Assessment of Response to Treatment of Osteoporosis With Oral Bisphosphonates in Patients With Muscular Dystrophy

Overview

By supplying an adequate amount of calcium and vitamin D with the addition of weekly bisphosphonate, the investigators will be able to increase bone mass and decrease the incidence of fragility fractures in these children with muscular dystrophy. The investigators think this treatment will also decrease the intensity of pain frequently present in these patients and slow the progression of scoliosis.

Full Title of Study: “Évaluation Multidimensionnelle de la réponse au Traitement de l’ostéoporose spontanée et Induite Par Les corticostéroïdes à l’Aide d’un Bisphosphonate à Administration Orale Chez Des Malades Porteurs d’Une Dystrophie Musculaire sévère.”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Prevention
    • Masking: None (Open Label)
  • Study Primary Completion Date: September 2007

Detailed Description

Children with muscular dystrophy, as well as children with other chronic diseases (e.g. cystic fibrosis, chronic inflammatory arthritis) are at risk to develop fragility fractures both due to the disease itself and to drugs (mostly corticosteroids) used to treat the diseases. In addition, children with muscular dystrophy frequently complain of diffuse pain making daily care more difficult and almost always develop structural scoliosis. The objective of the present protocol is to offer these children a preventive treatment aimed at maintaining or increasing their bone mass. We also propose that maintaining bone mass will decrease fracture rates, as well as pain and the rate of progression of scoliosis. Rigorous care to ensure adequate intake of calcium and vitamin D, as well as addition of a weekly bisphosphonate, are the central aspect of the project. In addition, we will monitor to avoid possible side-effects such as hypercalciuria and kidney stones.

Interventions

  • Drug: Bisphosphonate treatment
    • Treatment with Calcium, vitamin D and a weekly bisphosphonate. Comparison of bone mass, fracture rate, pain intensity and scoliosis progression before and after treatment

Arms, Groups and Cohorts

  • Experimental: Bisphosphonate treatment
    • Add a weekly bisphosphonate to adequate doses of calcium and vitamin D supplements

Clinical Trial Outcome Measures

Primary Measures

  • Increase in bone density according to osteodensitometry
    • Time Frame: Over 2 years of treatment
    • comparing successive bone densitometry

Secondary Measures

  • Decrease in bone pain
    • Time Frame: Over the first 2 years of treatment
    • comparing reports of bone pain
  • Retardation of scoliosis development
    • Time Frame: Over the first 2 years of treatment
    • computing how many patients had to have scoliosis surgery

Participating in This Clinical Trial

Inclusion Criteria

  • Osteoporosis or osteopenia – Severe muscular dystrophy or cystic fibrosis – May use corticosteroids Exclusion Criteria:

  • Inability to consent or to take drugs by mouth

Gender Eligibility: All

Minimum Age: 5 Years

Maximum Age: 18 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Gilles Boire
  • Collaborator
    • Procter and Gamble
  • Provider of Information About this Clinical Study
    • Sponsor-Investigator: Gilles Boire, Doctor Gilles Boire – Université de Sherbrooke
  • Overall Official(s)
    • Gilles Boire, MD, MSc, Principal Investigator, CHUS and Université de Sherbrooke

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