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| Sponsor: | FDA Office of Orphan Products Development |
|---|---|
| Collaborator: |
Children's Hospital Medical Center, Cincinnati |
| Information provided by: | FDA Office of Orphan Products Development |
| ClinicalTrials.gov Identifier: | NCT00004488 |
Purpose
OBJECTIVES:
I. Determine the efficacy of alendronate sodium in treating osteopenia (generalized bone density and focal bone lesions) in patients with Gaucher's disease.
| Condition | Intervention | Phase |
|---|---|---|
|
Gaucher's Disease Osteopenia |
Drug: alendronate sodium Drug: calcium carbonate Drug: cholecalciferol |
Phase II |
| Study Type: | Interventional |
| Study Design: | Treatment, Randomized, Double-Blind, Placebo Control, Single Group Assignment, Efficacy Study |
| Estimated Enrollment: | 82 |
| Study Start Date: | October 1998 |
PROTOCOL OUTLINE:
This is a randomized, double blind, placebo controlled study.
All patients receive oral calcium carbonate and cholecalciferol daily. Patients are randomized to receive oral alendronate sodium or placebo daily for 24 months.
Patients are followed every 6 months for 2 years.
Eligibility| Ages Eligible for Study: | 18 Years to 50 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Diagnosis of type 1 Gaucher's disease enzymatically proven to have acid beta glucosidase deficiency
Must have been on stable dose (8-60 U/kg/every 2 weeks) of alglucerase enzyme therapy (Cerezyme or Ceredase) for at least 24 months
Lumbar spine bone density below the mean for age, sex, and race
--Prior/Concurrent Therapy--
At least 6 months since prior medications that directly affect skeletal metabolism including, but not limited to, bisphosphonates, calcitonin, parathyroid hormone, or estrogen
--Patient Characteristics--
Renal: No chronic renal failure; No recurrent renal stones
Esophageal: No history of dysphagia; No frequent heartburn; No esophagitis requiring treatment
Other: No untreated hyperthyroidism or hypothyroidism; No concurrent hyperparathyroidism; No concurrent malignancy; No history of alcohol or drug abuse; Not pregnant; Negative pregnancy test; Fertile patients must use effective contraception
Contacts and Locations| United States, Ohio | |
| Children's Hospital Medical Center - Cincinnati | Recruiting |
| Cincinnati, Ohio, United States, 45229-3039 | |
| Contact: Richard J. Wenstrup 800-647-4805 | |
| Wright State University School of Medicine | Recruiting |
| Dayton, Ohio, United States, 45401 | |
| Contact: Shumei S. Guo 513-873-2933 | |
| Israel | |
| Shaare Zedek Medical Center | Recruiting |
| Jerusalem, Israel, 91031 | |
| Contact: Ari Zimran 972-2-6555-111 | |
| Study Chair: | Richard J. Wenstrup | Children's Hospital Medical Center, Cincinnati |
More Information
| Study ID Numbers: | 199/14269, CHMC-C-FDR001537, CHMC-C-498 |
| Study First Received: | October 18, 1999 |
| Last Updated: | June 23, 2005 |
| ClinicalTrials.gov Identifier: | NCT00004488 History of Changes |
| Health Authority: | United States: Federal Government |
|
Gaucher's disease inborn errors of metabolism rare disease sphingolipidoses |
|
Lipid Metabolism, Inborn Errors Sphingolipidoses Molecular Mechanisms of Pharmacological Action Physiological Effects of Drugs Bone Density Conservation Agents Calcium Carbonate Brain Diseases Bone Diseases Metabolism, Inborn Errors Musculoskeletal Diseases Alendronate Vitamins Micronutrients Brain Diseases, Metabolic, Inborn Cholecalciferol |
Reticuloendotheliosis Metabolic Diseases Lysosomal Storage Diseases, Nervous System Growth Substances Lysosomal Storage Diseases Nervous System Diseases Central Nervous System Diseases Bone Diseases, Metabolic Pharmacologic Actions Lymphatic Diseases Genetic Diseases, Inborn Antacids Lipidoses Gaucher Disease Brain Diseases, Metabolic |
