Neurofibromatosis Type 1 (NF1)Phase 2VitDBoneNF1NCT01968590
Vitamin D Supplementation for Adults With Neurofibromatosis Type 1 (NF1)
A Phase 2 study testing Cholecalciferol in people with Neurofibromatosis Type 1 (NF1), run by University of Utah at 3 sites in 2 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 25 to 40 years
- Conditions the study is for
- Neurofibromatosis Type 1 (NF1)
- What is being tested?
- Cholecalciferol
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 16 Aug 2017to 1 Jan 2022
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 25 to 40 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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 -
Who cannot join
- 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
Show all 14Show fewer
- 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
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
United States2 sites
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
University of Utah
Salt Lake City, Utah, 84108
Canada1 site
University of British Columbia
Vancouver, British Columbia, V6T 1Z4
About this study
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)
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.
Read more
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.
Read the 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.
- Conditions
- Neurofibromatosis Type 1 (NF1)
- Being tested
- Cholecalciferol
- Keywords
- Vitamin D, Cholecalciferol, Bone density
- Medical subject headings
- Neurofibromatosis 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CholecalciferolDrug
Also called Vitamin D, D Drops, Dietary Supplement
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.
| Study group | Role | What they receive |
|---|---|---|
| cholecalciferol 600 IU | Active comparator | cholecalciferol in Ddrops form at either 600 International units per day versus 4,000 IU per day Receives: Drug: Cholecalciferol |
| cholecalciferol 4,000 IU | Active comparator | Cholecalciferol at 4,000 IU per day in the form of liquid Ddrops Receives: Drug: Cholecalciferol |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Change from Day 1 to 2 years
Secondary outcomes (2)
Bone Fractures
Secondary outcome measure of history of bone fractures.
Measured: change from Day 1 through 2 years
Quality of life questionnaires
Secondary outcome measures include quality of life
Measured: change from Day 1 through 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Oct 2013 (estimated)
- Study start16 Aug 2017
- Primary completion1 Jan 2022
- Study completion1 Jan 2022
Why it stopped: unable to enroll
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Neurofibromatosis Type 1 (NF1)Studies of the same condition, open and under way.
- More trials run by University of UtahThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Utah
- Sponsor type
- University, hospital or other
- Collaborators
- U.S. Army Medical Research and Development Command, Universitätsklinikum Hamburg-Eppendorf, University of British Columbia, Children's Hospital Medical Center, Cincinnati
- Responsible party
- Principal investigator, David Viskochil, University of Utah
- Investigators
- David H. Viskochil, MD, PhD, Principal investigator, University of Utah
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01968590
- Sponsor's protocol ID
- DoD A-17236
- Other IDs
- W18XWH-12-1-0487
- Acronym
- VitDBoneNF1
- Status last verified
- May 2022
- First submitted
- 7 Oct 2013
- Last updated
- 25 May 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Publication of all study data will be written
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.
PubMed 23021159
Study at a glance
From the registry record, last updated by the sponsor on 25 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT01968590
- Acronym / sponsor ID
- VitDBoneNF1 / DoD A-17236
- Conditions
- Neurofibromatosis Type 1 (NF1)
- Keywords
- Vitamin D, Cholecalciferol, Bone density
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: unable to enroll
- Enrolment
- 26 participants (actual)
- Lead sponsor
- University of UtahUniversity, hospital or other
- Collaborators
- U.S. Army Medical Research and Development Command, Universitätsklinikum Hamburg-Eppendorf, University of British Columbia, Children's Hospital Medical Center, Cincinnati
- Study start
- 16 Aug 2017 (actual)
- Primary completion
- 1 Jan 2022 (actual)
- Study completion
- 1 Jan 2022 (actual)
- First posted
- 24 Oct 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| cholecalciferol 600 IU | Active comparator | Drug: Cholecalciferolcholecalciferol in Ddrops form at either 600 International units per day versus 4,000 IU per day |
| cholecalciferol 4,000 IU | Active comparator | Drug: CholecalciferolCholecalciferol at 4,000 IU per day in the form of liquid Ddrops |
Who may qualify
- Ages
- 25 to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (1 listed)
- 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 -
Key exclusion criteria (14 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CholecalciferolAlso: Vitamin D, D Drops, Dietary SupplementDDrops(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. | Drug | cholecalciferol 4,000 IU, cholecalciferol 600 IU |
Primary outcome
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.
2 secondary outcomes
Bone Fractures
Time frame: change from Day 1 through 2 years
Quality of life questionnaires
Time frame: change from Day 1 through 2 years
Results and safety
The study was stopped early (reason given: unable to enroll). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 May 2022.
- Background: PubMed 23021159Schnabel 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.
- Individual participant dataSharing planned: No. Publication of all study data will be written
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
David H. Viskochil, MD, PhD principal investigator
University of Utah
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Cholecalciferol in people with Neurofibromatosis Type 1 (NF1), run by University of Utah at 3 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 25 May 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: unable to enroll). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Bone mineral density (time frame: Change from Day 1 to 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01968590, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 May 2022.