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NCT Number: NCT01713231

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

* 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.

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Key information

Conditions

Osteogenesis Imperfecta Bone Diseases Bone Diseases, Developmental Collagen Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Musculoskeletal Diseases Osteochondrodysplasias Skin and Connective Tissue Diseases

Age range

6 year–19 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Shriners Hospitals for Children-Canada

Montreal, Quebec, H3G1A6, Canada

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

standard-dose vitamin D (400IU per day)

Dietary Supplement

high-dose vitamin D (2000 IU per day)

Dietary Supplement

Primary outcomes

  1. 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 .

Secondary outcomes

  1. 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').

Other outcomes

  1. Percentage change in lower extremity muscle power per body weight, as measured by jumping mechanography, relative to baseline.

    Time frame: baseline and 12 months

    A countermovement jump to maximal height ('single two-legged jump') will be evaluated. In patients who are unable to jump, the heel-rise test will be used to determine muscle power.

Sponsors and collaborators

Lead sponsor

Louis-Nicolas Veilleux Ph.D.

Other

Registry information

Important dates

Study start
2012
Primary completion
2013
Study completion
2014
First posted
Oct 24, 2012
Registry last updated
Sep 9, 2014

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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