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Results for “Gangliosidoses GM2”

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Showing 19 of 0 results

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
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 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
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)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
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
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
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
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
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 applicableWithdrawnNCT02298647
What this trial is testing

Biomarker for Gangliosidosis: BioGM1/BioGM2 (BioGM1/GM2)

Who this might be right for
Hepato-splenomegalyDysostosis MultiplexSeizures+1 more
CENTOGENE GmbH Rostock
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
Testing effectiveness (Phase 2)Study completedNCT05758922
What this trial is testing

Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

Who this might be right for
GM2 GangliosidosisNiemann-Pick Disease, Type C
Azafaros B.V. 13
Not applicableStudy completedNCT01869270
What this trial is testing

Gene Therapy for Tay-Sachs Disease

Who this might be right for
Tay Sachs DiseaseSandhoff DiseaseLate Onset Tay Sachs Disease
University of Minnesota 4
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 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
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 B.V. 31
Testing effectiveness (Phase 2)Looking for participantsNCT07399704
What this trial is testing

Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

Who this might be right for
GM2 GangliosidosisNiemann-Pick Type C Disease
Azafaros B.V. 21
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