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ClinicalTrials.gov public records Last synced May 21, 2026, 7:41 PM EDT

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Showing 25–29 of 5 matching trials from the live ClinicalTrials.gov search.

Local D1 index available.

Filters: Condition: Tay-Sachs Disease Clear all

Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Inborn Errors of Metabolism

NCT00176904

View directory record Official record

Conditions: Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease, Fucosidosis, Wolman Disease, Niemann-Pick Disease, Batten Disease, GM1 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease
Interventions: Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin; Procedure · Drug
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Not listed

Enrollment: 135 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1995 – 2010
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Source: ClinicalTrials.gov public record

Updated Dec 27, 2017 · Synced May 21, 2026, 7:41 PM EDT

Completed Phase 3 Interventional

Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses

NCT00672022

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Conditions: GM2 Gangliosidoses, Tay-Sachs, Sandhoff Disease
Interventions: Zavesca (Miglustat); Drug
Lead sponsor: Children's National Research Institute; Other
Eligibility: 6 Months to 5 Years

Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2007
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Source: ClinicalTrials.gov public record

Updated May 5, 2008 · Synced May 21, 2026, 7:41 PM EDT

Recruiting Phase 1 Interventional

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

NCT02254863

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Conditions: Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II, Leukodystrophy, Globoid Cell, Leukodystrophy, Metachromatic, Neimann Pick Disease, Pelizaeus-Merzbacher Disease, Sandhoff Disease, Tay-Sachs Disease, Brain Diseases, Metabolic, Inborn, Alpha-Mannosidosis, Sanfilippo Mucopolysaccharidoses
Interventions: DUOC-01; Biological
Lead sponsor: Joanne Kurtzberg, MD; Other
Eligibility: 1 Week to 22 Years

Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2026
U.S. locations: 1
States / cities: Durham, North Carolina

Source: ClinicalTrials.gov public record

Updated Sep 7, 2025 · Synced May 21, 2026, 7:41 PM EDT

Completed No phase listed Observational

Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2

NCT04470713

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Conditions: GM1 Gangliosidosis, GM2 Gangliosidosis, Gaucher Disease, Type 2, Tay-Sachs Disease, AB Variant Gangliosidosis GM2, Sandhoff Disease
Interventions: Not listed
Lead sponsor: Idorsia Pharmaceuticals Ltd.; Industry
Eligibility: 0 Months and older

Enrollment: 226 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2021
U.S. locations: 3
States / cities: Chicago, Illinois • Rochester, Minnesota • Fairfax, Virginia

Source: ClinicalTrials.gov public record

Updated Nov 7, 2021 · Synced May 21, 2026, 7:41 PM EDT

Terminated Phase 1Phase 2 Interventional

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

NCT01372228

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Conditions: Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III), Krabbe Disease (Globoid Leukodystrophy), Metachromatic Leukodystrophy (MLD), Adrenoleukodystrophy (ALD and AMN), Sandhoff Disease, Tay Sachs Disease, Pelizaeus Merzbacher (PMD), Niemann-Pick Disease, Alpha-mannosidosis
Interventions: hematopoietic stem cell infusion; Biological
Lead sponsor: Talaris Therapeutics Inc.; Industry
Eligibility: Not listed

Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2016
U.S. locations: 1
States / cities: Durham, North Carolina

Source: ClinicalTrials.gov public record

Updated Apr 11, 2023 · Synced May 21, 2026, 7:41 PM EDT