Effectiveness and Safety of Dysport® (abobotulinumtoxinA) in Paediatric Lower Limb Spasticity

Study Overview
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Brief Summary
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The purpose of this study is to assess the longitudinal attainment of subject centred and functional related goals (cumulated Goal Attainment Scale Total (GAS T) score) after abobotulinumtoxinA injection (including following repeated injection cycles where they occur) alongside spasticity management used in real life settings over a period of 18 months (and a maximum of six injection cycles).

Official Title
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A Phase IV, Prospective, Observational, Multicentre Study Evaluating the Effectiveness and Safety of Dysport® (abobotulinumtoxinA) in Paediatric Lower Limb Spasticity.

Conditions
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Lower Limb Spasticity

Intervention / Treatment
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* Biological: Botulinum toxin type A


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Female or male subjects aged 2 to 17 years inclusive Decision to prescribe abobotulinumtoxinA, to be made prior to and independently from the decision to enroll in the study Primary diagnosis of paediatric lower limb (PLL) spasticity and either: Previously untreated with BoNT (naïve to BoNT), or previously treated with a BoNT (i.e. non naïve to BoNT), and for those who were previously treated with BoNT-A, they should have responded to BoNT-A treatment according to the investigator's criteria For non naïve BoNT subjects, a minimum interval of 12 weeks since the last BoNT injection and in the presence of spasticity Exclusion Criteria: Known resistance to any BoNT or experienced serious safety issues with previous use of BoNT Concomitant treatment with other BoNT Known hypersensitivity to abobotulinumtoxinA or related compounds, or any component in the study drug formulation Subjects with any clinical (or subclinical) evidence of marked defective neuromuscular transmission (e.g. Lambert Eaton syndrome or myasthenia gravis) or persistent clinically significant neuromuscular disorders

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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Arms and Interventions

| Intervention/Treatment |
| --- |
|Biological: Botulinum toxin type A|This is an observational study designed to reflect real world clinical practice in the use of abobotulinumtoxinA. AbobotulinumtoxinA injected doses, frequency of injections, number of injection sites and volume injected per site are in accordance with the current USPI and physician's clinical practice.|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Cumulated GAS T score | Defined as the mean of the individual GAS T scores across all cycles will be used to measure progress towards individual therapy goals. If all goals are achieved as expected, the GAS T score is 50.0. | From day 1 up to 30 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| AbobotulinumtoxinA dose | During each injection cycle and overall | Day 1, then every 3.5 months (approximately) up to 30 months | 
| Time intervals between injections | During each injection cycle and overall | Day 1, then every 3.5 months (approximately) up to 30 months | 
| Number of injection points | During each injection cycle and overall | Day 1, then every 3.5 months (approximately) up to 30 months | 
| Muscle(s) injected | During each injection cycle and overall | Day 1, then every 3.5 months (approximately) up to 30 months | 
| Sedation used | During each injection cycle and overall | Day 1, then every 3.5 months (approximately) up to 30 months | 
| Type of injection guidance utilised | During each injection cycle and overall | Day 1, then every 3.5 months (approximately) up to 30 months | 
| Concomitant drug therapies | Listed and tabulated by frequency | From day 1 up to 30 months | 
| Non-drug therapies | Listed and tabulated by frequency | From day 1 up to 30 months | 
| Modified Ashworth scale (as applicable) in the injected muscle groups (gastrocnemius, soleus and others) at baseline and per the investigators' decision/routine practice during the course of the study. | This evaluation will not be mandatory during this study. Response over time will be presented using descriptive statistics. | From day 1 up to 30 months (per investigator's decision/routine practice) | 
| Average GAS T score | Per injection cycle | Day 1 then every 3.5 months (approximately) up to 30 months | 
| Percentage achievement of primary treatment goal | During each injection cycle and overall | Day 1 then every 3.5 months (approximately) up to 30 months | 
| Percentage achievement of primary treatment goal(s) per goal area(s) after repeated abobotulinumtoxinA injections | Per injection cycle | Day 1 then every 3.5 months (approximately) up to 30 months | 
| Incidence of adverse events and special situations collected | Adverse events will be coded using the Medical Dictionary for Regulatory Activities (MedDRA). | From day 1 up to 30 months | 
| Direct and indirect health care costs | Derived from the collected data, including concomitant treatments. | From day 1 up to 30 months |