Cerebral PalsyPhase 3NCT03469999
Effect of Dysport Injections on Energy Expenditure and Walking Efficiency in Children With Cerebral Palsy
A Phase 3 study testing Dysport Injectable Product in people with Cerebral Palsy, run by Columbia University at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 5 to 17 years
- Conditions the study is for
- Cerebral Palsy
- What is being tested?
- Dysport Injectable Product
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 12 Dec 2018to 26 May 2021
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
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- 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
Who cannot join
- 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
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- 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)
Trial locations and contacts
1 site in 1 country.
United States1 site
Columbia University Irving Medical Center
New York, New York, 10032
About this study
Official title: Effect of Single Event Multi Level Chemoneurolysis With Dysport on Energy Expenditure and Walking Efficiency
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.
Read the 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.
- Conditions
- Cerebral Palsy
- Being tested
- Dysport Injectable Product
- Keywords
- Botulinum toxin A, Walking efficiency, Spasticity, Dysport, Pediatric
- Medical subject headings
- Cerebral Palsy, Muscle Spasticity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dysport Injectable ProductDrug
Also called Botulinum toxin A
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.
| Study group | Role | What they receive |
|---|---|---|
| Dysport Injectable Product | Experimental | 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. Receives: Drug: Dysport Injectable Product |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
Secondary outcomes (4)
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.
Measured: Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
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.
Measured: Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
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.
Measured: Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
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.
Measured: Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Mar 2018
- Study start12 Dec 2018
- Primary completion26 May 2021
- Study completion26 May 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cerebral PalsyStudies of the same condition, open and under way.
- More trials run by Columbia UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Columbia University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Heakyung Kim, MD, Principal investigator, Columbia University Medical Center- Department of Rehabilitation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03469999
- Sponsor's protocol ID
- AAAR1322
- Status last verified
- May 2022
- First submitted
- 24 Jan 2018
- Last updated
- 1 Jun 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Single Event Multi Level Chemoneurolysis With Dysport on Energy Expenditure and Walking Efficiency
- ClinicalTrials.gov ID
- NCT03469999
- Acronym / sponsor ID
- AAAR1322
- Conditions
- Cerebral Palsy
- Keywords
- Botulinum toxin A, Walking efficiency, Spasticity, Dysport, Pediatric
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Columbia UniversityUniversity, hospital or other
- Study start
- 12 Dec 2018 (actual)
- Primary completion
- 26 May 2021 (actual)
- Study completion
- 26 May 2021 (actual)
- First posted
- 19 Mar 2018 (actual)
- Results
- Posted 1 Jun 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dysport Injectable Product | Experimental | Drug: Dysport Injectable ProductAll 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. |
Who may qualify
- Ages
- 5 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (11 listed)
- 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
Key exclusion criteria (13 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Dysport Injectable ProductAlso: Botulinum toxin ASelected 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. | Drug | Dysport Injectable Product |
Primary outcome
Oxygen Consumption (ml/kg/Min)
Time frame: Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
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.
4 secondary outcomes
Gait Velocity (cm/s)
Time frame: Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
Muscle Spasticity Using MAS
Time frame: Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
Muscle Spasticity Using MTS
Time frame: Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
Score on the Cerebral Palsy Quality of Life (CP QOL) Questionnaire
Time frame: Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jun 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Heakyung Kim, MD principal investigator
Columbia University Medical Center- Department of Rehabilitation
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Dysport Injectable Product in people with Cerebral Palsy, run by Columbia University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Jun 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 1 Jun 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Oxygen Consumption (ml/kg/Min) (time frame: Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Jun 2022.