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ClinicalTrials.gov public records Last synced May 22, 2026, 12:15 AM EDT

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Showing 1–6 of 6 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Allan-Herndon-Dudley Syndrome Clear all
Recruiting No phase listed Observational
View directory record Official record
Conditions
Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD Gene Mutation, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus Merzbacher Like Disease, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Not listed
Enrollment
12,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2030
U.S. locations
23
States / cities
Los Angeles, California • Orange, California • Palo Alto, California + 18 more
Source: ClinicalTrials.gov public record
Updated Oct 22, 2025 · Synced May 22, 2026, 12:15 AM EDT
Available No phase listed Expanded access
View directory record Official record
Conditions
Monocarboxylate Transporter 8 Deficiency, Allan-Herndon-Dudley Syndrome
Interventions
Tiratricol
Drug
Lead sponsor
Rare Thyroid Therapeutics International AB
Industry
Eligibility
Not listed
U.S. locations
17
States / cities
Birmingham, Alabama • Madera, California • San Diego, California + 13 more
Source: ClinicalTrials.gov public record
Updated May 4, 2026 · Synced May 22, 2026, 12:15 AM EDT
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
Allan-Herndon-Dudley Syndrome
Interventions
Tiratricol
Drug
Lead sponsor
Rare Thyroid Therapeutics International AB
Industry
Eligibility
Up to 30 Months · Male only
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
2
States / cities
Portland, Oregon • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 19, 2026 · Synced May 22, 2026, 12:15 AM EDT
Available No phase listed Expanded access
View directory record Official record
Conditions
Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency
Interventions
Diiodothyropropionic acid (DITPA)
Drug
Lead sponsor
Roy E. Weiss, M.D.
Other
Eligibility
Up to 18 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Dec 10, 2025 · Synced May 22, 2026, 12:15 AM EDT
Completed No phase listed Observational Results available
View directory record Official record
Conditions
Leukodystrophy, White Matter Disease, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts 1, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus-Merzbacher-Like Disease, 1, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 18 Years
Enrollment
236 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 9, 2025 · Synced May 22, 2026, 12:15 AM EDT
Completed Phase 3 Interventional
View directory record Official record
Conditions
Monocarboxylate Transporter 8 Deficiency, Allan-Herndon-Dudley Syndrome
Interventions
Tiratricol, Placebo
Drug
Lead sponsor
Rare Thyroid Therapeutics International AB
Industry
Eligibility
4 Years and older · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
6
States / cities
Kissimmee, Florida • Atlanta, Georgia • St Louis, Missouri + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 7, 2026 · Synced May 22, 2026, 12:15 AM EDT