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Sponsored by: |
Genzyme |
Information provided by: | Genzyme |
ClinicalTrials.gov Identifier: | NCT00358150 |
Gaucher disease is a genetic disease that results in a deficiency of an enzyme acid β-glucosidase, also known as glucocerebrosidase. This enzyme is needed to digest a substrate (lipid) called glucosylceramide and, to a lesser degree, glucosphingosine. In patients with Gaucher disease, the liver, spleen, bone marrow and brain show increases in lipid concentration, specifically in cells derived from the monocyte/macrophage system.
Genz-112638 is an oral drug that may regulate the Gaucher disease process by decreasing the synthesis of glucosylceramide. This study was designed to determine the efficacy, safety and pharmacokinetics of Genz-112638 in men and women with Type 1 Gaucher disease.
Each patient's duration of participation is expected to be approximately 26 months. After Day 1, each patient is required to return to the study center for 12 additional study visits post-Baseline for efficacy, safety and PK assessments.
Condition | Intervention | Phase |
Gaucher Disease, Type 1 Cerebroside Lipidosis Syndrome Glucocerebrosidase Deficiency Disease Glucosylceramide Beta-Glucosidase Deficiency Disease Gaucher Disease, Non-Neuronopathic Form |
Drug: Genz-112638 |
Phase II |
MedlinePlus related topics: | Gaucher's Disease |
Drug Information available for: | Alglucerase Imiglucerase |
Study Type: | Interventional |
Study Design: | Treatment, Open Label, Single Group Assignment, Safety/Efficacy Study |
Official Title: | A Phase 2, Open-Label, Multi-Center Study Evaluating the Efficacy, Safety and Pharmacokinetics of Genz-112638 in Gaucher Type 1 Patients |
Enrollment: | 23 |
Study Start Date: | June 2006 |
Estimated Study Completion Date: | October 2009 |
Ages Eligible for Study: | 18 Years to 65 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
INCLUSION CRITERIA:
EXCLUSION CRITERIA:
United States, New York | |||||
New York, New York, United States | |||||
Argentina | |||||
Buenos Aires, Argentina | |||||
Israel | |||||
Jerusalem, Israel | |||||
Haifa, Israel | |||||
Mexico | |||||
Mexico City, Mexico | |||||
Russian Federation | |||||
Moscow, Russian Federation |
Genzyme |
Study Director: | Judith Peterschmitt, M.D. (US Medical Monitor) | Genzyme |
Study Director: | Ana Cristina Scheidt Puga, M.D. Ph.D (EU Medical Monitor) | Genzyme |
Responsible Party: | Genzyme ( Medical Monitor ) |
Study ID Numbers: | GZGD00304 |
First Received: | July 27, 2006 |
Last Updated: | June 19, 2008 |
ClinicalTrials.gov Identifier: | NCT00358150 |
Health Authority: | United States: Food and Drug Administration; Russia: Pharmacological Committee, Ministry of Health; Israel: Israeli Health Ministry Pharmaceutical Administration; Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica; Mexico: Federal Commission for Protection Against Health Risks |
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