Lower Limb Spasticity
Conditions
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).
Interventions
BIOLOGICALBotulinum 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.
Sponsors
Ipsen
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
2 Years to 17 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Cumulated GAS T score | From day 1 up to 30 months | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Time intervals between injections | Day 1, then every 3.5 months (approximately) up to 30 months | During each injection cycle and overall |
| Number of injection points | Day 1, then every 3.5 months (approximately) up to 30 months | During each injection cycle and overall |
| Muscle(s) injected | Day 1, then every 3.5 months (approximately) up to 30 months | During each injection cycle and overall |
| Sedation used | Day 1, then every 3.5 months (approximately) up to 30 months | During each injection cycle and overall |
| Type of injection guidance utilised | Day 1, then every 3.5 months (approximately) up to 30 months | During each injection cycle and overall |
| Concomitant drug therapies | From day 1 up to 30 months | Listed and tabulated by frequency |
| AbobotulinumtoxinA dose | Day 1, then every 3.5 months (approximately) up to 30 months | During each injection cycle and overall |
| 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. | From day 1 up to 30 months (per investigator's decision/routine practice) | This evaluation will not be mandatory during this study. Response over time will be presented using descriptive statistics. |
| Average GAS T score | Day 1 then every 3.5 months (approximately) up to 30 months | Per injection cycle |
| Percentage achievement of primary treatment goal | Day 1 then every 3.5 months (approximately) up to 30 months | During each injection cycle and overall |
| Percentage achievement of primary treatment goal(s) per goal area(s) after repeated abobotulinumtoxinA injections | Day 1 then every 3.5 months (approximately) up to 30 months | Per injection cycle |
| Incidence of adverse events and special situations collected | From day 1 up to 30 months | Adverse events will be coded using the Medical Dictionary for Regulatory Activities (MedDRA). |
| Direct and indirect health care costs | From day 1 up to 30 months | Derived from the collected data, including concomitant treatments. |
| Non-drug therapies | From day 1 up to 30 months | Listed and tabulated by frequency |
Countries
United States
Outcome results
None listed