Effectiveness of Onabotulinumtoxin A (Botox) in Pediatric Patients Experiencing Migraines: A Randomized Double Blinded Placebo Cross-over Study in the Pediatric Pain Population
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 17
- 试验地点
- 2
- 主要终点
- Migraine Duration, in Hours
研究概览
简要总结
The purpose of the research is to examine the outcomes of pediatric patients receiving Botulinum toxin type A (Botox ®) for the treatment of migraine. There is limited literature on the effectiveness of Botox ® in the treatment of chronic neurological pain in pediatric patients, specifically in the treatment of migraines.
详细描述
Medical Literature approximates one in three children will experience chronic headaches in their lifetime, which increases as children reach adolescence. Migraines make up nearly 60% of all visits to a pediatric headache specialist. Studies have demonstrated the negative impact of having childhood migraine on overall quality of life is similar to pediatric cancer, heart disease and rheumatic disease. As the frequency of migraine attacks increase, so does proportionally the child's disability in lost school time and family and social interactions, all of which may lead in turn to economic disability. Studies estimate the health care costs are 70% higher for a family with a migraine than a non-migraine affected family, and direct medical costs for children with migraine are reported to be similar to those for adults. A study published in JAMA 2003 found that health care costs, work-related disability for parents and lost educational opportunity for the child leads to an annual economic impact in the US of approximately $36 billion due to both direct medical costs and lost productivity into adulthood.
Onaboutlinum (BOTOX) is currently FDA approved as a very successful treatment to prevent migraines in adults, however not yet children. Current treatments for migraine in children appear to be insufficient. No trials currently exist in literature prospectively studying onabotulinumtoxinA for efficacy and/or safety for indication of pediatric migraine, although significant contributions have been made by retrospective case series over the last 10 years.
This research will be the first investigator-initiated study to study BOTOX (R) in children prospectively in a randomized controlled placebo, cross-over study. The overriding rationale is to demonstrate efficacy, tolerability and safety of onabotulinumtoxinA for pediatric migraine and thereby potentially hasten the lengthy process to evaluate BOTOX® for approval in the pediatric population.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 8 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children aged 8 - 17 years of age with a history of migraine meeting the criteria established in ICHD-II (2004), Section
- •Patients will provide at least 28-day baseline data in the form in the daily diary and must have at least 15 days of reported headache during this period, with at least 4 distinct episodes lasting at least 4 hours each.
排除标准
- •Previous use of botulinum toxin of any serotype for any reason
- •Pregnancy.
- •Diagnosis of Myasthenia gravis, Eaton Lambert Syndrome, Amyotrophic Lateral Sclerosis
- •Treatment of headache using acupuncture, transcutaneous electrical stimulation (TENS), cranial traction, dental splints, or injection of anesthetics/steroids within 4 weeks prior to the week of screening visit
研究组 & 干预措施
OnabotulinumtoxinA/Saline Placebo
The AB subject group will receive OnabotulinumtoxinA in the first treatment and saline placebo in the second.Both groups will then receive OnabotulinumtoxinA in the last two treatments. There will be one treatment at the beginning of each 12 week block, meaning 4 treatments over the 48 week study period total. Progress check-ups will occur every 6 weeks during the study. Randomization will be via selection of sealed envelope.
干预措施: OnabotulinumtoxinA (Drug)
OnabotulinumtoxinA/Saline Placebo
The AB subject group will receive OnabotulinumtoxinA in the first treatment and saline placebo in the second.Both groups will then receive OnabotulinumtoxinA in the last two treatments. There will be one treatment at the beginning of each 12 week block, meaning 4 treatments over the 48 week study period total. Progress check-ups will occur every 6 weeks during the study. Randomization will be via selection of sealed envelope.
干预措施: Placebo (Saline) (Other)
Saline Placebo/OnabotulinumtoxinA
The BA subject group will receive saline and then Onabotulinumtoxin. A.Both groups will then receive OnabotulinumtoxinA in the last two treatments. There will be one treatment at the beginning of each 12 week block, meaning 4 treatments over the 48 week study period total. Progress check-ups will occur every 6 weeks during the study. Randomization will be via selection of sealed envelope.
干预措施: OnabotulinumtoxinA (Drug)
Saline Placebo/OnabotulinumtoxinA
The BA subject group will receive saline and then Onabotulinumtoxin. A.Both groups will then receive OnabotulinumtoxinA in the last two treatments. There will be one treatment at the beginning of each 12 week block, meaning 4 treatments over the 48 week study period total. Progress check-ups will occur every 6 weeks during the study. Randomization will be via selection of sealed envelope.
干预措施: Placebo (Saline) (Other)
结局指标
主要结局
Migraine Duration, in Hours
时间窗: Baseline (4 weeks) and 12 weeks post each, respective intervention
Duration of migraines in hours.
Pediatric Migraine Disability Score (PedMIDAS)
时间窗: Baseline (4 weeks) and 12 weeks post each, respective intervention
Pediatric Migraine Disability Score consists of 6 questions: 3 addressing school attendance and functioning, and 3 evaluating participation in events outside of school. The questionnaire is based on the patient's recall of the previous 3 months. The questionnaire produces a raw score (0-10, 11-30, 31-50, \>50) corresponding to a disability grade with increasing severity (little to non, mild, moderate, and severe) which was coded (1, 2, 3, and 4).
Frequency of Migraines
时间窗: Baseline (4 weeks) and 12 weeks post each, respective intervention
The frequency of migraines in days.
Intensity of Migraines
时间窗: Baseline (4 weeks) and 12 weeks post each, respective intervention
Median intensity of migraines based on 0-10 Pain Numeric Rating Score (NRS). The higher the score, the more intense the pain.
次要结局
- Hospital Admissions(Baseline (4 weeks) and 12 weeks post each, respective intervention)
- Duration of Benefit(Baseline (4 weeks) and 12 weeks post each, respective intervention)
- Concomitant Headache Medications(Baseline (4 weeks) and 12 weeks post each, respective intervention)
- Difficulty Sleeping(Baseline (4 weeks) and 12 weeks post each, respective intervention)
- School Plan Enrollment(Baseline (4 weeks) and 12 weeks post each, respective intervention)
- Functionality Improvement(Baseline (4 weeks) and 12 weeks post each, respective intervention)
- Emergency Department (ED) Visits(Baseline (4 weeks) and 12 weeks post each, respective intervention)
- Home School(Baseline (4 weeks) and 12 weeks post, respective intervention)
研究者
Shalini Suresh Shah
Vice Chair, Pain Management
University of California, Irvine