Clinical Trial: Vitamin D Supplementation for Adults With Neurofibromatosis Type 1 (NF1)

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: A Phase II Trial on the Effect of Low-Dose Versus High-Dose Vitamin D Supplementation on Bone Mass in Adults With Neurofibromatosis Type 1 (NF1

Brief Summary:

This clinical trial is conducted by one of 4 locations; University of British Columbia (Vancouver, CA), University of Utah (Salt Lake City, UT, USA), University of Cincinnati (Cincinnati, OH, USA), and University of Hamburg (Hamburg, Germany).

Adults with NF1 have a higher risk of osteopenia and osteoporosis, a condition of low bone density that can lead to fragile bones and bone breakage.

People with NF1 also have lower vitamin D levels than unaffected individuals. Vitamin D is important for normal bone health, but studies to improve bone health by vitamin D supplementation in people with NF1 have not been tried.

The purpose of this study is to treat adults with NF1 who have insufficient serum vitamin D levels with 2 different doses of vitamin D supplementation to determine if vitamin D supplementation ameliorates the usual loss of bone mineral density over 2 years.


Detailed Summary:

Individuals with NF1 and vitamin D insufficiency will be supplemented with 2 different doses of cholecalciferol (vitamin D) to see if their loss of bone mineral density is lessened over time. It is important to study vitamins as carefully as one would study medications, even though vitamins are generally safer than medications. For this reason, a carefully controlled trial of two doses of vitamin D will be performed over a 2-year period. It will include controlled doses and safety checks for participants' health to be sure that the vitamin D supplementation is safe for people with NF1.

Two different doses of cholecalciferol, either 600 IU or 4,000 IU (IU means "International Units" a standard measure of the strength of the dose), will be used for supplementation. The trial is "double blind". This means that neither participant nor investigator team know which dose an individual participant might be randomized to receive until after the trial ends. In an emergency, however, the investigator can find out.

Participants will be randomized to one or the other dose, and each participant will be provided a bottle of liquid called "D-drops" with a dropper that automatically drops out a measured amount when the bottle is turned upside down.

Both doses of vitamin D, 600 IU and 4,000 IU, have been approved in the general population; however, it is possible that the bodies of people with NF1 handle vitamin D differently. Therefore, safety measures are assessed during this trial. Any new medical issues or concerns throughout the 2-year period will need to be recorded and assessed with the study team.

The primary measure of this study is bone mineral density obtained by bone densitometry (DXA) using low-dose
Sponsor: University of Utah

Current Primary Outcome: Bone mineral density [ Time Frame: Change from Day 1 to 2 years ]

The primary outcome measure is a change in bone mineral density, which will be determined by bone densitometry at the beginning and at the end of the 2-year trial.


Original Primary Outcome: Bone mineral density [ Time Frame: Day 1 and at 2 years ]

The primary outcome measure is bone mineral density, which will be determined by bone densitometry at the beginning and at the end of the 2-year trial.


Current Secondary Outcome:

  • Bone Fractures [ Time Frame: change from Day 1 through 2 years ]
    Secondary outcome measure of history of bone fractures.
  • Quality of life questionnaires [ Time Frame: change from Day 1 throught 2 years ]
    Secondary outcome measures include quality of life


Original Secondary Outcome: Quality of life questionnaires [ Time Frame: Day 1, year 1 and at 2 years ]

Secondary outcome measures include diet analysis, activity level, quality of life, and history of bone fractures.


Information By: University of Utah

Dates:
Date Received: October 7, 2013
Date Started: May 2017
Date Completion: October 2019
Last Updated: May 2, 2017
Last Verified: April 2017