Expanded Access Program for Tiratricol in Patients With Monocarboxylate Transporter 8 Deficiency
1 other identifier
expanded_access
N/A
1 country
17
Brief 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.
Trial Health
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17 active sites
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Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 9, 2023
CompletedFirst Posted
Study publicly available on registry
June 22, 2023
CompletedMay 5, 2026
April 1, 2026
June 9, 2023
April 29, 2026
Conditions
Keywords
Interventions
Tiratricol (3,3',5-triiodothyroacetic acid) is available as 350 µg tablets for oral administration (suspended in water and, if needed, mixed with food) or by percutaneous endoscopic gastrostomy (PEG), nasogastric or jejunal tube.
Eligibility Criteria
You may qualify if:
- Each patient must meet all of the following criteria to be eligible:
- 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.
- 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.
- Patient is approved for enrolment by the sponsor RTT.
You may not qualify if:
- 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:
- 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.
- Patients with any contra-indication for treatment with tiratricol or any excipients in the program treatment.
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- AnovoRxcollaborator
- Egetis Therapeuticscollaborator
- Rare Thyroid Therapeutics International ABlead
Study Sites (17)
University of Alabama at Birmingham Hospital
Birmingham, Alabama, 35249, United States
Valley Children's Hospital
Madera, California, 93636-8762, United States
Rady Children's Hospital
San Diego, California, 92123, United States
Nemour's Children Hospital
Jacksonville, Florida, 32207, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, 30329, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
Louisiana State University Health Sciences Center and Children's Hospital
New Orleans, Louisiana, 70118, United States
Baystate Medical Center
Springfield, Massachusetts, 01199, United States
Gillette's Children's Specialty Healthcare
Saint Paul, Minnesota, 55101, United States
Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
Columbia University Irving Medical Center
New York, New York, 10032, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Le Bonheur Children's Hospital Foundation
Memphis, Tennessee, 38105, United States
Cook Children's Health Care System
Fort Worth, Texas, 76106, United States
MultiCare Mary Bridge Children's Hospital
Tacoma, Washington, 98403, United States
Children's Wisconsin
Milwaukee, Wisconsin, 53326, United States
Related Publications (2)
Groeneweg S, Peeters RP, Moran C, Stoupa A, Auriol F, Tonduti D, Dica A, Paone L, Rozenkova K, Malikova J, van der Walt A, de Coo IFM, McGowan A, Lyons G, Aarsen FK, Barca D, van Beynum IM, van der Knoop MM, Jansen J, Manshande M, Lunsing RJ, Nowak S, den Uil CA, Zillikens MC, Visser FE, Vrijmoeth P, de Wit MCY, Wolf NI, Zandstra A, Ambegaonkar G, Singh Y, de Rijke YB, Medici M, Bertini ES, Depoorter S, Lebl J, Cappa M, De Meirleir L, Krude H, Craiu D, Zibordi F, Oliver Petit I, Polak M, Chatterjee K, Visser TJ, Visser WE. Effectiveness and safety of the tri-iodothyronine analogue Triac in children and adults with MCT8 deficiency: an international, single-arm, open-label, phase 2 trial. Lancet Diabetes Endocrinol. 2019 Sep;7(9):695-706. doi: 10.1016/S2213-8587(19)30155-X. Epub 2019 Jul 31.
PMID: 31377265BACKGROUNDvan Geest FS, Groeneweg S, van den Akker ELT, Bacos I, Barca D, van den Berg SAA, Bertini E, Brunner D, Brunetti-Pierri N, Cappa M, Cappuccio G, Chatterjee K, Chesover AD, Christian P, Coutant R, Craiu D, Crock P, Dewey C, Dica A, Dimitri P, Dubey R, Enderli A, Fairchild J, Gallichan J, Garibaldi LR, George B, Hackenberg A, Heinrich B, Huynh T, Klosowska A, Lawson-Yuen A, Linder-Lucht M, Lyons G, Monti Lora F, Moran C, Muller KE, Paone L, Paul PG, Polak M, Porta F, Reinauer C, de Rijke YB, Seckold R, Menevse TS, Simm P, Simon A, Spada M, Stoupa A, Szeifert L, Tonduti D, van Toor H, Turan S, Vanderniet J, de Waart M, van der Wal R, van der Walt A, van Wermeskerken AM, Wierzba J, Zibordi F, Zung A, Peeters RP, Visser WE. Long-Term Efficacy of T3 Analogue Triac in Children and Adults With MCT8 Deficiency: A Real-Life Retrospective Cohort Study. J Clin Endocrinol Metab. 2022 Feb 17;107(3):e1136-e1147. doi: 10.1210/clinem/dgab750.
PMID: 34679181BACKGROUND
MeSH Terms
Conditions
Interventions
Central Study Contacts
Study Design
- Study Type
- expanded access
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 9, 2023
First Posted
June 22, 2023
Last Updated
May 5, 2026
Record last verified: 2026-04