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Results for “Gangliosidosis”

47 trials

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Showing 20 of 47 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
Not applicableStudy completedNCT04310163
What this trial is testing

Interviews and Video Capture in Patients With GM1 Gangliosidosis

Who this might be right for
GM1 Gangliosidosis
LYSOGENE 25
Testing effectiveness (Phase 2)Active Not RecruitingNCT04713475
What this trial is testing

Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

Who this might be right for
GM1 GangliosidosisGM1 Gangliosidosis, Type IGM1 Gangliosidosis, Type 2+1 more
Gemma Biotherapeutics 26
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 applicableStudy completedNCT04041102
What this trial is testing

Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

Who this might be right for
GM1 Gangliosidosis
University of Pennsylvania 31
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
Not applicableLooking for participantsNCT06539169
What this trial is testing

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Who this might be right for
Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more
xCures 1,000
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
Not applicableStudy completedNCT02699190
What this trial is testing

LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

Who this might be right for
LeukodystrophyWhite Matter Disease4H Syndrome+61 more
Children's Hospital of Philadelphia 236
Not applicableLooking for participantsNCT03047369
What this trial is testing

The Myelin Disorders Biorepository Project

Who this might be right for
LeukodystrophyWhite Matter DiseaseLeukoencephalopathies+63 more
Children's Hospital of Philadelphia 12,000
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
Large-scale testing (Phase 3)Looking for participantsNCT07082725
What this trial is testing

Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)

Who this might be right for
Niemann-Pick Type C Disease
Azafaros A.G. 72
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
Large-scale testing (Phase 3)Ended earlyNCT04395339
What this trial is testing

GM1 Prophylaxis for WBRT Related Cognitive Dysfunction

Who this might be right for
Brain MetastasesRadiotherapy Side EffectCognitive Impairment+2 more
Sun Yat-sen University 51
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
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