Gaucher's Disease, Osteopenia
Conditions
Keywords
Gaucher's disease, inborn errors of metabolism, rare disease, sphingolipidoses
Brief summary
OBJECTIVES: I. Determine the efficacy of alendronate sodium in treating osteopenia (generalized bone density and focal bone lesions) in patients with Gaucher's disease.
Detailed description
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
Interventions
Sponsors
Children's Hospital Medical Center, Cincinnati
Study design
Allocation
RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
DOUBLE
Eligibility
Sex/Gender
ALL
Age
18 Years to 50 Years
Healthy volunteers
No
Inclusion criteria
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
Countries
Israel, United States
Outcome results
None listed