A Randomized Controlled Pilot Trial of Baclofen for Children With Rumination Syndrome
概览
- 阶段
- 3 期
- 干预措施
- Baclofen
- 疾病 / 适应症
- Rumination Syndrome
- 发起方
- Nationwide Children's Hospital
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Vomiting once a week or less
- 状态
- 已完成
- 最后更新
- 上个月
概览
简要总结
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.
详细描述
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.
研究者
Peter Lu
Associate Professor of Clinical Pediatrics
Nationwide Children's Hospital
入排标准
入选标准
- •Children 4-18 years of age who meets Rome IV diagnostic criteria for rumination syndrome and are see at NCH GI clinic
排除标准
- •Patients who have contraindications to baclofen due to medical history and/or current medications
- •Patients who are non- English speaking
- •Patients who are pregnant
研究组 & 干预措施
Baclofen
Baclofen 0.5 mg/kg/day up to 15 mg/day divided three times a day in liquid formulation for 4 weeks.
干预措施: Baclofen
Placebo
Matching placebo three times a day in liquid formulation for 4 weeks.
干预措施: Placebo
结局指标
主要结局
Vomiting once a week or less
时间窗: The investigators will assess this specific metric at the end of four weeks.
Percentage of participants in each group who are vomiting once a week or less. This will be evaluated using a rumination severity survey.