Clinical Trials on GLYCOGEN STORAGE DISEASE IXa1

Total 7541 results

Ohio State University Comprehensive Cancer Center
National Cancer Institute (NCI)

Withdrawn

Pilot Trial of E Cigarettes in Pts Diagnosed With Cancer of the Head, Neck, and Lung

Head and Neck Squamous Cell Carcinoma | Lung Non-Small Cell Carcinoma | Stage III Lung Cancer AJCC v8 | Stage II Lung Cancer AJCC v8 | Stage IIA Lung Cancer AJCC v8 | Stage IIB Lung Cancer AJCC v8 | Stage IIIA Lung Cancer AJCC v8 | Stage IIIB Lung Cancer AJCC v8 | Stage I Lung Cancer AJCC v8 | Stage IA1... and other conditions

United States
Jonsson Comprehensive Cancer Center
Blue Note Therapeutics

Terminated

Cognitive-Behavioral Stress Management Device for the Treatment of Anxiety and Depressive Symptoms in Patients With Stage I-III Breast or Lung Cancer (BNT001)

Anatomic Stage I Breast Cancer AJCC v8 | Anatomic Stage IA Breast Cancer AJCC v8 | Anatomic Stage IB Breast Cancer AJCC v8 | Anatomic Stage II Breast Cancer AJCC v8 | Anatomic Stage IIA Breast Cancer AJCC v8 | Anatomic Stage IIB Breast Cancer AJCC v8 | Anatomic Stage III Breast Cancer AJCC v8 | Anatomic... 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
GOG Foundation
National Cancer Institute (NCI)

Active, not recruiting

Studying the Physical Function and Quality of Life Before and After Surgery in Patients With Stage I Cervical Cancer

Lymphedema | Cervical Adenocarcinoma | Cervical Adenosquamous Carcinoma | Cervical Squamous Cell Carcinoma | Stage IA2 Cervical Cancer AJCC v6 and v7 | Stage IB1 Cervical Cancer AJCC v6 and v7 | Stage IA1 Cervical Cancer AJCC v6 and v7

United States, Korea, Republic of, Canada
M.D. Anderson Cancer Center

Recruiting

Infertility Survey Among Reproductive Age Women With Gynecological and Breast Cancer

Anatomic Stage I Breast Cancer AJCC v8 | Anatomic Stage IA Breast Cancer AJCC v8 | Anatomic Stage IB Breast Cancer AJCC v8 | Anatomic Stage II Breast Cancer AJCC v8 | Anatomic Stage IIA Breast Cancer AJCC v8 | Anatomic Stage IIB Breast Cancer AJCC v8 | Anatomic Stage III Breast Cancer AJCC v8 | Anatomic... and other conditions

United States
Translational Research Center for Medical Innovation...

Recruiting

International Registry Study of Neutral Lipid Storage Disease (NLSD) / Triglyceride Deposit Cardiomyovasculopathy (TGCV) and Related Diseases

Neutral Lipid Storage Disease

China, United States, France, Austria, Germany, Italy, Japan, Netherlands, United Kingdom
Alexion Pharmaceuticals

Terminated

A Study to Identify and Characterize LAL-D Patients in High-risk Populations

Lysosomal Acid Lipase Deficiency

United States
Alexion Pharmaceuticals

Completed

Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency

Lysosomal Acid Lipase Deficiency

Spain, Germany, Italy, United States, Croatia, Canada, Russian Federation, Denmark, United Kingdom, Belgium, Mexico, Australia, Netherlands, Brazil, Turkey
Alexion Pharmaceuticals

Terminated

Screening for Lysosomal Acid Lipase Deficiency

Lysosomal Acid Lipase Deficiency

United States
Alexion Pharmaceuticals

No longer available

An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency

Lysosomal Acid Lipase Deficiency
SWOG Cancer Research Network
National Cancer Institute (NCI)

Active, not recruiting

Treatment Effects on Development of Chemotherapy-Induced Peripheral Neuropathy in Patients With Cancer

Anatomic Stage I Breast Cancer AJCC v8 | Anatomic Stage IA Breast Cancer AJCC v8 | Anatomic Stage IB Breast Cancer AJCC v8 | Anatomic Stage II Breast Cancer AJCC v8 | Anatomic Stage IIA Breast Cancer AJCC v8 | Anatomic Stage IIB Breast Cancer AJCC v8 | Anatomic Stage III Breast Cancer AJCC v8 | Anatomic... and other conditions

United States, Colombia, Mexico, Chile
Alexion Pharmaceuticals

Completed

Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency (ARISE)

Lysosomal Acid Lipase Deficiency

France, Poland, United Kingdom, Spain, Mexico, Turkey, Japan, Australia, Russian Federation, United States, Germany, Italy, Czechia, Argentina, Croatia
Massachusetts General Hospital
Alexion Pharmaceuticals

Unknown

Identification of Undiagnosed Lysosomal Acid Lipase Deficiency

Cholesterol Ester Storage Disease | Lysosomal Acid Lipase Deficiency

United States
M.D. Anderson Cancer Center
National Cancer Institute (NCI)

Recruiting

Role of Gut Microbiome and Fecal Transplant on Medication-Induced GI Complications in Patients With Cancer

Malignant Solid Neoplasm | Colitis | Stage IVA Lung Cancer AJCC v8 | Stage IVB Lung Cancer AJCC v8 | Lung Non-Small Cell Carcinoma | Stage III Lung Cancer AJCC v8 | Stage IV Lung Cancer AJCC v8 | Stage II Lung Cancer AJCC v8 | Stage IIA Lung Cancer AJCC v8 | Stage IIB Lung Cancer AJCC v8 | Stage IIIA Lung... and other conditions

United States
Orchard Therapeutics
Ospedale San Raffaele

Active, not recruiting

A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)

Lysosomal Storage Disease | Metachromatic Leukodystrophy

Italy
Orchard Therapeutics
Ospedale San Raffaele

Active, not recruiting

Gene Therapy for Metachromatic Leukodystrophy (MLD)

Lysosomal Storage Disease | Metachromatic Leukodystrophy

Italy
University of Aarhus
The Danish Dairy Research Foundation, Denmark

Completed

Breakfast for Young Women (GoDag)

Obesity | Wolman Disease | Nutrition

Denmark
Fondazione SISA (Societa Italiana per lo Studio...

Recruiting

Lipa Gene Mutation in PED-LIPIGEN (Pediatric FH Subjects)

Lysosomal Acid Lipase Deficiency

Italy
Genzyme, a Sanofi Company
CRL/Medinet

Terminated

Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

Fabry Disease | Chronic Kidney Disease, Stage IV (Severe)

United States
Assy Nimer

Unknown

Novel Association of Cholesterol Ester Storage Disease Due to Lysosomal Acid Lipase Deficiency and Non-Alcoholic Fatty Liver Disease: A Prospective Clinical Study

Non-alcoholic Fatty Liver Disease | Cholesterol Ester Storage Disease

Israel
Hospices Civils de Lyon

Unknown

Study of the Prevalence of Fabry Disease in French Dialysis Patients (FABRYDIAL)

End Stage Renal Disease | Fabry Disease | Renal Dialysis

France
Alexion

Completed

A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype

Wolman Disease | Lysosomal Acid Lipase Deficiency

United States, Canada, United Kingdom, Italy, France
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Wolman Disease (BioWolman) (BioWolman)

Cholesterol Ester Storage Disease | Acid Lipase Deficiency | Acid Cholesteryl Ester Hydrolase Deficiency, Wolman Type

Germany, India, Sri Lanka
M.D. Anderson Cancer Center

Active, not recruiting

Stereotactic Body Radiation Therapy With or Without Nivolumab in Treating Patients With Stage I-IIA or Recurrent Non-small Cell Lung Cancer

Lung Non-Small Cell Squamous Carcinoma | Stage IIA Lung Cancer AJCC v8 | Neuroendocrine Carcinoma | Lung Atypical Carcinoid Tumor | Minimally Invasive Lung Adenocarcinoma | Lung Neuroendocrine Neoplasm | Stage I Lung Cancer AJCC v8 | Stage IA1 Lung Cancer AJCC v8 | Stage IA2 Lung Cancer AJCC v8 | Stage... and other conditions

United States
Assiut University

Not yet recruiting

Prevalence and Risk Factors of Women Mental Health Disorders

Psychiatric Problem | Wolman Disease
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
Rutgers, The State University of New Jersey
National Cancer Institute (NCI)

Withdrawn

Web-Based Coping and Communication Skills Intervention in Improving Psychological Adaptation in Patients With Gynecological Cancer

Stage II Uterine Corpus Cancer AJCC v7 | Uterine Carcinosarcoma | Endometrial Carcinoma | Stage II Cervical Cancer AJCC v7 | Stage IIA Cervical Cancer AJCC v7 | Stage IIB Cervical Cancer AJCC v6 and v7 | Stage IIIB Cervical Cancer AJCC v6 and v7 | Stage IVA Cervical Cancer AJCC v6 and v7 | Stage 0 Fallopian... and other conditions

United States
Mayo Clinic
National Cancer Institute (NCI)

Active, not recruiting

Characterizing Chemo-Radiotherapy Treatment-Related Cardiac Changes

Stage III Lung Cancer AJCC v8 | Stage II Lung Cancer AJCC v8 | Stage IIA Lung Cancer AJCC v8 | Stage IIB Lung Cancer AJCC v8 | Stage IIIA Lung Cancer AJCC v8 | Stage IIIB Lung Cancer AJCC v8 | Stage I Lung Cancer AJCC v8 | Stage IA1 Lung Cancer AJCC v8 | Stage IA2 Lung Cancer AJCC v8 | Stage IA3 Lung Cancer... and other conditions

United States
Hospices Civils de Lyon

Unknown

Assessement of the Prevalence of Lysosomal Acid Lipase Deficiency in Liver Post-transplant Patients (LALTH-2)

Liver Post-transplant Patients

France
City of Hope Medical Center
National Cancer Institute (NCI)

Active, not recruiting

Remote Telemonitoring of Patient-Generated Physiologic Health Data and Patient-Reported Outcomes

Stage IV Prostate Cancer AJCC v8 | Hepatobiliary Neoplasm | Stage III Renal Cell Cancer AJCC v8 | Stage IV Renal Cell Cancer AJCC v8 | Stage III Uterine Corpus Cancer AJCC v8 | Stage IV Uterine Corpus Cancer AJCC v8 | Stage IVA Uterine Corpus Cancer AJCC v8 | Stage IVB Uterine Corpus Cancer AJCC v8 | Malignant Neoplasm and other conditions

United States
Hospices Civils de Lyon

Unknown

Assessment of the Prevalence of Lysosomal Acid Lipase Deficiency in Patients Waiting for a Liver Transplant. (LALTH-1)

Patients Waiting for a Liver Transplant.

France
O & O Alpan LLC

Unknown

Molecular and Cellular Mechanisms of Lysosomal Storage Diseases

Lysosomal Storage Disorders

United States
Takeda

Active, not recruiting

A Study Measuring Substances Potentially Indicating Bone Problems in Adults With Type 1 Gaucher Condition

Gaucher Disease

Spain
CENTOGENE GmbH Rostock

Active, not recruiting

Omics Gaucher Study: Multiomic Approach (OmicsGaucher)

Gaucher Disease

Germany
Lysosomal and Rare Disorders Research and Treatment...

Unknown

SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease

Gaucher Disease

United States
Pfizer

Completed

A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

Gaucher Disease

Israel, South Africa, Paraguay
Pfizer

Completed

A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease

Gaucher Disease

United States, Chile, United Kingdom, Israel, Canada, Spain, South Africa, Italy
Hospital St. Joseph, Marseille, France

Completed

Auto-antibodies Prevalence and CD1 Role in Gaucher Disease

Gaucher Disease

France
Assistance Publique - Hôpitaux de Paris

Completed

Cellular and Molecular Mechanisms Governing Bone Marrow Stem Cells in Gaucher Disease (MGMC)

Gaucher Disease

France
Amicus Therapeutics

Completed

Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease

Gaucher Disease

United States
Shire

Recruiting

Gaucher Disease Outcome Survey (GOS)

Gaucher Disease

United States
Shire

Terminated

A Study of VPRIV in Participants With Gaucher Disease Previously Treated With Other Enzyme Replacement Therapies or Substrate Reduction Therapies

Gaucher Disease

Canada
Ain Shams University

Completed

Role of Vitamin E in Gaucher Disease Patients

Gaucher Disease

Egypt
Takeda

Completed

A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy (SWITCH)

Gaucher Disease

United States
Genzyme, a Sanofi Company

Completed

Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies (EXOSKEL)

Gaucher Disease

Canada, Russian Federation, Tunisia
Shire

Completed

Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease

Gaucher Disease

Japan
Pfizer

Completed

Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

Gaucher Disease

United Kingdom, Canada, Israel, United States, Australia, Spain
Pfizer

Completed

Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

Gaucher Disease

Chile, United Kingdom, Canada, Spain, Israel, United States, Australia, South Africa
Pfizer

Completed

A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

Gaucher Disease

Israel, Paraguay, South Africa
Yasmeen Abd Elbaset Hamzaa

Unknown

Clinical Audit of Managment of Gausher Disease in Children

Gaucher Disease

Clinical Trials on GLYCOGEN STORAGE DISEASE IXa1

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CROs in Dominican Republic

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Rare Diseases

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