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

• Status: Recruiting
• Trial end date: 2022-10-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Utah
• Collaborators: Children's Hospital Medical Center, Cincinnati; U.S. Army Medical Research and Development Command; U.S. Army Medical Research and Materiel Command; Universitätsklinikum Hamburg-Eppendorf; University of British Columbia
• Treatments: Cholecalciferol; Ergocalciferols; Vitamin D; Vitamins

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