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

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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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.

Conditions
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Osteoporosis, Muscular Dystrophy, Cystic Fibrosis

Intervention / Treatment
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* Drug: Bisphosphonate treatment


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 5 Years
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Prevention
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Bisphosphonate treatment<br>Add a weekly bisphosphonate to adequate doses of calcium and vitamin D supplements | Drug: Bisphosphonate treatment<br>* Treatment with Calcium, vitamin D and a weekly bisphosphonate. Comparison of bone mass, fracture rate, pain intensity and scoliosis progression before and after treatment<br>* Other names: Alendronate;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Increase in bone density according to osteodensitometry | comparing successive bone densitometry | Over 2 years of treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Decrease in bone pain | comparing reports of bone pain | Over the first 2 years of treatment | 
| Retardation of scoliosis development | computing how many patients had to have scoliosis surgery | Over the first 2 years of treatment | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Osteoporosis, Severe muscular dystrophy, Cystic fibrosis