Expanded Access Program for Tiratricol in Patients With Monocarboxylate Transporter 8 Deficiency
NCT ID: NCT05911399
Last Updated: 2025-11-14
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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AVAILABLE
EXPANDED_ACCESS
Brief Summary
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Detailed Description
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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.
Conditions
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Keywords
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Interventions
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Tiratricol
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
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Inclusion Criteria
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
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.
ALL
No
Sponsors
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AnovoRx
UNKNOWN
Egetis Therapeutics
INDUSTRY
Rare Thyroid Therapeutics International AB
INDUSTRY
Responsible Party
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Locations
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University of Alabama at Birmingham Hospital
Birmingham, Alabama, United States
Valley Children's Hospital - Enrollment limited to patients within the hospital's health system
Madera, California, United States
Rady Children's Hospital
San Diego, California, United States
Nemour's Children Hospital
Jacksonville, Florida, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Louisiana State University Health Sciences Center and Children's Hospital
New Orleans, Louisiana, United States
Baystate Medical Center
Springfield, Massachusetts, United States
Gillette's Children's Specialty Healthcare
Saint Paul, Minnesota, United States
Oregon Health and Science University
Portland, Oregon, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Le Bonheur Children's Hospital Foundation
Memphis, Tennessee, United States
Cook Children's Health Care System
Fort Worth, Texas, United States
MultiCare Mary Bridge Children's Hospital
Tacoma, Washington, United States
Children's Wisconsin
Milwaukee, Wisconsin, United States
Countries
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Central Contacts
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Facility Contacts
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Leen Matalka, MD
Role: primary
Richard Sidlow, MD
Role: primary
Michael Gottschalk, MD
Role: primary
Larry Fox, MD
Role: primary
Kristina Cossen, MD
Role: primary
Apoorva Aekka, MD
Role: primary
Ricardo Gomez, MD
Role: primary
Bracha Goldsweig, MD
Role: primary
Lauren Yauch, MD
Role: primary
Lindsey Nicol, MD
Role: primary
Andrew Bauer, MD
Role: primary
Jordan Ross, MD
Role: primary
Paul Thornton, MD
Role: primary
Amy L. Yuen, MD
Role: primary
Bethany Auble, MD
Role: primary
References
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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.
van 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.
Other Identifiers
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Tiratricol-EAP-Pro MCT8-2022-4
Identifier Type: -
Identifier Source: org_study_id