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

This study has been completed.
Information provided by:
FDA Office of Orphan Products Development Identifier:
First received: October 18, 1999
Last updated: March 24, 2015
Last verified: March 2000


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
Primary Purpose: Treatment

Resource links provided by NLM:

Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment: 82
Study Start Date: October 1998
Estimated Study Completion Date: September 2002
Detailed Description:


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


Ages Eligible for Study:   18 Years to 50 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


--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

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Please refer to this study by its 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
Shaare Zedek Medical Center
Jerusalem, Israel, 91031
Sponsors and Collaborators
Children's Hospital Medical Center, Cincinnati
Study Chair: Richard J. Wenstrup Children's Hospital Medical Center, Cincinnati
  More Information Identifier: NCT00004488     History of Changes
Other Study ID Numbers: 199/14269
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

Additional relevant MeSH terms:
Bone Diseases, Metabolic
Gaucher Disease
Bone Diseases
Musculoskeletal Diseases
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
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
Calcium Carbonate
Growth Substances
Physiological Effects of Drugs
Bone Density Conservation Agents
Molecular Mechanisms of Pharmacological Action
Gastrointestinal Agents processed this record on September 21, 2017