BOTOX® Treatment in Pediatric Upper Limb Spasticity: Double-blind Study
Trial Snapshot
- Phase
- Phase 3
- Status
- Completed
- Sponsor
- Allergan
- Enrollment
- 235
- Locations
- 45
- Primary Endpoint
- Average Change From Baseline in Modified Ashworth Scale-Bohannon (MAS-B) Score of the Principal Muscle Group at Weeks 4 and 6
Study Overview
Brief Summary
This study will evaluate the safety and efficacy of BOTOX® (botulinum toxin Type A) in pediatric patients with upper limb spasticity.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 2 Years to 16 Years (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Minimum weight of 10 kg/22 lb
- •Upper limb spasticity due to cerebral palsy or stroke
Exclusion Criteria
- •Muscular dystrophy, myasthenia gravis, Eaton-Lambert syndrome, amyotrophic lateral sclerosis, or mitochondrial disease
- •Uncontrolled epilepsy
- •Botulinum Toxin therapy of any serotype for any condition within the last 6 months
- •Previous surgical treatment of the study limb (except tendon lengthening), or planned surgery of the study limb during the study
- •Previous casting of the study limb for spasticity within 6 months or with a dynamic splint within 3 months, or planned casting or dynamic splinting for spasticity of the study limb or affected lower limb during the study
Arms & Interventions
BOTOX® 3 U/kg
Participants received intramuscular injections of BOTOX® (botulinum toxin Type A) 3 units (U) per kilogram (kg) of body weight (3 U/kg) into specified muscles of the upper limb on Day 1. Participants received weekly occupational therapy (OT).
Intervention: botulinum toxin Type A (Biological)
BOTOX® 6 U/kg
Participants received intramuscular injections of BOTOX® (botulinum toxin Type A) 6 U per kg of body weight (6 U/kg) into specified muscles of the upper limb on Day 1. Participants received weekly OT.
Intervention: botulinum toxin Type A (Biological)
Placebo
Participants received intramuscular injections of normal saline (placebo) into specified muscles of the upper limb on Day 1. Participants received weekly OT.
Intervention: Normal Saline (Placebo) (Drug)
Outcomes
Primary Outcomes
Average Change From Baseline in Modified Ashworth Scale-Bohannon (MAS-B) Score of the Principal Muscle Group at Weeks 4 and 6
Time Frame: Baseline (Day 1) to Weeks 4 and 6
The MAS-B was used to evaluate spasticity based on grading the resistance encountered in the principal muscle group (elbow and wrist) by means of passively moving a limb through its range of motion at a study specified velocity. The resistance encountered to passive stretch was graded using a 6-point scale where: 0=no increase in muscle tone (best) to 4=affected part(s) rigid in flexion or extension (worst). For analysis purposes, the MAS-B was recoded as follows: 0=1, 1=1, 1+=2, 2=3, 3=4, 4=5. The scores at Weeks 4 and 6 were averaged. A Mixed Model Repeated Measures (MMRM) model was used for analysis. A negative change from Baseline indicates improvement.
Average Clinical Global Impression (CGI) of Overall Change by Physician at Weeks 4 and 6
Time Frame: Weeks 4 and 6
The CGI of overall change (improvement or worsening) was assessed by the physician considering the participant's clinical condition and severity of side effects using a 9-point scale where: -4=very marked worsening to +4=very marked improvement. The scores at Weeks 4 and 6 were averaged. A MMRM model was used for analysis.
Secondary Outcomes
- Average Change From Baseline in MAS-B Score of the Finger Flexor Muscle Group at Weeks 4 and 6(Baseline (Day 1) to Weeks 4 and 6)
- Goal Attainment Score (GAS) as Assessed by Physician Using a 6-Point Scale(Week 8 and 12)
- Change From Baseline in Severity of Spasticity of the Principal Muscle Group (R2-R1) Calculated Using the Modified Tardieu Scale (MTS)(Baseline (Day 1) to Week 6)