Cerebral Palsy, Muscle Spasticity, Pediatrics
Conditions
Brief summary
This study will evaluate the long-term safety of BOTOX® (botulinum toxin Type A) for the treatment of pediatric lower limb spasticity.
Interventions
BIOLOGICALBotulinum Toxin Type A
Participants received intramuscular injections of botulinum toxin Type A into the lower limb muscles and/or upper limb muscles at a minimum of 12 weeks apart for a maximum of 5 treatments.
Sponsors
Allergan
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
* Minimum weight of 10 kilograms (kg)/22 pounds (lb) * Cerebral palsy with dynamic muscle contracture of the ankle
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 3 months * History of surgical intervention of the lower study leg within 1 year, or planned surgery of any limb during the study
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With at Least One Treatment-emergent Adverse Event (TEAE) | From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks) | An adverse event (AE) was any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. A TEAE was an AE that occurred after receiving the first dose of investigational product or an AE present prior to first dose but increased in severity during the Treatment Period. |
Countries
Hungary, Italy, Philippines, Poland, Russia, South Korea, Thailand, Turkey (Türkiye), United States
Participant flow
Pre-assignment details
Pediatric participants with lower limb spasticity who were previously treated with BOTOX® in study 191622-111 \[NCT01603628\] and de novo participants received up to 5 BOTOX® treatments in this study.
Participants by arm
| Arm | Count |
|---|---|
| BOTOX® Participants received maximum of 5 treatments of intramuscular injections of BOTOX® (botulinum toxin Type A) into a single lower limb muscles or divided between both lower limb muscles or into the lower limb muscles and/or upper limb muscles at a minimum of 12 weeks apart. Treatment dosing was according to investigator judgment not to exceed a maximum of 8 unit per kilogram (U/kg) of body weight (not to exceed 300 U) in treatment Cycle 1. Dose could be increased to a maximum of 10 U/kg (not to exceed 340 U) in treatment Cycles 2-5. Participants received intramuscular injections of BOTOX® (botulinum toxin Type A) 4 or 8 U/kg into the lower limb in the previous study or were de novo participants who were not enrolled in the previous study. | 367 |
| Total | 367 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 1 |
| Overall Study | Did not Receive Treatment | 3 |
| Overall Study | Lack of Efficacy | 1 |
| Overall Study | Lost to Follow-up | 6 |
| Overall Study | Other Miscellaneous Reasons | 6 |
| Overall Study | Personal Reasons | 18 |
Baseline characteristics
| Characteristic | BOTOX® |
|---|---|
| Age, Continuous | 6.9 years STANDARD_DEVIATION 3.8 |
| Race/Ethnicity, Customized Asian | 109 Participants |
| Race/Ethnicity, Customized Black | 9 Participants |
| Race/Ethnicity, Customized Hispanic | 21 Participants |
| Race/Ethnicity, Customized Other | 4 Participants |
| Race/Ethnicity, Customized White | 224 Participants |
| Sex: Female, Male Female | 167 Participants |
| Sex: Female, Male Male | 200 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 367 |
| other Total, other adverse events | 145 / 367 |
| serious Total, serious adverse events | 24 / 367 |
Outcome results
Primary
Percentage of Participants With at Least One Treatment-emergent Adverse Event (TEAE)
An adverse event (AE) was any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. A TEAE was an AE that occurred after receiving the first dose of investigational product or an AE present prior to first dose but increased in severity during the Treatment Period.
Time frame: From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Population: Safety population included all treated participants.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| BOTOX® | Percentage of Participants With at Least One Treatment-emergent Adverse Event (TEAE) | 65.4 percentage of participants |