NCT01882400

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
11

participants targeted

Target at below P25 for phase_4

Timeline
Completed

Started May 2001

Longer than P75 for phase_4

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 1, 2001

Completed
6.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2007

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2007

Completed
5.8 years until next milestone

First Submitted

Initial submission to the registry

June 17, 2013

Completed
3 days until next milestone

First Posted

Study publicly available on registry

June 20, 2013

Completed
Last Updated

January 9, 2018

Status Verified

January 1, 2018

Enrollment Period

6.3 years

First QC Date

June 17, 2013

Last Update Submit

January 6, 2018

Conditions

Keywords

OsteoporosisSevere muscular dystrophyCystic fibrosis

Outcome Measures

Primary Outcomes (1)

  • Increase in bone density according to osteodensitometry

    comparing successive bone densitometry

    Over 2 years of treatment

Secondary Outcomes (2)

  • Decrease in bone pain

    Over the first 2 years of treatment

  • Retardation of scoliosis development

    Over the first 2 years of treatment

Other Outcomes (1)

  • Side effects of treatment

    Over 2 years

Study Arms (1)

Bisphosphonate treatment

EXPERIMENTAL

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

Drug: Bisphosphonate treatment

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

Also known as: Risedronate, Alendronate
Bisphosphonate treatment

Eligibility Criteria

Age5 Years - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Osteoporosis or osteopenia
  • Severe muscular dystrophy or cystic fibrosis
  • May use corticosteroids

You may not qualify if:

  • Inability to consent or to take drugs by mouth

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Centre hospitalier universitaire de Sherbrooke

Sherbrooke, Quebec, J1H 5N4, Canada

Location

Related Publications (1)

  • 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.

MeSH Terms

Conditions

OsteoporosisMuscular DystrophiesCystic Fibrosis

Interventions

Risedronic AcidAlendronate

Condition Hierarchy (Ancestors)

Bone Diseases, MetabolicBone DiseasesMusculoskeletal DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesMuscular Disorders, AtrophicMuscular DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesPancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesInfant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

DiphosphonatesOrganophosphonatesOrganophosphorus CompoundsOrganic ChemicalsPyridinesHeterocyclic Compounds, 1-RingHeterocyclic Compounds

Study Officials

  • Gilles Boire, MD, MSc

    CHUS and Université de Sherbrooke

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NA
Masking
NONE
Purpose
PREVENTION
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Doctor Gilles Boire

Study Record Dates

First Submitted

June 17, 2013

First Posted

June 20, 2013

Study Start

May 1, 2001

Primary Completion

September 1, 2007

Study Completion

September 1, 2007

Last Updated

January 9, 2018

Record last verified: 2018-01

Locations