Intra-rectal Botulinum Toxin Injection for Intractable Non-retentive Fecal Incontinence in Children - an Open Label Pilot Study
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 14
- 试验地点
- 1
- 主要终点
- median number of FI episodes/week at 1-month post-intervention compared to baseline
研究概览
简要总结
Background: Fecal Incontinence (FI) is a frustrating and prevalent GI condition with profound social implications and a marked effect on quality of life. Treatment options are limited for children whose FI is not secondary to constipation (overflow incontinence), and they are defined as having non-retentive fecal incontinence (NRFI). Rectal botulinum injections (RBI) have recently shown promise for the treatment of FI in adults, following a large, randomized placebo-controlled trial, but no data exists regarding efficacy in children.
Objectives: To evaluate the efficacy and safety of RBI in children with non-retentive fecal incontinence.
Methods: A prospective open-label pilot study. Children with intractable NRFI will be screened using anorectal manometry and a colonic transit study. Eligible patients will receive one course of RBI and data regarding FI frequency will be prospectively collected during a 15-week period.
Significance: New treatment options for children with intractable fecal incontinence are highly in need. The current study aims to introduce a new treatment modality into pediatric research and patient care.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 4 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children 4-18 years old with fecal incontinence for a period greater than 6 months.
- •FI frequency of ≥ 3 episodes/week.
- •After appropriate medical evaluation, FI cannot be explained by another medical condition.
- •Normal colonic transit study, defined as passage of 80% of markers on day
- •Normal RAIR on anorectal manometry
排除标准
- •Patients currently fulfilling rome IV criteria for functional constipation.
- •Patients with evidence of fecal retention.
- •Patients who had had good response to treatment for overflow incontinence.
- •Absent RAIR on anorectal manometry.
- •Any radiologic evidence of dochylosigmoid or distended colon.
- •Any known organic condition that may affect bowel transit.
研究组 & 干预措施
Botulinum toxin
干预措施: Botulinum Toxin A (Botox ) (Drug)
结局指标
主要结局
median number of FI episodes/week at 1-month post-intervention compared to baseline
时间窗: 3-months post-intervention
median number of FI episodes/week at 1-month post-intervention compared to baseline. The median FI episodes/week will be calculated based on the 21-day diary. FI episode minimal definition: any episode of soiling requiring change of underwear/clothes/washing-up.
次要结局
- Median number of FI episodes/week at 3-months post-intervention, compared to baseline diary.(3-months post-intervention)
- Rate of patients with a 50% decrease in mean FI episodes/week compared to baseline diary.(3-months post-intervention)
- Rate of patients with daily FI episodes (at least 1 episode/day) compared to baseline.(3-months post-intervention)
- Mean number of complete bowel movements/week compared to baseline according to diary.(3-months post-intervention)
- Parents and patient's general impression of the treatment using the following questions: "Was the treatment useful/beneficial?"; "Did the treatment improve your quality of life"?(3-months post-intervention)
- Rate of RBI-associated major and minor adverse events. Patients will be questioned according to list, as well as allowed to add adverse events not listed.(3-months post-intervention)
研究者
Yogev Dotan
Dr Dotan Yogev
Shaare Zedek Medical Center