Search clinical trials for: GM1 Gangliosidosis

Total 63 results

National Human Genome Research Institute (NHGRI)
Sio Gene Therapies

Recruiting

A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis

Gangliosidosis | Lysosomal Diseases | GM1

United States
University of Pennsylvania
Passage Bio, Inc.

Recruiting

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

GM1 Gangliosidosis

United States, Turkey, Brazil, Canada, United Kingdom
General Hospital of Shenyang Military Region
The Third Xiangya Hospital of Central South University

Completed

An Exploratory Study of Ganglioside GM1 in Acute Ischemic Stroke

Stroke, Ischemic

China
Fudan University

Unknown

Treatment of Cerebral Radiation Necrosis With GM1, a Prospective Study

MRI | Nasopharyngeal Carcinoma | Radiotherapy | Cerebral Radiation Necrosis

China
Sun Yat-sen University

Completed

Effect of GM1 in Prevention of Taxanes Induced Neurotoxicity in Operable Breast Cancer

Breast Cancer

China
Meir Medical Center

Unknown

17 Alfa Hydroxyprogesterone Caproate Versus Natural Progesterone for the Prevention of Preterm Labor

Pregnancy

Israel
Thomas Jefferson University
National Institute of Neurological Disorders and Stroke (NINDS)

Completed

GM1 Ganglioside Effects on Parkinson's Disease

Parkinson Disease

United States
Alliance for Clinical Trials in Oncology
National Cancer Institute (NCI)

Recruiting

Finding an Effective Dose of GM1 to Reduce or Prevent Neuropathy (Numbness or Weakness) Due to Treatment With Paclitaxel (Phase II)

Anatomic Stage IV Breast Cancer AJCC v8 | Chemotherapy-Induced Peripheral Neuropathy | Metastatic Breast Carcinoma

United States
Yuhong Li

Completed

Effect of GM1 in Prevention of Oxaliplatin Induced Neurotoxicity in Stage II/III Colorectal Cancer

Colorectal Cancer | Chemotherapy-induced Neutropenia

China
Peking University Cancer Hospital & Institute
Peking University First Hospital; Peking Union Medical College Hospital; Cancer... and other collaborators

Unknown

Monosialoganglioside Preventing Neurotoxicity Induced by Albumin-bound Paclitaxel Chemotherapy in Lung Cancer Patients

Neurotoxicity

China
InnoMedica Schweiz AG

Not yet recruiting

Estimates of the Short-term Efficacy of Talineuren (TLN) and Placebo in Patients With Parkinson Disease

Parkinson Disease

Switzerland
InnoMedica Schweiz AG

Active, not recruiting

Safety Evaluation of Intravenous Talineuren (TLN) in Parkinson's Disease-affected Patients (NEON)

Parkinson Disease

Switzerland
Sun Yat-sen University

Unknown

Efficacy of Monosialotetrahexosylganglioside in the Prophylactic Treatment of Bortezomib-induced Peripheral Neuropathy

Multiple Myeloma

China
Passage Bio, Inc.

Active, not recruiting

Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis (Imagine-1)

GM1 Gangliosidosis | GM1 Gangliosidosis, Type I | GM1 Gangliosidosis, Type 2 | Beta-Galactosidase-1 (GLB1) Deficiency

United States, Canada, United Kingdom, Turkey, Brazil
Sun Yat-sen University
Southern Medical University, China

Unknown

Monosialoganglioside(GM1) Preventing Neurotoxicity Induced by Cisplatin Contained Chemotherapy in NSCLC Patients

Non-small Cell Lung Cancer

China
LYSOGENE
Casimir, LLC; Cure GM1 Foundation

Completed

Interviews and Video Capture in Patients With GM1 Gangliosidosis

GM1 Gangliosidosis

United States
University of British Columbia
Medical University of Graz; Hospital de Clinicas de Porto Alegre

Unknown

Natural History of Morquio B and Late-Onset of GM1 Gangliosidosis

Morquio B Disease | GM1 Gangliosidosis Type III

Canada
LYSOGENE

Terminated

A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis

GM1 Gangliosidosis

United Kingdom, United States, France
University of Minnesota
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators

Terminated

Synergistic Enteral Regimen for Treatment of the Gangliosidoses (Syner-G)

Tay-Sachs Disease | Sandhoff Disease | GM1 Gangliosidoses | GM2 Gangliosidoses

United States
Idorsia Pharmaceuticals Ltd.

Completed

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

GM2 Gangliosidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Gaucher Disease, Type 2 | AB Variant Gangliosidosis GM2

United States, Spain, Germany, Italy, Belgium, Brazil, France, Portugal, Switzerland, United Kingdom
Sun Yat-sen University

Recruiting

GM1 Prophylaxis for WBRT Related Cognitive Dysfunction (GLORY)

Breast Cancer | Cognitive Impairment | Brain Metastases | Radiotherapy Side Effect | Gangliosidosis, GM1

China
University of Minnesota
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators

Recruiting

A Natural History Study of the Gangliosidoses

GM2 Gangliosidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Late Onset Tay-Sachs Disease

United States
Azafaros A.G.

Active, not recruiting

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

GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease

United States, Brazil, France, Germany, Italy, United Kingdom
Memorial Sloan Kettering Cancer Center
National Cancer Institute (NCI)

Completed

Immunization With a Pentavalent Vaccine Composed of KLH-conjugates of GD2L, GD3L, Globo H, Fucosyl GM1, and N-Propionylated Polysialic Acid

Lung Cancer

United States
Qilu Pharmaceutical Co., Ltd.

Not yet recruiting

A Multicenter, Randomized, Double-blind Phase II Trial to Evaluate GM1 Prevention of Peripheral Neuropathy in Patients With Breast Cancer

To Evaluate the Effectiveness of GM1 in the Prevention of Albumin-paclitaxel Therapy Induced Peripheral Neuropathy in Breast Cancer Patients
Henan Cancer Hospital

Withdrawn

GM1 in the Treatment of Neurotoxicity Induced by Albumin-bound Paclitaxel

Early Breast Cancer

China
Iovance Biotherapeutics, Inc.

Recruiting

A Study to Investigate the Efficacy and Safety of an Infusion of IOV-4001 in Adult Participants With Unresectable or Metastatic Melanoma or Stage III or IV Non-small-cell Lung Cancer

Metastatic Melanoma | Unresectable Melanoma | Stage IV Non-small Cell Lung Cancer | Stage III Non-small Cell Lung Cancer

United States
Sutro Biopharma, Inc.

Recruiting

Study of STRO-002, an Anti-Folate Receptor Alpha (FolRα) Antibody Drug Conjugate in Ovarian & Endometrial Cancers

Ovarian Cancer | Fallopian Tube Cancer | Endometrial Cancer | Ovarian Carcinoma | Endometrioid Adenocarcinoma | Ovary Cancer | Primary Peritoneal Carcinoma

United States, Spain
Sun Yat-sen University

Recruiting

GM1 Prophylaxis for Post-chemotherapy Cognitive Impairment in Patients With Early Operable Breast Cancer

Breast Cancer | Chemotherapy-Related Cognitive Impairment

China
SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt)

Recruiting

Registry Gangliosidoses

Gangliosidoses | Galactosialidosis | Sialidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Morquio B Disease | Gm2-Gangliosidosis, Variant B1 | GM2 Activator Deficiency

Germany
Orphalan

Completed

Study Comparing Once Daily Dose of 900mg of TETA 4HCL Against Cuprior® (450mg Trientine Base, Twice Daily).

Wilson's Disease

United Kingdom
National Human Genome Research Institute (NHGRI)

Recruiting

Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders

Myoclonus | Neurological Regression | Cherry Red Spot | Brain Atrophy

United States
Bristol-Myers Squibb

Completed

BMS-986012 in Relapsed/Refractory SCLC

Small Cell Lung Cancer

United States, Belgium, Canada, Puerto Rico, Australia, Korea, Republic of, Netherlands
Masonic Cancer Center, University of Minnesota

Completed

HSCT for High Risk Inherited Inborn Errors

Wolman Disease | Adrenoleukodystrophy | Metachromatic Leukodystrophy | Sanfilippo Syndrome | GM1 Gangliosidosis | Globoid Cell Leukodystrophy | Tay Sachs Disease | Sandhoffs Disease | I-Cell Disease

United States
University of Pittsburgh

Recruiting

Longitudinal Study of Neurodegenerative Disorders

Lysosomal Storage Diseases | Leukodystrophy | Niemann-Pick Diseases | Batten Disease | Gaucher Disease | Krabbe Disease | Alpha-Mannosidosis | Osteopetrosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | GM1 Gangliosidoses | MPS I | MPS II | Vanishing White Matter Disease | Multiple Sulfatase Deficiency... and other conditions

United States
Bristol-Myers Squibb

Recruiting

A Study of BMS-986012 in Combination With Carboplatin, Etoposide, and Nivolumab as First-line Therapy in Extensive-stage Small Cell Lung Cancer

Extensive-stage Small Cell Lung Cancer

Netherlands, Belgium, United States, Australia, Greece, Italy, Canada, Japan, Poland, Romania, Spain
Masonic Cancer Center, University of Minnesota

Completed

Stem Cell Transplant for Inborn Errors of Metabolism

Niemann-Pick Disease | Wolman Disease | Adrenoleukodystrophy | Batten Disease | Metachromatic Leukodystrophy | GM1 Gangliosidosis | Fucosidosis | Globoid Cell Leukodystrophy | Gaucher's Disease | Tay Sachs Disease | Sandhoff Disease

United States
Masonic Cancer Center, University of Minnesota

Completed

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

Lysosomal Storage Disease | Peroxisomal Disorder

United States
Albert Einstein College of Medicine
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Enrolling by invitation

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program (ScreenPlus)

Mucopolysaccharidosis II | Fabry Disease | Niemann-Pick Disease, Type C | Mucopolysaccharidosis VI | Lysosomal Acid Lipase Deficiency | Metachromatic Leukodystrophy | Gaucher Disease | Cerebrotendinous Xanthomatosis | Mucopolysaccharidosis IV A | GM1 Gangliosidosis | Mucopolysaccharidosis VII | Acid Sphingomyelinase... and other conditions

United States
Aldagen

Terminated

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases (UCBT-002)

Mucopolysaccharidosis | Inborn Errors of Metabolism | Lysosomal Storage Disorders | Inherited Metabolic Diseases | Peroxisomal Storage Diseases

United States
Tehran University of Medical Sciences
Mashhad University of Medical Sciences; Kashan University of Medical Sciences

Recruiting

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

Supportive Care | GM2 Gangliosidosis

Iran, Islamic Republic of
Children's Hospital of Philadelphia
Eli Lilly and Company; University of Pennsylvania; Takeda; National Institute of... and other collaborators

Recruiting

The Myelin Disorders Biorepository Project (MDBP)

Mucopolysaccharidoses | Leukoencephalopathies | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2... and other conditions

United States
Joanne Kurtzberg, MD
The Marcus Foundation

Recruiting

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells (DUOC-01)

Mucopolysaccharidosis II | Brain Diseases, Metabolic, Inborn | Adrenoleukodystrophy | Batten Disease | Leukodystrophy, Metachromatic | Leukodystrophy, Globoid Cell | Alpha-Mannosidosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | Neimann Pick Disease | Sanfilippo Mucopolysaccharidoses

United States
Children's Hospital of Philadelphia
Illumina, Inc.

Active, not recruiting

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

Mucopolysaccharidoses | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2 Gangliosidosis | Zellweger... and other conditions

United States
Massachusetts General Hospital

Active, not recruiting

A Natural History of Late Onset Tay-Sachs Disease

GM2 Gangliosidosis

United States
IntraBio Inc

Completed

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

GM2 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease

United States, Germany, Spain, United Kingdom
Azafaros A.G.

Active, not recruiting

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 (RAINBOW)

Niemann-Pick Disease, Type C | GM2 Gangliosidosis

Brazil
Genzyme, a Sanofi Company

Active, not recruiting

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

Tay-Sachs Disease | Sandhoff Disease

United States, Argentina, Czechia, Portugal, Russian Federation, Turkey, United Kingdom, Brazil, France, Germany, Italy, Japan, Spain
Terence Flotte
Massachusetts General Hospital; University of Massachusetts, Worcester

Active, not recruiting

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

Tay-Sachs Disease | Sandhoff Disease

United States
Dr. Anupam Sehgal
Taysha Gene Therapies, Inc.; GlycoNet

Active, not recruiting

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

Infantile GM2 Gangliosidosis (Disorder)

Canada

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