Clinical trial NCT03017729: Effectiveness and Safety of Dysport® (abobotulinumtoxinA) in Paediatric Lower Limb Spasticity
Official title: A Phase IV, Prospective, Observational, Multicentre Study Evaluating the Effectiveness and Safety of Dysport® (abobotulinumtoxinA) in Paediatric Lower Limb Spasticity.
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Lower Limb Spasticity
Lead sponsor: Ipsen
Sponsor class: INDUSTRY
First posted: 2017-01-11
Enrollment: 242
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Pediatric patients with lower limb spasticity

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).


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

Primary outcomes:
measure: Cumulated GAS T score
timeFrame: From day 1 up to 30 months
Secondary outcomes:
measure: AbobotulinumtoxinA dose
timeFrame: Day 1, then every 3.5 months (approximately) up to 30 months
measure: Time intervals between injections
timeFrame: Day 1, then every 3.5 months (approximately) up to 30 months
measure: Number of injection points
timeFrame: Day 1, then every 3.5 months (approximately) up to 30 months
measure: Muscle(s) injected
timeFrame: Day 1, then every 3.5 months (approximately) up to 30 months
measure: Sedation used
timeFrame: Day 1, then every 3.5 months (approximately) up to 30 months
measure: Type of injection guidance utilised
timeFrame: Day 1, then every 3.5 months (approximately) up to 30 months
measure: Concomitant drug therapies
timeFrame: From day 1 up to 30 months
measure: Non-drug therapies
timeFrame: From day 1 up to 30 months
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.
timeFrame: From day 1 up to 30 months (per investigator's decision/routine practice)
measure: Average GAS T score
timeFrame: Day 1 then every 3.5 months (approximately) up to 30 months
measure: Percentage achievement of primary treatment goal
timeFrame: Day 1 then every 3.5 months (approximately) up to 30 months
measure: Percentage achievement of primary treatment goal(s) per goal area(s) after repeated abobotulinumtoxinA injections
timeFrame: Day 1 then every 3.5 months (approximately) up to 30 months
measure: Incidence of adverse events and special situations collected
timeFrame: From day 1 up to 30 months
measure: Direct and indirect health care costs
timeFrame: From day 1 up to 30 months
Other outcomes:
Locations:
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Miami
country: United States
facility: Nicklaus Children's Hospital
state: Florida
city: North Palm Beach
country: United States
facility: Laszlo J. Mate, M.D.
state: Florida
city: Atlanta
country: United States
facility: Clinical Integrative Research Central Atlanta
state: Georgia
city: Baltimore
country: United States
facility: Mt. Washington Pediatric Hospital
state: Maryland
city: Royal Oak
country: United States
facility: William Beaumont Hospital Pediatric Research
state: Michigan
city: Saint Paul
country: United States
facility: Gillette Children's Specialty Healthcare
state: Minnesota
city: Columbia
country: United States
facility: University of Missouri
state: Missouri
city: Saint Louis
country: United States
facility: Washington University School Of Medicine
state: Missouri
city: Ridgewood
country: United States
facility: Valley Health System
state: New Jersey
city: Bronx
country: United States
facility: Montefiore Medical Center
state: New York
city: Akron
country: United States
facility: Akron Children's Hospital
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Dayton
country: United States
facility: Dayton Children's Hospital
state: Ohio
city: Bethany
country: United States
facility: The Children's Center
state: Oklahoma
city: Portland
country: United States
facility: Shriners Hospitals for Children
state: Oregon
city: Allentown
country: United States
facility: Good Shepherd Rehabilitation Network
state: Pennsylvania
city: Philadelphia
country: United States
facility: Shriners Hospitals for Children
state: Pennsylvania
city: Greenville
country: United States
facility: Shriners Hospitals for Children
state: South Carolina
city: Chattanooga
country: United States
facility: Children's Hospital at Erlanger
state: Tennessee
city: Dallas
country: United States
facility: Scottish Rite Hospital for Children
state: Texas
city: Houston
country: United States
facility: Shriners Hospitals for Children
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Plano
country: United States
facility: Texas Children's
state: Texas
city: San Antonio
country: United States
facility: The Children's Hospital of San Antonio
state: Texas
city: Murray
country: United States
facility: Utah Neuro Rehabilitation
state: Utah
city: Milwaukee
country: United States
facility: Children's Hospital of Wisconsin
state: Wisconsin
Study officials:
affiliation: Ipsen
name: Ipsen Medical Director
role: STUDY_DIRECTOR
References: