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Results for “Tay-Sachs”

30 trials

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Showing 20 of 30 results

Not applicableStudy completedNCT01626092
What this trial is testing

Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders

Who this might be right for
Lysosomal Storage DiseasePeroxisomal Disorder
Masonic Cancer Center, University of Minnesota 3
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 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 applicableLooking for participantsNCT05007990
What this trial is testing

Caregiving Networks Across Disease Context and the Life Course

Who this might be right for
Inherited Metabolic DisordersUndiagnosed DiseasesBatten's Disease+2 more
National Human Genome Research Institute (NHGRI) 2,800
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)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)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
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
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
Early research (Phase 1)Ended earlyNCT04669535
What this trial is testing

A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease

Who this might be right for
Tay-Sachs DiseaseSandhoff Disease
Terence Flotte 9
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
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 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 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 applicableStudy completedNCT00006057
What this trial is testing

Diagnostic and Screening Study of Genetic Disorders

Who this might be right for
Tay-Sachs DiseasePorphyria, ErythropoieticLeukodystrophy, Globoid Cell+1 more
National Center for Research Resources (NCRR) 50
Not applicableLooking for participantsNCT00668187
What this trial is testing

A Natural History Study of the Gangliosidoses

Who this might be right for
Tay-Sachs DiseaseSandhoff DiseaseLate Onset Tay-Sachs Disease+2 more
University of Minnesota 52
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
Not applicableStudy completedNCT04470713
What this trial is testing

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

Who this might be right for
GM1 GangliosidosisGM2 GangliosidosisGaucher Disease, Type 2+3 more
Idorsia Pharmaceuticals Ltd. 226
Not applicableActive Not RecruitingNCT02851862
What this trial is testing

A Natural History of Late Onset Tay-Sachs Disease

Who this might be right for
GM2 Gangliosidosis
Massachusetts General Hospital 10
Not applicableStudy completedNCT01999257
What this trial is testing

Efficacy Study of an Online Educational Module Before Carrier Genetic Screening in Persons of Ashkenazi Jewish Descent.

Who this might be right for
Tay Sachs DiseaseCanavan DiseaseFamilial Dysautonomia
McGill University Health Centre/Research Institute of the McGill University Health Centre 60
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