Brief Summary
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).
Brief Title
Effectiveness and Safety of Dysport® (abobotulinumtoxinA) in Paediatric Lower Limb Spasticity
Categories
Completion Date
Completion Date Type
Actual
Conditions
Lower Limb Spasticity
Eligibility Criteria
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
* 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
Inclusion Criteria
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
* 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
Gender
All
Gender Based
false
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Maximum Age
17 Years
Minimum Age
2 Years
NCT Id
NCT03017729
Org Class
Industry
Org Full Name
Ipsen
Org Study Id
A-US-52120-330
Overall Status
Completed
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
A Phase IV, Prospective, Observational, Multicentre Study Evaluating the Effectiveness and Safety of Dysport® (abobotulinumtoxinA) in Paediatric Lower Limb Spasticity.
Primary Outcomes
Outcome Description
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.
Outcome Measure
Cumulated GAS T score
Outcome Time Frame
From day 1 up to 30 months
Secondary Outcomes
Outcome Description
During each injection cycle and overall
Outcome Time Frame
Day 1, then every 3.5 months (approximately) up to 30 months
Outcome Measure
AbobotulinumtoxinA dose
Outcome Description
During each injection cycle and overall
Outcome Time Frame
Day 1, then every 3.5 months (approximately) up to 30 months
Outcome Measure
Time intervals between injections
Outcome Description
During each injection cycle and overall
Outcome Time Frame
Day 1, then every 3.5 months (approximately) up to 30 months
Outcome Measure
Number of injection points
Outcome Description
During each injection cycle and overall
Outcome Time Frame
Day 1, then every 3.5 months (approximately) up to 30 months
Outcome Measure
Muscle(s) injected
Outcome Description
During each injection cycle and overall
Outcome Time Frame
Day 1, then every 3.5 months (approximately) up to 30 months
Outcome Measure
Sedation used
Outcome Description
During each injection cycle and overall
Outcome Time Frame
Day 1, then every 3.5 months (approximately) up to 30 months
Outcome Measure
Type of injection guidance utilised
Outcome Description
Listed and tabulated by frequency
Outcome Time Frame
From day 1 up to 30 months
Outcome Measure
Concomitant drug therapies
Outcome Description
Listed and tabulated by frequency
Outcome Time Frame
From day 1 up to 30 months
Outcome Measure
Non-drug therapies
Outcome Description
This evaluation will not be mandatory during this study. Response over time will be presented using descriptive statistics.
Outcome Time Frame
From day 1 up to 30 months (per investigator's decision/routine practice)
Outcome Measure
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.
Outcome Description
Per injection cycle
Outcome Time Frame
Day 1 then every 3.5 months (approximately) up to 30 months
Outcome Measure
Average GAS T score
Outcome Description
During each injection cycle and overall
Outcome Time Frame
Day 1 then every 3.5 months (approximately) up to 30 months
Outcome Measure
Percentage achievement of primary treatment goal
Outcome Description
Per injection cycle
Outcome Time Frame
Day 1 then every 3.5 months (approximately) up to 30 months
Outcome Measure
Percentage achievement of primary treatment goal(s) per goal area(s) after repeated abobotulinumtoxinA injections
Outcome Description
Adverse events will be coded using the Medical Dictionary for Regulatory Activities (MedDRA).
Outcome Time Frame
From day 1 up to 30 months
Outcome Measure
Incidence of adverse events and special situations collected
Outcome Description
Derived from the collected data, including concomitant treatments.
Outcome Time Frame
From day 1 up to 30 months
Outcome Measure
Direct and indirect health care costs
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Estimated
First Submit Date
First Submit QC Date
Study Population
Pediatric patients with lower limb spasticity
Std Ages
Child
Maximum Age Number (converted to Years and rounded down)
17
Minimum Age Number (converted to Years and rounded down)
2
Investigators
Investigator Type
Principal Investigator
Investigator Name
Yuxi Chen
Investigator Email
yuchen@montefiore.org
Investigator Phone