Clinical Trials on Glycogen Storage Disease Type VI

Total 58641 results

National Institute of Neurological Disorders and...

Completed

The Classification and Cause of Leukodystrophies of Unknown Cause

Lysosomal Storage Disease

United States, France, Israel, Netherlands
Maastricht University Medical Center

Completed

The Effect of Fibrate Therapy in Two Patients With Neutral Lipid Storage Disease With Myopathy (NLSDM) (NLSDM)

Neutral Lipid Storage Disease

Netherlands
Alexion Pharmaceuticals

Completed

Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency

Lysosomal Acid Lipase Deficiency | Cholesterol Ester Storage Disease(CESD) | LAL-Deficiency

France, United States, United Kingdom, Czechia
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
Alexion Pharmaceuticals

Completed

Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency

Lysosomal Acid Lipase Deficiency | LAL-Deficiency | Cholesterol Ester Storage Disease (CESD)

France, United Kingdom, United States, Canada, Czechia
Lawson Health Research Institute
Sequana Medical N.V.

Recruiting

Interdialytic Peritoneal UltraFiltration in HemoDialysis Patients (IPUF-HD)

Hemodialysis | Peritoneal Dialysis | End Stage Renal Disease on Dialysis | Nonosmotic Sodium Storage

Canada
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
Northern Care Alliance NHS Foundation Trust
Brighter AB

Completed

Actiste® Diabetes Management as a Service (ADMS) Clinical Investigation (ADMSUK01)

Diabetes type1 | Diabetes type2

United Kingdom
New York Medical College

Completed

Human Placental-Derived Stem Cell Transplantation (HPDSC)

Mucopolysaccharidosis I | Myelodysplastic Syndrome | Acute Lymphocytic Leukemia | Acute Myelogenous Leukemia | Niemann-Pick Disease | Mucopolysaccharidosis VI | Wolman Disease | Adrenoleukodystrophy | Batten Disease | Metachromatic Leukodystrophy | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Fucosidosis | Gaucher... and other conditions

United States
Eisai Inc.

Terminated

Phase 2 Study of the Poly(ADP-Ribose) Polymerase Inhibitor E7016 in Combination With Temozolomide in Subjects With Wild Type BRAF Stage IV or Unresectable Stage III Melanoma

Unresectable Stage III Melanoma | Wild Type BRAF Stage IV Melanoma

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

Recruiting

Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1)

Gaucher Disease Type 1

United States
Chung-Hsing Wang
Sanofi

Not yet recruiting

Screening for Gaucher Disease and Acid Sphingomyelinase Deficiency

Gaucher Disease

Taiwan
LYSOGENE

Completed

Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy

Mucopolysaccharidosis Type III A | Sanfilippo Disease Type A

France
LYSOGENE

Completed

Intracerebral Gene Therapy for Sanfilippo Type A Syndrome

Mucopolysaccharidosis Type III A | Sanfilippo Disease Type A

France
Eline C. B. Eskes

Recruiting

Biomarkers Of Lung InVolvement In ASMD (BOLIVIA)

Asmd, Visceral Type

Netherlands
University of Minnesota

Recruiting

Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease

Inflammation | Oxidative Stress | Gaucher Disease Type I

United States
Charite University, Berlin, Germany

Completed

Morphometric, Clinical and Histological Study on One Stage Bone Augmentation Using BoneCeramic (BCP) and Collagen Membranes (BCP+Collagen)

Dehiscent Type of Alveolar Bone Defect | One Stage Bone Augmentation
Istanbul University
Biruni University

Not yet recruiting

Daily Living Activities and Exercise Capacity in Lipid Storage Diseases

Lipid Storage Disease

Turkey
Sanofi

Recruiting

Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France (OPERA)

Acid Sphingomyelinase Deficiency (ASMD)

France
Sanofi

Completed

A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)

Sphingomyelin Lipidosis

Italy, Czechia, Belgium, France, Portugal, Turkey, United States, Argentina, Brazil, Chile, Germany, Romania, Spain
National Institute of Neurological Disorders and...

Completed

The Natural History and Pathogenesis of Mucolipidosis Type IV

Mucolipidosis Type IV

United States
National Taiwan University Hospital

Recruiting

The Natural History of Sialidosis Type I

Observational Study

Taiwan
Jonsson Comprehensive Cancer Center
National Cancer Institute (NCI); Genzyme, a Sanofi Company

Withdrawn

Sargramostim or Hypertonic Saline Before Sentinel Lymph Node Biopsy in Treating Patients With Stage IB-II Melanoma

Stage IIB Melanoma | Stage IIC Melanoma | Stage IB Melanoma | Stage IIA Melanoma

United States
Sanofi

Approved for marketing

Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)

Sphingomyelin Lipidosis
Genzyme, a Sanofi Company

Completed

Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency (ASCEND-Peds)

Sphingomyelin Lipidosis

Brazil, France, Italy, United States, Germany, United Kingdom
Masonic Cancer Center, University of Minnesota

Completed

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Mucopolysaccharidosis | Hurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Krabbe Disease | Adrenoleukodystrophy (ALD) | Hunter Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Metachromatic Leukodystrophy (MLD) | Maroteaux-Lamy Syndrome

United States
Cinnagen

Completed

Efficacy and Safety of P046 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients

Mucopolysaccharidosis Type 1

Iran, Islamic Republic of
Nationwide Children's Hospital
Sanfilippo Children's Research Foundation; The Sanfilippo Research Foundation; The Children's Medical Research Foundation

Completed

Natural History Studies of Mucopolysaccharidosis III

Mucopolysaccharidosis Type IIIA | Mucopolysaccharidosis Type IIIB

United States
Genzyme, a Sanofi Company

Completed

Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency (ASCEND)

Sphingomyelin Lipidosis

United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Chile, France, Germany, Italy, Japan, Netherlands, Portugal, Spain, Tunisia, Turkey, United Kingdom
Genzyme, a Sanofi Company

Completed

A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

Sphingomyelin Lipidosis

United States, Belgium, Brazil, France, Germany, Italy, United Kingdom
Genzyme, a Sanofi Company

Completed

Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients

Human Acid Sphingomyelinase Deficiency

United Kingdom, United States
National Cancer Institute (NCI)

Completed

Vaccine Therapy in Treating HLA-A2 Positive Patients With Melanoma

Recurrent Melanoma | Stage IIIA Melanoma | Stage IIIB Melanoma | Stage IIIC Melanoma | Stage IIB Melanoma | Stage IIC Melanoma | Stage IA Melanoma | Stage IB Melanoma | Stage IIA Melanoma

United States
Fudan University

Unknown

Nab-P and Gem Compared With Gem and Tegafur in Adjuvant Chemotherapy After Radical Resection of Pancreatic Cancer

Stage IA Pancreatic Adenocarcinoma | Stage IB Pancreatic Adenocarcinoma | Stage IIA Pancreatic Adenocarcinoma | Stage IIB Pancreatic Adenocarcinoma

China
Hanmi Pharmaceutical Company Limited

Unknown

Multiple Ascending Dose Study of HM12470 in Type 1 and Type 2 Diabetes Mellitus

Type2 Diabetes Mellitus | Type1 Diabetes Mellitus

United States
Eunice Kennedy Shriver National Institute of Child...

Recruiting

Study of Inborn Errors of Cholesterol Synthesis and Related Disorders

Lysosomal Storage Disease | Cholesterol Metabolism

United States
Albert Einstein College of Medicine
National Cancer Institute (NCI)

Terminated

Gemcitabine Hydrochloride, Docetaxel, and Radiation Therapy in Treating Patients With Uterine Sarcoma That Has Been Removed By Surgery

Uterine Corpus Leiomyosarcoma | Stage IIA Uterine Sarcoma | Stage IIB Uterine Sarcoma | Stage IIIA Uterine Sarcoma | Stage IIIB Uterine Sarcoma | Stage IIIC Uterine Sarcoma | Stage IVA Uterine Sarcoma | Stage IVB Uterine Sarcoma | Stage IA Uterine Sarcoma | Stage IB Uterine Sarcoma | Stage IC Uterine Sarcoma

United States
Mayo Clinic
National Cancer Institute (NCI)

Completed

Vaccine Therapy and Resiquimod in Treating Patients With Stage II-IV Melanoma That Has Been Removed By Surgery

Recurrent Melanoma | Stage IV Melanoma | Stage IIIA Melanoma | Stage IIIB Melanoma | Stage IIIC Melanoma | Stage IIB Melanoma | Stage IIC Melanoma | Stage IIA Melanoma

United States
Joanne Kurtzberg, MD
Aldagen

Withdrawn

Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases (IUHST-001)

Lysosomal Storage Diseases | Inborn Errors of Metabolism

United States
Bo Feldt-Rasmussen
Novo Nordisk A/S; The GCP unit at Copenhagen University Hospital

Completed

Liraglutide Treatment to Patients With Severe Renal Insufficiency

Type 2 Diabetes Mellitus | End-stage Renal Disease

Denmark
National Human Genome Research Institute (NHGRI)

Enrolling by invitation

Genetic Studies of Lysosomal Storage Disorders

Parkinson Disease | Gaucher Disease | Lysosomal Storage Disorders

United States
Ohio State University Comprehensive Cancer Center
Alliance for Clinical Trials in Oncology

Recruiting

Telephone-Based Intervention in Increasing Adherence to Adjuvant Hormonal Therapy in Patients With Breast Cancer

Stage 0 Breast Cancer | Stage IIIA Breast Cancer | Stage IIIB Breast Cancer | Stage IA Breast Cancer | Stage IB Breast Cancer | Stage IIA Breast Cancer | Stage IIB Breast Cancer | Stage IIIC Breast Cancer

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

Active, not recruiting

Busulfan, Melphalan, and Stem Cell Transplant After Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma

Stage 4S Neuroblastoma | Ganglioneuroblastoma | Stage 2A Neuroblastoma | Stage 2B Neuroblastoma | Stage 3 Neuroblastoma | Stage 4 Neuroblastoma | Stage 1 Neuroblastoma | Stage 2 Neuroblastoma

United States, Canada, Australia, New Zealand
National Cancer Institute (NCI)

Withdrawn

RO4929097 in Treating Patients With Stage IIIB, Stage IIIC, or Stage IV Melanoma That Can Be Removed by Surgery

Stage IV Melanoma | Stage IIIB Melanoma | Stage IIIC Melanoma

United States
Sanofi

Recruiting

A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen (OPPUS)

Acid SphingoMyelinase Deficiency | Gaucher Disease, Splenomegaly

France
Xinhua Hospital, Shanghai Jiao Tong University...

Unknown

Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease

Niemann-Pick Disease, Type C1

China
Allievex Corporation

Completed

A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Mucopolysaccharidosis Type IIIB | Mucopolysaccharidosis Type 3 B | MPS III B | MPS 3 B

United States, Spain, Turkey, Taiwan, Australia, Colombia, Germany, United Kingdom
Rutgers, The State University of New Jersey
National Cancer Institute (NCI)

Completed

Web-Based Decision Aid in Improving Informed Decisions in Patients With Stage 0-IIIA Breast Cancer Considering Contralateral Prophylactic Mastectomy

Stage 0 Breast Cancer | Stage I Breast Cancer | Stage II Breast Cancer | Stage IIIA Breast Cancer | Stage IA Breast Cancer | Stage IB Breast Cancer | Stage IIA Breast Cancer | Stage IIB Breast Cancer

United States
University Medical Center Groningen

Not yet recruiting

Sentinel Lymph Node Detection in Patients With Stage Ib-III Melanoma Using MSOT and ICG (MelanooMSOT)

Melanoma Stage III | Melanoma, Stage II | Melanoma, Stage I
Masonic Cancer Center, University of Minnesota

Recruiting

Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.

Mucopolysaccharidosis Type I | Hematopoietic Cell Transplantation

United States
Oxford University Hospitals NHS Trust
University of Oxford; Barts & The London NHS Trust; Ninewells Hospital

Completed

Exploring Unmet Needs and Attitudes to Skin Self-examination in Melanoma Survivors (EUNASS)

Melanoma Stage III | Melanoma, Stage II | Melanoma, Stage I

United Kingdom

Clinical Trials on Glycogen Storage Disease Type VI

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