louisiana, 70118, United States

  • MultiCare Mary Bridge Children's Hospital

    AVAILABLE

    Tacoma, Washington, 98403, United States

  • Nemour's Children Hospital

    AVAILABLE

    Jacksonville, Florida, 32207, United States

  • Oregon Health and Science University

    AVAILABLE

    Portland, Oregon, 97239, United States

  • Rady Children's Hospital

    AVAILABLE

    San Diego, California, 92123, United States

  • University of Alabama at Birmingham Hospital

    AVAILABLE

    Birmingham, Alabama, 35249, United States

  • Valley Children's Hospital

    AVAILABLE

    Madera, California, 93636-8762, United States

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