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DOI: 10.1055/s-0037-1603014
Phase 3 Trial to Evaluate AbobotulinumtoxinA (Dysport) Injections in Children with Upper Limb Spasticity Due to Cerebral Palsy: A Study Design
Publication History
Publication Date:
26 April 2017 (online)
Introduction: Although use of botulinum toxin-A (BoNT-A) to treat upper limb spasticity (ULS) in cerebral palsy (CP) is long established, most clinical trials in CP have been small, of limited duration, and without adequate control.
Objective: This pediatric study assesses the efficacy and safety of abobotulinumtoxinA (aboBoNT-A) at 8 and 16 U/kg versus aboBoNT-A 2 U/kg (control group) for the treatment of ULS due to CP (four treatment cycles, 1 year).
Methods: Approximately 210 children (2–17 years) with ULS due to CP (Gross Motor Function Classification System [GMFCS] levels I–IV) will be recruited by specialist centers. Patients will have a Modified Ashworth Scale (MAS) score 32 in the primary targeted muscle group (PTMG; elbow or wrist flexors). Patients will be randomized (1:1:1) to injections of aboBoNT-A 2U/kg, aboBoNT-A 8U/kg, or aboBoNT-A 16U/kg in cycle 1, using a prespecified injection protocol under electrical stimulation/ultrasound guidance. Patients will perform home exercises (under trained care supervision) consistent with the chosen treatment goals throughout the study. Control patients (aboBoNT-A 2 U/kg in cycle 1) will be re-randomized to either aboBoNT-A8 U/kg or aboBoNT-A-16 U/kg in subsequent cycles; double blind will be maintained. Eligibility to move to next cycle will be assessed at Week 16 of each cycle. Primary endpoint will be change from baseline to Week 6 of cycle 1 in muscle tone.
Results: Patient recruitment: ongoing.
Summary/Conclusion: This will be one of the largest and most robust studies simultaneously evaluating effects of BoNT-A on muscle tone, spasticity, and function in pediatric ULS due to CP.
Keywords: Abobotulinumtoxin A, cerebral palsy, upper limb spasticity