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Conditions
Sandhoff Disease

Clinical trial condition

Sandhoff Disease

Explore clinical trials studying Sandhoff Disease. Study availability and eligibility vary by location and protocol.

15 public trials 3 recruiting studies

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Sandhoff Disease trial results

Completed

Gene Therapy for Tay-Sachs Disease

NCT01869270

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Data Management and Coordinating Center (DMCC), Univ. of South Florida Tampa, Florida, United States

University of Minnesota Minneapolis, Minnesota, United States

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Completed

Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)

NCT01102686

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +17 more
Locations: The Hospital for Sick Children Toronto, Ontario, Canada

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Completed

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

NCT00672022

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
Locations: Children's National Medical Center Washington D.C., District of Columbia, United States

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Active, Not Recruiting

Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease

NCT06614569

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: UMass Chan Medical School Worcester, Massachusetts, United States

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Active, Not Recruiting

First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis

NCT04798235

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
Locations: Queen's University/Kingston Health Sciences Centre Kingston, Ontario, Canada

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