Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

Vitamin D Supplementation for Adults With Neurofibromatosis Type 1 (NF1) (VitDBoneNF1)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01968590
Recruitment Status : Recruiting
First Posted : October 24, 2013
Last Update Posted : January 29, 2020
Sponsor:
Collaborators:
U.S. Army Medical Research and Development Command
Universitätsklinikum Hamburg-Eppendorf
University of British Columbia
Children's Hospital Medical Center, Cincinnati
Information provided by (Responsible Party):
David Viskochil, University of Utah

Tracking Information
First Submitted Date  ICMJE October 7, 2013
First Posted Date  ICMJE October 24, 2013
Last Update Posted Date January 29, 2020
Actual Study Start Date  ICMJE August 16, 2017
Estimated Primary Completion Date October 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 10, 2016)
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 Measures  ICMJE
 (submitted: October 19, 2013)
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.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 27, 2020)
  • 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 through 2 years ]
    Secondary outcome measures include quality of life
Original Secondary Outcome Measures  ICMJE
 (submitted: October 19, 2013)
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.
Current Other Pre-specified Outcome Measures
 (submitted: November 10, 2016)
  • Parathyroid hormone with calcium [ Time Frame: change from day1 through year 2 ]
    Serum samples will be analyzed for intact parathyroid hormone with calcium.
  • 25(OH) vitamin D [ Time Frame: change from day1 through year 2 ]
    Serum samples will be analyzed for 25(OH) vitamin D
Original Other Pre-specified Outcome Measures
 (submitted: October 19, 2013)
Serum samples [ Time Frame: samples at day1 , year 1, and year 2 ]
Serum samples will be analyzed for 25(OH) vitamin D, calcium, and intact parathyroid hormone.
 
Descriptive Information
Brief Title  ICMJE Vitamin D Supplementation for Adults With Neurofibromatosis Type 1 (NF1)
Official Title  ICMJE 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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Prevention
Condition  ICMJE Neurofibromatosis Type 1 (NF1)
Intervention  ICMJE Drug: Cholecalciferol
DDrops(trademark) prepared two sets of bottles, one set has 300IU per metered dose and the other set has 2,000IU per metered dose. Participants are randomized to either 600 IU or 4,000 IU, and they take two measured drops orally, once a day. There is a dropper that automatically drops out a measured amount when the bottle is turned upside down.
Other Names:
  • Vitamin D
  • D Drops
  • Dietary Supplement
Study Arms  ICMJE
  • Active Comparator: cholecalciferol 600 IU
    cholecalciferol in Ddrops form at either 600 International units per day versus 4,000 IU per day
    Intervention: Drug: Cholecalciferol
  • Active Comparator: cholecalciferol 4,000 IU
    Cholecalciferol at 4,000 IU per day in the form of liquid Ddrops
    Intervention: Drug: Cholecalciferol
Publications * Schnabel C, Jett K, Friedman JM, Frieling I, Kruse HP, Mautner V. Effect of vitamin D3 treatment on bone density in neurofibromatosis 1 patients: a retrospective clinical study. Joint Bone Spine. 2013 May;80(3):315-9. doi: 10.1016/j.jbspin.2012.07.010. Epub 2012 Sep 26.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: October 19, 2013)
320
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE October 2022
Estimated Primary Completion Date October 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 25 Years to 40 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Carrie Bailey, BS 8015873605 carrie.bailey@hsc.utah.edu
Contact: Sarah L Couchon, BS, CCRC 8015853822 Sarah.Couchon@hsc.utah.edu
Listed Location Countries  ICMJE Canada,   United States
Removed Location Countries Germany
 
Administrative Information
NCT Number  ICMJE NCT01968590
Other Study ID Numbers  ICMJE DoD A-17236
W18XWH-12-1-0487 ( Other Grant/Funding Number: Department of Defense NF Research Program )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Plan Description: Publication of all study data will be written
Responsible Party David Viskochil, University of Utah
Study Sponsor  ICMJE University of Utah
Collaborators  ICMJE
  • U.S. Army Medical Research and Development Command
  • Universitätsklinikum Hamburg-Eppendorf
  • University of British Columbia
  • Children's Hospital Medical Center, Cincinnati
Investigators  ICMJE
Principal Investigator: David H. Viskochil, MD, PhD University of Utah
PRS Account University of Utah
Verification Date January 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP