Clinical Trials on GM3 Synthase-deficiëntie

Total 98 results

Mendel Tuchman
Children's Hospital of Philadelphia; University of California, Los Angeles; Icahn... and other collaborators

Completed

Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia (STO)

Methylmalonic Acidemia | Carbamoyl-Phosphate Synthase I Deficiency Disease | Propionic Acidemia, Type I and/or Type II | Ornithine Carbamoyltransferase Deficiency

United States
Children's Oncology Group
National Cancer Institute (NCI)

Completed

Biomarkers in Bone Marrow Samples From Patients With Acute Promyelocytic Leukemia

Adult Acute Promyelocytic Leukemia (M3) | Childhood Acute Promyelocytic Leukemia (M3) | Adult Acute Myeloid Leukemia With t(15;17)(q22;q12) | Adult Acute Myeloid Leukemia

United States
University of Jena

Unknown

Jena Sepsis Registry (JenaSepsisReg)

Severe Sepsis or Septic Shock (ICD-10-GM, R65.0, R65.1)

Germany
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
National Institute of Allergy and Infectious Diseases...

Active, not recruiting

Natural History of Severe Allergic Inflammation and Reactions

PGM3 Deficiency | Eosinophilic and/or Atopic Dermatitis | OSMR Deficiency | Primary Localized Cutaneous Amyloidosis | Hereditary Alpha-tryptasemia

United States
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
AIO-Studien-gGmbH
Novartis Pharmaceuticals; Assign Data Management and Biostatistics GmbH

Completed

Safety and Tolerability of Everolimus as Second-line Treatment in Poorly Differentiated Neuroendocrine Carcinoma / Neuroendocrine Carcinoma G3 (WHO 2010) and Neuroendocrine Tumor G3 - an Investigator Initiated Phase II Study (EVINEC)

Neuroendocrine Carcinoma, Grade 3 | Poorly Differentiated Malignant Neuroendocrine Carcinoma | Neuroendocrine Carcinoma, Grade 1 [Well-differentiated Neuroendocrine Carcinoma] That Switched to G3 | Neuroendocrine Carcinoma, Grade 2 [Moderately Differentiated Neuroendocrine Carcinoma]... and other conditions

Germany
Cabaletta Bio

Recruiting

RESET-MG: A Study to Evaluate the Safety and Efficacy of CABA-201 in Participants With Generalized Myasthenia Gravis

Generalized Myasthenia Gravis (gMG)

United States
Alexion Pharmaceuticals, Inc.

Recruiting

Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG)

Generalized Myasthenia Gravis | gMG

Spain, Italy, United States, France, Switzerland, Serbia, Japan, Netherlands
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
University of Minnesota
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators

Completed

Gene Therapy for Tay-Sachs Disease

Tay Sachs Disease | Sandhoff Disease | Late Onset Tay Sachs Disease

United States
Wake Forest University Health Sciences
National Cancer Institute (NCI)

Approved for marketing

Gemtuzumab Ozogamicin in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Acute Promyelocytic Leukemia

Recurrent Adult Acute Myeloid Leukemia | Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid Leukemia With Del(5q) | Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) | Adult Acute Myeloid Leukemia With t(16;16)(p13;q22) | Adult Acute Myeloid Leukemia With t(8... and other conditions

United States
Sun Yat-sen University

Recruiting

The Effect of GM-CSF to Preventing Oral Mucositis for Patients With Nasopharyngeal Carcinoma Receiving Radiotherapy

GM-CSF

China
National Center for Research Resources (NCRR)
Icahn School of Medicine at Mount Sinai

Completed

Diagnostic and Screening Study of Genetic Disorders

Metabolism, Inborn Errors | Leukodystrophy, Globoid Cell | Tay-Sachs Disease | Porphyria, Erythropoietic

United States
Merck Sharp & Dohme LLC

Completed

Immunogenicity and Safety of Concomitant and Non-Concomitant Administration of RotaTeq® (V260) and Inactivated Poliomyelitis Vaccine in Healthy Chinese Infants (V260-074)

Prevention of Rotavirus Gastroenteritis in Infants and Children Caused by Serotypes G1, G2, G3, G4, and G9

China
National Cancer Institute (NCI)

Active, not recruiting

Talimogene Laherparepvec and Radiation Therapy in Treating Patients With Newly Diagnosed Soft Tissue Sarcoma That Can Be Removed by Surgery

Soft Tissue Sarcoma | Leiomyosarcoma | Liposarcoma | Undifferentiated Pleomorphic Sarcoma | Soft Tissue Sarcoma of the Trunk and Extremities | Stage I Soft Tissue Sarcoma AJCC v7 | Stage II Soft Tissue Sarcoma AJCC v7 | Sarcoma G2 | Sarcoma G3

United States, Canada
Chia Tai Tianqing Pharmaceutical Group Co., Ltd.

Unknown

A Study of Anlotinib in Patients With Gastroenteropancreatic Neuroendocrine Tumor G3

Gastroenteropancreatic Neuroendocrine Tumor G3

China
McGill University Health Centre/Research Institute...

Completed

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

Familial Dysautonomia | Tay Sachs Disease | Canavan Disease

Canada
National Cancer Institute (NCI)

Completed

Selumetinib in Treating Patients With Recurrent or Refractory Acute Myeloid Leukemia

Myelodysplastic Syndromes | Recurrent Adult Acute Myeloid Leukemia | Secondary Acute Myeloid Leukemia | Adult Acute Promyelocytic Leukemia (M3) | Adult Acute Myeloid Leukemia With t(15;17)(q22;q12) | Myelodysplastic/Myeloproliferative Neoplasms

United States
Rigshospitalet, Denmark

Unknown

Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy

Carbohydrate Metabolism, Inborn Errors | Metabolism, Inborn Errors | Lipid Metabolism, Inborn Errors | Glycogen Storage Disease Type II | Glycogen Storage Disease Type V | VLCAD Deficiency | Glycogen Storage Disease Type III | Phosphoglycerate Kinase Deficiency | Neutral Lipid Storage Disease | Carnitine... and other conditions

Denmark
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
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 Washington
National Cancer Institute (NCI)

Completed

Clofarabine, Cytarabine, and G-CSF in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia

Acute Myeloid Leukemia | Recurrent Adult Acute Myeloid Leukemia | Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) | Adult Acute Myeloid Leukemia With t(16;16)(p13;q22) | Adult Acute Myeloid Leukemia With t(8;21)(q22;q22) | Adult... and other conditions

United States
Alexion Pharmaceuticals, Inc.

Not yet recruiting

Study of Ultomiris® (Ravulizumab) Safety in Pregnancy

Pregnancy | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Atypical Hemolytic Uremic Syndrome (aHUS) | Ultomiris-exposed Pregnant/ Postpartum | Generalized Myasthenia Gravis (gMG) | Neuromyelitis Optica Spectrum Disorder (NMOSD)
Wake Forest University Health Sciences
National Cancer Institute (NCI)

Withdrawn

Gemtuzumab Ozogamicin in Treating Patients With Acute Myeloid Leukemia

Recurrent Adult Acute Myeloid Leukemia | Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid Leukemia With Del(5q) | Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) | Adult Acute Myeloid Leukemia With t(16;16)(p13;q22) | Adult Acute Myeloid Leukemia With t(8... and other conditions
National Cancer Institute (NCI)

Completed

Romidepsin in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia

Recurrent Adult Acute Myeloid Leukemia | Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid Leukemia With Del(5q) | Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) | Adult Acute Myeloid Leukemia With t(16;16)(p13;q22) | Adult Acute Myeloid Leukemia With t(8... and other conditions

United States
National Cancer Institute (NCI)

Terminated

Decitabine in Treating Children With Relapsed or Refractory Acute Myeloid Leukemia or Acute Lymphoblastic Leukemia

Secondary Acute Myeloid Leukemia | Childhood Acute Myeloblastic Leukemia With Maturation (M2) | Childhood Acute Promyelocytic Leukemia (M3) | Recurrent Childhood Acute Lymphoblastic Leukemia | Recurrent Childhood Acute Myeloid Leukemia

United States
Centre Hospitalier Universitaire de Liege
Sanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaborators

Recruiting

Baby Detect : Genomic Newborn Screening

Congenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditions

Belgium
National Human Genome Research Institute (NHGRI)

Recruiting

A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders

Metabolic Disease | Purine-Pyrimidine Metabolism | AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2 | UNG, OMIM *191525, Hyper-IgM Syndrome 5 | NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency | UMPS, OMIM *613891, Orotic Aciduria | DHODH, OMIM *126064,... and other conditions

United States
National Cancer Institute (NCI)

Completed

Bortezomib in Treating Young Patients With Refractory or Recurrent Leukemia

Childhood Acute Promyelocytic Leukemia (M3) | Recurrent Childhood Acute Lymphoblastic Leukemia | Recurrent Childhood Acute Myeloid Leukemia | Blastic Phase Chronic Myelogenous Leukemia

United States
RTI International
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Enrolling by invitation

Early Check: Expanded Screening in Newborns

Primary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease 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
Fred Hutchinson Cancer Center
National Cancer Institute (NCI)

Terminated

Bortezomib in Treating Patients With High-Risk Acute Myeloid Leukemia in Remission

Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome | Adult Acute Minimally Differentiated Myeloid Leukemia (M0) | Adult Acute Myeloblastic Leukemia Without Maturation (M1) | Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid... and other conditions

United States
National Cancer Institute (NCI)

Completed

PS-341 in Treating Patients With Refractory or Relapsed Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Chronic Myeloid Leukemia in Blast Phase, or Myelodysplastic Syndrome

Recurrent Adult Acute Myeloid Leukemia | Adult Acute Promyelocytic Leukemia (M3) | Previously Treated Myelodysplastic Syndromes | Recurrent Adult Acute Lymphoblastic Leukemia | Relapsing Chronic Myelogenous Leukemia | Refractory Anemia With Excess Blasts | Blastic Phase Chronic Myelogenous Leukemia and other conditions

United States
National Cancer Institute (NCI)

Completed

BMS-214662 in Treating Patients With Acute Leukemia, Myelodysplastic Syndrome, or Chronic Myeloid Leukemia

Recurrent Adult Acute Myeloid Leukemia | Adult Acute Promyelocytic Leukemia (M3) | Childhood Myelodysplastic Syndromes | Previously Treated Myelodysplastic Syndromes | Recurrent Adult Acute Lymphoblastic Leukemia | Recurrent Childhood Acute Lymphoblastic Leukemia | Recurrent Childhood Acute Myeloid... and other conditions

United States
Talaris Therapeutics Inc.
Duke University

Terminated

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

Niemann-Pick Disease | Alpha-mannosidosis | Tay Sachs Disease | Sandhoff Disease | Metachromatic Leukodystrophy (MLD) | Hurler-Scheie Syndrome | Hurler Syndrome (MPS I) | Hunter Syndrome (MPS II) | Sanfilippo Syndrome (MPS III) | Krabbe Disease (Globoid Leukodystrophy) | Adrenoleukodystrophy (ALD and AMN) | Pelizaeus...

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
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
Nohla Therapeutics, Inc.
National Cancer Institute (NCI); Fred Hutchinson Cancer Center

Completed

Clofarabine, Cytarabine, and Filgrastim Followed by Infusion of Non-HLA Matched Ex Vivo Expanded Cord Blood Progenitors in Treating Patients With Acute Myeloid Leukemia

Recurrent Adult Acute Myeloid Leukemia | Adult Acute Megakaryoblastic Leukemia (M7) | Adult Acute Minimally Differentiated Myeloid Leukemia (M0) | Adult Acute Monoblastic Leukemia (M5a) | Adult Acute Monocytic Leukemia (M5b) | Adult Acute Myeloblastic Leukemia With Maturation (M2) | Adult Acute... and other conditions

United States
National Cancer Institute (NCI)

Completed

Vorinostat in Treating Patients With Acute Myeloid Leukemia

Recurrent Adult Acute Myeloid Leukemia | Adult Acute Megakaryoblastic Leukemia (M7) | Adult Acute Minimally Differentiated Myeloid Leukemia (M0) | Adult Acute Monoblastic Leukemia (M5a) | Adult Acute Monocytic Leukemia (M5b) | Adult Acute Myeloblastic Leukemia With Maturation (M2) | Adult Acute... and other conditions

United States
National Cancer Institute (NCI)

Completed

Vorinostat and Idarubicin in Treating Patients With Relapsed or Refractory Leukemia or Myelodysplastic Syndromes

Recurrent Adult Acute Myeloid Leukemia | Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome | Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) | Adult Acute Myeloid Leukemia With t(16;16)(p13... and other conditions

United States
National Cancer Institute (NCI)

Completed

Vorinostat With or Without Isotretinoin in Treating Young Patients With Recurrent or Refractory Solid Tumors, Lymphoma, or Leukemia

Unspecified Childhood Solid Tumor, Protocol Specific | Juvenile Myelomonocytic Leukemia | Recurrent Childhood Medulloblastoma | Childhood Atypical Teratoid/Rhabdoid Tumor | Childhood Acute Promyelocytic Leukemia (M3) | Childhood Chronic Myelogenous Leukemia | Recurrent Childhood Acute Lymphoblastic... and other conditions

United States
St. Jude Children's Research Hospital
Novartis Pharmaceuticals

Completed

SJDAWN: St. Jude Children's Research Hospital Phase 1 Study Evaluating Molecularly-Driven Doublet Therapies for Children and Young Adults With Recurrent Brain Tumors

Glioma | Neoplasms | Glioblastoma | Neoplasms, Neuroepithelial | Central Nervous System Neoplasms | Brain Cancer | Brain Tumor | Ependymoma | Medulloblastoma | Anaplastic Astrocytoma | Anaplastic Ganglioglioma | Anaplastic Ependymoma | Anaplastic Oligodendroglioma | Pineoblastoma | Recurrent Medulloblastoma | CNS Tumor | Pediatric... and other conditions

United States
National Cancer Institute (NCI)

Completed

7-Hydroxystaurosporine and Perifosine in Treating Patients With Relapsed or Refractory Acute Leukemia, Chronic Myelogenous Leukemia or High Risk Myelodysplastic Syndromes

Recurrent Adult Acute Myeloid Leukemia | Adult Acute Megakaryoblastic Leukemia (M7) | Adult Acute Minimally Differentiated Myeloid Leukemia (M0) | Adult Acute Monoblastic Leukemia (M5a) | Adult Acute Monocytic Leukemia (M5b) | Adult Acute Myeloblastic Leukemia With Maturation (M2) | Adult Acute... and other conditions

United States
National Cancer Institute (NCI)

Completed

SB-715992 in Treating Patients With Acute Leukemia, Chronic Myelogenous Leukemia, or Advanced Myelodysplastic Syndromes

Recurrent Adult Acute Myeloid Leukemia | Adult Acute Megakaryoblastic Leukemia (M7) | Adult Acute Minimally Differentiated Myeloid Leukemia (M0) | Adult Acute Monoblastic Leukemia (M5a) | Adult Acute Monocytic Leukemia (M5b) | Adult Acute Myeloblastic Leukemia With Maturation (M2) | Adult Acute... and other conditions

United States
Case Comprehensive Cancer Center

Completed

High-Dose Busulfan and High-Dose Cyclophosphamide Followed By Donor Bone Marrow Transplant in Treating Patients With Leukemia, Myelodysplastic Syndrome, Multiple Myeloma, or Recurrent Hodgkin or Non-Hodgkin Lymphoma

Recurrent Adult Acute Myeloid Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma | Recurrent Adult Diffuse Large Cell Lymphoma | Recurrent Adult Diffuse Mixed Cell Lymphoma | Recurrent... and other conditions

United States
National Cancer Institute (NCI)

Completed

Vorinostat, Cytarabine, and Etoposide in Treating Patients With Relapsed and/or Refractory Acute Leukemia or Myelodysplastic Syndromes or Myeloproliferative Disorders

Primary Myelofibrosis | Polycythemia Vera | Essential Thrombocythemia | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Adult Acute Megakaryoblastic Leukemia (M7) | Adult Acute Minimally Differentiated Myeloid Leukemia (M0) | Adult Acute Monoblastic Leukemia (M5a) | Adult... and other conditions

United States
City of Hope Medical Center
National Cancer Institute (NCI)

Completed

Vaccine Therapy in Preventing Cytomegalovirus Infection in Patients With Hematological Malignancies Undergoing Donor Stem Cell Transplant

Primary Myelofibrosis | Polycythemia Vera | Essential Thrombocythemia | Stage I Multiple Myeloma | Stage II Multiple Myeloma | Stage III Multiple Myeloma | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid... and other conditions

United States

Clinical Trials on GM3 Synthase-deficiëntie

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