Clinical trial NCT03055767: Effectiveness of Onabotulinumtoxin A (Botox) in Pediatric Patients Experiencing Migraines: A Study in the Pediatric Pain Population
Official title: Effectiveness of Onabotulinumtoxin A (Botox) in Pediatric Patients Experiencing Migraines: A Randomized Double Blinded Placebo Cross-over Study in the Pediatric Pain Population
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Migraine Disorders
Headache, Migraine
Pediatrics
Lead sponsor: University of California, Irvine
Sponsor class: OTHER
Collaborators: name: American Society of Regional Anesthesia
First posted: 2017-02-16
Enrollment: 17
Sex: ALL
Minimum age (years): 8
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* Children aged 8 - 17 years of age with a history of migraine meeting the criteria established in ICHD-II (2004), Section 1. 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.

Exclusion Criteria:

* 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

Primary outcomes:
measure: Frequency of Migraines
timeFrame: Baseline (4 weeks) and 12 weeks post each, respective intervention
measure: Intensity of Migraines
timeFrame: Baseline (4 weeks) and 12 weeks post each, respective intervention
measure: Migraine Duration, in Hours
timeFrame: Baseline (4 weeks) and 12 weeks post each, respective intervention
measure: Pediatric Migraine Disability Score (PedMIDAS)
timeFrame: Baseline (4 weeks) and 12 weeks post each, respective intervention
Secondary outcomes:
measure: Functionality Improvement
timeFrame: Baseline (4 weeks) and 12 weeks post each, respective intervention
measure: Difficulty Sleeping
timeFrame: Baseline (4 weeks) and 12 weeks post each, respective intervention
measure: Hospital Admissions
timeFrame: Baseline (4 weeks) and 12 weeks post each, respective intervention
measure: Emergency Department (ED) Visits
timeFrame: Baseline (4 weeks) and 12 weeks post each, respective intervention
measure: Home School
timeFrame: Baseline (4 weeks) and 12 weeks post, respective intervention
measure: School Plan Enrollment
timeFrame: Baseline (4 weeks) and 12 weeks post each, respective intervention
measure: Duration of Benefit
timeFrame: Baseline (4 weeks) and 12 weeks post each, respective intervention
measure: Concomitant Headache Medications
timeFrame: Baseline (4 weeks) and 12 weeks post each, respective intervention
Other outcomes:
measure: Frequency of Migraines | Open-label Period
timeFrame: 24-48 weeks post-baseline period
measure: Intensity of Migraines | Open-Label Period
timeFrame: 24-48 weeks post-baseline period
measure: Pediatric Migraine Disability Score (PedMIDAS) | Open-Label Period
timeFrame: 24-48 weeks post-baseline period
measure: Duration of Migraine | Open-Label Period
timeFrame: 24-48 weeks post-baseline period
Locations:
city: Irvine
country: United States
facility: Gottschalk Medical Plaza
state: California
city: Orange
country: United States
facility: UC Irvine Medical Center
state: California
Study officials:
affiliation: Assistant Clinical Professor
name: Shalini S Shah, MD
role: PRINCIPAL_INVESTIGATOR
References:
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citation: Shah S, Calderon MD, Crain N, Pham J, Rinehart J. Effectiveness of onabotulinumtoxinA (BOTOX) in pediatric patients experiencing migraines: a randomized, double-blinded, placebo-controlled crossover study in the pediatric pain population. Reg Anesth Pain Med. 2021 Jan;46(1):41-48. doi: 10.1136/rapm-2020-101605. Epub 2020 Oct 26.