4 completed, 3 recruiting, 1 active not recruiting, 1 approved for marketing. 3 Phase 2, 2 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 3 | Active not recruiting 1, Completed 1, Unknown 1 |
| Phase 3 | 2 | Completed 1, Unknown 1 |
Follow Allan-Herndon-Dudley syndrome: new trials by email Literature Review of Allan-Herndon-Dudley syndrome trials Research Report on Allan-Herndon-Dudley syndrome
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Withdrawal of Tiratricol Treatment in Males With Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency) Tiratricol, Placebo - Monocarboxylate Transporter 8 Deficiency, Allan-Herndon-Dudley Syndrome | Phase 3 | Completed | Rare Thyroid Therapeutics International AB | 20 | 2023-07-21 | 2026-04-08 |
| MCT8 Deficiency Caregiver Study Monocarboxylate Transporter 8 (MCT8) Deficiency, Allan-Herndon-Dudley Syndrome | Completed | Rare Thyroid Therapeutics International AB | 21 | 2022-08-23 | 2024-04-25 | |
| Register for Patients With Thyroid Hormone Resistance. no intervention - Hypothyroidism, Global Developmental Delay | Recruiting | Charite University, Berlin, Germany | 200 | 2021-07-01 | 2024-08-22 | |
| Glycerol-Phenylbutyrate Treatment in Children With MCT Mutation (Allan-Herndon- Dudley Syndrome) Glycerol Phenylbutyrate 1100 MG/ML - Monocarboxylate Transporter 8 Deficiency | Phase 2 / Phase 3 | Unknown | Kaplan Medical Center | 6 | 2021-06-30 | 2021-08-24 |
| Triac Trial II in MCT8 Deficiency Patients Tiratricol - Allan-Herndon-Dudley Syndrome | Phase 2 | Active not recruiting | Rare Thyroid Therapeutics International AB | 22 | 2020-12-07 | 2026-02-20 |
| GROWing Up With Rare GENEtic Syndromes Retrospective file studies - Prader-Willi Syndrome, PWS-like Syndrome | Recruiting | dr. Laura C. G. de Graaff-Herder | 600 | 2018-10-01 | 2023-09-06 | |
| LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies Leukodystrophy, White Matter Disease | Completed | Children's Hospital of Philadelphia | 236 | 2017-01-06 | 2025-11-10 | |
| The Myelin Disorders Biorepository Project Leukodystrophy, White Matter Disease | Recruiting | Children's Hospital of Philadelphia | 12000 | 2016-12-08 | 2025-10-23 | |
| Thyroid Hormone Analog Therapy in MCT8 Deficiency: Triac Trial Patients Triac - Allan-Herndon-Dudley Syndrome | Phase 2 | Completed | Erasmus Medical Center | 46 | 2014-10-01 | 2019-04-16 |
| Rescue of Infants With MCT8 Deficiency Diiodothyropropionic acid (DITPA) - Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency | Available | Roy E. Weiss, M.D. | 2025-12-11 | |||
| Expanded Access Program for Tiratricol in Patients With Monocarboxylate Transporter 8 Deficiency Tiratricol - Monocarboxylate Transporter 8 Deficiency, Allan-Herndon-Dudley Syndrome | Approved for marketing | Rare Thyroid Therapeutics International AB | 2026-10-06 |
Adrenoleukodystrophy (2) · Aicardi-Goutieres syndrome (2) · Alexander Disease (2) · Alexanders leukodystrophy (2) · Attention Deficit Disorder with Hyperactivity (2) · CADASIL (2) · Canavan Disease (2) · Charcot-Marie-Tooth Disease (2) · Cockayne Syndrome (2) · Gangliosidoses (2) · Gangliosidoses, GM2 (2) · Leukodystrophy, Globoid Cell (2) · Leukodystrophy, Hypomyelinating, 5 (2) · Leukodystrophy, Hypomyelinating, 6 (2)
Congenital Abnormalities (4) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Endocrine System Diseases (4) · Musculoskeletal Diseases (4) · Nervous System Diseases (4) · Nervous System Malformations (4) · Neurobehavioral Manifestations (4) · Neurologic Manifestations (4) · Pathological Conditions, Signs and Symptoms (4) · Abnormalities, Multiple (3) · Adrenal Gland Diseases (3) · Bone Diseases (3) · Bone Diseases, Developmental (3) · Brain Diseases (3) · Cardiovascular Diseases (3)
3,3',5-triiodothyroacetic acid (4) · 3,5-diiodothyropropionic acid (1) · glycerol phenylbutyrate (1)
Rare Thyroid Therapeutics International AB (4) · Children's Hospital of Philadelphia (2) · Charite University, Berlin, Germany (1) · Erasmus Medical Center (1) · Kaplan Medical Center (1) · Roy E. Weiss, M.D. (1) · dr. Laura C. G. de Graaff-Herder (1)
United States (6) · Netherlands (4) · Germany (2) · United Kingdom (2) · Czechia (1) · Israel (1)
Adeline Vanderver, MD (2) · Amnon Zung, MD (1) · Andrew Bauer, MD (1) · Andrew J. Bauer, MD (1) · Heiko Krude (1) · Heiko Krude, MD (1) · Jan Lebl (1) · Lindsey Nicol (1) · Mark Larkin, PhD (1) · Roy E Weiss, M.D. (1) · W. E. Visser, MD (1) · W.E. Visser, MD, PhD (1) · W.E. Visser, dr, (1)
PMID 31377265, PMID 32342562, PMID 32749716, PMID 34969638, PMID 37881807, PMID 38469646
2014 (1), U01NS106845 (1), U54NS115052 (1)