Baclofen for Children With Rumination Syndrome

A Randomized Controlled Pilot Trial of Baclofen for Children With Rumination Syndrome

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.

Study Overview

• Status: Recruiting
• Conditions: Rumination Syndrome
• Intervention / Treatment: Drug: Baclofen; Drug: Placebo

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.

• Study Type: Interventional
• Enrollment (Estimated): 50
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Peter Lu, MD, MS; Phone Number: 6147223450; Email: peter.lu@nationwidechildrens.org
• Study Contact Backup: Name: Janice Khoo, MD; Phone Number: 6147223450; Email: janice.khoo@nationwidechildrens.org

Study Locations

• United States
  • Ohio
    • Columbus, Ohio, United States, 73205
      • Recruiting
      • Nationwide Children's Hospital
      • Contact: Janice Khoo, MD; Phone Number: 614-722-3450

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: Yes

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Baclofen; Baclofen 0.5 mg/kg/day up to 15 mg/day divided three times a day in liquid formulation for 4 weeks.	Drug: Baclofen; 0.5 mg/kg/day up to 15 mg/day divided three times a day for 4 weeks
Placebo Comparator: Placebo; Matching placebo three times a day in liquid formulation for 4 weeks.	Drug: Placebo; Placebo three times a day for 4 weeks

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Vomiting once a week or less	Percentage of participants in each group who are vomiting once a week or less. This will be evaluated using a rumination severity survey.	The investigators will assess this specific metric at the end of four weeks.

Collaborators and Investigators

• Sponsor: Nationwide Children's Hospital

Study record dates

Study Major Dates

• Study Start (Actual): January 31, 2023
• Primary Completion (Estimated): December 1, 2023
• Study Completion (Estimated): April 1, 2024

Study Registration Dates

• First Submitted: July 27, 2023
• First Submitted That Met QC Criteria: July 27, 2023
• First Posted (Actual): August 4, 2023

Study Record Updates

• Last Update Posted (Actual): September 21, 2023
• Last Update Submitted That Met QC Criteria: September 18, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Digestive System Diseases; Pathologic Processes; Disease; Feeding and Eating Disorders; Gastrointestinal Diseases; Syndrome; Rumination Syndrome; Feeding and Eating Disorders of Childhood; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Peripheral Nervous System Agents; GABA Agents; Neuromuscular Agents; Muscle Relaxants, Central; GABA Agonists; GABA-B Receptor Agonists; Baclofen
• Other Study ID Numbers: STUDY00002491

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• 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.: Yes