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

NCT ID: NCT01882400

Last Updated: 2018-01-09

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 11 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2001-05-31
• Study Completion Date: 2007-09-30

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.

Conditions

Osteoporosis; Muscular Dystrophy; Cystic Fibrosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: PREVENTION
• Blinding Strategy: NONE

Study Groups

Bisphosphonate treatment

Add a weekly bisphosphonate to adequate doses of calcium and vitamin D supplements

Group Type: EXPERIMENTAL

Bisphosphonate treatment

Intervention Type: DRUG

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

Interventions

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

Intervention Type: DRUG

Other Intervention Names

Risedronate; Alendronate

Eligibility Criteria

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

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Procter and Gamble

INDUSTRY

Sponsor Role: collaborator

Gilles Boire

OTHER

Sponsor Role: lead

Responsible Party

Gilles Boire

Doctor Gilles Boire

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Gilles Boire, MD, MSc

Role: PRINCIPAL_INVESTIGATOR

CHUS and Université de Sherbrooke

Locations

Centre hospitalier universitaire de Sherbrooke

Sherbrooke, Quebec, Canada

Countries

Canada

References

Jeffery TC, Chang AB, Conwell LS. Bisphosphonates for osteoporosis in people with cystic fibrosis. Cochrane Database Syst Rev. 2023 Jan 10;1(1):CD002010. doi: 10.1002/14651858.CD002010.pub5.

Reference Type: DERIVED

PMID: 36625789 (View on PubMed)

Other Identifiers

Unrestricted grant

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

01-12

Identifier Type: -

Identifier Source: org_study_id