Baclofen for Children With Rumination Syndrome

NCT ID: NCT05975684

Last Updated: 2024-07-26

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 50 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-01-31
• Study Completion Date: 2024-07-31

Brief Summary

The goal of this clinical trial is to learn about baclofen in pediatric patients with rumination syndrome. The main question it aims to answer is whether baclofen is effective in treating children with rumination syndrome. Participants will be asked to take baclofen or placebo for 4 weeks and fill out surveys regarding symptoms.

Detailed Description

Rumination syndrome is a functional gastrointestinal disorder characterized by effortless, repetitive regurgitation of recently ingested meal. The objective of this study is to evaluate the efficacy of baclofen, a GABA-b agonist, in children with rumination syndrome. The investigators hypothesize that children with rumination syndrome who are treated with baclofen in addition to standard behavioral treatment will have greater clinical improvement compared to those who receive placebo.

After enrollment into the study, participants will be randomized into one of two parallel groups in a 1:1 ratio: (a) baclofen or (b) placebo control. The research team will be blinded to randomization. Participants will subsequently meet with investigational pharmacist and receive four-week supply of either baclofen 0.5 mg/kg/day up to 15 mg divided three times a day or matching placebo three times a day in liquid formulation. At baseline, the research team will collect information on demographics, medical, and surgical history, relevant diagnostic testing, and relevant past treatments (including working with a psychologist). Patients/families will complete a baseline questionnaire (rumination severity questions, PedsQL, route of nutrition, medications) and then the patient will start taking study drug or placebo in addition to their usual care as determined by the patient's primary gastroenterologist. Participants/families will complete follow-up questionnaires at week 1, week 2, week 4, and then week 8. The questionnaires will be filled out by the patient or guardian electronically via REDCap. The research team will closely monitor for side effects. In addition to asking about any side effects at follow-up timepoints, the research team will also emphasize that families should contact the team using the research team phone number with any instances of suspected side effects even before the next follow-up timepoint. After the 4-week drug or placebo period, participants are free to start or continue baclofen, or use other treatment modalities, as determined by their primary GI team.

Conditions

Rumination Syndrome

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

Baclofen

Baclofen 0.5 mg/kg/day up to 15 mg/day divided three times a day in liquid formulation for 4 weeks.

Group Type: ACTIVE_COMPARATOR

Baclofen

Intervention Type: DRUG

0.5 mg/kg/day up to 15 mg/day divided three times a day for 4 weeks

Placebo

Matching placebo three times a day in liquid formulation for 4 weeks.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Placebo three times a day for 4 weeks

Interventions

Baclofen

0.5 mg/kg/day up to 15 mg/day divided three times a day for 4 weeks

Intervention Type: DRUG

Placebo

Placebo three times a day for 4 weeks

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Children 4-18 years of age who meets Rome IV diagnostic criteria for rumination syndrome and are see at NCH GI clinic

Exclusion Criteria

• Patients who have contraindications to baclofen due to medical history and/or current medications
• Patients who are non- English speaking
• Patients who are pregnant

• Minimum Eligible Age: 4 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Nationwide Children's Hospital

OTHER

Sponsor Role: lead

Responsible Party

Peter Lu

Associate Professor of Clinical Pediatrics

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Peter Lu, MD, MS

Role: CONTACT

peter.lu@nationwidechildrens.org

6147223450

Janice Khoo, MD

Role: CONTACT

janice.khoo@nationwidechildrens.org

6147223450

Facility Contacts

Janice Khoo, MD

Role: primary

614-722-3450

Other Identifiers

STUDY00002491

Identifier Type: -

Identifier Source: org_study_id