Bone Health in Duchenne Muscular Dystrophy- a randomised controlled study of Risedronate use
Conditions
Interventions
Trade Name: Actonel Once a week 5mg film coated tablets
Product Name: Risedronate sodium
Product Code: Risedronate
Pharmaceutical Form: Film-coated tablet
Trade Name: Calcichew D3 Forte
Product Name:
Sponsors
The Central Remedial Clinic and The Children's University Hospital
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1.>/4 years old at time of initiation of study 2. Z score as measured by DXA >1.0 S.D. below the mean 3. boys only with DMD Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Hypersensitivity to Risedronate sodium 2. <4years at time of initiation of study 3. Previous bisphosphonate use 4. Uncorrected hypocalcaemia, uncorrected low Vitamin D 5. Inability to stay in an upright position (sitting or standing) for at least 30minutes 6. Severe renal impairment (creatinine clearance <30ml/min) 7.Undergoing invasive dental treatment 8. History of recent or active oesophageal or upper gastrointestinal problems 9.History of oesophageal disorders which delay oesophageal transit or emptying e.g. stricture or achalasia 10. Involvement in clinical trial in the preceding 12 weeks. 11. Learning difficulties in carer resulting in difficulties comprehending treatment
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: (1)To prospectively determine, using a randomized controlled trial, the effects of a targeted intervention on improving BMD in this population using oral bisphosphonate and calcium and Vitamin D supplementation (Calcichew D3 Forte) versus Calcium and Vitamin D supplementation with Calcichew D3 Forte alone. ;Secondary Objective: (2)To assess bone health in Irish population of children and young adults with DMD a) Baseline calcium and vitamin D status and optimize prior to onset of trial b) Baseline bone mineral density in ambulant and nonambulant boys with DMD, using DXA scan and markers of bone formation and resorption. DXA performed up to six months prior to the onset of the trail will be used as a baseline measurement. c) Determine the incidence of long bone and vertebral fractures and bone pain in this group and whether there is any correlation with disease severity/steroid use or genetic mutations. (3) To establish guidelines on management of bone health in the DMD population to reduce fracture risk. ;Primary end point(s): Primary Outcome: Efficacy of bisphosphonates and Calcium and Vitamin D supplementation with Calcichew D3 Forte, compared with Calcium and Vitamin D supplementation (Calchichew D3 Forte) alone a. Change /% change in areal/volumetric BMD Z score of lumbar spine following intervention over 12 months is the primary endpoint. Criteria for success of treatment will be an increase of 0.5SD in lumbar spine Z score as measured by DXA scan. The doctor reporting the DXA scans (MMcK) will be blinded to what treatment each patient is receiving. Our study is large enough to detect a difference of 0.8SD in lumbar spine DXA score between treatment and control groups. b. Change /% change in areal/volumetric BMD Z score of lateral distal femoral head, distal third of radius, total body excluding head as measured by DXA Our power calculation is based on the primary end point of lumbar spine BMD Z-score changes. Because stratified | — |
Countries
Ireland
Outcome results
None listed