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

Lower Limb Spasticity Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03017729
• Other study ID #: A-US-52120-330
• Status: Completed
• Start date: February 10, 2017
• Est. completion date: November 30, 2020

Study information

• Verified date: December 2020
• Source: Ipsen
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 242
• Est. completion date: November 30, 2020
• Est. primary completion date: November 30, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 17 Years

Eligibility

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

Related Conditions & MeSH terms

• Lower Limb Spasticity
• Muscle Spasticity

Intervention

Biological:
• Botulinum 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.

Locations

Country	Name	City	State
United States	Akron Children's Hospital	Akron	Ohio
United States	Good Shepherd Rehabilitation Network	Allentown	Pennsylvania
United States	Clinical Integrative Research Central Atlanta	Atlanta	Georgia
United States	Mt. Washington Pediatric Hospital	Baltimore	Maryland
United States	The Children's Center	Bethany	Oklahoma
United States	Montefiore Medical Center	Bronx	New York
United States	Children's Hospital at Erlanger	Chattanooga	Tennessee
United States	Cleveland Clinic	Cleveland	Ohio
United States	University of Missouri	Columbia	Missouri
United States	Scottish Rite Hospital for Children	Dallas	Texas
United States	Dayton Children's Hospital	Dayton	Ohio
United States	Shriners Hospitals for Children	Greenville	South Carolina
United States	Shriners Hospitals for Children	Houston	Texas
United States	Texas Children's Hospital	Houston	Texas
United States	Nicklaus Children's Hospital	Miami	Florida
United States	Children's Hospital of Wisconsin	Milwaukee	Wisconsin
United States	Utah Neuro Rehabilitation	Murray	Utah
United States	Laszlo J. Mate, M.D.	North Palm Beach	Florida
United States	Shriners Hospitals for Children	Philadelphia	Pennsylvania
United States	Texas Children's	Plano	Texas
United States	Shriners Hospitals for Children	Portland	Oregon
United States	Valley Health System	Ridgewood	New Jersey
United States	William Beaumont Hospital Pediatric Research	Royal Oak	Michigan
United States	Washington University School Of Medicine	Saint Louis	Missouri
United States	Gillette Children's Specialty Healthcare	Saint Paul	Minnesota
United States	The Children's Hospital of San Antonio	San Antonio	Texas
United States	Children's National Medical Center	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Ipsen

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	AbobotulinumtoxinA dose	During each injection cycle and overall	Day 1, then every 3.5 months (approximately) up to 30 months
Secondary	Time intervals between injections	During each injection cycle and overall	Day 1, then every 3.5 months (approximately) up to 30 months
Secondary	Number of injection points	During each injection cycle and overall	Day 1, then every 3.5 months (approximately) up to 30 months
Secondary	Muscle(s) injected	During each injection cycle and overall	Day 1, then every 3.5 months (approximately) up to 30 months
Secondary	Sedation used	During each injection cycle and overall	Day 1, then every 3.5 months (approximately) up to 30 months
Secondary	Type of injection guidance utilised	During each injection cycle and overall	Day 1, then every 3.5 months (approximately) up to 30 months
Secondary	Concomitant drug therapies	Listed and tabulated by frequency	From day 1 up to 30 months
Secondary	Non-drug therapies	Listed and tabulated by frequency	From day 1 up to 30 months
Secondary	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)
Secondary	Average GAS T score	Per injection cycle	Day 1 then every 3.5 months (approximately) up to 30 months
Secondary	Percentage achievement of primary treatment goal	During each injection cycle and overall	Day 1 then every 3.5 months (approximately) up to 30 months
Secondary	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
Secondary	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
Secondary	Direct and indirect health care costs	Derived from the collected data, including concomitant treatments.	From day 1 up to 30 months