Trial record 5 of 38 for:    Open Studies | "Neurofibromatosis 1"

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

This study is not yet open for participant recruitment. (see Contacts and Locations)
Verified October 2013 by University of Utah
Sponsor:
Collaborators:
U.S. Army Medical Research and Materiel Command
Universitätsklinikum Hamburg-Eppendorf
University of British Columbia
Children's Hospital Medical Center, Cincinnati
Information provided by (Responsible Party):
Dave Viskochil, University of Utah
ClinicalTrials.gov Identifier:
NCT01968590
First received: October 7, 2013
Last updated: October 19, 2013
Last verified: October 2013
  Purpose

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.


Condition Intervention Phase
Neurofibromatosis Type 1 (NF1)
Drug: Cholecalciferol
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Prevention
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)

Resource links provided by NLM:


Further study details as provided by University of Utah:

Primary Outcome Measures:
  • Bone mineral density [ Time Frame: Day 1 and at 2 years ] [ Designated as safety issue: No ]
    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.


Secondary Outcome Measures:
  • Quality of life questionnaires [ Time Frame: Day 1, year 1 and at 2 years ] [ Designated as safety issue: No ]
    Secondary outcome measures include diet analysis, activity level, quality of life, and history of bone fractures.


Other Outcome Measures:
  • Serum samples [ Time Frame: samples at day1 , year 1, and year 2 ] [ Designated as safety issue: No ]
    Serum samples will be analyzed for 25(OH) vitamin D, calcium, and intact parathyroid hormone.


Estimated Enrollment: 320
Study Start Date: March 2014
Estimated Study Completion Date: December 2016
Estimated Primary Completion Date: June 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: cholecalciferol 600 IU
cholecalciferol in Ddrops form at either 600 International units per day versus 4,000 IU per day
Drug: Cholecalciferol
A pharmacist employed by DDrops(trademark) prepares two sets of bottles, one set has 600IU per metered dose and the other set has 4,000IU per metered dose. Participants are randomized to one or the other dose, and they take one measured drop 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
Active Comparator: cholecalciferol 4,000 IU
Cholecalciferol at 4,000 IU per day in the form of liquid Ddrops
Drug: Cholecalciferol
A pharmacist employed by DDrops(trademark) prepares two sets of bottles, one set has 600IU per metered dose and the other set has 4,000IU per metered dose. Participants are randomized to one or the other dose, and they take one measured drop 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

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. Each bottle lasts 6 months and will be resupplied at the mid-year evaluation.

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 diet, activity level, 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.

  Eligibility

Ages Eligible for Study:   25 Years to 40 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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. lack of NF1 diagnostic criteria on physical examination
  2. they foresee that they will be unable to comply with the 2-year study protocol
  3. they are pregnant at the time of DXA scanning
  4. their initial screening 25(OH)D level is >29 ng/ml (sufficient)
  5. their initial screening 25(OH)D level is <8 ng/ml (deficient)
  6. lack of NF1 diagnostic criteria on physical examination
  7. vitamin D supplementation in the last 6 months equal to or greater than 600 IU per day
  8. high-dose steroid use
  9. bisphosphonate therapy
  10. thyroid therapy
  11. calcium supplementation in last 6 months >1000mg per day
  12. malignant peripheral nerve sheath tumor
  13. history of kidney stones and hypercalciuria,
  14. individuals with metal instrumentation in spine or hip that preclude accurate DXA interpretation.
  15. inability to obtain blood samples on routine venipuncture
  16. anti-epileptic therapy
  17. anticoagulant therapy
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01968590

Contacts
Contact: Heather Hanson, CCRC 801-587-9017 heather.hanson@hsc.utah.edu

Locations
United States, Ohio
Cincinnati Children's Hospital Medical Center Not yet recruiting
Cincinnati,, Ohio, United States, 45229
Contact: Sara Manning, MPH, CCRC    513-803-0077    Sara.Manning@cchmc.org   
Sub-Investigator: Elizabeth Schorry, MD         
Canada, British Columbia
University of British Columbia Not yet recruiting
Vancouver, British Columbia, Canada, V6T 1Z4
Contact: Patricia Birch, RN    1 604-822-2211    birch@interchange.ubc.ca   
Sub-Investigator: Jan Friedman, MD PhD         
Germany
University Medical Center Hamburg-Eppendorf Not yet recruiting
Hamburg, Germany, D - 20246
Contact: Claudia Wargel    (040) 7410-53273    nfambulanz@uke.uni-hamburg.de   
Sub-Investigator: Victor Mautner, MD         
Sponsors and Collaborators
University of Utah
U.S. Army Medical Research and Materiel Command
Universitätsklinikum Hamburg-Eppendorf
University of British Columbia
Children's Hospital Medical Center, Cincinnati
Investigators
Principal Investigator: David H. Viskochil, MD, PhD University of Utah
  More Information

Publications:
Responsible Party: Dave Viskochil, David H .Viskochil M.D. , Ph.D professor of pediatrics, University of Utah
ClinicalTrials.gov Identifier: NCT01968590     History of Changes
Other Study ID Numbers: DoD A-17236, W18XWH-12-1-0487
Study First Received: October 7, 2013
Last Updated: October 19, 2013
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
Germany: Federal Institute for Drugs and Medical Devices

Keywords provided by University of Utah:
Vitamin D
Cholecalciferol
Bone density

Additional relevant MeSH terms:
Neurofibromatoses
Neurofibromatosis 1
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
Genetic Diseases, Inborn
Peripheral Nervous System Diseases
Neuromuscular Diseases
Cholecalciferol
Vitamin D
Ergocalciferols
Vitamins
Micronutrients
Growth Substances
Physiological Effects of Drugs
Pharmacologic Actions
Bone Density Conservation Agents

ClinicalTrials.gov processed this record on August 28, 2014