Rifaximin for Treatment of Bloating in Children and Adults With Cystic Fibrosis
Primary Purpose
Cystic Fibrosis, Abdominal Pain, Small Bowel Disease
Status
Not yet recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Rifaximin 550 MG Oral Tablet [XIFAXAN]
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Cystic Fibrosis focused on measuring Cystic Fibrosis, Cystic Fibrosis in Children, Rifaximin, Bloating, Abdominal distension, Abdominal pain, Small intestinal bacterial overgrowth, Intestinal Dysbiosis, People with Cystic Fibrosis
Eligibility Criteria
Inclusion Criteria:
- Confirmed CF diagnosis who are enrolled in the CFF registry.
- Patient ages ≥12 years and ≥ 30 kilograms (~66.15 lbs)
- Ability to provide informed consent
Exclusion Criteria:
- Patients who have previously been allergic to rifaximin or had a hypersensitivity to rifamycin
- Patients with FEV1 < 40 will be excluded from the study given potential risks in patients with advanced lung disease
- Patients who have taken any probiotics or new antibiotic for treatment of an acute pulmonary infection 4 weeks prior or during the study period
- Patients with a recent pulmonary exacerbation defined as 4 weeks prior to screening will not be enrolled
- Patients who are on probiotics will be asked to discontinue the use of probiotics 14 days prior to study enrolment as probiotics can alter the gut microbiome and cause bloating
- Patients with newly initiated CFTR modulator treatments within one month prior to the study
- Patients with new onset of DIOS (Distal Intestinal Obstruction Syndrome 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
- Patients with bilirubin or alkaline phosphatase elevations >2x normal at baseline will be excluded as this may be related to CFTR modulator use
Sites / Locations
- Atrium Health
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Treatment
Placebo
Arm Description
Participants in this arm will receive Rifaximin 550 mg three times daily for 14 days.
Participants in this arm will receive placebo three times daily for 14 days.
Outcomes
Primary Outcome Measures
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
Secondary Outcome Measures
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"
Full Information
NCT ID
NCT05408910
First Posted
June 2, 2022
Last Updated
September 28, 2023
Sponsor
Wake Forest University Health Sciences
Collaborators
Nationwide Children's Hospital, University of Minnesota, University of Texas Southwestern Medical Center
1. Study Identification
Unique Protocol Identification Number
NCT05408910
Brief Title
Rifaximin for Treatment of Bloating in Children and Adults With Cystic Fibrosis
Official Title
Rifaximin for Treatment of Bloating in Children and Adults With Cystic Fibrosis
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
November 2023 (Anticipated)
Primary Completion Date
January 2024 (Anticipated)
Study Completion Date
January 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Wake Forest University Health Sciences
Collaborators
Nationwide Children's Hospital, University of Minnesota, University of Texas Southwestern Medical Center
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes
5. Study Description
Brief 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. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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, People with Cystic Fibrosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
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
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
100 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Treatment
Arm Type
Experimental
Arm Description
Participants in this arm will receive Rifaximin 550 mg three times daily for 14 days.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Participants in this arm will receive placebo three times daily for 14 days.
Intervention Type
Drug
Intervention Name(s)
Rifaximin 550 MG Oral Tablet [XIFAXAN]
Intervention Description
Participants in this arm will receive Rifaximin 550 mg three times daily for 14 days.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Participants in this arm will receive placebo three times daily for 14 days.
Primary Outcome Measure Information:
Title
Rifaximin Treatment Group Improvement of Symptoms
Description
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
Time Frame
Two weeks
Secondary Outcome Measure Information:
Title
Improved ePROS scores
Description
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"
Time Frame
42 days
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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)
Exclusion Criteria:
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
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Baha Moshiree, MD
Phone
704-355-0244
Email
bmoshire@wakehealth.edu
First Name & Middle Initial & Last Name or Official Title & Degree
Jamie Crawford, BSN
Phone
704-355-0282
Email
jamie.crawford@atriumhealth.org
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Baha Moshiree, MD
Organizational Affiliation
Wake Forest University Health Sciences
Official's Role
Principal Investigator
Facility Information:
Facility Name
Atrium Health
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28204
Country
United States
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jamie Crawford, BSN
Phone
704-355-0282
Email
jamie.crawford@atriumhealth.org
First Name & Middle Initial & Last Name & Degree
Katelyn Glen, BS
Phone
704.355.0282
Email
katelyn.glen@atriumhealth.org
12. IPD Sharing Statement
Citations:
PubMed Identifier
30232597
Citation
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.
Results Reference
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Rifaximin for Treatment of Bloating in Children and Adults With Cystic Fibrosis
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