Effect of Botulinum Toxin Type A on Motor Function in Children With Cerebral Palsy

Official title

Effect of Botulinum Toxin Type A on Functionality and Quality of Life in Children and Adolescents With Spastic Hemiparetic Cerebral Palsy GMFCS I-II at Teletón Santiago: A Prospective Single-Group Trial

Overview

Project Identification and Researchers Title: "Changes in functionality and quality of life when applying the botulinum toxin type A infiltration protocol in children and adolescents with cerebral palsy of the spastic hemiparesis type GMFCS I and II, from the Teletón Institute Santiago: A prospective single-group trial." This project is led by a group of physiatrists from the Teletón Institute, along with a team of co-investigators specialized in kinesiology, occupational therapy, and nursing. Study Relevance The study aims to generate scientific evidence on the effectiveness of the BoNT-A infiltration protocol in the functionality and quality of life of children with spastic hemiparesis. The results could optimize therapeutic interventions at the Teletón Institute, improve patient well-being, and expand the coverage of therapeutic services for spasticity. Problem Statement Cerebral palsy (CP) is the most common motor disability in childhood, with an estimated prevalence of 1.6 per 1,000 live births in developed countries and between 1 and 2 per 1,000 live births in Chile. Spastic CP requires multidisciplinary management, including BoNT-A infiltration. Although BoNT-A has been shown to reduce spasticity, there is a gap in knowledge regarding its impact on overall functionality and quality of life. Research Question What changes occur in functionality and quality of life when applying the BoNT-A infiltration protocol from the Teletón Institute Santiago in children aged 8 to 17 years, diagnosed with cerebral palsy of the spastic hemiparesis type GMFCS I and II? Study Objectives General Objective: To compare the results in functionality and quality of life before BoNT-A infiltration, at 4 weeks, and at 12 weeks, in children and adolescents diagnosed with cerebral palsy of the spastic hemiparesis type GMFCS I and II. Specific Objectives: To evaluate the effect of the infiltration protocol on the functionality of the upper and lower limbs, and on the quality of life of the children and their families. Hypothesis The BoNT-A infiltration protocol has a positive effect on the functionality of the upper and lower limbs and on the quality of life of children with spastic cerebral palsy. Method The study is a prospective single-group trial, following participants over time to compare the results of the BoNT-A infiltration protocol. The target population includes children with spastic hemiparesis cerebral palsy GMFCS I-II referred to the botulinum toxin infiltration clinic at the Teletón Institute Santiago. A non-probabilistic convenience sampling will be used, selecting individuals who meet the inclusion and exclusion criteria. Procedure Patients will be selected and evaluated before infiltration, at 4 weeks, and at 12 weeks post-infiltration. The infiltration will be performed under ultrasound and electrostimulation guidance. Evaluations will include functionality and quality of life scales, such as the Ashworth Scale, Tardieu Scale, Bilan 400 Points Functional Hand Scale, GMFM-88, KIDSCREEN-27, and Goal Attainment Scale. Data Collection Data collection will be carried out through clinical record review, clinical observation, and application of scales. A homologation protocol will be implemented among operators to standardize the use of the scales. Variables Variables include age, sex, laterality, GMFCS, infiltrated muscles, infiltration dose, educational level, participation in a school integration program, physical activity, manual functionality, lower limb functionality, quality of life, and goal attainment. Statistical Analysis A database will be created in Microsoft Excel, and qualitative and quantitative variables will be analyzed. Chi-square tests, repeated measures ANOVA, and Friedman tests will be used to compare measurements over time. Ethical Considerations The study complies with ethical principles of scientific validity, social utility, researcher competence, favorable risk-benefit ratio, equitable selection of subjects, informed consent, and protection of the privacy and confidentiality of participants.

Interventions

• Procedure Botulinum toxin infiltration
  Botulinum toxin is a medication that is injected directly into the muscles selected by a physiatrist, based on a clinical evaluation. The doses are determined according to the clinical condition of the spasticity, the patient's age, and weight. The procedure is performed under deep sedation and uses ultrasound and electrostimulation to ensure greater safety and precision.

Primary outcome measures

• Bilan 400 Points Functional Hand Scale [Time frame: Baseline, 4 weeks, and 12 weeks after intervention]
• Gross Motor Function Measure (GMFM-88). D and E dimensions [Time frame: Baseline, 4 weeks, and 12 weeks after intervention]
• KIDSCREEN-27 [Time frame: Baseline and 12 weeks after intervention]
• Goal Attainment Scale [Time frame: Baseline and 12 weeks after intervention]

Secondary outcome measures (3)

• Goniometry [Time frame: Baseline, 4 weeks, and 12 weeks after intervention]
• Ashworth Scale [Time frame: Baseline, 4 weeks, and 12 weeks after intervention]
• Tardieu Scale [Time frame: Baseline, 4 weeks, and 12 weeks after intervention]

Eligibility criteria

Inclusion criteria

• Spastic hemiparesis type Cerebral Palsy
• Gross Motor Classification System I-II
• Age 8 to 17 years (based on validation ages for scales to be used)
• Minimum of 6 months since last botulinum toxin injection
• Informed consent signed by legal guardian and assent from users aged 12 to 17 years

Exclusion criteria

• Cognitive impairment that prevents following instructions for functional assessments
• Limitation of joint range not caused by spasticity
• Patients diagnosed with other diseases causing spasticity, different from cerebral palsy
• Patients with other neuromuscular diseases

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Supportive care

Study locations

Chile · 1 center

• Instituto Teleton Santiago — Santiago

Identifiers

NCT: NCT07034547 · TeletonChile

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗