Rifaximin for Treatment of Bloating in Children and Adults With Cystic Fibrosis

Sponsor

Wake Forest University Health Sciences (Other)

Overall Status

Not yet recruiting

CT.gov ID

NCT05408910

Collaborator

Emory University (Other), University of Minnesota (Other), University of Texas Southwestern Medical Center (Other)

100

Enrollment

Location

Arms

Anticipated Duration (Months)

6.7

Patients Per Site Per Month

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis Abdominal Pain Small Bowel Disease	Drug: Rifaximin 550 MG Oral Tablet [XIFAXAN] Drug: Placebo	Phase 2/Phase 3

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

Study Type:

Interventional

Anticipated Enrollment :

100 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Intervention 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.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 (Participant, Care Provider, Investigator)

Primary Purpose:

Treatment

Official Title:

Rifaximin for Treatment of Bloating in Children and Adults With Cystic Fibrosis

Anticipated Study Start Date :

Sep 1, 2022

Anticipated Primary Completion Date :

Sep 1, 2023

Anticipated Study Completion Date :

Dec 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Treatment Participants in this arm will receive Rifaximin 550 mg three times daily for 14 days.	Drug: Rifaximin 550 MG Oral Tablet [XIFAXAN] Participants in this arm will receive Rifaximin 550 mg three times daily for 14 days.
Placebo Comparator: Placebo Participants in this arm will receive placebo three times daily for 14 days.	Drug: Placebo Participants in this arm will receive placebo three times daily for 14 days.

Arm

Intervention/Treatment

Experimental: Treatment

Participants in this arm will receive Rifaximin 550 mg three times daily for 14 days.

Drug: Rifaximin 550 MG Oral Tablet [XIFAXAN]

Participants in this arm will receive Rifaximin 550 mg three times daily for 14 days.

Placebo Comparator: Placebo

Participants in this arm will receive placebo three times daily for 14 days.

Drug: Placebo

Participants in this arm will receive placebo three times daily for 14 days.

Outcome Measures

Primary Outcome Measures

Rifaximin Treatment Group Improvement of Symptoms [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

Secondary Outcome Measures

Improved ePROS scores [42 days]
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

Eligibility Criteria

Criteria

Ages Eligible for Study:

12 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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