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

Primary Purpose

Neurofibromatosis Type 1 (NF1)

Status
Terminated
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Cholecalciferol
Sponsored by
University of Utah
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional prevention trial for Neurofibromatosis Type 1 (NF1) focused on measuring Vitamin D, Cholecalciferol, Bone density

Eligibility Criteria

25 Years - 40 Years (Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Cincinnati Children's Hospital Medical Center
  • University of Utah
  • University of British Columbia

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Active Comparator

Arm Label

cholecalciferol 600 IU

cholecalciferol 4,000 IU

Arm Description

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

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

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Bone Fractures
Secondary outcome measure of history of bone fractures.
Quality of life questionnaires
Secondary outcome measures include quality of life

Full Information

First Posted
October 7, 2013
Last Updated
May 17, 2022
Sponsor
University of Utah
Collaborators
U.S. Army Medical Research and Development Command, Universitätsklinikum Hamburg-Eppendorf, University of British Columbia, Children's Hospital Medical Center, Cincinnati
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1. Study Identification

Unique Protocol Identification Number
NCT01968590
Brief Title
Vitamin D Supplementation for Adults With Neurofibromatosis Type 1 (NF1)
Acronym
VitDBoneNF1
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)
Study Type
Interventional

2. Study Status

Record Verification Date
May 2022
Overall Recruitment Status
Terminated
Why Stopped
unable to enroll
Study Start Date
August 16, 2017 (Actual)
Primary Completion Date
January 1, 2022 (Actual)
Study Completion Date
January 1, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University of Utah
Collaborators
U.S. Army Medical Research and Development Command, Universitätsklinikum Hamburg-Eppendorf, University of British Columbia, Children's Hospital Medical Center, Cincinnati

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neurofibromatosis Type 1 (NF1)
Keywords
Vitamin D, Cholecalciferol, Bone density

7. Study Design

Primary Purpose
Prevention
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
26 (Actual)

8. Arms, Groups, and Interventions

Arm Title
cholecalciferol 600 IU
Arm Type
Active Comparator
Arm Description
cholecalciferol in Ddrops form at either 600 International units per day versus 4,000 IU per day
Arm Title
cholecalciferol 4,000 IU
Arm Type
Active Comparator
Arm Description
Cholecalciferol at 4,000 IU per day in the form of liquid Ddrops
Intervention Type
Drug
Intervention Name(s)
Cholecalciferol
Other Intervention Name(s)
Vitamin D, D Drops, Dietary Supplement
Intervention Description
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.
Primary Outcome Measure Information:
Title
Bone mineral density
Description
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.
Time Frame
Change from Day 1 to 2 years
Secondary Outcome Measure Information:
Title
Bone Fractures
Description
Secondary outcome measure of history of bone fractures.
Time Frame
change from Day 1 through 2 years
Title
Quality of life questionnaires
Description
Secondary outcome measures include quality of life
Time Frame
change from Day 1 through 2 years
Other Pre-specified Outcome Measures:
Title
Parathyroid hormone with calcium
Description
Serum samples will be analyzed for intact parathyroid hormone with calcium.
Time Frame
change from day1 through year 2
Title
25(OH) vitamin D
Description
Serum samples will be analyzed for 25(OH) vitamin D
Time Frame
change from day1 through year 2

10. Eligibility

Sex
All
Minimum Age & Unit of Time
25 Years
Maximum Age & Unit of Time
40 Years
Accepts Healthy Volunteers
No
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: 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
David H. Viskochil, MD, PhD
Organizational Affiliation
University of Utah
Official's Role
Principal Investigator
Facility Information:
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84108
Country
United States
Facility Name
University of British Columbia
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V6T 1Z4
Country
Canada

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing Plan Description
Publication of all study data will be written
Citations:
PubMed Identifier
23021159
Citation
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.
Results Reference
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Vitamin D Supplementation for Adults With Neurofibromatosis Type 1 (NF1)

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