Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease
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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 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Intervention Model: Single Group Assignment Masking: Double-Blind Primary Purpose: Treatment |
| Estimated Enrollment: | 82 |
| Study Start Date: | October 1998 |
| Estimated Study Completion Date: | September 2002 |
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.
Completion date provided represents the completion date of the grant per OOPD records
Eligibility| Ages Eligible for Study: | 18 Years to 50 Years (Adult) |
| Sexes Eligible for Study: | All |
| 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 LocationsPlease refer to this study by its ClinicalTrials.gov identifier: NCT00004488
| United States, Ohio | |
| Children's Hospital Medical Center - Cincinnati | |
| Cincinnati, Ohio, United States, 45229-3039 | |
| Wright State University School of Medicine | |
| Dayton, Ohio, United States, 45401 | |
| Israel | |
| Shaare Zedek Medical Center | |
| Jerusalem, Israel, 91031 | |
| Study Chair: | Richard J. Wenstrup | Children's Hospital Medical Center, Cincinnati |
More Information
| ClinicalTrials.gov Identifier: | NCT00004488 History of Changes |
| Other Study ID Numbers: |
199/14269 CHMC-C-FDR001537 CHMC-C-498 |
| Study First Received: | October 18, 1999 |
| Last Updated: | March 24, 2015 |
Keywords provided by FDA Office of Orphan Products Development:
|
Gaucher's disease inborn errors of metabolism rare disease sphingolipidoses |
Additional relevant MeSH terms:
|
Bone Diseases, Metabolic Gaucher Disease Bone Diseases Musculoskeletal Diseases Sphingolipidoses Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Metabolism, Inborn Errors Genetic Diseases, Inborn Lipidoses Lipid Metabolism, Inborn Errors |
Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders Alendronate Cholecalciferol Calcium Carbonate Bone Density Conservation Agents Physiological Effects of Drugs Antacids Molecular Mechanisms of Pharmacological Action Gastrointestinal Agents Vitamins Micronutrients Growth Substances |
ClinicalTrials.gov processed this record on May 25, 2017