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

Sponsor

Children's Hospital Medical Center, Cincinnati (Other)

Overall Status

Completed

CT.gov ID

NCT00004488

Collaborator

(none)

Enrollment

Locations

Duration (Months)

27.3

Patients Per Site

0.6

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

OBJECTIVES:

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

Study Type:

Interventional

Anticipated Enrollment :

82 participants

Allocation:

Randomized

Intervention Model:

Single Group Assignment

Masking:

Double

Primary Purpose:

Treatment

Study Start Date :

Oct 1, 1998

Study Completion Date :

Sep 1, 2002

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 50 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Locations

	Site	City	State	Country	Postal Code
1	Children's Hospital Medical Center - Cincinnati	Cincinnati	Ohio	United States	45229-3039
2	Wright State University School of Medicine	Dayton	Ohio	United States	45401
3	Shaare Zedek Medical Center	Jerusalem		Israel	91031