Dysport® Pediatric Lower Limb Spasticity Study

Study Overview
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Brief Summary
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The purpose of this research study is to determine whether Dysport® is effective in the treatment of increased stiffness of the calf muscles and to evaluate the safety of this treatment in children with Cerebral Palsy. In addition this study will also check whether Dysport® can lessen the pain caused by spasticity and improve the child's wellbeing.

Official Title
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A Phase III, Multicentre, Double Blind, Prospective, Randomised, Placebo Controlled Study Assessing the Efficacy and Safety of DYSPORT® Used in the Treatment of Lower Limb Spasticity in Children With Dynamic Equinus Foot Deformity Due to Cerebral Palsy

Conditions
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Cerebral Palsy, Muscle Spasticity, Children

Intervention / Treatment
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* Biological: Botulinum toxin type A
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Children aged 2 to 17 years with cerebral palsy Equinus foot position Ambulatory Intensity of muscle tone equal to or greater than 2 in affected lower limb, as measured on the Modified Ashworth Scale Exclusion Criteria: Fixed contracture Previous phenol, alcohol injection or surgical intervention Other neurological / neuromuscular disorder Severe athetoid or dystonic movements

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Dysport 10 U/Kg<br>10 U/Kg per lower limb. Either one or both lower limbs can be treated. Total volume injected, 2ml per leg. | Biological: Botulinum toxin type A<br>* I.M. (in the muscle) injection on day 1 of a single treatment cycle.<br>* Other names: AbobotulinumtoxinA (Dysport®);|
| Experimental: Dysport 15 U/Kg<br>15 U/Kg per lower limb. Either one or both lower limbs can be treated. Total volume injected, 2ml per leg. | Biological: Botulinum toxin type A<br>* I.M. (in the muscle) injection on day 1 of a single treatment cycle.<br>* Other names: AbobotulinumtoxinA (Dysport®);|
| Placebo Comparator: Placebo<br>Total volume to be injected per lower limb - 2ml. Either one or both lower limbs can be treated. | Drug: Placebo<br>* I.M. injection on day 1 of a single treatment cycle.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in MAS Score in the Gastrocnemius-soleus Complex (GSC) at the Ankle Joint of the (Most) Affected Lower Limb | The MAS is a 6-point scale which measures the intensity of muscle tone by measuring the resistance of the muscle to passive lengthening or stretching. Investigator will grade muscle tone in the GSC from 0 (no increase in tone) to 4 (affected parts rigid in flexion or extension). | Change from baseline to Week 4 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Physician's Global Assessment (PGA) of the Treatment Response. | PGA Scale of the Treatment Response: Global assessment of treatment response assessed by asking the Investigator the following question: how would you rate the response to treatment in the subject's lower limb(s) since the last injection? Answers will be made on a 9 point rating scale (-4: markedly worse, -3: much worse, -2: worse, -1: slightly worse, 0: no change, +1: slightly improved, +2: improved, +3: much improved, +4: markedly improved). | Week 4 | 
| Goal Attainment Scale (GAS) Score | GAS is a functional scale used to measure progress towards individual therapy goals. Individual goals defined for each subject by the physician, and the child's parents (caregiver) where applicable, prior to treatment. Post-baseline, the GAS for each goal rated using a defined scale (-2: Much less than expected outcome, -1: somewhat less than expected outcome, 0: expected outcome, 1: somewhat more than expected outcome, and 2: Much more than expected outcome). | Week 4 |