Clinical Trials on Cholesterol Ester Storage Disease

Total 7654 results

Thomas Jefferson University

Suspended

A Vaccine (Ad5.F35-hGCC-PADRE) for the Treatment of Gastrointestinal Adenocarcinoma

Malignant Solid Neoplasm | Gastric Adenocarcinoma | Pancreatic Ductal Adenocarcinoma | Stage II Pancreatic Cancer AJCC v8 | Stage III Pancreatic Cancer AJCC v8 | Colorectal Adenocarcinoma | Small Intestinal Adenocarcinoma | Clinical Stage III Gastric Cancer AJCC v8 | Stage IV Colorectal Cancer AJCC v8 | Stage IVA Colorectal Cancer AJCC... and other conditions

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

Recruiting

Endocrine Therapy Fulvestrant & Palbociclib or Aromatase Inhibitor Therapy in Treating Older Patients With Hormone Responsive Breast Cancer That Cannot Be Removed by Surgery

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 | HER2/Neu Negative | Estrogen Receptor and/or Progesterone Receptor Positive

United States
Academic and Community Cancer Research United
National Cancer Institute (NCI)

Active, not recruiting

Panitumumab, Regorafenib, or TAS-102, in Treating Patients With Metastatic and/or Unresectable RAS Wild-Type Colorectal Cancer

Metastatic Colon Adenocarcinoma | Metastatic Colorectal Carcinoma | Metastatic Rectal Adenocarcinoma | Stage III Colon Cancer AJCC v8 | Stage III Rectal Cancer AJCC v8 | Stage IIIA Colon Cancer AJCC v8 | Stage IIIA Rectal Cancer AJCC v8 | Stage IIIB Colon Cancer AJCC v8 | Stage IIIB Rectal Cancer AJCC... and other conditions

United States
University of Washington
National Cancer Institute (NCI)

Terminated

IGFBP-2 Vaccine and Combination Chemotherapy in Treating Patients With Stage III-IV Ovarian, Fallopian Tube, or Primary Peritoneal Cancer Undergoing Surgery

Stage IIIA Fallopian Tube Cancer | Stage IIIA Ovarian Cancer | Stage IIIA Primary Peritoneal Cancer | Stage IIIB Fallopian Tube Cancer | Stage IIIB Ovarian Cancer | Stage IIIB Primary Peritoneal Cancer | Stage IIIC Fallopian Tube Cancer | Stage IIIC Ovarian Cancer | Stage IIIC Primary Peritoneal Cancer and other conditions

United States
Jonsson Comprehensive Cancer Center
Bayer; Translational Research in Oncology-U.S. Inc. (TRIO-US)

Terminated

Copanlisib, Letrozole, and Palbociclib in Treating Patients With Hormone Receptor Positive HER2 Negative Stage I-IV Breast Cancer

Stage I Breast Cancer | Stage IV Breast Cancer | Stage II Breast Cancer | Stage IIIA Breast Cancer | Stage IIIB Breast Cancer | Invasive Breast Carcinoma | Stage IA Breast Cancer | Stage IB Breast Cancer | Stage IIA Breast Cancer | Stage IIB Breast Cancer | Stage IIIC Breast Cancer | Estrogen Receptor Positive | HER2/Neu Negative and other conditions

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

Active, not recruiting

Online Psychosocial Intervention in Improving Social Well-Being and Support in Women With Stage I-IV Non-small Cell Lung Cancer Undergoing Treatment

Stage IVA Lung Cancer AJCC v8 | Stage IVB Lung Cancer AJCC v8 | 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 Cancer AJCC v8 | Stage IIIB Lung Cancer AJCC v8 | Stage I Lung Cancer... and other conditions

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

Active, not recruiting

Nab-paclitaxel and Alpelisib for the Treatment of Anthracycline Refractory Triple Negative Breast Cancer With PIK3CA or PTEN Alterations

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

Active, not recruiting

Intensity Modulated Radiation Therapy or 3-Dimensional Conformal Radiation Therapy in Treating Patients With Lymph-Node Positive Breast Cancer

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 Stage IIIA Breast Cancer AJCC v8 | Anatomic Stage IIIB Breast Cancer AJCC v8 | Anatomic Stage IIIC Breast Cancer AJCC... and other conditions

United States
M.D. Anderson Cancer Center

Recruiting

Canakinumab for the Prevention of Lung Cancer, the Can-Prevent-Lung Trial

Stage III Lung Cancer AJCC v8 | Lung Carcinoma | 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... and other conditions

United States
Radboud University Medical Center

Unknown

Muscle Relaxation in Myopathies With Positive Muscle Phenomena

Myotonic Dystrophy Type 2 | McArdle Disease | Nemaline Myopathy Type 6
Children's Hospital of Fudan University

Not yet recruiting

Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease

Fabry Disease

China
Westfälische Wilhelms-Universität Münster
Chiesi GmbH

Recruiting

German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa (GoPEG)

Fabry Disease

Germany
ISU Abxis Co., Ltd.

Recruiting

Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease

Fabry Disease

Korea, Republic of
Sanofi

Recruiting

Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan

Fabry Disease

Taiwan
Bio Sidus SA

Recruiting

Switch Over Study of Biosimilar AGA for Fabry Disease (SMILE)

Fabry Disease

Argentina
Amicus Therapeutics

Recruiting

A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

Fabry Disease

United States
University Health Network, Toronto
Ozmosis Research Inc.

Completed

Autologous Stem Cell Transplantation of Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease

Fabry Disease

Canada
4D Molecular Therapeutics

Active, not recruiting

4D-310 in Adults With Fabry Disease and Cardiac Involvement

Fabry Disease

Taiwan, Australia
Bio Sidus SA

Completed

PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion

Fabry Disease

Argentina
Takeda

Active, not recruiting

Early Detection of Fabry Disease

Fabry Disease

Germany
Takeda

Completed

A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease

Fabry Disease

China
AVROBIO

Terminated

Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease

Fabry Disease

United States, Australia, Brazil
National and Kapodistrian University of Athens

Completed

Pulsatility Index, Vasomotor Reactivity and Leukoencephalopathy in Fabry Patients

Fabry Disease

Greece
Genzyme, a Sanofi Company

Completed

China Post-marketing Surveillance (PMS) Study of Fabrazyme®

Fabry Disease

China
Protalix
Chiesi Farmaceutici S.p.A.

Completed

Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks (BRIGHT)

Fabry Disease

United States, Denmark, Belgium, United Kingdom, Norway, Italy, Czechia
Protalix
Chiesi Farmaceutici S.p.A.

Completed

Extension Study of PRX-102 for up to 60 Months

Fabry Disease

United States, Spain, United Kingdom, Paraguay
Sanofi

Withdrawn

To Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease (BCLEAR1)

Fabry Disease
Chiesi Farmaceutici S.p.A.
Iqvia Pty Ltd; Protalix

Completed

Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion (PEOPLE)

Fabry Disease

United States, Belgium, Denmark, Italy, United Kingdom
University of Utah
Genzyme, a Sanofi Company

Completed

Pre-Clinical White Matter Changes and Associated Connectivity Effects in Fabry Disease

Fabry Disease

United States
Amicus Therapeutics

Completed

Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)

Fabry Disease

United Kingdom, United States
Shire

Completed

Fabry Outcome Survey (FOS) (FOS)

Fabry Disease

United States
Manchester University NHS Foundation Trust
Northern Care Alliance NHS Foundation Trust

Completed

Characterisation of Heart Involvement in Fabry Disease With T1 Mapping (T1)

Fabry Disease

United Kingdom
Children's Hospital of Fudan University
First Affiliated Hospital, Sun Yat-Sen University; Wuhan Union Hospital, China and other collaborators

Withdrawn

Fabry Aim Children Early (ACE) Project

Fabry Disease

China
Shire

Completed

Replagal Enzyme Replacement Therapy for Children With Fabry Disease

Fabry Disease

Canada, United States
Shire

Completed

Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease

Fabry Disease

United States, United Kingdom, Australia, Czechia, Finland, Paraguay, Poland, Slovenia
Shire

Completed

Safety Study of Replagal® Therapy in Children With Fabry Disease

Fabry Disease

United States
Shire

Completed

Sophisticated Assessment of Disease Burden in Patients With Fabry Disease (SOPHIA)

Fabry Disease

Finland, Sweden, Germany, Belgium, Czechia, Denmark
CENTOGENE GmbH Rostock
Shire Human Genetic Therapies, Inc.

Completed

Stroke in Young Fabry Patients (sifap2): Characterization of the Stroke Rehabilitation (sifap2)

Fabry Disease | Cerebrovascular Accident

Austria, Croatia, France, Germany, Poland, Portugal, Georgia
Shire

Completed

Home Therapy With Replagal in Fabry Disease

Fabry Disease

Austria, Germany
Amicus Therapeutics

Approved for marketing

Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease

Fabry Disease

United States, Australia
Amicus Therapeutics

Completed

A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

Fabry Disease

Australia, Brazil
Amicus Therapeutics

Completed

A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

Fabry Disease

United States
Amicus Therapeutics

Terminated

Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study

Fabry Disease

United Kingdom, United States, Australia, Brazil, France
Amicus Therapeutics

Completed

A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

Fabry Disease

France, United Kingdom
Chiayi Christian Hospital
Sanofi

Unknown

Epidemiological Study of Fabry Disease in Taiwan Young Stroke Patients

Fabry Disease
National Institute of Neurological Disorders and...

Completed

Study to Collect Data on Fabry Disease Patients With Enhanceable Alpha-Galactosidase A Activity

Fabry Disease

United States
Spital Linth
University of Zurich; National University, Singapore

Unknown

Lipidomics and Functional Analyses of Platelets in Fabry Disease

Fabry Disease

Switzerland
University Hospital, Bordeaux

Unknown

Study of the Relation Between Lipid Myocardial Overload Evaluated by Cardiac Magnetic Resonance Imaging (MRI), Alteration of Longitudinal Myocardial Deformations by Echocardiography, and Clinical Achievements (Functional, Biological and Electrical) in Fabry Disease, and Its Outcomes. (FABRY-Image)

Fabry Disease

France
O & O Alpan LLC

Completed

Immune Response in Subjects With Fabry Disease Who Are Switching From Agalsidase Alfa to Agalsidase Beta

Fabry Disease

United States
Ohio State University
Genzyme, a Sanofi Company

Completed

Establishment of Biomarkers for Fabry Disease

Fabry Disease

United States

Clinical Trials on Cholesterol Ester Storage Disease

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Cameroon

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations