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Effect of High-Dose Vitamin D on Bone Density in Osteogenesis Imperfecta

Phase 4
Completed
Conditions
Osteogenesis Imperfecta
Interventions
Dietary Supplement: standard-dose vitamin D (400IU per day)
Dietary Supplement: high-dose vitamin D (2000 IU per day)
Registration Number
NCT01713231
Lead Sponsor
Louis-Nicolas Veilleux Ph.D.
Brief Summary

* Overall Objective: To test the hypothesis that oral vitamin D supplementation at higher than currently prescribed doses has a beneficial effect on the skeleton of young patients with osteogenesis imperfecta (OI).

* Specific Aims: 1. To determine whether 12 months of high-dose vitamin D supplementation, compared to standard-dose vitamin D supplementation, increases areal bone mineral density z-scores at the lumbar spine. 2. To examine the effectiveness of high-dose vitamin D supplementation to increase trabecular and cortical bone mineral density at the radius. 3. To examine whether high-dose vitamin D supplementation has an effect on physiological determinants of bone mass (parathyroid hormone, activity of bone metabolism, muscle function).

* Background: In a preliminary cross-sectional study of 282 OI patients we observed an inverse relationship between serum 25-hydroxyvitamin D and parathyroid hormone levels and a positive relationship between circulating levels of 25-hydroxyvitamin D and lumbar spine areal bone mineral density z-scores. This suggested that high-dose vitamin D supplementation would have a beneficial effect on bone density. Most OI patients currently receive oral vitamin D supplementation of 400 International Units per day, but doses of 2000 International Units per day are safe and have been shown to be beneficial in studies on healthy adolescents.

* Study Design: This is a parallel-group double-blind randomized controlled trial of 12 months duration on 60 children and adolescents aged 6 to 19 years with a clinical diagnosis of OI. One group of 30 participants will be randomized to receive vitamin D3 at a dose of 2000 international units per day ('high-dose group'). The other group of 30 participants will be randomized to receive vitamin D3 at a dose of 400 international units per day ('standard-dose group'). Randomization will be stratified according to pubertal status and bisphosphonate treatment status.

* Clinical Relevance: The proposed study aims at direct improvements in the care of OI patients. If a simple and low-cost 'intervention' such as high-dose vitamin D supplementation can be shown to be effective in relieving some of the disease burden associated with OI, the benefit to OI patients worldwide would be substantial.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
60
Inclusion Criteria
  • Clinical diagnosis of OI of any type.
Exclusion Criteria
  • Any condition that renders bone density measurements at the lumbar spine impossible. An example for this is prior spinal fusion surgery.
  • Bisphosphonate therapy for less than two years duration.
  • Use of medication, other than bisphosphonates, known to affect bone metabolism or 25OHD serum concentrations. Examples are anti-epileptics, active vitamin D metabolites, corticosteroids and thyroid hormones.
  • Liver and renal disease known to interfere with vitamin D metabolism.
  • Any other disorder of calcium and phosphate metabolism (apart from vitamin D deficiency) that might interfere with PTH.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
standard-dose vitamin Dstandard-dose vitamin D (400IU per day)one group of 30 participants will be randomized to receive vitamin D3 at a dose of 400 international units per day ('standard-dose group').
high-dose vitamin Dhigh-dose vitamin D (2000 IU per day)One group of 30 participants will be randomized to receive vitamin D3 at a dose of 2000 international units per day ('high-dose group').
Primary Outcome Measures
NameTimeMethod
Change in areal bone mineral density z-score of the lumbar spineat baseline and 12 months

LS-aBMD z-score will be used as the primary outcome.The lumbar spine is the standard site of measurement both in the clinical follow up of OI patients .

Secondary Outcome Measures
NameTimeMethod
Change in trabecular and cortical volumetric bone mineral density z-scores at the radius, as measured by pQCT, relative to baseline.at baseline and at 12 months

Trabecular bone is analyzed at the distal radial metaphysis ('4% site'). Cortical bone is analyzed at the radial diaphysis ('65% site').

Trial Locations

Locations (1)

Shriners Hospitals for Children-Canada

🇨🇦

Montréal, Quebec, Canada

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