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

Primary Purpose

Osteoporosis, Muscular Dystrophy, Cystic Fibrosis

Status

Completed

Phase

Phase 4

Locations

Canada

Study Type

Interventional

Intervention

Bisphosphonate treatment

About this trial

This is an interventional prevention trial for Osteoporosis focused on measuring Osteoporosis, Severe muscular dystrophy, Cystic fibrosis

Eligibility Criteria

5 Years - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Centre hospitalier universitaire de Sherbrooke

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Bisphosphonate treatment

Arm Description

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

Outcomes

Primary Outcome Measures

Increase in bone density according to osteodensitometry

comparing successive bone densitometry

Secondary Outcome Measures

Decrease in bone pain

comparing reports of bone pain

Retardation of scoliosis development

computing how many patients had to have scoliosis surgery

Full Information

NCT ID

NCT01882400

First Posted

June 17, 2013

Last Updated

January 6, 2018

Sponsor

Gilles Boire

Collaborators

Procter and Gamble

1. Study Identification

Unique Protocol Identification Number

NCT01882400

Brief Title

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

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.

Study Type

Interventional

2. Study Status

Record Verification Date

January 2018

Overall Recruitment Status

Completed

Study Start Date

May 2001 (undefined)

Primary Completion Date

September 2007 (Actual)

Study Completion Date

September 2007 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor-Investigator

Name of the Sponsor

Gilles Boire

Collaborators

Procter and Gamble

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Osteoporosis, Muscular Dystrophy, Cystic Fibrosis

Keywords

Osteoporosis, Severe muscular dystrophy, Cystic fibrosis

7. Study Design

Primary Purpose

Prevention

Study Phase

Phase 4

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

11 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Bisphosphonate treatment

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

Bisphosphonate treatment

Other Intervention Name(s)

Risedronate, Alendronate

Intervention Description

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

Primary Outcome Measure Information:

Title

Increase in bone density according to osteodensitometry

Description

comparing successive bone densitometry

Time Frame

Over 2 years of treatment

Secondary Outcome Measure Information:

Title

Decrease in bone pain

Description

comparing reports of bone pain

Time Frame

Over the first 2 years of treatment

Title

Retardation of scoliosis development

Description

computing how many patients had to have scoliosis surgery

Time Frame

Over the first 2 years of treatment

Other Pre-specified Outcome Measures:

Title

Side effects of treatment

Description

Look for increases in kidney stones or hypercalciuria

Time Frame

Over 2 years

10. Eligibility

Sex

All

Minimum Age & Unit of Time

5 Years

Maximum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Gilles Boire, MD, MSc

Organizational Affiliation

CHUS and Université de Sherbrooke

Official's Role

Principal Investigator

Facility Information:

Facility Name

Centre hospitalier universitaire de Sherbrooke

City

Sherbrooke

State/Province

Quebec

ZIP/Postal Code

J1H 5N4

Country

Canada

12. IPD Sharing Statement

Learn more about this trial

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

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