NCT05911399|Unknown
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Sponsor study identifier
expanded_access
N/A
1 country
17
Study Start
Not reported
Date not reportedPrimary Completion
Not reported
Date not reportedStudy Completion
Not reported
Date not reportedThe 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.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
Not reported
Date not reportedClick on a node to explore related trials.
Some registry-reported names shown below are not yet linked in this graph.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
June 9, 2023
Actual (reported)June 22, 2023
Actual (reported)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.
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
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
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.
First Submitted
June 9, 2023
First Posted
June 22, 2023
Last Updated
October 6, 2026
Record last verified: 2026-10
Download the complete ClinicalTrials.gov record imported by Syfrah, including fields not shown in this page. The registry may contain a newer revision.
Source: ClinicalTrials.gov.
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Source retrieved:
MCT8 Deficiency Caregiver Study
Contact the study team to confirm eligibility.
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