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

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Primary Purpose

Status

Completed

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

alendronate sodium

calcium carbonate

cholecalciferol

About this trial

This is an interventional treatment trial for Gaucher's Disease focused on measuring Gaucher's disease, inborn errors of metabolism, rare disease, sphingolipidoses

Eligibility Criteria

18 Years - 50 Years (Adult)All SexesDoes not accept 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

Sites / Locations

Children's Hospital Medical Center - Cincinnati
Wright State University School of Medicine
Shaare Zedek Medical Center

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

NCT ID

NCT00004488

First Posted

October 18, 1999

Last Updated

March 24, 2015

Sponsor

Children's Hospital Medical Center, Cincinnati

1. Study Identification

Unique Protocol Identification Number

NCT00004488

Brief Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

March 2000

Overall Recruitment Status

Completed

Study Start Date

October 1998 (undefined)

Primary Completion Date

undefined (undefined)

Study Completion Date

September 2002 (undefined)

3. Sponsor/Collaborators

Name of the Sponsor

Children's Hospital Medical Center, Cincinnati

4. Oversight

5. 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.

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Gaucher's Disease, Osteopenia

Keywords

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

Double

Allocation

Randomized

Enrollment

82 (Anticipated)

8. Arms, Groups, and Interventions

Intervention Type

Drug

Intervention Name(s)

alendronate sodium

Intervention Type

Drug

Intervention Name(s)

calcium carbonate

Intervention Type

Drug

Intervention Name(s)

cholecalciferol

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

50 Years

Accepts Healthy Volunteers

No

Eligibility 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Richard J. Wenstrup

Organizational Affiliation

Children's Hospital Medical Center, Cincinnati

Official's Role

Study Chair

Facility Information:

Facility Name

Children's Hospital Medical Center - Cincinnati

City

Cincinnati

State/Province

Ohio

ZIP/Postal Code

45229-3039

Country

United States

Facility Name

Wright State University School of Medicine

City

Dayton

State/Province

Ohio

ZIP/Postal Code

45401

Country

United States

Facility Name

Shaare Zedek Medical Center

City

Jerusalem

ZIP/Postal Code

91031

Country

Israel

Gaucher's Disease, Osteopenia

About this trial

Eligibility Criteria

Sites / Locations

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

Learn more about this trial