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

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ClinicalTrials.gov Identifier: NCT00004488

Recruitment Status : Completed

First Posted : October 19, 1999

Last Update Posted : March 25, 2015

Sponsor:

Information provided by:

FDA Office of Orphan Products Development

Study Description

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

Resource links provided by the National Library of Medicine

MedlinePlus Genetics related topics: Gaucher disease

MedlinePlus related topics: Gaucher Disease

Drug Information available for: Alendronate sodium

Genetic and Rare Diseases Information Center resources: Gaucher Disease Sphingolipidosis

U.S. FDA Resources

Arms and Interventions

Outcome Measures

Eligibility Criteria

Information from the National Library of Medicine

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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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00004488

Locations

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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

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

More Information

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ClinicalTrials.gov Identifier:	NCT00004488
Other Study ID Numbers:	199/14269 CHMC-C-FDR001537 CHMC-C-498
First Posted:	October 19, 1999 Key Record Dates
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:

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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 Calcium Calcium-Regulating Hormones and Agents Physiological Effects of Drugs Vitamins Micronutrients Bone Density Conservation Agents Antacids Molecular Mechanisms of Pharmacological Action Gastrointestinal Agents

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