Trial Outcomes & Findings for BOTOX® Open-Label Treatment in Pediatric Lower Limb Spasticity (NCT NCT01603641)
NCT ID: NCT01603641
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.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
370 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 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.
Participant milestones
| Measure |
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.
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|---|---|
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Overall Study
STARTED
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370
|
|
Overall Study
Safety Population (Treated)
|
367
|
|
Overall Study
Modified Intent-to-treat Population
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366
|
|
Overall Study
COMPLETED
|
335
|
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Overall Study
NOT COMPLETED
|
35
|
Reasons for withdrawal
| Measure |
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.
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|---|---|
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Overall Study
Did not Receive Treatment
|
3
|
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Overall Study
Adverse Event
|
1
|
|
Overall Study
Lack of Efficacy
|
1
|
|
Overall Study
Lost to Follow-up
|
6
|
|
Overall Study
Personal Reasons
|
18
|
|
Overall Study
Other Miscellaneous Reasons
|
6
|
Baseline Characteristics
BOTOX® Open-Label Treatment in Pediatric Lower Limb Spasticity
Baseline characteristics by cohort
| Measure |
BOTOX®
n=367 Participants
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.
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|---|---|
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Age, Continuous
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6.9 years
STANDARD_DEVIATION 3.8 • n=5 Participants
|
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Sex: Female, Male
Female
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167 Participants
n=5 Participants
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Sex: Female, Male
Male
|
200 Participants
n=5 Participants
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Race/Ethnicity, Customized
White
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224 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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109 Participants
n=5 Participants
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Race/Ethnicity, Customized
Hispanic
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21 Participants
n=5 Participants
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Race/Ethnicity, Customized
Other
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4 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.
Outcome measures
| Measure |
BOTOX®
n=367 Participants
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.
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|---|---|
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Percentage of Participants With at Least One Treatment-emergent Adverse Event (TEAE)
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65.4 percentage of participants
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Adverse Events
BOTOX®
Serious events: 24 serious events
Other events: 145 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
BOTOX®
n=367 participants at risk
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.
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|---|---|
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Cardiac disorders
Arrhythmia
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0.27%
1/367 • From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Safety Population included all treated participants.
|
|
Eye disorders
Entropion
|
0.27%
1/367 • From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Safety Population included all treated participants.
|
|
Eye disorders
Strabismus
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0.27%
1/367 • 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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Eye disorders
Cataract
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0.27%
1/367 • 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
Pharyngitis
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1.1%
4/367 • 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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1.1%
4/367 • 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
Laryngitis
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0.27%
1/367 • 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
Tonsillitis
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0.27%
1/367 • 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
Bronchitis
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0.54%
2/367 • 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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Investigations
Aspartate aminotransferase increased
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0.27%
1/367 • 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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Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Renal cancer
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0.27%
1/367 • 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
Febrile convulsion
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1.1%
4/367 • 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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0.54%
2/367 • 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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0.54%
2/367 • 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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0.54%
2/367 • 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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0.27%
1/367 • 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
Asthma
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0.27%
1/367 • 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=367 participants at risk
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.
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|---|---|
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General disorders
Pyrexia
|
7.4%
27/367 • From first dose of study drug up to 12 weeks post last dose (Up to 60 weeks)
Safety Population included all treated participants.
|
|
Infections and infestations
Upper respiratory tract infection
|
18.3%
67/367 • 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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17.7%
65/367 • 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
Bronchitis
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5.7%
21/367 • 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