Does Botulinum Toxin A Make Walking Easier in Children With Cerebral Palsy?
Condition: Cerebral Palsy; Muscle Spasticity
MeSH terms: Muscle Spasticity; Cerebral Palsy
Study type: Interventional
Phase: Phase 4

Summary: 
      In Norway, about 60% of all children with cerebral palsy (CP) are being treated with
      botulinum toxin A (BoNT-A) at 6 years of age, mainly in the legs. Despite this widespread use
      of the drug, the evidence for a positive effect on walking is insufficient. Moreover, large
      variation in effect is seen by clinicians. The main objective of the present study is to
      investigate whether injections with BoNT-A in the calf muscles make walking easier in
      children with spastic CP within 6 months, reflected by reduced energy cost during walking.
    

Detailed description: 
      This is an industry independent multicentre clinical trial. The randomization will be done by
      a computer number random generator and will be carried out and held by the unit of Applied
      Clinical Research at NTNU. Two strata: age and center.

      The study will be conducted according to Consort guidelines and guidelines for Good Clinical
      Practice. It is approved by the local Ethical committee (REK Nord) and the Norwegian Drug
      Agency.

      Primary research question is: Do BoNT-A injections in the calf muscles make walking easier in
      children with CP? Secondary research questions: 1) Do BoNT-A injections in the calf muscles
      increase activity? 2) Do BoNT-A injections in the calf muscles improve walking capacity 3) Do
      BoNT-A injections in the calf muscles improve perceived performance and satisfaction related
      to mobility tasks and 4) Do BoNT-A injections in the calf muscles reduce recurrent
      musculoskeletal pain? The participants will receive the treatment with both local anaesthesia
      and conscious sedation with oral or nasal benzodiazepines.Outcome measures are made at
      baseline and 4, 12 and 24 weeks after treatment, with primary endpoint at 12 weeks.

      Data will be analyzed using a linear mixed model (LMM). The difference in change in the
      primary outcome measure (energy cost during walking) between the treated and placebo groups
      will be done using a post hoc test following the LMM. Secondary, the same model will be used
      to test for an effect also at 4 and 24 weeks post injection. Age, GMFCS Level, number of
      prior BoNT-A treatments and study center will be considered as potential covariates.

      A substudy will be conducted within the frames of this RCT, aiming to identify
      characteristics of those who respond to the treatment compared to those who do not respond
      (outcome measures 6, 7,8 and 9).
    

Intervention name(s): botox; placebo
Intervention type(s): Drug; Drug
Intervention description(s): The agent will be given only once at point zero in the time scheme for the project.; The agent will be given only once at point zero in the time scheme for the project.
Other intervention name(s): botulinum toxin A; sodium chloride
Intervention MeSH terms: Botulinum Toxins; Botulinum Toxins, Type A
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Botulinum Toxins, Type A; Walking