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

NCT00004488 | Completed Phase 2

• 3 other identifiers: 199/14269CHMC-C-FDR001537CHMC-C-498
• Study Type: interventional
• Target: 82
• Locations: 2 countries
• Sites: 3

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.

Conditions

Gaucher's Disease; Osteopenia

Keywords

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

Interventions

alendronate sodium DRUG

calcium carbonate DRUG

cholecalciferol DRUG

Eligibility Criteria

• Age: 18 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead

Study Sites (3)

Children's Hospital Medical Center - Cincinnati

Cincinnati, Ohio, 45229-3039, United States

Location

Wright State University School of Medicine

Dayton, Ohio, 45401, United States

Location

Shaare Zedek Medical Center

Jerusalem, 91031, Israel

Location

MeSH Terms

Conditions

Gaucher Disease; Bone Diseases, Metabolic; Metabolism, Inborn Errors; Rare Diseases; Sphingolipidoses

Interventions

Alendronate; Calcium Carbonate; Cholecalciferol

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders; Bone Diseases; Musculoskeletal Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Diphosphonates; Organophosphonates; Organophosphorus Compounds; Organic Chemicals; Calcium Compounds; Inorganic Chemicals; Carbonates; Carbonic Acid; Carbon Compounds, Inorganic; Minerals; Cholestenes; Cholestanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Sterols; Vitamin D; Secosteroids; Membrane Lipids; Lipids

Study Officials

• Richard J. Wenstrup

  Children's Hospital Medical Center, Cincinnati

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: October 18, 1999
• First Posted: October 19, 1999
• Study Start: October 1, 1998
• Study Completion: September 1, 2002
• Last Updated: March 25, 2015

Record last verified: 2000-03

Locations

IL (1); US (2)