Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease

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.

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