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

Cystic Fibrosis Clinical Trial

Official title:

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.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01882400
• Other study ID #: 01-12
• Secondary ID: Unrestricted gra
• Status: Completed
• Phase: Phase 4
• First received: June 17, 2013
• Last updated: January 6, 2018
• Start date: May 2001
• Est. completion date: September 2007

Study information

• Verified date: January 2018
• Source: Université de Sherbrooke
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 11
• Est. completion date: September 2007
• Est. primary completion date: September 2007
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis
• Muscular Dystrophies
• Muscular Dystrophy
• Osteoporosis

Intervention

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

Locations

Country	Name	City	State
Canada	Centre hospitalier universitaire de Sherbrooke	Sherbrooke	Quebec

Sponsors (2)

Lead Sponsor	Collaborator
Gilles Boire	Procter and Gamble

Country where clinical trial is conducted

Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Side effects of treatment	Look for increases in kidney stones or hypercalciuria	Over 2 years
Primary	Increase in bone density according to osteodensitometry	comparing successive bone densitometry	Over 2 years of treatment
Secondary	Decrease in bone pain	comparing reports of bone pain	Over the first 2 years of treatment
Secondary	Retardation of scoliosis development	computing how many patients had to have scoliosis surgery	Over the first 2 years of treatment