Crick
HomeTrialsGenesDrugs23andMeGraphBlogContact
  • Home
  • Trials
  • Genes
  • Drugs
  • 23andMe
  • Graph
  • Blog
  • Contact
Crick

An open-source clinical intelligence platform. Explore clinical trials, gene networks, and molecular structures using public data sources.

Data Sources

  • ClinicalTrials.gov
  • OpenTargets
  • ClinVar
  • PubChem

Links

  • Contact
  • About
  • Privacy

© 2026 Crick. All rights reserved.

Crick is for educational purposes only. Not medical advice.

Results for “Sandhoff Disease”

26 trials

Filters

Phase
Early Phase 1
Phase 1
Phase 2
Phase 3
Phase 4
Status
Recruiting
Active, Not Recruiting
Not Yet Recruiting
Completed
Enrolling by Invitation

Showing 20 of 26 results

Testing effectiveness (Phase 2)Active Not RecruitingNCT04798235
What this trial is testing

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

Who this might be right for
Infantile GM2 Gangliosidosis (Disorder)
Dr. Anupam Sehgal 3
Testing effectiveness (Phase 2)Study completedNCT00418847
What this trial is testing

Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis

Who this might be right for
Gangliosidoses GM2
The Hospital for Sick Children 5
Early research (Phase 1)WithdrawnNCT01003912
What this trial is testing

Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases

Who this might be right for
Lysosomal Storage DiseasesInborn Errors of Metabolism
Joanne Kurtzberg, MD
Large-scale testing (Phase 3)Study completedNCT00176904
What this trial is testing

Stem Cell Transplant for Inborn Errors of Metabolism

Who this might be right for
AdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+8 more
Masonic Cancer Center, University of Minnesota 135
Testing effectiveness (Phase 2)Study completedNCT01102686
What this trial is testing

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

Who this might be right for
Gangliosidoses, GM2Sandhoff DiseaseTay-Sachs Disease
The Hospital for Sick Children 20
Not applicableActive Not RecruitingNCT06614569
What this trial is testing

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

Who this might be right for
GM2 GangliosidosisTay Sachs DiseaseSandhoff Disease
Terence Flotte 7
Testing effectiveness (Phase 2)Study completedNCT00383448
What this trial is testing

HSCT for High Risk Inherited Inborn Errors

Who this might be right for
AdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+6 more
Masonic Cancer Center, University of Minnesota 38
Not applicableStudy completedNCT05109793
What this trial is testing

GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)

Who this might be right for
GM1 GangliosidosisSandhoff DiseaseTay-Sachs Disease
Azafaros A.G. 31
Not applicableLooking for participantsNCT00029965
What this trial is testing

Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders

Who this might be right for
Neurological RegressionMyoclonusCherry Red Spot+1 more
National Human Genome Research Institute (NHGRI) 200
Not applicableNot Yet RecruitingNCT07445490
What this trial is testing

Translational Potential of ex Vivo Gene Therapy in GM2 Gangliosidosis

Who this might be right for
Tay-Sachs Disease GangliosideSandhoff Disease Ganglioside
Assistance Publique - Hôpitaux de Paris 6
Large-scale testing (Phase 3)Ended earlyNCT03822013
What this trial is testing

Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD)

Who this might be right for
GM2 GangliosidosisSupportive Care
Tehran University of Medical Sciences 30
Not applicableUnknownNCT04624789
What this trial is testing

Registry Gangliosidoses

Who this might be right for
GangliosidosesGM1 GangliosidosisMorquio B Disease+6 more
SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt) 40
Large-scale testing (Phase 3)Study completedNCT00672022
What this trial is testing

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

Who this might be right for
GM2 GangliosidosesTay-SachsSandhoff Disease
Children's National Research Institute 10
Not applicableLooking for participantsNCT03333200
What this trial is testing

Longitudinal Study of Neurodegenerative Disorders

Who this might be right for
MLDKrabbe DiseaseALD+24 more
University of Pittsburgh 1,500
Early research (Phase 1)WithdrawnNCT00679744
What this trial is testing

A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis

Who this might be right for
G(M2) GangliosideTay-Sachs Disease GangliosideSandhoff Disease Ganglioside
Exsar Corporation
Testing effectiveness (Phase 2)Ended earlyNCT01372228
What this trial is testing

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

Who this might be right for
Hurler Syndrome (MPS I)Hurler-Scheie SyndromeHunter Syndrome (MPS II)+9 more
Talaris Therapeutics Inc. 3
Large-scale testing (Phase 3)Ended earlyNCT00654433
What this trial is testing

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

Who this might be right for
Inherited Metabolic DiseasesLysosomal Storage DisordersPeroxisomal Storage Diseases+2 more
Aldagen 40
Post-approval studies (Phase 4)Ended earlyNCT02030015
What this trial is testing

Synergistic Enteral Regimen for Treatment of the Gangliosidoses

Who this might be right for
GM1 GangliosidosesGM2 GangliosidosesTay-Sachs Disease+1 more
University of Minnesota 16
Testing effectiveness (Phase 2)Study completedNCT03759665
What this trial is testing

N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)

Who this might be right for
GM2 GangliosidosisTay-Sachs DiseaseSandhoff Disease
IntraBio Inc 30
Large-scale testing (Phase 3)Ended earlyNCT04221451
What this trial is testing

A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2

Who this might be right for
Tay-Sachs DiseaseSandhoff Disease
Genzyme, a Sanofi Company 75
Load More Results