Effect of Dysport Injections on Energy Expenditure and Walking Efficiency in Children With Cerebral Palsy

Cerebral Palsy Clinical Trial

Official title:

Effect of Single Event Multi Level Chemoneurolysis With Dysport on Energy Expenditure and Walking Efficiency

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03469999
• Other study ID #: AAAR1322
• Status: Completed
• Phase: Phase 3
• Start date: December 12, 2018
• Est. completion date: May 26, 2021

Study information

• Verified date: May 2022
• Source: Columbia University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 12
• Est. completion date: May 26, 2021
• Est. primary completion date: May 26, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years to 17 Years

Eligibility

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)

Related Conditions & MeSH terms

• Cerebral Palsy

Intervention

Drug:
• Dysport Injectable Product
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.

Locations

Country	Name	City	State
United States	Columbia University Irving Medical Center	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Columbia University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
Secondary	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.	Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
Secondary	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.	Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
Secondary	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.	Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
Secondary	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.	Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection