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

NCT01968590 | Terminated Phase 2 FDA Drug

• 2 other identifiers: DoD A-17236W18XWH-12-1-0487
• Study Type: interventional
• Target: 26
• Locations: 2 countries
• Sites: 3

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.

Conditions

Neurofibromatosis Type 1 (NF1)

Keywords

Vitamin D; Cholecalciferol; Bone density

Outcome Measures

Primary Outcomes (1)

• Bone mineral density

  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.

  Change from Day 1 to 2 years

Secondary Outcomes (2)

• Bone Fractures

  change from Day 1 through 2 years

• Quality of life questionnaires

  change from Day 1 through 2 years

Other Outcomes (2)

• Parathyroid hormone with calcium

  change from day1 through year 2

• 25(OH) vitamin D

  change from day1 through year 2

Study Arms (2)

cholecalciferol 600 IU

ACTIVE COMPARATOR

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

Drug: Cholecalciferol

cholecalciferol 4,000 IU

ACTIVE COMPARATOR

Cholecalciferol at 4,000 IU per day in the form of liquid Ddrops

Drug: Cholecalciferol

Interventions

Cholecalciferol DRUG

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.

Also known as: Vitamin D, D Drops, Dietary Supplement

cholecalciferol 4,000 IU; cholecalciferol 600 IU

Eligibility Criteria

• Age: 25 Years - 40 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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 -

You may not qualify if:

• diagnosis of Paget's disease, hyperthyroidism, hyperparathyroidism, or other medical condition that affects bone health
• they foresee that they will be unable to comply with the two-year study protocol
• Pregnant, planning to conceive within the next two years, or is less than 6 months post-delivery or lactation.
• vitamin D supplementation in the last 3 months equal to or greater than 600IU per day
• oral or IV glucocorticoid use for over 3 months
• bisphosphonate therapy for more than 3 months
• calcitonin therapy for more than 3 months
• calcium supplementation in last 3 months equal to or greater than 1000mg per day
• malignant peripheral nerve sheath tumor (MPNST)
• history of kidney stones in last 5 years
• individuals with metal instrumentation in spine or hip that preclude accurate DXA interpretation.
• inability to obtain blood samples on routine venipuncture
• anti-epileptic medical therapy
• anticoagulant medical therapy

Sponsors & Collaborators

• University of Utah: lead
• U.S. Army Medical Research and Development Command: collaborator
• Universitätsklinikum Hamburg-Eppendorf: collaborator
• University of British Columbia: collaborator
• Children's Hospital Medical Center, Cincinnati: collaborator

Study Sites (3)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

University of Utah

Salt Lake City, Utah, 84108, United States

Location

University of British Columbia

Vancouver, British Columbia, V6T 1Z4, Canada

Location

Related Publications (1)

• 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.

  PMID: 23021159 BACKGROUND

MeSH Terms

Conditions

Neurofibromatosis 1

Interventions

Cholecalciferol; Vitamin D; Dietary Supplements

Condition Hierarchy (Ancestors)

Neurofibromatoses; Neurofibroma; Nerve Sheath Neoplasms; Neoplasms, Nerve Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Cholestenes; Cholestanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Sterols; Secosteroids; Membrane Lipids; Lipids; Food; Diet, Food, and Nutrition; Physiological Phenomena; Food and Beverages

Study Officials

• David H. Viskochil, MD, PhD

  University of Utah

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: David H .Viskochil M.D. , Ph.D professor of pediatrics

Study Record Dates

• First Submitted: October 7, 2013
• First Posted: October 24, 2013
• Study Start: August 16, 2017
• Primary Completion: January 1, 2022
• Study Completion: January 1, 2022
• Last Updated: May 25, 2022

Record last verified: 2022-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (2); CA (1)