Clinical trial NCT03469999: Effect of Dysport Injections on Energy Expenditure and Walking Efficiency in Children With Cerebral Palsy
Official title: Effect of Single Event Multi Level Chemoneurolysis With Dysport on Energy Expenditure and Walking Efficiency
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Cerebral Palsy
Keywords: Botulinum toxin A
Walking efficiency
Spasticity
Dysport
Pediatric
Lead sponsor: Columbia University
Sponsor class: OTHER
First posted: 2018-03-19
Enrollment: 12
Sex: ALL
Minimum age (years): 5
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study aims to evaluate the efficacy of single event multi level chemoneurolysis with the abobotulinumtoxin A drug, Dysport®, on walking energy expenditure and gait in children with spastic diplegia cerebral palsy.

Detailed description: Cerebral Palsy (CP) is the leading cause of disability in children, with the most widespread type of CP being spastic CP which negatively affects physical function. Specifically it is reported that there is an increase in energy expenditure and oxygen consumption in children with CP. Single event multi level chemoneurolysis with abobotulinumtoxinA has been found to be an effective treatment for patients with spasticity to reduce energy expenditure by increasing walking efficiency; however many of these studies have conflicting methodological approaches. Therefore this study aims to evaluate the single event multilevel chemoneurolysis with Dysport® on energy expenditure and gait in children with spastic diplegia CP.

Eligibility criteria: Inclusion Criteria:

* Age: 5-17 years. Must be \<18 prior to injection.
* \>10 kilograms at screening and injection visits
* Diagnosis of spastic diplegia or mild- to moderate spastic quadriplegia Cerebral Palsy
* Gross Motor Function Classification System level: I, II, III
* Ability to ambulate independently without aid, equinus gait
* Absent of joint or bone deformities
* Eligible to receive single-event multi-level chemoneurolysis (SEMLC)
* Cooperative and tolerant to testing procedures during clinic screening
* Presence of spasticity in one or both legs
* Be on a stable dose and regimen if on any prescribed medication/s
* Parent must have signed written informed consent and the Patient Authorization for Use and Release of Health and Research Study Information

Exclusion Criteria:

* Ankle contractures no more than -10 degrees with the knee extended
* Hemiplegia
* Wheelchair dependent
* Received Botulinum toxin within previous 4 months
* Uncontrolled epilepsy or certain types of seizures
* Fracture in the study limb within previous 12 months
* Infection or skin disorder at planned injection site
* Shortness of breath or other respiratory issues
* Uncontrolled clinically significant medical condition
* Received phenol or alcohol block in the study limb within previous 6 months
* Surgery in the study limb within previous 12 months
* Serial casting within previous 12 months
* New physiotherapy and/or orthotic regimen \<1 month before study start. (physiotherapy and/or orthotic regimen will be permitted if it began \>1 month before study start and maintained throughout study)

Primary outcomes:
measure: Oxygen Consumption (ml/kg/Min)
timeFrame: Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
Secondary outcomes:
measure: Gait Velocity (cm/s)
timeFrame: Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
measure: Muscle Spasticity Using MAS
timeFrame: Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
measure: Muscle Spasticity Using MTS
timeFrame: Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
measure: Score on the Cerebral Palsy Quality of Life (CP QOL) Questionnaire
timeFrame: Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
Other outcomes:
Locations:
city: New York
country: United States
facility: Columbia University Irving Medical Center
state: New York
Study officials:
affiliation: Columbia University Medical Center- Department of Rehabilitation
name: Heakyung Kim, MD
role: PRINCIPAL_INVESTIGATOR
References: