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

This study has been completed.

Sponsor:

Information provided by:

FDA Office of Orphan Products Development

ClinicalTrials.gov Identifier:

NCT00004488

First received: October 18, 1999

Last updated: March 24, 2015

Last verified: March 2000

History of Changes

Purpose

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	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:

Genetics Home Reference 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

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:

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

Eligibility


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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004488

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


ClinicalTrials.gov Identifier:	NCT00004488 History of Changes
Other Study ID Numbers:	199/14269 CHMC-C-FDR001537 CHMC-C-498
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 sphingolipidoses

Additional relevant MeSH terms:


Bone Diseases, Metabolic Gaucher Disease Bone Diseases Musculoskeletal 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 Metabolic 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

ClinicalTrials.gov processed this record on September 20, 2017

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