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

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Brief Title

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

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 Description

      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 xrays. Bone mineral density will be tested at the beginning and the end of the
      trial focusing on density of bones in the hip and spine. Secondary measures of this trial
      include questionnaires that measure quality of life and history of bone fractures.

      When someone is insufficient in serum vitamin D and supplementation is recommended, it is
      also supplemented with calcium. Therefore, participants will be given a calcium supplement of
      400 mg elemental calcium per day. A diary to keep daily track of vitamin D and calcium
      supplementation and documentation of fractures, should they occur, is provided.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Bone mineral density

Secondary Outcome

 Bone Fractures

Condition

Neurofibromatosis Type 1 (NF1)

Intervention

Cholecalciferol

Study Arms / Comparison Groups

 cholecalciferol 600 IU
Description:  cholecalciferol in Ddrops form at either 600 International units per day versus 4,000 IU per day

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

320

Start Date

August 16, 2017

Completion Date

October 2022

Primary Completion Date

October 2022

Eligibility Criteria

        Inclusion Criteria:

        All individuals with NF1 (neurofibromatosis type 1), based on NIH diagnostic criteria, who
        are between the ages of 25 and 40 years, may participate in this study. All participants
        will be enrolled in the study and then screened for serum 25(OH)D levels, and all
        individuals with levels between 9 and 29ng/ml (inclusive) are eligible for vitamin D
        supplementation -

        Exclusion Criteria:

          1. diagnosis of Paget's disease, hyperthyroidism, hyperparathyroidism, or other medical
             condition that affects bone health

          2. they foresee that they will be unable to comply with the two-year study protocol

          3. Pregnant, planning to conceive within the next two years, or is less than 6 months
             post-delivery or lactation.

          4. vitamin D supplementation in the last 3 months equal to or greater than 600IU per day

          5. oral or IV glucocorticoid use for over 3 months

          6. bisphosphonate therapy for more than 3 months

          7. calcitonin therapy for more than 3 months

          8. calcium supplementation in last 3 months equal to or greater than 1000mg per day

          9. malignant peripheral nerve sheath tumor (MPNST)

         10. history of kidney stones in last 5 years

         11. individuals with metal instrumentation in spine or hip that preclude accurate DXA
             interpretation.

         12. inability to obtain blood samples on routine venipuncture

         13. anti-epileptic medical therapy

         14. anticoagulant medical therapy
      

Gender

All

Ages

25 Years - 40 Years

Accepts Healthy Volunteers

No

Contacts

David H. Viskochil, MD, PhD, 8015873605, [email protected]

Location Countries

Canada

Location Countries

Canada

Administrative Informations


NCT ID

NCT01968590

Organization ID

DoD A-17236

Secondary IDs

W18XWH-12-1-0487

Responsible Party

Principal Investigator

Study Sponsor

University of Utah

Collaborators

 U.S. Army Medical Research and Development Command

Study Sponsor

David H. Viskochil, MD, PhD, Principal Investigator, University of Utah


Verification Date

April 2021