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	Treatment or Intervention	Phase
Gaucher's Disease Osteopenia	Drug: alendronate sodium  Drug: calcium carbonate  Drug: cholecalciferol	Phase II

Further Study Details: 

Expected Total Enrollment:  82

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.

Ages Eligible for Study:  18 Years   -   50 Years,  Genders Eligible for Study:  Both

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

Ohio
      Children's Hospital Medical Center - Cincinnati, Cincinnati,  Ohio,  45229-3039,  United States; Recruiting
      Wright State University School of Medicine, Dayton,  Ohio,  45401,  United States; Recruiting
Israel
      Shaare Zedek Medical Center, Jerusalem,  91031,  Israel; Recruiting

Study chairs or principal investigators

Richard J. Wenstrup,  Study Chair,  Children's Hospital Medical Center - Cincinnati   

Study ID Numbers:  199/14269; CHMC-C-FDR001537; CHMC-C-498
Record last reviewed:  March 2000
Last Updated:  October 13, 2004
Record first received:  October 18, 1999
ClinicalTrials.gov Identifier:  NCT00004488
Health Authority: United States: Federal Government
ClinicalTrials.gov processed this record on 2005-04-08