Cystic FibrosisPhase 2/3NCT05408910
Rifaximin for Treatment of Bloating in Children and Adults With Cystic Fibrosis
A Phase 2/3 study testing Rifaximin 550 MG Oral Tablet [XIFAXAN] in people with Cystic Fibrosis and Abdominal Pain, run by Wake Forest University Health Sciences.
Trial summary
Gastrointestinal symptoms are commonly reported in as much as 65% of people with CF even independent of pancreatic enzyme replacement therapy (PERT) and the most frequent of these symptoms are bloating/distension, flatulence, abdominal pain and bowel habit changes. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Cystic Fibrosis, Abdominal Pain, Small Bowel Disease
- What is being tested?
- Rifaximin 550 MG Oral Tablet [XIFAXAN]
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Jul 2025to Sep 2025
- Phase
- Phase 2/3
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Confirmed CF diagnosis who are enrolled in the CFF registry.
- Mild to severe symptom severity defined as abdominal Distention score ≥ 2 and/or bloating score ≥ 2 on a Likert Scale of 0-6)
- Patient age ≥12 years and ≥ 30 kilograms (~66.15 lbs)
- Ability to provide informed consent or presence of legally authorized representative (LAR)
- Ability to take drug or placebo by mouth (Pill must be intact. May not be opened, crushed, or modified to aid in ingestion)
Who cannot join
- Subjects who have previously been allergic to rifaximin or had a hypersensitivity to rifamycin or used rifaximin for any reason within three months (12 weeks) of the study start date
- Subjects with FEV1 < 40 (as measured within the last 12 months) will be excluded from the study given potential risks in subjects with advanced lung disease
- Subjects who have received a new antibiotic for treatment of an acute pulmonary infection, or antibiotics for any other infection within 4 weeks prior to randomization or during the study period. Cyclic Antibiotics- Inhaled cyclic antibiotics are allowed at any timepoint. Oral or systemic cyclic antibiotics are exclusionary except for prophylactic antibiotics (e.g., azithromycin) which are allowed. New prophylactic antibiotics cannot be started within 4 weeks of randomization.
- Subjects with a recent pulmonary exacerbation defined as 4 weeks prior to screening will not be enrolled
- Subjects who are on probiotics will be asked to discontinue the use of probiotics 14 days prior to randomization as probiotics can alter the gut microbiome and cause bloating
- Subjects with newly initiated cystic fibrosis transmembrane conductance regulator (CFTR) modulator treatments within one month prior to the study
Show all 13Show fewer
- Subjects with new onset of distal intestinal obstruction syndrome (DIOS) or constipation
- Subjects with advanced liver disease defined by:
- portal hypertension and/or child Pugh B or C cirrhosis
- or those with elevated liver enzymes-both AST/ALT > 3 times the upper limit of normal at screening
- Subjects with bilirubin or alkaline phosphatase elevations > 2 times the upper limit of normal at screening will be excluded as this may be related to CFTR modulator use
- Women of childbearing potential who are pregnant, trying to become pregnant, breastfeeding, or not using an acceptable method of contraception as described in Section 6.2.
- Known clostridium difficile colitis. Colonization with c. difficile is not exclusionary.
About this study
Gastrointestinal symptoms are commonly reported in as much as 65% of people with CF even independent of pancreatic enzyme replacement therapy (PERT) and the most frequent of these symptoms are bloating/distension, flatulence, abdominal pain and bowel habit changes. An alteration in the intestinal microbiome due to intestinal dysmotility, inflammation or other changes including pH changes in the intestine related to CFTR gene mutation may cause intestinal dysbiosis leading to a bacterial overgrowth in the proximal small intestine which may explain some of the findings of distension and bloating in CF.
Our small pilot study aims to investigate use of the only FDA-approved antibiotic, rifaximin for a GI syndrome- IBS, to treat bloating and global GI symptoms in CF patients with bloating and distension. Our goal is to recruit patients >12 years and age/sex matched into rifaximin and placebo arms with total of 100 recruited subjects recruited.
Read the detailed description
Gastrointestinal symptoms are commonly reported in persons with cystic fibrosis-both adults and pediatrics- and these symptoms cause distress, impact patients quality of life, and can lead to poor nutrition. One of the findings from our largest US study of patient-reported GI symptom outcomes in CF called GALAXY, which enrolled 402 adults and children with CF, was the finding of high rates of patient dissatisfaction with current GI treatments as well as commonly reported symptoms of bloating (48%), fullness (67%) with meals and abdominal distension (50%). This study aims to evaluate if this same-nonsystemically absorbed and noninvasive antibiotic can be used to treat similar commonly reported symptoms in people with CF.
- Conditions
- Cystic Fibrosis, Abdominal Pain, Small Bowel Disease
- Being tested
- Rifaximin 550 MG Oral Tablet [XIFAXAN], Placebo
- Keywords
- Cystic Fibrosis, Cystic Fibrosis in Children, Rifaximin, Bloating, Abdominal distension, Abdominal pain, Small intestinal bacterial overgrowth, Intestinal Dysbiosis, People with Cystic Fibrosis
- Medical subject headings
- Cystic Fibrosis, Abdominal Pain
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Rifaximin 550 MG Oral Tablet [XIFAXAN]Drug
Also called XIFAXAN
Participants in this arm will receive Rifaximin 550 mg three times daily for 14 days.
PlaceboDrug
Participants in this arm will receive placebo three times daily for 14 days.
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | Participants in this arm will receive Rifaximin 550 mg three times daily for 14 days. Receives: Drug: Rifaximin 550 MG Oral Tablet [XIFAXAN] |
| Placebo | Placebo comparator | Participants in this arm will receive placebo three times daily for 14 days. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rifaximin Treatment Group Improvement of Symptoms
The primary aim and outcome of our study is to show a significant improvement in individual symptoms of abdominal bloating and/or distension, in PwCF (People with Cystic Fibrosis) treated with rifaximin as compared to placebo
Measured: Two weeks
Secondary outcomes (1)
Improved ePROS scores
Our secondary aim and outcome will be based on improvements in the overall ePROS scores and sub-scores for bloating and distension as obtained from GALAXY. These questionnaires will be on a Likert scale ranging from 0-6 where 0 is "Not at all" and 6 is "A very great deal"
Measured: 42 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jun 2022
- Study startJul 2025 (estimated)
- Primary completionSep 2025 (estimated)
- Study completionSep 2025 (estimated)
Why it stopped: new drug formulation by the manufacturer
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 Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Wake Forest University Health SciencesThe 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This is a parallel-designed multicenter randomized controlled trial of rifaximin versus placebo done in 100 patients with CF age 12 and up and over 30 kg of weight recruited based on bloating and distension symptoms.
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Wake Forest University Health Sciences
- Sponsor type
- University, hospital or other
- Collaborators
- Nationwide Children's Hospital, University of Minnesota, University of Texas Southwestern Medical Center
- Responsible party
- Sponsor
- Investigators
- Baha Moshiree, MD, Principal investigator, Wake Forest University Health Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05408910
- Sponsor's protocol ID
- IRB00086338
- Other IDs
- MOSHIR21-A0-I
- Status last verified
- Jun 2025
- First submitted
- 2 Jun 2022
- Last updated
- 27 Jun 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Furnari M, De Alessandri A, Cresta F, Haupt M, Bassi M, Calvi A, Haupt R, Bodini G, Ahmed I, Bagnasco F, Giannini EG, Casciaro R. The role of small intestinal bacterial overgrowth in cystic fibrosis: a randomized case-controlled clinical trial with rifaximin. J Gastroenterol. 2019 Mar;54(3):261-270. doi: 10.1007/s00535-018-1509-4. Epub 2018 Sep 19.
PubMed 30232597
Study at a glance
From the registry record, last updated by the sponsor on 27 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Rifaximin for Treatment of Bloating in Children and Adults With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT05408910
- Acronym / sponsor ID
- IRB00086338
- Conditions
- Cystic Fibrosis, Abdominal Pain, Small Bowel Disease
- Keywords
- Cystic Fibrosis, Cystic Fibrosis in Children, Rifaximin, Bloating, Abdominal distension, Abdominal pain, Small intestinal bacterial overgrowth, Intestinal Dysbiosis
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: new drug formulation by the manufacturer
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Wake Forest University Health SciencesUniversity, hospital or other
- Collaborators
- Nationwide Children's Hospital, University of Minnesota, University of Texas Southwestern Medical Center
- Study start
- Jul 2025 (estimated)
- Primary completion
- Sep 2025 (estimated)
- Study completion
- Sep 2025 (estimated)
- First posted
- 7 Jun 2022 (actual)
- 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.
Gastrointestinal symptoms are commonly reported in as much as 65% of people with CF even independent of pancreatic enzyme replacement therapy (PERT) and the most frequent of these symptoms are bloating/distension, flatulence, abdominal pain and bowel habit changes. An alteration in the intestinal microbiome due to intestinal dysmotility, inflammation or other changes including pH changes in the intestine related to CFTR gene mutation may cause intestinal dysbiosis leading to a bacterial overgrowth in the proximal small intestine which may explain some of the findings of distension and bloating in CF.
Our small pilot study aims to investigate use of the only FDA-approved antibiotic, rifaximin for a GI syndrome- IBS, to treat bloating and global GI symptoms in CF patients with bloating and distension. Our goal is to recruit patients >12 years and age/sex matched into rifaximin and placebo arms with total of 100 recruited subjects recruited.
Detailed description
Gastrointestinal symptoms are commonly reported in persons with cystic fibrosis-both adults and pediatrics- and these symptoms cause distress, impact patients quality of life, and can lead to poor nutrition. One of the findings from our largest US study of patient-reported GI symptom outcomes in CF called GALAXY, which enrolled 402 adults and children with CF, was the finding of high rates of patient dissatisfaction with current GI treatments as well as commonly reported symptoms of bloating (48%), fullness (67%) with meals and abdominal distension (50%). This study aims to evaluate if this same-nonsystemically absorbed and noninvasive antibiotic can be used to treat similar commonly reported symptoms in people with CF.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
- Model description
- This is a parallel-designed multicenter randomized controlled trial of rifaximin versus placebo done in 100 patients with CF age 12 and up and over 30 kg of weight recruited based on bloating and distension symptoms.
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Drug: Rifaximin 550 MG Oral Tablet [XIFAXAN]Participants in this arm will receive Rifaximin 550 mg three times daily for 14 days. |
| Placebo | Placebo comparator | Drug: PlaceboParticipants in this arm will receive placebo three times daily for 14 days. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Confirmed CF diagnosis who are enrolled in the CFF registry.
- Mild to severe symptom severity defined as abdominal Distention score ≥ 2 and/or bloating score ≥ 2 on a Likert Scale of 0-6)
- Patient age ≥12 years and ≥ 30 kilograms (~66.15 lbs)
- Ability to provide informed consent or presence of legally authorized representative (LAR)
- Ability to take drug or placebo by mouth (Pill must be intact. May not be opened, crushed, or modified to aid in ingestion)
Key exclusion criteria (13 listed)
- Subjects who have previously been allergic to rifaximin or had a hypersensitivity to rifamycin or used rifaximin for any reason within three months (12 weeks) of the study start date
- Subjects with FEV1 < 40 (as measured within the last 12 months) will be excluded from the study given potential risks in subjects with advanced lung disease
- Subjects who have received a new antibiotic for treatment of an acute pulmonary infection, or antibiotics for any other infection within 4 weeks prior to randomization or during the study period. Cyclic Antibiotics- Inhaled cyclic antibiotics are allowed at any timepoint. Oral or systemic cyclic antibiotics are exclusionary except for prophylactic antibiotics (e.g., azithromycin) which are allowed. New prophylactic antibiotics cannot be started within 4 weeks of randomization.
- Subjects with a recent pulmonary exacerbation defined as 4 weeks prior to screening will not be enrolled
- Subjects who are on probiotics will be asked to discontinue the use of probiotics 14 days prior to randomization as probiotics can alter the gut microbiome and cause bloating
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 |
|---|---|---|
| Rifaximin 550 MG Oral Tablet [XIFAXAN]Also: XIFAXANParticipants in this arm will receive Rifaximin 550 mg three times daily for 14 days. | Drug | Treatment |
| PlaceboParticipants in this arm will receive placebo three times daily for 14 days. | Drug | Placebo |
Primary outcome
Rifaximin Treatment Group Improvement of Symptoms
Time frame: Two weeks
The primary aim and outcome of our study is to show a significant improvement in individual symptoms of abdominal bloating and/or distension, in PwCF (People with Cystic Fibrosis) treated with rifaximin as compared to placebo
1 secondary outcome
Improved ePROS scores
Time frame: 42 days
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 27 Jun 2025.
- Background: PubMed 30232597Furnari M, De Alessandri A, Cresta F, Haupt M, Bassi M, Calvi A, Haupt R, Bodini G, Ahmed I, Bagnasco F, Giannini EG, Casciaro R. The role of small intestinal bacterial overgrowth in cystic fibrosis: a randomized case-controlled clinical trial with rifaximin. J Gastroenterol. 2019 Mar;54(3):261-270. doi: 10.1007/s00535-018-1509-4. Epub 2018 Sep 19.
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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.
Baha Moshiree, MD principal investigator
Wake Forest University Health Sciences
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Rifaximin 550 MG Oral Tablet [XIFAXAN] in people with Cystic Fibrosis and Abdominal Pain, run by Wake Forest University Health Sciences.
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 5 inclusion and 13 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 27 Jun 2025. 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. 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 combined 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.
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?
Rifaximin Treatment Group Improvement of Symptoms (time frame: Two weeks). 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 NCT05408910, 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 27 Jun 2025.