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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

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 uni
  

  Detailed Summary:
  Sponsor: Louis-Nicolas Veilleux Ph.D.
  

  Current Primary Outcome: Change in areal bone mineral density z-score of the lumbar spine [ Time Frame: at 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 .
  
  
  

  Original Primary Outcome: Same as current
  
  

  Current Secondary Outcome: Change in trabecular and cortical volumetric bone mineral density z-scores at the radius, as measured by pQCT, relative to baseline. [ Time Frame: 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').
  
  
  

  Original Secondary Outcome: Same as current
  
  Information By: Shriners Hospitals for Children
  

  Dates:
  Date Received: October 21, 2012
  Date Started: September 2012
  Date Completion:
  Last Updated: September 8, 2014
  Last Verified: September 2014