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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Dysport Injectable Product | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Muscle Spasticity Using MAS |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Heakyung Kim, MD | Columbia University Medical Center- Department of Rehabilitation | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Columbia University Irving Medical Center | New York | New York | 10032 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | Dysport Injectable Product | 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. 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
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| ID | Title | Description |
|---|---|---|
| BG000 | Dysport Injectable Product | 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. 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | All participants who received Dysport and had VO2 level measured at baseline, week 4 and week 12. | Posted | Mean | 95% Confidence Interval | ml/kg/min | Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection |
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Each participant was followed for up to 12 weeks post-injection for occurrence of any adverse events, serious adverse events and/or death. Adverse event data was collected over a period of approximately 1 year and 1 month, from December 12, 2018 to January 29, 2020.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Dysport Injectable Product | 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. Dysport Injectable Product: All participants will receive Dysport injection at multi levels in the spastic lower limb(s). |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Heakyung Kim | Columbia University Irving Medical Center | 212-305-9416 | hk2641@cumc.columbia.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Oct 8, 2021 | Mar 22, 2022 | Prot_SAP_000.pdf |
| ICF | No | No | Yes | Informed Consent Form | Oct 2, 2019 | Mar 22, 2022 | ICF_001.pdf |
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| ID | Term |
|---|---|
| D002547 | Cerebral Palsy |
| D009128 | Muscle Spasticity |
| ID | Term |
|---|---|
| D001925 | Brain Damage, Chronic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
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| ID | Term |
|---|---|
| D019274 | Botulinum Toxins, Type A |
| ID | Term |
|---|---|
| D001905 | Botulinum Toxins |
| D008666 | Metalloendopeptidases |
| D010450 | Endopeptidases |
| D010447 | Peptide Hydrolases |
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|
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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 |
| 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 |
| 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 |
| years |
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| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
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| Gross Motor Function Classification System (GMFCS) Diagnosis | The Gross Motor Function Classification System (GMFCS) looks at movements such as sitting, walking and use of mobility devices. Grading is based on physical examination and physician's asssessents of a patient's clinical presentation. Grading ranges from GMFCS Level I, which includes children who walk without limitations but with some degree of difficulty with speed, balance, and coordination, (better outcome), to GMFCS Level V, which includes children who are transported in a manual wheelchair and have difficulty in head/trunk control (worse outcome). | Count of Participants | Participants |
|
|
|
| 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. | Only includes participants who received Dysport and had gait analyzed at baseline, week 4 and week 12. | Posted | Mean | 95% Confidence Interval | cm/sec | 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. | Only includes participants who received Dysport and completed the MAS at baseline, week 4 and week 12. | Posted | Mean | 95% Confidence Interval | score on a scale | 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. | Data was not collected for any of the participants. | Posted | 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. | Data was not collected for any of the participants. | Posted | Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection |
|
|
| 0 |
| 12 |
| 0 |
| 12 |
| 0 |
| 12 |
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| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009122 | Muscle Hypertonia |
| D020879 | Neuromuscular Manifestations |
| D009461 | Neurologic Manifestations |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D006867 |
| Hydrolases |
| D004798 | Enzymes |
| D045762 | Enzymes and Coenzymes |
| D045726 | Metalloproteases |
| D001426 | Bacterial Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D001427 | Bacterial Toxins |
| D014118 | Toxins, Biological |
| D001685 | Biological Factors |
| Title | Measurements |
|---|---|
|
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| MAS L Hip Adduction Baseline |
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| MAS L Hip Adduction 4 week |
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| MAS L Hip Adduction 12 week |
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| MAS R Knee Flexion Baseline |
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| MAS R knee Flexion 4 week |
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| MAS R Knee Flexion 12 week |
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| MAS L Knee Flexion Baseline |
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| MAS L Knee Flexion 4 week |
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| MAS L Knee Flexion 12 week |
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| MAS R Dorsiflexion Baseline |
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| MAS R Dorsiflexion 4 week |
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| MAS R Dorsiflexion 12 week |
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| MAS L Dorsiflexion Baseline |
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| MAS L Dorsiflexion 4 week |
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| MAS L dorsiflexion 12 week |
|