Effects of Low-level Mechanical Vibration on Bone Density in Ambulant Children Affected by Duchenne Muscular Dystrophy

Not Applicable

Completed

• Conditions: Duchenne Muscular Dystrophy; Osteoporosis
• Interventions: Device: Low-level mechanical vibrations WITH vertical sinusoidal acceleration; Device: Low-level mechanical vibrations WITHOUT vertical sinusoidal acceleration

• Registration Number: NCT05281120
• Lead Sponsor: Istituto Auxologico Italiano

Brief Summary

Duchenne muscular dystrophy (DMD) is a X-linked recessive disorder due to a mutation of the dystrophin gene (Xp21). Dystrophin is a sarcolemmal protein of skeletal and cardiac muscle, and its absence causes progressive muscle degeneration and substitution with fat and connective tissue. The progressive muscle degeneration leads to loss of autonomous walking before the age of 15 years and death for cardiac and/or respiratory failure. There are no specific treatment for DMD, and the standard of care is now based on long-term corticosteroid (CS) use. The studies on bone mass in DMD are very few, but they agree in reporting the presence of a reduced bone mass and an increased rate of fractures probably due to long-term steroid therapy and disuse-osteopenia. The aim of this study, involving 20 ambulant DMD boys (age 7-10 years) has been the evaluation of the effects of low-level mechanical vibrations on bone in a group of ambulant DMD children for 1 year, with RDA-adjusted dietary calcium intake and 25OH vitamin D supplementation.

Detailed Description

All children were instructed to have a daily intake of calcium equal to the 100% of the RDA and were supplemented with calcifediol (0.7 mcg/kg/die).

The 20 boys were randomly assigned to two groups:

group 1 (mechanical intervention group) = a mechanical device (a small platform designed to induce vertical, sinusoidal acceleration) was installed in the home of each boy of group 1.

group 2 (placebo control group) = a placebo device was installed in the home of each boy of group 2

All boys were instructed to stand on the platform for 10 minutes each day for 12 months.

Compliance was followed and stimulated through weekly telephone contacts with parents and children.

Study Timeline

• Study Start: 2006-11
• Primary Completion: 2007-05
• Study Completion: 2007-11
• Study First Submitted: 2022-02-18
• Status Verified: 2022-03
• Study First Submitted QC: 2022-03-10
• Last Update Submitted: 2022-03-10
• Study First Posted: 2022-03-16
• Last Update Posted: 2022-03-16

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 20

Inclusion Criteria

• Diagnosis of DMD
• Ability to stand up and walk (some balance assistance allowed, but full weight-bearing necessary)
• All the children must already be on glucocorticoid therapy for at least 6 months before the start of the study.

Exclusion Criteria

• Presence of other diseases interfering with bone density and bone turnover
• The inability to regularly use the vibratory platform.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
mechanical intervention group	Low-level mechanical vibrations WITH vertical sinusoidal acceleration	20 subjects participated to the study (1 year double-blind, randomized, placebo controlled, parallel group study) and 10 subjects were randomized to this arm. Intervention: to stand on the active platform (inducing vertical, sinusoidal acceleration) for 10 minutes each day for 12 months.
mechanical placebo group	Low-level mechanical vibrations WITHOUT vertical sinusoidal acceleration	20 subjects participated to the study (1 year double-blind, randomized, placebo controlled, parallel group study) and 10 subjects were randomized to this arm. Intervention: to stand on the placebo platform (not inducing vertical, sinusoidal acceleration) for 10 minutes each day for 12 months.

Primary Outcome Measures

Name	Time	Method
Change in bone mineral density at lumbar spine.	baseline and 12th month	Bone mineral density evaluated by DXA. Bone mineral apparent density calculated to correct for bone size (growing subjects). Z-score calculated.; Measurements: baseline and 12 months.

Secondary Outcome Measures

Name	Time	Method
1,25(OH)2 vitamin D	baseline and 12th month	changes in serum 1,25(OH)2 vitamin D (ng/L)
25-OH vitamin D	baseline and 12th month	changes in serum 25-OH vitamin D (µg/L)
Magnesium	baseline and 12th month	changes in serum magnesium (mg/dL)
Parathyroid Hormone	baseline and 12th month	changes in serum parathyroid hormone (ng/L)
Phosphate	baseline and 12th month	changes in serum phosphate (mg/dL)
Bone Alkaline Phosphatase	baseline and 12th month	changes in serum bone alkaline phosphatase (µg/L)
Osteocalcin	baseline and 12th month	changes in serum osteocalcin (µg/L)
Creatinine	baseline and 12th month	changes in serum creatinine (mg/dL)
Calcium	baseline and 12th month	changes in serum calcium (mg/dL)