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Sponsored by: |
Amicus Therapeutics |
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Information provided by: | Amicus Therapeutics |
ClinicalTrials.gov Identifier: | NCT00446550 |
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 |
Estimated Study Completion Date: | May 2009 |
Estimated Primary Completion Date: | May 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.
Ages Eligible for Study: | 18 Years to 74 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
United States, California | |
University of California San Francisco | Not yet recruiting |
San Francisco, California, United States, 94143-0749 | |
Contact: Seymour Packman, MD 415-476-2871 spackman@peds.ucsf.edu | |
Principal Investigator: Seymour Packman, MD | |
Tower Hematology Oncology Medical Group - Comprehensive Gaucher Treatment Center | Recruiting |
Beverly Hills, California, United States, 90211 | |
Contact: Cheryl Elzinga 888-248-4456 ElzingaC@toweroncology.com | |
Principal Investigator: Barry Rosenbloom, MD | |
United States, Florida | |
University Research Foundation for Lysosomal Storage Diseases, Inc. | Recruiting |
Coral Springs, Florida, United States, 33065 | |
Contact: Lisa Constantin 954-755-1904 ext 115 lisa@nwoncology.com | |
Principal Investigator: Neal Weinreb, MD | |
United States, Georgia | |
Emory University Lysosomal Storage Disease Ctr | Recruiting |
Decatur, Georgia, United States, 30033 | |
Contact: Karen Grinzaid 404-778-8565 kgrinzaid@genetics.emory.edu | |
Principal Investigator: Paul Fernhoff, MD | |
United States, Iowa | |
University of Iowa | Recruiting |
Iowa City, Iowa, United States, 52242 | |
Contact: Myrl Holida 319-356-2007 myrl-holida@uiowa.edu | |
Principal Investigator: Thomas Loew, MD | |
United States, Kansas | |
University of Kansas Medical Center | Recruiting |
Kansas City, Kansas, United States, 66160 | |
Contact: Majed Dasouki, MD 913-588-6326 mdasouki@kumc.edu | |
Principal Investigator: Majed Dasouki, MD | |
United States, Massachusetts | |
Genetics & Metabolism Center for Human Genetics | Recruiting |
Boston, Massachusetts, United States, 02114 | |
Contact: Virginia Clarke 617-726-8830 vclarke1@partners.org | |
Principal Investigator: Marsha Browning, MD | |
United States, Minnesota | |
University of Minnesota | Recruiting |
Minneapolis, Minnesota, United States, 55455 | |
Contact: Lisa Hostetler 612-825-7422 hoste005@umn.edu | |
Principal Investigator: Chester Whitley, MD PhD | |
United States, New York | |
New York University School of Medicine | Not yet recruiting |
New York, New York, United States, 10016 | |
Contact: Michele Ford 212-263-8344 Michele.Ford@nyumc.org | |
Principal Investigator: Greg Pastores, MD | |
United States, Ohio | |
Lysosomal Storage Disease Center | Not yet recruiting |
Cincinnati, Ohio, United States, 45229 | |
Contact: Laurie Bailey 513-636-4507 laurie.bailey@cchmc.org | |
Principal Investigator: Nancy Leslie, MD | |
United States, Pennsylvania | |
Children's Hospital of Pittsburgh | Recruiting |
Pittsburgh, Pennsylvania, United States, 15213 | |
Contact: Michele Graham 800-334-7980 michele.graham@chp.edu | |
Principal Investigator: David Feingold, MD | |
Children's Hospital of Philadelphia | Recruiting |
Philadelphia, Pennsylvania, United States, 19104 | |
Contact: Kathy Gilmartin 267-426-5441 gilmartin@email.chop.edu | |
Principal Investigator: Can Ficicioglu, MD | |
Germany | |
Universitats-Kinderklinik | Recruiting |
Mainz, Germany, 55101 | |
Contact: Michael Beck, MD 49 6131 172398 beck@kinder.klinik.uni-mainz.de | |
Principal Investigator: Michael Beck, MD | |
Israel | |
Rambam Health Care Campus - Hematology and Bone Marrow Transplant Department | Recruiting |
Haifa, Israel | |
Contact: Hanna Rosenbaum, MD 972-4-8542343 h_rosenbaum@rambam.health.gov.il | |
Principal Investigator: Hanna Rosenbaum, MD | |
Schneider Children's Medical Center of Israel | Recruiting |
Petah-Tikvah, Israel | |
Contact: Talli Shrieber 972-3-9253781 tallish@clalit.org.il | |
Principal Investigator: Ian Cohen, MD | |
United Kingdom | |
Addenbrooke's Hospital, Cambridge University Hospitals NHS Trust | Recruiting |
Cambridge, United Kingdom, CB2 2QQ | |
Contact: Elizabeth Morris 44 1223 336864 liz.morris@addenbrookes.nhs.uk | |
Principal Investigator: Timothy Cox, MD, PhD | |
The Royal Free Hospital | Recruiting |
London, United Kingdom, WC1N 3BG | |
Contact: Alan Milligan 44 2074 726409 alan.milligan@royalfree.nhs.uk | |
Principal Investigator: Atul Mehta, MD |
Study Director: | Eugene Schneider, MD | Amicus Therapeutics |
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: | November 24, 2008 |
ClinicalTrials.gov Identifier: | NCT00446550 |
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 |
Lipid Metabolism, Inborn Errors Sphingolipidoses Metabolic Diseases Lysosomal Storage Diseases Sphingolipidosis Central Nervous System Diseases Brain Diseases Lymphatic Diseases |
Metabolism, Inborn Errors Genetic Diseases, Inborn Brain Diseases, Metabolic, Inborn Lipidoses Metabolic disorder Gaucher Disease Lipid Metabolism Disorders Brain Diseases, Metabolic |
Reticuloendotheliosis Lysosomal Storage Diseases, Nervous System Nervous System Diseases |