Effectiveness and Safety of Dysport® (abobotulinumtoxinA) in Paediatric Lower Limb Spasticity
- Conditions
- Lower Limb Spasticity
- Registration Number
- NCT03017729
- Lead Sponsor
- Ipsen
- 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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 242
- 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
- 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
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method Time intervals between injections 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
Sedation used Day 1, then every 3.5 months (approximately) up to 30 months During each injection cycle and overall
Non-drug therapies From day 1 up to 30 months Listed and tabulated by frequency
Direct and indirect health care costs From day 1 up to 30 months Derived from the collected data, including concomitant treatments.
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.
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
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
Percentage achievement of primary treatment goal 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
Average GAS T score 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).
Trial Locations
- Locations (24)
Children's Hospital at Erlanger
🇺🇸Chattanooga, Tennessee, United States
Utah Neuro Rehabilitation
🇺🇸Murray, Utah, United States
Texas Children's
🇺🇸Plano, Texas, United States
The Children's Center
🇺🇸Bethany, Oklahoma, United States
Good Shepherd Rehabilitation Network
🇺🇸Allentown, Pennsylvania, United States
Scottish Rite Hospital for Children
🇺🇸Dallas, Texas, United States
Clinical Integrative Research Central Atlanta
🇺🇸Atlanta, Georgia, United States
Laszlo J. Mate, M.D.
🇺🇸North Palm Beach, Florida, United States
William Beaumont Hospital Pediatric Research
🇺🇸Royal Oak, Michigan, United States
University of Missouri
🇺🇸Columbia, Missouri, United States
Gillette Children's Specialty Healthcare
🇺🇸Saint Paul, Minnesota, United States
Washington University School Of Medicine
🇺🇸Saint Louis, Missouri, United States
Valley Health System
🇺🇸Ridgewood, New Jersey, United States
Akron Children's Hospital
🇺🇸Akron, Ohio, United States
Mt. Washington Pediatric Hospital
🇺🇸Baltimore, Maryland, United States
Cleveland Clinic
🇺🇸Cleveland, Ohio, United States
Nicklaus Children's Hospital
🇺🇸Miami, Florida, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States
The Children's Hospital of San Antonio
🇺🇸San Antonio, Texas, United States
Shriners Hospitals for Children
🇺🇸Houston, Texas, United States
Dayton Children's Hospital
🇺🇸Dayton, Ohio, United States
Children's Hospital of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
Children's National Medical Center
🇺🇸Washington, District of Columbia, United States
Montefiore Medical Center
🇺🇸Bronx, New York, United States