Abstract
BackgroundIntramuscular injections of botulinum toxin A (BoNT-A) have been a cornerstone in the treatment of spasticity for the last 20 years. In Norway, the treatment is now offered to two out of three children with spastic cerebral palsy (CP). However, despite its common use, the evidence for its functional effects is limited and inconclusive. The objective of this study is to determine whether BoNT-A makes walking easier in children with CP. We hypothesize that injections with BoNT-A in the calf muscles will reduce energy cost during walking, improve walking capacity, increase habitual physical activity, reduce pain and improve self-perceived performance and satisfaction.Methods/designThis randomized, double-blinded, placebo-controlled, multicenter trial is conducted in a clinical setting involving three health regions in Norway. Ninety-six children with spastic CP, referred for single-level injections with BoNT-A in the calf muscles, will be invited to participate. Those who are enrolled will be randomized to receive either injections with BoNT-A (Botox®) or 0.9% saline in the calf muscles. Stratification according to age and study center will be made. The allocation ratio will be 1:1. Main inclusion criteria are (1) age 4 − 17.5 years, (2) Gross Motor Function Classification System levels I and II, (3) no BoNT-A injections in the lower limbs during the past 6 months and (4) no orthopedic surgery to the lower limbs during the past 2 years. The outcome measures will be made at baseline and 4, 12 (primary endpoint) and 24 weeks after injections. Primary outcome is change in energy cost during walking. Secondary outcomes are change in walking capacity, change in activity, perceived change in performance and satisfaction in mobility tasks, and pain. The primary analysis will use a linear mixed model to test for difference in change in the outcome measures between the groups. The study is approved by the Regional Ethical Committee and The Norwegian Medicines Agency. Recruitment started in September 2015.DiscussionThe evaluation of effect is comprehensive and includes objective standardized tests and measures on both impairment and activity level. Results are to be expected by spring 2019.Trial registrationClinicalTrials.gov, NCT02546999. Registered on 9 September 2015.
Highlights
Intramuscular injections of botulinum toxin A (BoNT-A) have been a cornerstone in the treatment of spasticity for the last 20 years
In a substudy of this randomized control trials (RCTs), we aim to identify different cerebral palsy (CP) phenotypes of responders to BoNT-A treatment and, to gain insight into the processes underlying BoNT-A-induced changes in walking
The gross motor function of the participants should be at levels I or II according to the Gross Motor Function Classification System (GMFCS) [22] and they should not have been treated with BoNT-A in the lower limbs during the past 6 months or have undergone orthopedic surgery to the lower limbs in the last 2 years prior to study participation
Summary
Intramuscular injections of botulinum toxin A (BoNT-A) have been a cornerstone in the treatment of spasticity for the last 20 years. Cerebral palsy (CP) is the most common cause of neurological disability in childhood, and the motor signs associated with the condition include primary neuromuscular deficits, such as spasticity, muscle weakness, and decreased selective motor control, and secondary musculoskeletal problems such as bony malformations and contractures [1]. Impaired walking is a main clinical feature of CP, and approximately 70% of children with CP are able to walk, they all experience varying degrees of restrictions related to this function [2]. Such restrictions include reduced walking speed [3], impaired balance [4] and high energy costs (ECs) during walking [5]. Children with CP are in general less active than their peers, and they less often take part in social activities [11]
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