Trial Outcomes & Findings for BOTOX® Open-Label Treatment in Pediatric Upper Limb Spasticity (NCT NCT01603615)
NCT ID: NCT01603615
Last Updated: 2019-08-21
Results Overview
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. Safety population included all treated participants.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
220 participants
Primary outcome timeframe
From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Results posted on
2019-08-21
Participant Flow
Pediatric participants with Upper Limb Spasticity who were previously treated with BOTOX® in study 191622-101 \[NCT01603602\] and de novo participants received up to 5 BOTOX® treatments in this study.
Participant milestones
| Measure |
BOTOX®
Participants received a maximum of 5 treatments of intramuscular injections of BOTOX® (botulinum toxin Type A) into upper limb and/or lower limb muscles at a minimum of 12 weeks apart. Treatment dosing was according to investigator judgment, de novo participants received at least 6 U/kg of body weight or a maximum of 8 U/kg of body weight (not to exceed 300 U). Rollover participants received up to a maximum of 8 U/kg of body weight (not to exceed 300 U) for treatment Cycle 1. Dose could be increased to a maximum of 10 U/kg (not to exceed 340 U) in treatment Cycles 2-5. Rolled over participants received intramuscular injections of BOTOX® (botulinum toxin Type A) 3 or 6 U/kg into upper limb in previous study or were de novo participants who were not enrolled in previous study.
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|---|---|
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Overall Study
STARTED
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220
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Overall Study
Safety Population (Treated)
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213
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Overall Study
COMPLETED
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186
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Overall Study
NOT COMPLETED
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34
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Reasons for withdrawal
| Measure |
BOTOX®
Participants received a maximum of 5 treatments of intramuscular injections of BOTOX® (botulinum toxin Type A) into upper limb and/or lower limb muscles at a minimum of 12 weeks apart. Treatment dosing was according to investigator judgment, de novo participants received at least 6 U/kg of body weight or a maximum of 8 U/kg of body weight (not to exceed 300 U). Rollover participants received up to a maximum of 8 U/kg of body weight (not to exceed 300 U) for treatment Cycle 1. Dose could be increased to a maximum of 10 U/kg (not to exceed 340 U) in treatment Cycles 2-5. Rolled over participants received intramuscular injections of BOTOX® (botulinum toxin Type A) 3 or 6 U/kg into upper limb in previous study or were de novo participants who were not enrolled in previous study.
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|---|---|
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Overall Study
Did not Receive Treatment
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7
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Overall Study
Lost to Follow-up
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9
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Overall Study
Personal Reasons
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17
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Overall Study
Other Miscellaneous
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1
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Baseline Characteristics
BOTOX® Open-Label Treatment in Pediatric Upper Limb Spasticity
Baseline characteristics by cohort
| Measure |
BOTOX®
n=213 Participants
Participants received a maximum of 5 treatments of intramuscular injections of BOTOX® (botulinum toxin Type A) into upper limb and/or lower limb muscles at a minimum of 12 weeks apart. Treatment dosing was according to investigator judgment, de novo participants received at least 6 U/kg of body weight or a maximum of 8 U/kg of body weight (not to exceed 300 U). Rollover participants received up to a maximum of 8 U/kg of body weight (not to exceed 300 U) for treatment Cycle 1. Dose could be increased to a maximum of 10 U/kg (not to exceed 340 U) in treatment Cycles 2-5. Rolled over participants received intramuscular injections of BOTOX® (botulinum toxin Type A) 3 or 6 U/kg into upper limb in previous study or were de novo participants who were not enrolled in previous study.
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|---|---|
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Age, Continuous
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8.3 years
STANDARD_DEVIATION 4.1 • n=5 Participants
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Sex: Female, Male
Female
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85 Participants
n=5 Participants
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Sex: Female, Male
Male
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128 Participants
n=5 Participants
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Race/Ethnicity, Customized
White
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130 Participants
n=5 Participants
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Race/Ethnicity, Customized
Black
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9 Participants
n=5 Participants
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Race/Ethnicity, Customized
Asian
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61 Participants
n=5 Participants
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Race/Ethnicity, Customized
Hispanic
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10 Participants
n=5 Participants
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Race/Ethnicity, Customized
Other
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3 Participants
n=5 Participants
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PRIMARY outcome
Timeframe: From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)Population: Safety population included all treated participants.
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. Safety population included all treated participants.
Outcome measures
| Measure |
BOTOX®
n=213 Participants
Participants received a maximum of 5 treatments of intramuscular injections of BOTOX® (botulinum toxin Type A) into upper limb and/or lower limb muscles at a minimum of 12 weeks apart. Treatment dosing was according to investigator judgment, de novo participants received at least 6 U/kg of body weight or a maximum of 8 U/kg of body weight (not to exceed 300 U). Rollover participants received up to a maximum of 8 U/kg of body weight (not to exceed 300 U) for treatment Cycle 1. Dose could be increased to a maximum of 10 U/kg (not to exceed 340 U) in treatment Cycles 2-5. Rolled over participants received intramuscular injections of BOTOX® (botulinum toxin Type A) 3 or 6 U/kg into upper limb in previous study or were de novo participants who were not enrolled in previous study.
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|---|---|
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Percentage of Participants With at Least One Treatment- Emergent Adverse Event (TEAE)
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58.7 percentage of participants
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Adverse Events
BOTOX®
Serious events: 13 serious events
Other events: 57 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
BOTOX®
n=213 participants at risk
Participants received a maximum of 5 treatments of intramuscular injections of BOTOX® (botulinum toxin Type A) into upper limb and/or lower limb muscles at a minimum of 12 weeks apart. Treatment dosing was according to investigator judgment, de novo participants received at least 6 U/kg of body weight or a maximum of 8 U/kg of body weight (not to exceed 300 U). Rollover participants received up to a maximum of 8 U/kg of body weight (not to exceed 300 U) for treatment Cycle 1. Dose could be increased to a maximum of 10 U/kg (not to exceed 340 U) in treatment Cycles 2-5. Rolled over participants received intramuscular injections of BOTOX® (botulinum toxin Type A) 3 or 6 U/kg into upper limb in previous study or were de novo participants who were not enrolled in previous study.
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|---|---|
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Congenital, familial and genetic disorders
Cryptorchism
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0.47%
1/213 • From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Safety population included all treated participants.
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Gastrointestinal disorders
Food poisoning
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0.47%
1/213 • From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Safety population included all treated participants.
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Gastrointestinal disorders
Dental caries
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0.47%
1/213 • From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Safety population included all treated participants.
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Infections and infestations
Gastroenteritis
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0.47%
1/213 • From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Safety population included all treated participants.
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Infections and infestations
Pneumonia
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0.47%
1/213 • From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Safety population included all treated participants.
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Infections and infestations
Respiratory tract infection viral
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0.47%
1/213 • From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Safety population included all treated participants.
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Infections and infestations
Meningitis
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0.47%
1/213 • From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Safety population included all treated participants.
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Metabolism and nutrition disorders
Dehydration
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0.47%
1/213 • From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Safety population included all treated participants.
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Metabolism and nutrition disorders
Hypoglycaemia
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0.47%
1/213 • From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Safety population included all treated participants.
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Musculoskeletal and connective tissue disorders
Joint contracture
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0.47%
1/213 • From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Safety population included all treated participants.
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Musculoskeletal and connective tissue disorders
Juvenile idiopathic arthritis
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0.47%
1/213 • From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Safety population included all treated participants.
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Nervous system disorders
Epilepsy
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0.94%
2/213 • From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Safety population included all treated participants.
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Nervous system disorders
Hemiplegia
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0.47%
1/213 • From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Safety population included all treated participants.
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Nervous system disorders
Seizure
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0.47%
1/213 • From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Safety population included all treated participants.
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Nervous system disorders
Status epilepticus
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0.47%
1/213 • From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Safety population included all treated participants.
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Nervous system disorders
Partial seizures
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0.47%
1/213 • From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Safety population included all treated participants.
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Other adverse events
| Measure |
BOTOX®
n=213 participants at risk
Participants received a maximum of 5 treatments of intramuscular injections of BOTOX® (botulinum toxin Type A) into upper limb and/or lower limb muscles at a minimum of 12 weeks apart. Treatment dosing was according to investigator judgment, de novo participants received at least 6 U/kg of body weight or a maximum of 8 U/kg of body weight (not to exceed 300 U). Rollover participants received up to a maximum of 8 U/kg of body weight (not to exceed 300 U) for treatment Cycle 1. Dose could be increased to a maximum of 10 U/kg (not to exceed 340 U) in treatment Cycles 2-5. Rolled over participants received intramuscular injections of BOTOX® (botulinum toxin Type A) 3 or 6 U/kg into upper limb in previous study or were de novo participants who were not enrolled in previous study.
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|---|---|
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Infections and infestations
Upper respiratory tract infection
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13.6%
29/213 • From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Safety population included all treated participants.
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Infections and infestations
Nasopharyngitis
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10.3%
22/213 • From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Safety population included all treated participants.
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Respiratory, thoracic and mediastinal disorders
Cough
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6.1%
13/213 • From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Safety population included all treated participants.
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee A disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 90 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
- Publication restrictions are in place
Restriction type: OTHER