Bone Health in Duchenne Muscular Dystrophy- a case controlled study of Risedronate use - Risedronate in DMD

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2009-017649-67-IE
• Date of registration: 13/01/2010
• Prospective Registration: Yes
• Primary sponsor: The Central Remedial Clinic and The Children's University Hospital
• Public title: Bone Health in Duchenne Muscular Dystrophy- a case controlled study of Risedronate use - Risedronate in DMD
• Scientific title: Bone Health in Duchenne Muscular Dystrophy- a case controlled study of Risedronate use - Risedronate in DMD
• Date of first enrolment: 22/06/2010
• Target sample size: 70
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-017649-67
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: no Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: yes Placebo: no Other: no

Countries of recruitment

Ireland

Key inclusion & exclusion 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:

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




Age minimum:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

Bone Health in Duchenne Muscular Dystrophy- a randomised controlled study of Risedronate use

Intervention(s)

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: Calcichew D3 Forte
Product Code: Calcichew D3 Forte
Pharmaceutical Form: Chewable tablet

Primary Outcome(s)

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 randomization has only a minor effect on power for a superiority trials such as this standard two-group comparison power calculations were used
Based on a two-sided significance level of 5% and a power of 80%, a trial with 25 patients in each group would be able to detect a difference in BMD lumbar spine Z-score changes of 0.81 standard deviations (SD) or greater after 12months treatment.
This change in BMD lumbar spine Z score has been reported by previous groups using similar dose regime.

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 ID(s)

nnisbjl2009