Neuropediatrics 2017; 48(S 01): S1-S45
DOI: 10.1055/s-0037-1603014
P – Poster
Georg Thieme Verlag KG Stuttgart · New York

Phase 3 Trial to Evaluate AbobotulinumtoxinA (Dysport) Injections in Children with Upper Limb Spasticity Due to Cerebral Palsy: A Study Design

M. R. Delgado
1   Texas Scottish Rite Hospital for Children, Dallas, Texas, United States
,
A. Tilton
2   LSUHSC and Children's Hospital New Orleans, New Orleans, Louisiana, United States
,
J. Carranza
3   Hospital San José Celaya, Celaya, Guanajuato, Mexico
,
M. Bonikowski
4   Non-Public Healthcare Unit, Mazovian Neurorehabilitation and Psychiatry Center in Zagorze, Wiazowna, Poland
,
N. Dursun
5   Kocaeli University Medical Faculty, Izmit, Turkey
,
F. Catus
6   Ipsen, Les Ulis, France
,
P. Picaut
6   Ipsen, Les Ulis, France
› Author Affiliations
Further Information

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