Cystic FibrosisPhase 4NCT04118010
Vitamin D and Prebiotics for Intestinal Health in Cystic Fibrosis
A Phase 4 study testing Vitamin D3 and Inulin in people with Cystic Fibrosis and Dysbiosis, run by Emory University at 1 site in United States.
Trial summary
The study will assess if administration of high-dose vitamin D and a commonly used prebiotic (inulin) is effective to reduce gastrointestinal dysbiosis and to improve critical intestinal functions in Cystic Fibrosis with the additive or synergistic effects of the combination of vitamin D + inulin. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis, Dysbiosis
- What is being tested?
- Vitamin D3 and Inulin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 1 siteUnited States
- Study dates
- 13 Mar 2020to 31 Dec 2022
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- male and female patients (age > 18 years) with confirmed CF by genetic mutation and/or sweat chloride testing,
- not currently on oral or systemic antibiotics for pulmonary exacerbation,
- vitamin D deficient/insufficient (25(OH)D, 6 - 30 ng/mL) with most recent 25(OH)D in the past 12 months,
- use of CFTR modulator therapy is allowed
Who cannot join
- severe vitamin D deficiency 25(OH)D ≤ 5 ng/mL or hypocalcemia or hypercalcemia,
- active GI disease, abdominal pain and/or diarrhea,
- chronic kidney disease worse than stage 3 (eGFR < ml/min per 1.73 m2),
- any vitamin D supplement use >2,000 IU or vitamin D analogue (patients who are taking more than 2,000 IU of vitamin D must agree to stop the vitamin D for 6 weeks and take less than 2,000 IU of vitamin D during the study),
- use of immunosuppressants or history of organ transplantation,
- current use of probiotics or prebiotics
Trial locations and contacts
1 site in 1 country.
About this study
The study will assess if administration of high-dose vitamin D and a commonly used prebiotic (inulin) is effective to reduce gastrointestinal dysbiosis and to improve critical intestinal functions in Cystic Fibrosis with the additive or synergistic effects of the combination of vitamin D + inulin.
Read the detailed description
Cystic fibrosis (CF) is the most common life-shortening genetic condition among Caucasians in the United States. Individuals with CF have an altered gastrointestinal (GI) microbiota, which may be a result of chronic systemic inflammation and infection, frequent use of antibiotics, and/or medically prescribed and habitual high-fat/high-calorie diets.
The study will assess if administration of high-dose vitamin D and a commonly used prebiotic (inulin) is effective to reduce gastrointestinal dysbiosis and to improve critical intestinal functions in Cystic Fibrosis.
- Conditions
- Cystic Fibrosis, Dysbiosis
- Being tested
- Vitamin D3, Placebo vitamin D3, Inulin, Placebo Inulin
- Keywords
- Cystic Fibrosis, Gastrointestinal Dysbiosis, Vitamin D, Prebiotic, Inulin, Gut microbiome, Intestinal inflammation, Intestinal absorption
- Medical subject headings
- Cystic Fibrosis, Dysbiosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vitamin D3Drug
High-dose vitamin D3 50,000 IU /week for 12 weeks
Placebo vitamin D3Drug
Matching to Vitamin D3 placebo capsules for 12 weeks
InulinDrug
Chicory-derived prebiotic inulin 12 g/day for 12 weeks
Placebo InulinDrug
Corn-derived maltodextrin 12g/day as the prebiotic placebo for 12 weeks
| Study group | Role | What they receive |
|---|---|---|
| Vitamin D3 and Inulin | Active comparator | Vitamin D3 50,000 IU/week and 12 g/day chicory-derived prebiotic inulin for 12 weeks Receives: Drug: Vitamin D3; Drug: Inulin |
| Vitamin D3 and placebo Inulin | Active comparator | Vitamin D3 50,000 IU/week and 12 g/day corn-derived maltodextrin/day as the prebiotic placebo for 12 weeks Receives: Drug: Vitamin D3; Drug: Placebo Inulin |
| Placebo vitamin D3 and Inulin | Active comparator | Matching to Vitamin D3 placebo capsules, and 12 g/day chicory-derived prebiotic inulin for 12 weeks Receives: Drug: Placebo vitamin D3; Drug: Inulin |
| Placebo vitamin D3 and placebo Inulin | Placebo comparator | Matching to Vitamin D3 placebo capsules, and 12 g/day corn-derived maltodextrin/day as the prebiotic placebo for 12 weeks Receives: Drug: Placebo vitamin D3; Drug: Placebo Inulin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Shannon Index
The Shannon Index is a measure of diversity of microbial species that takes into account both abundance (the number of species present) and evenness (how close the numbers for each species are). The Shannon index can be calculated using the following equation: H= -∑(i=1)^s pi ln(pi). A value of zero for H indicates that a community has only one species. The higher the value of H, the higher the diversity of species in a particular community.
Sputum microbiota analysis was measured using this ecological diversity measure. Sputum samples were collected via a sputum kit.
Measured: Baseline, 12 weeks post-intervention
Change in Species Richness Index From Baseline
Stool microbiota analysis will be measured using this ecological diversity measure. Stool samples will be collected using a stool kit provided to the participant.
Measured: Baseline, 12 weeks post-intervention
Secondary outcomes (3)
Change in GI Microbiota Diversity
Changes in GI microbiota diversity will be determined using 16S rRNA gene sequencing and microbiome-dependent metabolites pathways in stool and plasma using high-resolution metabolomics analysis.Changes in GI microbiota diversity will be reported as the Shannon Index.
Measured: Baseline, 12 weeks post-intervention
Change in GI Microbiota Richness
Changes in GI microbiota richness will be determined using 16S rRNA gene sequencing and microbiome-dependent metabolites pathways in stool and plasma using high-resolution metabolomics analysis.Changes in GI microbiota richness will be reported as a number of populations of microorganisms.
Measured: Baseline, 12 weeks post-intervention
Change in GI Microbiota Composition
Changes in GI microbiota composition will be determined using 16S rRNA gene sequencing and microbiome-dependent metabolites pathways in stool and plasma using high-resolution metabolomics analysis.Changes in GI microbiota composition will be reported as a percentage of bacteria.
Measured: Baseline, 12 weeks post-intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Oct 2019
- Study start13 Mar 2020
- Primary completion31 Dec 2022
- Study completion31 Dec 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe 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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Vin Tangpricha, Emory University
- Investigators
- Vin Tangpricha, MD, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04118010
- Sponsor's protocol ID
- IRB00114230
- Status last verified
- Jan 2024
- First submitted
- 4 Oct 2019
- Last updated
- 7 Feb 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Individual de-identified participant data (including data dictionaries) be shared with listed investigators
Study at a glance
From the registry record, last updated by the sponsor on 7 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Vitamin D and Prebiotics for Intestinal Health in Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT04118010
- Acronym / sponsor ID
- IRB00114230
- Conditions
- Cystic Fibrosis, Dysbiosis
- Keywords
- Cystic Fibrosis, Gastrointestinal Dysbiosis, Vitamin D, Prebiotic, Inulin, Gut microbiome, Intestinal inflammation, Intestinal absorption
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Study start
- 13 Mar 2020 (actual)
- Primary completion
- 31 Dec 2022 (actual)
- Study completion
- 31 Dec 2022 (actual)
- First posted
- 7 Oct 2019 (actual)
- Results
- Posted 7 Feb 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study will assess if administration of high-dose vitamin D and a commonly used prebiotic (inulin) is effective to reduce gastrointestinal dysbiosis and to improve critical intestinal functions in Cystic Fibrosis with the additive or synergistic effects of the combination of vitamin D + inulin.
Detailed description
Cystic fibrosis (CF) is the most common life-shortening genetic condition among Caucasians in the United States. Individuals with CF have an altered gastrointestinal (GI) microbiota, which may be a result of chronic systemic inflammation and infection, frequent use of antibiotics, and/or medically prescribed and habitual high-fat/high-calorie diets.
The study will assess if administration of high-dose vitamin D and a commonly used prebiotic (inulin) is effective to reduce gastrointestinal dysbiosis and to improve critical intestinal functions in Cystic Fibrosis.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Vitamin D3 and Inulin | Active comparator | Drug: Vitamin D3, Drug: InulinVitamin D3 50,000 IU/week and 12 g/day chicory-derived prebiotic inulin for 12 weeks |
| Vitamin D3 and placebo Inulin | Active comparator | Drug: Vitamin D3, Drug: Placebo InulinVitamin D3 50,000 IU/week and 12 g/day corn-derived maltodextrin/day as the prebiotic placebo for 12 weeks |
| Placebo vitamin D3 and Inulin | Active comparator | Drug: Placebo vitamin D3, Drug: InulinMatching to Vitamin D3 placebo capsules, and 12 g/day chicory-derived prebiotic inulin for 12 weeks |
| Placebo vitamin D3 and placebo Inulin | Placebo comparator | Drug: Placebo vitamin D3, Drug: Placebo InulinMatching to Vitamin D3 placebo capsules, and 12 g/day corn-derived maltodextrin/day as the prebiotic placebo for 12 weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- male and female patients (age > 18 years) with confirmed CF by genetic mutation and/or sweat chloride testing,
- not currently on oral or systemic antibiotics for pulmonary exacerbation,
- vitamin D deficient/insufficient (25(OH)D, 6 - 30 ng/mL) with most recent 25(OH)D in the past 12 months,
- use of CFTR modulator therapy is allowed
Key exclusion criteria (6 listed)
- severe vitamin D deficiency 25(OH)D ≤ 5 ng/mL or hypocalcemia or hypercalcemia,
- active GI disease, abdominal pain and/or diarrhea,
- chronic kidney disease worse than stage 3 (eGFR < ml/min per 1.73 m2),
- any vitamin D supplement use >2,000 IU or vitamin D analogue (patients who are taking more than 2,000 IU of vitamin D must agree to stop the vitamin D for 6 weeks and take less than 2,000 IU of vitamin D during the study),
- use of immunosuppressants or history of organ transplantation,
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 |
|---|---|---|
| Vitamin D3High-dose vitamin D3 50,000 IU /week for 12 weeks | Drug | Vitamin D3 and Inulin, Vitamin D3 and placebo Inulin |
| Placebo vitamin D3Matching to Vitamin D3 placebo capsules for 12 weeks | Drug | Placebo vitamin D3 and Inulin, Placebo vitamin D3 and placebo Inulin |
| InulinChicory-derived prebiotic inulin 12 g/day for 12 weeks | Drug | Placebo vitamin D3 and Inulin, Vitamin D3 and Inulin |
| Placebo InulinCorn-derived maltodextrin 12g/day as the prebiotic placebo for 12 weeks | Drug | Placebo vitamin D3 and placebo Inulin, Vitamin D3 and placebo Inulin |
Primary outcome
Shannon Index
Time frame: Baseline, 12 weeks post-intervention
The Shannon Index is a measure of diversity of microbial species that takes into account both abundance (the number of species present) and evenness (how close the numbers for each species are). The Shannon index can be calculated using the following equation: H= -∑(i=1)^s pi ln(pi). A value of zero for H indicates that a community has only one species. The higher the value of H, the higher the diversity of species in a particular community. Sputum microbiota analysis was measured using this ecological diversity measure. Sputum samples were collected via a sputum kit.
Change in Species Richness Index From Baseline
Time frame: Baseline, 12 weeks post-intervention
Stool microbiota analysis will be measured using this ecological diversity measure. Stool samples will be collected using a stool kit provided to the participant.
3 secondary outcomes
Change in GI Microbiota Diversity
Time frame: Baseline, 12 weeks post-intervention
Change in GI Microbiota Richness
Time frame: Baseline, 12 weeks post-intervention
Change in GI Microbiota Composition
Time frame: Baseline, 12 weeks post-intervention
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Feb 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. Individual de-identified participant data (including data dictionaries) be shared with listed investigators
Related trials for Cystic Fibrosis
Active, not recruitingObservational study
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
Cystic Fibrosis, Bone Loss
RecruitingPhase 2
A Study of 24 to 52 Weeks Treatment to Evaluate The Efficacy And Safety of Galvokimig in Adult Study Participants With Non-Cystic Fibrosis Bronchiectasis
Non-cystic Fibrosis Bronchiectasis
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
Active, not recruiting
Personalized Mobile Health Platform to Promote Physical Activity in Adolescents and Young Adults With Cystic Fibrosis
Cystic Fibrosis (CF)
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.
Vin Tangpricha, MD principal investigator
Emory University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Vitamin D3 and Inulin in people with Cystic Fibrosis and Dysbiosis, run by Emory University at 1 site in United States.
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 6 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: finished, as of the sponsor's last update on 7 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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. Some participants in this study may receive a placebo. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Feb 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Shannon Index (time frame: Baseline, 12 weeks post-intervention). The study lists 2 primary outcomes in all. 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 7 Feb 2024; AzTrials last refreshed its copy on 8 Nov 2026.