Cystic FibrosisPhase 3NCT00762918
Vitamin D3 for the Treatment of Low Vitamin D in Cystic Fibrosis
A Phase 3 study testing cholecalciferol in people with Cystic Fibrosis and Vitamin D Deficiency, run by Children's Hospital of Philadelphia.
Trial summary
Vitamin D deficiency is common in cystic fibrosis. Vitamin D deficiency frequently persists despite aggressive treatment with ergocalciferol, a vitamin D preparation also known as vitamin D2. Cholecalciferol, a vitamin D preparation also known as vitamin D3,may work better to increase vitamin D levels. Read more about this study
- Who is it for?
- Ages 10 to 25 years
- Conditions the study is for
- Cystic Fibrosis, Vitamin D Deficiency
- What is being tested?
- cholecalciferol
- Type of treatment
- Pill or oral medicine
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Mar 2008to Sep 2008
- Phase
- Phase 3
- 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 10 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subjects age 10-25 years
- cystic fibrosis
- 25-(OH)-D < 20 ng/mL
- FEV1 > 40% -
Who cannot join
- inability to perform pulmonary function or hand-grip tests
- liver disease (including cirrhosis and portal hypertension) or baseline liver enzymes 21/2-fold greater than the upper limit of normal
- acute use of glucocorticoids at time of testing
- acute pulmonary exacerbation at time of testing
- known non-adherence to enzyme replacement
- hypercalcemia
- engages in "suntanning
About this study
Official title: Vitamin D and Its Non-Classic Roles in Cystic Fibrosis
Vitamin D deficiency is common in cystic fibrosis. Vitamin D deficiency frequently persists despite aggressive treatment with ergocalciferol, a vitamin D preparation also known as vitamin D2. Cholecalciferol, a vitamin D preparation also known as vitamin D3,may work better to increase vitamin D levels.
Vitamin D is important for absorption of calcium from the diet and bone health. Vitamin D more recently has been found to play a role in regulating the normal inflammatory process. Since cystic fibrosis is a state of excessive inflammation, vitamin D may be playing a role in cystic fibrosis.
We hypothesize: cholecalciferol will work better to increase vitamin D levels in patients iwth cystic fibrosis and that it will have an effect on markers of inflammation.
Read the detailed description
Vitamin D deficiency is common in cystic fibrosis (CF) and persists despite relatively high doses of ergocalciferol, vitamin D2. Replacement has traditionally been focused upon maintenance of calcium and phosphorus homeostasis and bone health. However, non-classic roles of vitamin D have become increasingly recognized and the contribution of vitamin D deficiency to non-bone disorders has become apparent. Vitamin D deficiency has been associated with increased risk of a variety of cancers, autoimmune diseases such as Type 1 diabetes and multiple sclerosis, Type 2 diabetes, tuberculosis, and myopathy. The connection between vitamin D and these disease states likely reflects vitamin D's role as a transcriptional regulator: it participates in cell cycle regulation and in the innate immune system mediates cathelicidin production following activation of toll-like receptors.One hallmark of CF is pulmonary hyper-inflammation with recurrent infections. Additionally, malnutrition and decreased lean muscle mass threaten pulmonary function in CF. While vitamin D and its relation to bone has been explored in CF, the role of vitamin D in inflammation, lean body mass and strength, and pulmonary muscle strength has not been investigated. Moreover, vitamin D replacement has traditionally been with ergocalciferol, vitamin D2. Vitamin D3, cholecalciferol, has a longer half-life and is considered more potent. Thus, cholecalciferol treatment of children and young adults with CF and vitamin D deficiency may be useful for attaining normal vitamin D status and for exploring the impact of vitamin D upon lean body mass, pulmonary muscle strength, and inflammation.
- Conditions
- Cystic Fibrosis, Vitamin D Deficiency
- Being tested
- cholecalciferol
- Keywords
- cystic fibrosis, vitamin d, inflammation
- Medical subject headings
- Cystic Fibrosis, Vitamin D Deficiency, Inflammation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
cholecalciferolDietary supplement
Also called Vitamin D3
cholecalciferol 5000 IU capsule by mouth daily for 3 months
What is being measured
The primary outcome is the main question the study is designed to answer.
serum 25-hydroxy vitamin D levels
Measured: 3 months
Secondary outcomes (3)
body composition
Measured: 3 months
inflammatory markers
Measured: 3 months
muscles strength
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Sep 2008 (estimated)
- Study startMar 2008
- Primary completionSep 2008
- Study completionSep 2008 (estimated)
Why it stopped: Recruitment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Children's Hospital of PhiladelphiaThe 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Philadelphia
- Sponsor type
- University, hospital or other
- Investigators
- Andrea Kelly, MD, Principal investigator, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00762918
- Sponsor's protocol ID
- 2007-12-5688
- Status last verified
- Feb 2010
- First submitted
- 26 Sep 2008
- Last updated
- 22 Feb 2010
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 22 Feb 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Vitamin D and Its Non-Classic Roles in Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT00762918
- Acronym / sponsor ID
- 2007-12-5688
- Conditions
- Cystic Fibrosis, Vitamin D Deficiency
- Keywords
- cystic fibrosis, vitamin d, inflammation
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Recruitment
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Children's Hospital of PhiladelphiaUniversity, hospital or other
- Study start
- Mar 2008
- Primary completion
- Sep 2008 (actual)
- Study completion
- Sep 2008 (estimated)
- First posted
- 30 Sep 2008 (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.
Vitamin D deficiency is common in cystic fibrosis. Vitamin D deficiency frequently persists despite aggressive treatment with ergocalciferol, a vitamin D preparation also known as vitamin D2. Cholecalciferol, a vitamin D preparation also known as vitamin D3,may work better to increase vitamin D levels.
Vitamin D is important for absorption of calcium from the diet and bone health. Vitamin D more recently has been found to play a role in regulating the normal inflammatory process. Since cystic fibrosis is a state of excessive inflammation, vitamin D may be playing a role in cystic fibrosis.
We hypothesize: cholecalciferol will work better to increase vitamin D levels in patients iwth cystic fibrosis and that it will have an effect on markers of inflammation.
Detailed description
Vitamin D deficiency is common in cystic fibrosis (CF) and persists despite relatively high doses of ergocalciferol, vitamin D2. Replacement has traditionally been focused upon maintenance of calcium and phosphorus homeostasis and bone health. However, non-classic roles of vitamin D have become increasingly recognized and the contribution of vitamin D deficiency to non-bone disorders has become apparent. Vitamin D deficiency has been associated with increased risk of a variety of cancers, autoimmune diseases such as Type 1 diabetes and multiple sclerosis, Type 2 diabetes, tuberculosis, and myopathy. The connection between vitamin D and these disease states likely reflects vitamin D's role as a transcriptional regulator: it participates in cell cycle regulation and in the innate immune system mediates cathelicidin production following activation of toll-like receptors.One hallmark of CF is pulmonary hyper-inflammation with recurrent infections. Additionally, malnutrition and decreased lean muscle mass threaten pulmonary function in CF. While vitamin D and its relation to bone has been explored in CF, the role of vitamin D in inflammation, lean body mass and strength, and pulmonary muscle strength has not been investigated. Moreover, vitamin D replacement has traditionally been with ergocalciferol, vitamin D2. Vitamin D3, cholecalciferol, has a longer half-life and is considered more potent. Thus, cholecalciferol treatment of children and young adults with CF and vitamin D deficiency may be useful for attaining normal vitamin D status and for exploring the impact of vitamin D upon lean body mass, pulmonary muscle strength, and inflammation.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 10 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Subjects age 10-25 years
- cystic fibrosis
- 25-(OH)-D < 20 ng/mL
- FEV1 > 40% -
Key exclusion criteria (7 listed)
- inability to perform pulmonary function or hand-grip tests
- liver disease (including cirrhosis and portal hypertension) or baseline liver enzymes 21/2-fold greater than the upper limit of normal
- acute use of glucocorticoids at time of testing
- acute pulmonary exacerbation at time of testing
- known non-adherence to enzyme replacement
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 D3cholecalciferol 5000 IU capsule by mouth daily for 3 months | Dietary supplement | – |
Primary outcome
serum 25-hydroxy vitamin D levels
Time frame: 3 months
3 secondary outcomes
body composition
Time frame: 3 months
inflammatory markers
Time frame: 3 months
muscles strength
Time frame: 3 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Feb 2010.
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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.
Andrea Kelly, MD principal investigator
Children's Hospital of Philadelphia
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing cholecalciferol in people with Cystic Fibrosis and Vitamin D Deficiency, run by Children's Hospital of Philadelphia.
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 4 inclusion and 7 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: withdrawn, as of the sponsor's last update on 22 Feb 2010. Status can change between updates, so confirm with the study contact before making plans.
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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 withdrawn before enrolling participants, so there are no results.
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?
serum 25-hydroxy vitamin D levels (time frame: 3 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 22 Feb 2010.