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

NCT05408910 | Withdrawn Phase 2 DMC FDA Drug

• 2 other identifiers: IRB00086338MOSHIR21-A0-I
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

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; People with Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• 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

  Two weeks

Secondary Outcomes (1)

• Improved ePROS scores

  42 days

Study Arms (2)

Treatment

EXPERIMENTAL

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

Drug: Rifaximin 550 MG Oral Tablet [XIFAXAN]

Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

Rifaximin 550 MG Oral Tablet [XIFAXAN] DRUG

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

Also known as: XIFAXAN

Treatment

Placebo DRUG

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

Placebo

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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 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.

Sponsors & Collaborators

• Wake Forest University Health Sciences: lead
• Nationwide Children's Hospital: collaborator
• University of Minnesota: collaborator
• University of Texas Southwestern Medical Center: collaborator

Related Publications (1)

• 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.

  PMID: 30232597 BACKGROUND

MeSH Terms

Conditions

Cystic Fibrosis; Abdominal Pain

Interventions

Rifaximin; Tablets

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Pain; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Signs and Symptoms, Digestive

Intervention Hierarchy (Ancestors)

Rifamycins; Heterocyclic Compounds, 4 or More Rings; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds; Lactams, Macrocyclic; Macrocyclic Compounds; Polycyclic Compounds; Dosage Forms; Pharmaceutical Preparations

Study Officials

• Baha Moshiree, MD

  Wake Forest University Health Sciences

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: June 2, 2022
• First Posted: June 7, 2022
• Study Start: July 1, 2025
• Primary Completion: September 1, 2025
• Study Completion: September 1, 2025
• Last Updated: June 27, 2025

Record last verified: 2025-06