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

Study Description

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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.

Condition or disease	Intervention/treatment	Phase
Gaucher's Disease; Osteopenia	Drug: alendronate sodium; Drug: calcium carbonate; Drug: cholecalciferol	Phase 2

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

Study Design

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Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	82 participants
Allocation:	Randomized
Intervention Model:	Single Group Assignment
Masking:	Double
Primary Purpose:	Treatment
Study Start Date :	October 1998
Study Completion Date :	September 2002

Arms and Interventions

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Outcome Measures

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Eligibility Criteria

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

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

Contacts and Locations

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Locations

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
Israel
Shaare Zedek Medical Center
Jerusalem, Israel, 91031

Sponsors and Collaborators

Children's Hospital Medical Center, Cincinnati

Investigators

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

More Information

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

ClinicalTrials.gov Identifier:	NCT00004488 History of Changes
Other Study ID Numbers:	199/14269; CHMC-C-FDR001537; CHMC-C-498
First Posted:	October 19, 1999
Last Update Posted:	March 25, 2015
Last Verified:	March 2000

Keywords provided by FDA Office of Orphan Products Development:

Gaucher's disease; inborn errors of metabolism; rare disease; sphingolipidoses

Additional relevant MeSH terms:

Bone Diseases, Metabolic; Gaucher Disease; Bone Diseases; Musculoskeletal Diseases; Metabolic Diseases; Sphingolipidoses; 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; Lipidoses	Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Cholecalciferol; Alendronate; Calcium Carbonate; Vitamins; Micronutrients; Growth Substances; Physiological Effects of Drugs; Bone Density Conservation Agents; Antacids; Molecular Mechanisms of Pharmacological Action; Gastrointestinal Agents