Clinical trial NCT05281120: Effects of Low-level Mechanical Vibration on Bone Density in Ambulant Children Affected by Duchenne Muscular Dystrophy
Official title: Low-level Mechanical Vibration, Bone Density, Bone Resorption and Muscular Strength in Ambulant Children Affected by Duchenne Muscular Dystrophy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Osteoporosis
Duchenne Muscular Dystrophy
Keywords: Duchenne Muscular Dystrophy
children
low bone mass
osteoporosis
low-level mechanical vibration
bone turnover markers
Lead sponsor: Istituto Auxologico Italiano
Sponsor class: OTHER
First posted: 2022-03-16
Enrollment: 20
Sex: MALE
Minimum age (years): 7
Maximum age (years): 10
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Change in bone mineral density at lumbar spine.
timeFrame: baseline and 12th month
Secondary outcomes:
measure: Calcium
timeFrame: baseline and 12th month
measure: Phosphate
timeFrame: baseline and 12th month
measure: Magnesium
timeFrame: baseline and 12th month
measure: Creatinine
timeFrame: baseline and 12th month
measure: Bone Alkaline Phosphatase
timeFrame: baseline and 12th month
measure: Osteocalcin
timeFrame: baseline and 12th month
measure: Parathyroid Hormone
timeFrame: baseline and 12th month
measure: 25-OH vitamin D
timeFrame: baseline and 12th month
measure: 1,25(OH)2 vitamin D
timeFrame: baseline and 12th month
Other outcomes:
Locations:
Study officials:
affiliation: Istituto Auxologico Italiano
name: Maria Luisa Bianchi
role: PRINCIPAL_INVESTIGATOR
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