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

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

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).

Study Overview

• Status: Completed
• Conditions: Lower Limb Spasticity
• Intervention / Treatment: Biological: Botulinum toxin type A

• Study Type: Observational
• Enrollment (Actual): 242

Contacts and Locations

Study Locations

• United States
  • District of Columbia
    • Washington, District of Columbia, United States, 20010
      • Children's National Medical Center
  • Florida
    • Miami, Florida, United States, 33155
      • Nicklaus Children's Hospital
    • North Palm Beach, Florida, United States, 33408
      • Laszlo J. Mate, M.D.
  • Georgia
    • Atlanta, Georgia, United States, 30328
      • Clinical Integrative Research Central Atlanta
  • Maryland
    • Baltimore, Maryland, United States, 21209
      • Mt. Washington Pediatric Hospital
  • Michigan
    • Royal Oak, Michigan, United States, 48073
      • William Beaumont Hospital Pediatric Research
  • Minnesota
    • Saint Paul, Minnesota, United States, 55101
      • Gillette Children's Specialty Healthcare
  • Missouri
    • Columbia, Missouri, United States, 65212
      • University of Missouri
    • Saint Louis, Missouri, United States, 63110
      • Washington University School of Medicine
  • New Jersey
    • Ridgewood, New Jersey, United States, 07450
      • Valley Health System
  • New York
    • Bronx, New York, United States, 10467
      • Montefiore Medical Center
  • Ohio
    • Akron, Ohio, United States, 44308
      • Akron Children's Hospital
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic
    • Dayton, Ohio, United States, 45404
      • Dayton Children's Hospital
  • Oklahoma
    • Bethany, Oklahoma, United States, 73008
      • The Children's Center
  • Oregon
    • Portland, Oregon, United States, 97239
      • Shriners Hospitals for Children
  • Pennsylvania
    • Allentown, Pennsylvania, United States, 18103
      • Good Shepherd Rehabilitation Network
    • Philadelphia, Pennsylvania, United States, 19140
      • Shriners Hospitals for Children
  • South Carolina
    • Greenville, South Carolina, United States, 29605
      • Shriners Hospitals for Children
  • Tennessee
    • Chattanooga, Tennessee, United States, 37403
      • Children's Hospital at Erlanger
  • Texas
    • Dallas, Texas, United States, 75219
      • Scottish Rite Hospital for Children
    • Houston, Texas, United States, 77030
      • Texas Children's Hospital
    • Houston, Texas, United States, 77030
      • Shriners Hospitals for Children
    • Plano, Texas, United States, 75024
      • Texas Children's
    • San Antonio, Texas, United States, 78207
      • The Children's Hospital of San Antonio
  • Utah
    • Murray, Utah, United States, 84124
      • Utah Neuro Rehabilitation
  • Wisconsin
    • Milwaukee, Wisconsin, United States, 53226
      • Children's Hospital of Wisconsin

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 17 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Pediatric patients with lower limb spasticity

Description

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

Study Plan

How is the study designed?

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Ipsen

Study record dates

Study Major Dates

• Study Start (ACTUAL): February 10, 2017
• Primary Completion (ACTUAL): November 30, 2020
• Study Completion (ACTUAL): November 30, 2020

Study Registration Dates

• First Submitted: December 30, 2016
• First Submitted That Met QC Criteria: January 9, 2017
• First Posted (ESTIMATE): January 11, 2017

Study Record Updates

• Last Update Posted (ACTUAL): December 19, 2020
• Last Update Submitted That Met QC Criteria: December 17, 2020
• Last Verified: December 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neurologic Manifestations; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Manifestations; Muscle Hypertonia; Muscle Spasticity; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Peripheral Nervous System Agents; Cholinergic Agents; Membrane Transport Modulators; Acetylcholine Release Inhibitors; Neuromuscular Agents; Botulinum Toxins; Botulinum Toxins, Type A; abobotulinumtoxinA
• Other Study ID Numbers: A-US-52120-330

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No