Baclofen for Children With Rumination Syndrome
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 3 |
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. We 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, we 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. We will closely monitor for side effects. In addition to asking about any side effects at follow-up timepoints, we will also emphasize that families should contact us 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 Design
Arms and Interventions
| 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
|
Outcome Measures
Primary Outcome Measures
- Vomiting once a week or less [We will assess this specific metric at the end of four weeks.]
Percentage of patients in each group who are vomiting once a week or less. This will be evaluated using a rumination severity survey.
Eligibility Criteria
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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Nationwide Children's Hospital | Columbus | Ohio | United States | 73205 |
Sponsors and Collaborators
- Nationwide Children's Hospital
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- STUDY00002491