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| Sponsor: | Amicus Therapeutics |
|---|---|
| Information provided by: | Amicus Therapeutics |
| ClinicalTrials.gov Identifier: | NCT00446550 |
Purpose
This study is being conducted to evaluate the safety and effects of AT2101 in patients with type I Gaucher disease who are not receiving ERT or SRT.
| Condition | Intervention | Phase |
|---|---|---|
|
Gaucher Disease, Type 1 |
Drug: AT2101 |
Phase II |
| Study Type: | Interventional |
| Study Design: | Treatment, Randomized, Open Label, Dose Comparison, Parallel Assignment, Safety Study |
| Official Title: | A Randomized, Open-label Study To Assess the Safety and Tolerability of AT2101 in Treatment-naive Adult Patients With Type I Gaucher Disease |
| Estimated Enrollment: | 16 |
| Study Start Date: | December 2007 |
| Study Completion Date: | August 2009 |
| Primary Completion Date: | August 2009 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
1: Experimental
AT2101 dose regimen 1
|
Drug: AT2101
Arm 1: AT2101 oral capsules, dose 1, regimen 1 Arm 2: AT2101 oral capsules, dose 1, regimen 2 |
|
2: Experimental
AT2101 dose regimen 2
|
Drug: AT2101
Arm 1: AT2101 oral capsules, dose 1, regimen 1 Arm 2: AT2101 oral capsules, dose 1, regimen 2 |
Gaucher disease is a lysosomal storage disorder resulting from a deficiency in the key enzyme beta-glucocerebrosidase (GCase). The enzyme deficiency is caused by genetic mutations, which can result in the production of misfolded GCase. AT2101 is designed to act as a pharmacological chaperone by selectively binding to the misfolded GCase and helping it fold correctly, which may restore GCase activity.
This study is being conducted to test the safety of AT2101 in patients with type I Gaucher disease who have not already received enzyme replacement therapy (ERT) or substrate reduction therapy (SRT), or who have not received ERT or SRT in the 12 months before screening. This study will also evaluate the effects of AT2101 on parameters that are commonly abnormal in Gaucher disease. The study will involve 9 visits over 29 weeks.
Eligibility| Ages Eligible for Study: | 18 Years to 74 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| United States, California | |
| University of California San Francisco | |
| San Francisco, California, United States, 94143-0749 | |
| Tower Hematology Oncology Medical Group - Comprehensive Gaucher Treatment Center | |
| Beverly Hills, California, United States, 90211 | |
| United States, Florida | |
| University Research Foundation for Lysosomal Storage Diseases, Inc. | |
| Coral Springs, Florida, United States, 33065 | |
| United States, Georgia | |
| Emory University Lysosomal Storage Disease Ctr | |
| Decatur, Georgia, United States, 30033 | |
| United States, Iowa | |
| University of Iowa | |
| Iowa City, Iowa, United States, 52242 | |
| United States, Kansas | |
| University of Kansas Medical Center | |
| Kansas City, Kansas, United States, 66160 | |
| United States, Massachusetts | |
| Genetics & Metabolism Center for Human Genetics | |
| Boston, Massachusetts, United States, 02114 | |
| United States, Minnesota | |
| University of Minnesota | |
| Minneapolis, Minnesota, United States, 55455 | |
| United States, New York | |
| New York University School of Medicine | |
| New York, New York, United States, 10016 | |
| United States, Ohio | |
| Lysosomal Storage Disease Center | |
| Cincinnati, Ohio, United States, 45229 | |
| United States, Pennsylvania | |
| Children's Hospital of Pittsburgh | |
| Pittsburgh, Pennsylvania, United States, 15213 | |
| Children's Hospital of Philadelphia | |
| Philadelphia, Pennsylvania, United States, 19104 | |
| Germany | |
| Universitats-Kinderklinik | |
| Mainz, Germany, 55101 | |
| Israel | |
| Rambam Health Care Campus - Hematology and Bone Marrow Transplant Department | |
| Haifa, Israel | |
| Schneider Children's Medical Center of Israel | |
| Petah-Tikvah, Israel | |
| South Africa | |
| Morningside Clinic | |
| Johannesburg, South Africa | |
| United Kingdom | |
| Addenbrooke's Hospital, Cambridge University Hospitals NHS Trust | |
| Cambridge, United Kingdom, CB2 2QQ | |
| The Royal Free Hospital | |
| London, United Kingdom, WC1N 3BG | |
| Study Director: | Eugene Schneider, MD | Amicus Therapeutics |
More Information
| Responsible Party: | Amicus Therapeutics ( Eugene Schneider, MD / Medical Director, Clinical Research ) |
| Study ID Numbers: | GAU-CL-202 |
| Study First Received: | March 9, 2007 |
| Last Updated: | September 10, 2009 |
| ClinicalTrials.gov Identifier: | NCT00446550 History of Changes |
| Health Authority: | United States: Food and Drug Administration; United Kingdom: Medicines and Healthcare Products Regulatory Agency; Germany: Federal Institute for Drugs and Medical Devices; Israel: Ministry of Health; South Africa: Medicines Control Council |
|
Lipid Metabolism, Inborn Errors Sphingolipidoses Metabolic Diseases Reticuloendotheliosis Lysosomal Storage Diseases, Nervous System Lysosomal Storage Diseases Nervous System Diseases Central Nervous System Diseases Brain Diseases |
Lymphatic Diseases Metabolism, Inborn Errors Genetic Diseases, Inborn Brain Diseases, Metabolic, Inborn Lipidoses Gaucher Disease Lipid Metabolism Disorders Brain Diseases, Metabolic |