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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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
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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

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Gaucher Disease

Arms and Interventions
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Outcome Measures
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Eligibility Criteria
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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
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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
Vitamin D
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