Back to resultsEffect of Dysport Injections on Energy Expenditure and Walking Efficiency in Children With Cerebral Palsy
Primary Purpose
Cerebral Palsy
Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Dysport Injectable Product
Sponsored by
About this trial
This is an interventional treatment trial for Cerebral Palsy focused on measuring Botulinum toxin A, Walking efficiency, Spasticity, Dysport, Pediatric
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)
Sites / Locations
- Columbia University Irving Medical Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Dysport Injectable Product
Arm Description
All participants will participate in baseline data collection of energy expenditure, gait analysis, and lower limb spasticity assessment. All participants will receive single event multi level chemoneurolysis with Dysport and will have repeat data collection at 4 weeks and 12 weeks post injection.
Outcomes
Primary Outcome Measures
Oxygen Consumption (ml/kg/Min)
Energy expenditure (oxygen consumption (VO2)) will be measured during the 6 minute walk test using a portable metabolic cart. The participants will use a 10 meter walkway to walk back and forth during the 6 minutes to coverage as much distance as possible. During this time they will be wearing a portable spirometric device that collects the oxygen consumption (VO2) per minute.
Secondary Outcome Measures
Gait Velocity (cm/s)
Gait analysis will be measured by the GaitMat system while the child walks on the mat back and forth for five trials.
Muscle Spasticity Using MAS
The Modified Ashworth Scale (MAS) measures muscle spasticity during passive stretching. Scores are on a 6-point scale and include 0, 1, 1+, 2, 3, and 4. Scores range from 0 (better outcome), which indicates no increase in muscle tone/muscle spasticity to a score of 4 (worse outcome), which indicates a rigid muscle or maximum spasticity. Each subscale is independently scored and not combined to create a composite score. When calculating the mean score, 1+ is equivalent to a value of 1.5.
Muscle Spasticity Using MTS
The Modified Tardieu Scale (MTS) measures muscle spasticity during a passive stretch at both slow and fast speeds. Scores range from 0, which indicates no resistance throughout the course of the passive movement (better outcome), to 5, which indicates that the joint is immovable (worse outcome). The first measure determines the maximum range of motion of a target muscle group in degrees and the second measure determines the angle where muscle resistance is felt during a rapid velocity stretch, recorded in degrees. The total score is calculated by subtracting measure one from measure two. A greater difference between measures indicates less muscle spasticity. The Modified Tardieu Scale will be performed in the hamstrings, gastrocnemius, and soleus muscles.
Score on the Cerebral Palsy Quality of Life (CP QOL) Questionnaire
The Cerebral Palsy Quality of Life (CP QOL) Questionnaire consists of 66 items ranging from 1 to 9 with a lower score indicating a higher quality of life. The total score will be calculated as well as an individual score for the seven subscale domains: Social well-being and acceptance, functioning, Participation and physical health, Emotional well-being, Access to services, pain and feeling about disability, and family health. Scoring involves 2 steps. First, items are transformed to a scale with a possible range of 0-100. The scores are recoded as follows 1 to 0, 2 to 12.5, 3 to 25, 4 to 37.5, 5 to 50, 6 to 6.2, 7 to 75, 8 to 87.5, 9 to 100. Then the algebraic mean of item values is computed from the composite score for each domain. Once rescored, the final score ranges from 0 to 100, with 0 indicating a worse outcome.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03469999
Brief Title
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
2. Study Status
Record Verification Date
May 2022
Overall Recruitment Status
Completed
Study Start Date
December 12, 2018 (Actual)
Primary Completion Date
May 26, 2021 (Actual)
Study Completion Date
May 26, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Columbia University
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cerebral Palsy
Keywords
Botulinum toxin A, Walking efficiency, Spasticity, Dysport, Pediatric
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Dysport Injectable Product
Arm Type
Experimental
Arm Description
All participants will participate in baseline data collection of energy expenditure, gait analysis, and lower limb spasticity assessment. All participants will receive single event multi level chemoneurolysis with Dysport and will have repeat data collection at 4 weeks and 12 weeks post injection.
Intervention Type
Drug
Intervention Name(s)
Dysport Injectable Product
Other Intervention Name(s)
Botulinum toxin A
Intervention Description
Selected dose of medication will be determined by affected muscle(s), severity of spasticity, and the patient's body weight. We will follow the recommended total Dysport dose of 10-15 units/kg per limb, not to exceed 15 units/kg for unilateral lower limb, 30 units/kg for bilateral lower limb, or a total of 1000 units, whichever is lower in a given session.
Primary Outcome Measure Information:
Title
Oxygen Consumption (ml/kg/Min)
Description
Energy expenditure (oxygen consumption (VO2)) will be measured during the 6 minute walk test using a portable metabolic cart. The participants will use a 10 meter walkway to walk back and forth during the 6 minutes to coverage as much distance as possible. During this time they will be wearing a portable spirometric device that collects the oxygen consumption (VO2) per minute.
Time Frame
Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
Secondary Outcome Measure Information:
Title
Gait Velocity (cm/s)
Description
Gait analysis will be measured by the GaitMat system while the child walks on the mat back and forth for five trials.
Time Frame
Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
Title
Muscle Spasticity Using MAS
Description
The Modified Ashworth Scale (MAS) measures muscle spasticity during passive stretching. Scores are on a 6-point scale and include 0, 1, 1+, 2, 3, and 4. Scores range from 0 (better outcome), which indicates no increase in muscle tone/muscle spasticity to a score of 4 (worse outcome), which indicates a rigid muscle or maximum spasticity. Each subscale is independently scored and not combined to create a composite score. When calculating the mean score, 1+ is equivalent to a value of 1.5.
Time Frame
Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
Title
Muscle Spasticity Using MTS
Description
The Modified Tardieu Scale (MTS) measures muscle spasticity during a passive stretch at both slow and fast speeds. Scores range from 0, which indicates no resistance throughout the course of the passive movement (better outcome), to 5, which indicates that the joint is immovable (worse outcome). The first measure determines the maximum range of motion of a target muscle group in degrees and the second measure determines the angle where muscle resistance is felt during a rapid velocity stretch, recorded in degrees. The total score is calculated by subtracting measure one from measure two. A greater difference between measures indicates less muscle spasticity. The Modified Tardieu Scale will be performed in the hamstrings, gastrocnemius, and soleus muscles.
Time Frame
Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
Title
Score on the Cerebral Palsy Quality of Life (CP QOL) Questionnaire
Description
The Cerebral Palsy Quality of Life (CP QOL) Questionnaire consists of 66 items ranging from 1 to 9 with a lower score indicating a higher quality of life. The total score will be calculated as well as an individual score for the seven subscale domains: Social well-being and acceptance, functioning, Participation and physical health, Emotional well-being, Access to services, pain and feeling about disability, and family health. Scoring involves 2 steps. First, items are transformed to a scale with a possible range of 0-100. The scores are recoded as follows 1 to 0, 2 to 12.5, 3 to 25, 4 to 37.5, 5 to 50, 6 to 6.2, 7 to 75, 8 to 87.5, 9 to 100. Then the algebraic mean of item values is computed from the composite score for each domain. Once rescored, the final score ranges from 0 to 100, with 0 indicating a worse outcome.
Time Frame
Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
10. Eligibility
Sex
All
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Heakyung Kim, MD
Organizational Affiliation
Columbia University Medical Center- Department of Rehabilitation
Official's Role
Principal Investigator
Facility Information:
Facility Name
Columbia University Irving Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Effect of Dysport Injections on Energy Expenditure and Walking Efficiency in Children With Cerebral Palsy
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