Cystic Fibrosis, Muscular Dystrophy, Osteoporosis
Conditions
Keywords
Osteoporosis, Severe muscular dystrophy, Cystic fibrosis
Brief summary
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
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
Treatment with Calcium, vitamin D and a weekly bisphosphonate. Comparison of bone mass, fracture rate, pain intensity and scoliosis progression before and after treatment
Sponsors
Procter and Gamble
Gilles Boire
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
PREVENTION
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
5 Years to 18 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Increase in bone density according to osteodensitometry | Over 2 years of treatment | comparing successive bone densitometry |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Decrease in bone pain | Over the first 2 years of treatment | comparing reports of bone pain |
| Retardation of scoliosis development | Over the first 2 years of treatment | computing how many patients had to have scoliosis surgery |
Other
| Measure | Time frame | Description |
|---|---|---|
| Side effects of treatment | Over 2 years | Look for increases in kidney stones or hypercalciuria |
Countries
Canada
Outcome results
None listed