Gaucher's Disease | Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease
Gaucher's Disease research study
What is the primary objective of this study?
OBJECTIVES: I. Determine the efficacy of alendronate sodium in treating osteopenia (generalized bone density and focal bone lesions) in patients with Gaucher's disease.
Who is eligible to participate?
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
Which medical condition, disease, disorder, syndrome, illness, or injury is researched?
Interventions can include giving participants drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available or noninvasive approaches such as surveys, education, and interviews.
Research studies and clinical trials typically have two or more research arms. An arm is a group of people who receive the same treatment in the study.
Start Date: October 1998
Completed Date: September 2002
Phase: Phase 2
Study sponsors, principal investigator, and references
Principal Investigator: Richard J. Wenstrup
Lead Sponsor: Children's Hospital Medical Center, Cincinnati