The use of Botulinum Toxin A in ambulant children with cerebral palsy
Research type
Research Study
Full title
Understanding Clinical and Patient Reported Response of Children and Young People with Cerebral Palsy to Botulinum Toxin A: a Longitudinal Observational Study
IRAS ID
211617
Contact name
Lesley Katchburian
Contact email
Sponsor organisation
UCL Great Ormond Street Institute of child health
Duration of Study in the UK
4 years, 8 months, 29 days
Research summary
Children and young people (CYP) with cerebral palsy (CP) often have difficulty moving and taking part in activities due to stiffness (spasticity/dystonia) in their muscles.
Botulinum Toxin A (BoNT-A) is a drug (sometimes known as ‘botox’ or ‘toxin’) that can be injected into muscles to reduce stiffness and make movement easier. The effect of BoNT-A is temporary; this means injections often need to be repeated. The time between injections varies between 4 - 12 months and in some cases longer.What we do not know yet is whether reducing the stiffness in the muscles actually makes life easier for CYP with CP in terms of how much they can do (activity), how much they can join in at home, at school and in the community (participation) or how long these changes last. We also do not know if the standardised measures clinicians use are sensitive enough to pick up the important things that matter to CYP with CP and their families. The goal of this study is to learn about the factors that influence a response to toxin injections for CYP with CP who are able to walk.
CYP and their parents coming to our clinic will be asked to carry out repeated assessments at up to 4 times over 12 months (as per usual clinic procedure). Often clinic assessments focus on a patient’s impairment (tightness). In addition this study will look at participant’s activity levels, participation and quality of life. This will involve a clinical examination, tests to look at gross motor function (as per usual procedure) and also the completion of 2 questionnaires (one for CYP and one for parents/carers) followed by goal setting before treatment and a review of the same goals after treatment. A subgroup of CYP and their parents will also take part in an interview, which will allow them to share views about their experience of receiving BoNT-A treatment and its impact on their life.
NICE have recommended further research on this topic in order to inform decisions that clinicians and families have to make about the use of toxin. Clinicians and families have also identified this as a priority for research. This research will help to address uncertainty about treatment effects.
REC name
London - Central Research Ethics Committee
REC reference
17/LO/0579
Date of REC Opinion
4 May 2017
REC opinion
Favourable Opinion