Detailed Outcome Data

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Clinical Review Report: abobotulinumtoxinA (Dysport Therapeutic): (Ipsen Biopharmaceuticals Canada Inc.): Indication: For the symptomatic treatment of lower-limb spasticity in pediatric patients 2 years of age and older [Internet]. Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2018 Aug.

Cover of Clinical Review Report: abobotulinumtoxinA (Dysport Therapeutic)

Clinical Review Report: abobotulinumtoxinA (Dysport Therapeutic): (Ipsen Biopharmaceuticals Canada Inc.): Indication: For the symptomatic treatment of lower-limb spasticity in pediatric patients 2 years of age and older [Internet].

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Appendix 4Detailed Outcome Data

Table 18Modified Ashworth Scale Score in the (Most) Affected Leg, Change From Baseline at all Time Points (Except Week 4) (Dose per Leg) for Study 141 — ITT Population

	AboBoNTA 10 U/kg/leg (N = 79)	AboBoNTA 15 U/kg/leg (N = 79)	Placebo (N = 77)
MAS score at baseline
Mean (SD)	▬	▬	▬
Week 12
N (%)	▬	▬	▬
Mean change (SD)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change versus placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Week 16
N (%)	▬	▬	▬
Mean change (SD)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change versus placebo (95% CI)	▬	▬	▬
Week 22
N (%)	▬	▬	▬
Mean change (SD)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change versus placebo (95% CI)	▬	▬	▬
Week 28^a
N (%)	▬	▬	▬
Mean change (SD)	▬	▬	▬

: aboBoNTA = abobotulinumtoxinA; ANCOVA = analysis of covariance; BoNT = botulinum toxin; CI = confidence interval; ITT = intention-to-treat; LS = least squares; MAS = Modified Ashworth Scale; N = number of patients in group; n = number of patients with data; SD = standard deviation; U = unit.
: ▬▬▬▬▬
: ▬▬▬▬▬
: ▬▬▬▬▬
: Source: Study 141 Clinical Study Report.²²

Table 19Modified Ashworth Scale Score in the (Most) Affected Leg, Change From Baseline at Week 4, by Botulinum Toxin Status and Treatment Group (Dose per Leg) for Study 141 — ITT Population

	AboBoNTA10 U/kg/leg (N = 79)	AboBoNTA15 U/kg/leg (N = 79)	Placebo (N = 77)
Botulinum toxin status = naive
N (%)	▬	▬	▬
MAS score at baseline
Mean (SD)	▬	▬	▬
MAS score at week 4
Mean (SD)	▬	▬	▬
Change in MAS score from baseline to week 4
Mean (SD)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
Botulinum toxin status = experienced
N (%)	▬	▬	▬
MAS score at baseline
Mean (SD)	▬	▬	▬
MAS score at week 4
Mean (SD)	▬	▬	▬
Change in MAS score from baseline to week 4
Mean (SD)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬

: aboBoNTA = abobotulinumtoxinA; BoNT = botulinum toxin; CI = confidence interval; ITT = intention-to-treat; LS = least squares; MAS = Modified Ashworth Scale; N = number of patients in group; SD = standard deviation; U = unit.
: ▬▬▬▬▬
: Source: Study 141 Clinical Study Report.²²

Table 20Modified Ashworth Scale Score Responders in the (Most) Affected Leg (One Grade Improvement) (Dose per Leg) for Study 141 — ITT Population

	AboBoNTA 10 U/kg/leg (N = 79)	AboBoNTA 15 U/kg/leg (N = 79)	Placebo (N = 77)
Week 4
n	▬	▬	▬
Responders (%)	▬	▬	▬
Odds ratio versus placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Week 12
n	▬	▬	▬
Responders (%)	▬	▬	▬
Odds ratio versus placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Week 16
n	▬	▬	▬
Responders (%)	▬	▬	▬
Odds ratio versus placebo (95% CI)	▬	▬	▬
Week 22
n	▬	▬	▬
Responders (%)	▬	▬	▬
Odds ratio versus placebo (95% CI)	▬	▬	▬
Week 28
n	▬	▬	▬
Responders (%)	▬	▬	▬

: aboBoNTA = abobotulinumtoxinA; BoNT = botulinum toxin; CI = confidence interval; ITT = intention-to-treat; MAS = Modified Ashworth Scale; N = number of patients in group; n = number of patients with data; U = unit.
: ▬▬▬▬▬
: Source: Study 141 Clinical Study Report.²²

Table 21Tardieu Scale: Angle of Arrest and Angle of Catch in the (Most) Affected Leg, Change From Baseline at all Time Points (Dose per Leg) — ITT Population

	AboBoNTA 10 U/kg/leg (N = 79)	AboBoNTA 15 U/kg/leg (N = 79)	Placebo (N = 77)
Angle of arrest (X_V1) in degrees
Baseline
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
Week 4
n (%)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change versus placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Week 12
n (%)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change versus placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Week 16
n (%)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change versus placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Week 22
n (%)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change versus placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Angle of catch (X_V3) in degrees
Baseline
N (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
Week 4
n (%)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change versus placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Week 12
n (%)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change versus placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Week 16
n (%)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change versus placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Week 22
n (%)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change versus placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬

: aboBoNTA = abobotulinumtoxinA; ANCOVA = analysis of covariance; BoNT = botulinum toxin; CI = confidence interval; ITT = intention-to-treat; LS = least squares; MAS = Modified Ashworth Scale; N = number of patients in group; n = number of patients with data; NA = not applicable; SD = standard deviation; U = unit.
: ▬▬▬▬▬
: Source: Study 141 Clinical Study Report.²²

Table 22Tardieu Scale: Spasticity Angle and Spasticity Grade in the (Most) Affected Leg, Change From Baseline at all Time Points (Dose per Leg) for Study 141 — ITT Population

	AboBoNTA 10 U/kg/leg (N = 79)	AboBoNTA 15 U/kg/leg (N = 79)	Placebo (N = 77)
Spasticity angle (X) in degrees
Baseline
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
Week 4
n (%)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change versus placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Week 12
n (%)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change versus placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Week 16
n (%)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change versus placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Week 22
n (%)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change versus placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Spasticity grade (Y)
Baseline
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
Week 4
n (%)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change versus placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Week 12
n (%)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change versus placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Week 16
n (%)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change versus placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Week 22
n (%)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change versus placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬

: aboBoNTA = abobotulinumtoxinA; BoNT = botulinum toxin; CI = confidence interval; ITT = intention-to-treat; LS = least squares; MAS = Modified Ashworth Scale; N = number of patients in group; n = number of patients with data; SD = standard deviation; U = unit.
: ▬▬▬▬▬
: Source: Study 141 Clinical Study Report.²²

Table 23Goal Attainment Scaling: Summary of Goals Selected at Baseline for Study 141, (ITT Population)

	AboBoNTA 10 U/kg/leg (N = 79)	AboBoNTA 15 U/kg/leg (N = 79)	Placebo (N = 77)
Improved walking pattern	48 (60.8)	63 (79.7)	54 (70.1)
Improved balance	31 (39.2)	26 (32.9)	19 (24.7)
Decreased frequency of falling	22 (27.8)	26 (32.9)	25 (32.5)
Decreased frequency of tripping	16 (20.3)	17 (21.5)	13 (16.9)
Improved endurance	18 (22.8)	11 (13.9)	11 (14.3)
Other	10 (12.7)	12 (15.2)	18 (23.4)
Decreased foot pain	6 (7.6)	5 (6.3)	10 (13.0)
Increased walking speed	6 (7.6)	9 (11.4)	3 (3.9)
Improved tolerance of the AFO	7 (8.9)	4 (5.1)	5 (6.5)
Looks better	2 (2.5)	5 (6.3)	7 (9.1)
Longer shoe wear	1 (1.3)	2 (2.5)	1 (1.3)
Improved ease in putting on the AFO	0	1 (1.3)	2 (2.6)

: aboBoNTA = abobotulinumtoxinA; AFO = ankle-foot orthoses; ITT = intention-to-treat; N = number of patients in group; n = number of patients with data; U = unit.
: Source: Study 141 Clinical Study Report.²²

Table 24Goal Attainment Scaling Total Score at all Time Points (Except Week 4) (Dose per Leg) for Study 141 — ITT Population

	AboBoNTA 10 U/kg/leg (N = 79)	AboBoNTA 15 U/kg/leg (N = 79)	Placebo (N = 77)
Week 12
n (%)	▬	▬	▬
Mean change (SD)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change vs. placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Week 16
n (%)	▬	▬	▬
Mean change (SD)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change vs. placebo (95% CI)	▬	▬	▬
Week 22
n (%)	▬	▬	▬
Mean change (SD)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change vs. placebo (95% CI)	▬	▬	▬
Week 28^a
n (%)	▬	▬	▬
Mean change (SD)	▬	▬	▬

: aboBoNTA = abobotulinumtoxinA; ANOVA = analysis of variance; BoNT = botulinum toxin; Cl = confidence interval; ITT = intention-to-treat; LS = least squares; N = number of patients in group; n = number of patients with data; SD = standard deviation; U = unit; vs. = versus.
: ▬▬▬▬▬
: ▬▬▬▬▬
: Source: Study 141 Clinical Study Report.²²

Table 25Goal Attainment Scaling — Top Three Most Commonly Selected Individual Goals (Dose per Leg) for Study 141 — ITT Population

	AboBoNTA 10 U/kg/leg (N = 79)	AboBoNTA 15 U/kg/leg (N = 79)	Placebo (N = 77)
Improved walking pattern
n (%)	▬	▬	▬
Mean total score (SD)	▬	▬	▬
Improved balance
n (%)	▬	▬	▬
Mean total score (SD)	▬	▬	▬
Decreased frequency of falling
n (%)	▬	▬	▬
Mean total score (SD)	▬	▬	▬

: aboBoNTA = abobotulinumtoxinA; ITT = intention-to-treat; N = number of patients in group; n = number of patients with data; SD = standard deviation; U = unit.
: ▬▬▬▬▬
: Source: Study 141 Clinical Study Report.²²

Table 26Goal Attainment Scaling Total Score at Week 4 by Botuhnum Toxin Status and Group (Dose per Leg) for Study 141 — ITT Population

	AboBoNTA 10 U/kg/leg (N = 79)	AboBoNTA 15 U/kg/leg (N = 79)	Placebo (N = 77)
BoNT Status = Naive
GAS score at week 4
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
BoNT Status = Experienced
GAS score at week 4
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬

: BoNT = botulinum toxin; CI = confidence interval; GAS = goal attainment scaling; ITT = intention-to-treat; LS = least squares; N = number of patients in group; n = number of patients with data; SD = standard deviation; U = unit.
: ▬▬▬▬▬
: Source: Study 141 Clinical Study Report.²²

Table 27Pediatric Quality of Life Inventory Generic Core Scores, Change From Baseline (Dose per Leg) for Study 141 — ITT Population

	AboBoNTA 10 U/kg/leg (N = 79)	AboBoNTA 15 U/kg/leg (N = 79)	Placebo (N = 77)
Score = Physical Health Summary
At baseline
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
Change from baseline to week 12
n (%)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
Change from baseline to end of study
n (%)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
Score = Psychosocial Health Summary
At baseline
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
Change from baseline to week 12
n (%)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
Change from baseline to end of study
n (%)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
Score = Total Scale
At baseline
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
Change from baseline to week 12
n (%)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
Change from baseline to end of study
n (%)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬

: aboBoNTA = abobotulinumtoxinA; BoNT = botulinum toxin; CI = confidence interval; ITT = intention-to-treat; LS = least squares; SD = standard deviation.
: ▬▬▬▬▬
: Source: Study 141 Clinical Study Report.²²

Table 28Pediatnc Quality of Life Inventory Cerebral Palsy Module Scores, Change From Baseline (Dose per Leg) for Study 141 — ITT Population

	AboBoNTA 10 U/kg/leg (N = 79)	AboBoNTA 15 U/kg/leg (N = 79)	Placebo (N = 77)
Score = Daily Activities
At baseline
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
Change from baseline to week 12
n (%)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
Change from baseline to end of study
n (%)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
Score = Eating Activities
At baseline
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
Change from baseline to week 12
n (%)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
Change from baseline to end of study
n (%)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
Score = Fatigue
At baseline
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
Change from baseline to week 12
n (%)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
Change from baseline to end of study
n (%)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
Score = Movement and Balance
At baseline
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
Change from baseline to week 12
n (%)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
Change from baseline to end of study
n (%)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
Score = Pain and Hurt
At baseline
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
Change from baseline to week 12
n (%)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
Change from baseline to end of study
n (%)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
Score = School Activities
At baseline
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
Change from baseline to week 12
n (%)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
Change from baseline to end of study
n (%)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
Score = Speech and Communication
At baseline
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
Change from baseline to week 12
n	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
Change from baseline to end of study
n (%)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬

: aboBoNTA = abobotulinumtoxinA; BoNT = botulinum toxin; CI = confidence interval; LS = least squares; SD = standard deviation.
: ▬▬▬▬▬
: Source: Study 141 Clinical Study Report.²²

Table 29Lower-Limb Pain — Faces Pain Scale, Change From Baseline (Dose per Leg) for Study 141 (ITT Population)

	AboBoNTA 10 U/kg/leg (N = 79)	AboBoNTA 15 U/kg/leg (N = 79)	Placebo (N = 77)
At baseline
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
At Week 4
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
Change from baseline to week 4
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
At Week 12
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
Change from baseline to week 12
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
At Week 16
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
Change from baseline to week 16
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
At Week 22
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
Change from baseline to end of study
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
At end of study
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
Change from baseline to week 22
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬

: aboBoNTA = abobotulinumtoxinA; BoNT = botulinum toxin; Cl = confidence interval LS = least squares; SD = standard deviation.
: ▬▬▬▬▬
: Source: Study 141 Clinical Study Report.²²

Table 30Physician’s Global Assessment of Treatment Response at All Time Points (Except Week 4) (Dose per Leg) for Study 141 — ITT Population

	AboBoNTA 10 U/kg/leg (N = 79)	AboBoNTA 15 U/kg/leg (N = 79)	Placebo (N = 77)
Week 12
n (%)	▬	▬	▬
Mean change (SD)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change vs. placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Week 16
n (%)	▬	▬	▬
Mean change (SD)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change vs. placebo (95% CI)	▬	▬	▬
Week 22
n (%)	▬	▬	▬
Mean change (SD)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change vs. placebo (95% CI)	▬	▬	▬
Week 28^a
n (%)	▬	▬	▬
Mean change (SD)	▬	▬	▬

: aboBoNTA = abobotulinumtoxinA; ANOVA = analysis of variance; BoNT = botulinum toxin; CI = confidence interval; ITT = intention-to-treat; LS = least squares; N = number of patients in group; n = number of patients with data; SD = standard deviation; U = unit; vs. = versus.
: ▬▬▬▬▬
: ▬▬▬▬▬
: Source: Study 141 Clinical Study Report.²²

Table 31Physician’s Global Assessment of Treatment Response at Week 4, by Botulinum Toxin Status and Treatment Group (Dose per Leg) for Study 141 — ITT Population

	AboBoNTA 10 U/kg/leg (N = 79)	AboBoNTA 15 U/kg/leg (N = 79)	Placebo (N = 77)
Botulinum Toxin Status = Naive
n (%)	▬	▬	▬
PGA score at week 4
Mean (SD)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬
Botulinum Toxin Status = Experienced
n (%)	▬	▬	▬
PGA score at week 4
Mean (SD)	▬	▬	▬
LS mean (95% CI)	▬	▬	▬
Comparison with placebo
Difference in LS mean (95% CI)	▬	▬	▬
P value	▬	▬	▬

: aboBoNTA = abobotulinumtoxinA; BoNT = botulinum toxin; CI = confidence interval; ITT = intention-to-treat; LS = least squares; N = number of patients in group; PGA = Physician’s Global Assessment; SD = standard deviation; U = unit.
: ▬▬▬▬▬
: Source: Study 141 Clinical Study Report.²²

Table 32Observational Gait Scale in the (Most) Affected Leg, Change From Baseline at All Time Points (Dose per Leg) for Study 141 — ITT Population

	AboBoNTA 10 U/kg/leg (N = 79)	AboBoNTA 15 U/kg/leg (N = 79)	Placebo (N = 77)
Baseline
n (%)	▬	▬	▬
Mean (SD)	▬	▬	▬
Week 4
n (%)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change vs. placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Week 12
n (%)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change vs. placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Week 16
n (%)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change vs. placebo (95% CI)	▬	▬	▬
Week 22
n (%)	▬	▬	▬
LS mean change (95% CI)	▬	▬	▬
LS mean change vs. placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬

: aboBoNTA = abobotulinumtoxinA; BoNT = botulinum toxin; CI = confidence interval; ITT = intention-to-treat; LS = least squares; N = number of patients in group; n = number of patients with data; SD = standard deviation; U = unit; vs.= versus.
: ▬▬▬▬▬
: Source: Study 141 Clinical Study Report.²²

Table 33Observational Gait Scale Responders (One Grade Improvement in the “Initial Foot Contact” Subsection of the OGS in the [Most] Affected Leg) (Dose per Leg) for Study 141 — ITT Population

	AboBoNTA 10 U/kg/leg (N = 79)	AboBoNTA 15 U/kg/leg (N = 79)	Placebo (N = 77)
Week 4
n (%)	▬	▬	▬
Responders (%)	▬	▬	▬
Odds ratio vs. placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Week 12
n (%)	▬	▬	▬
Responders (%)	▬	▬	▬
Odds ratio vs. placebo (95% CI)	▬	▬	▬
P value	▬	▬	▬
Week 16
n (%)	▬	▬	▬
Responders (%)	▬	▬	▬
Odds ratio vs. placebo (95% CI)	▬	▬	▬
Week 22
n (%)	▬	▬	▬
Responders (%)	▬	▬	▬
Odds ratio vs. placebo (95% CI)	▬	▬	▬
Week 28
n (%)	▬	▬	▬
Responders (%)	▬	▬	▬

: aboBoNTA = abobotulinumtoxinA; BoNT = botulinum toxin; CI = confidence interval; ITT = intention-to-treat; N = number of patients in group; n = number of patients with data; vs.= versus; U = unit.
: ▬▬▬▬▬
: Source: Study 141 Clinical Study Report.²²

Table 34GMFM overall score at Week 4 for Study 701 — APT Population

	AboBoNTA 30 U/kg (N = 26)	Placebo (N = 26)
GMFM overall score at baseline
Mean (SD)	87 (10)	76 (18)
GMFM overall score at week 4
Mean (SD)	89 (10)	79 (16)
Change from baseline
Mean (SD)	2.0 (1.8)	2.9 (4.3)
Comparison with placebo
Difference in mean (95% CI)	0.49 (−1.21 to 2.18)
P value	0.566

: aboBoNTA = abobotulinumtoxinA; APT = all patients treated; GMFM = Gross Motor Function Measure; SD = standard deviation.
: Source: Study 701 Clinical Study Report.³⁰

Table 35Change in GMFM Overall and Goal-Total Scores Post-Treatment for Study 701 — APT Population

	AboBoNTA 30 U/kg (N = 26)	Placebo (N = 26)
Overall Score
Week 8
Mean (SD)	2.4 (2.7)	3.8 (4.9)
P value	0.980
Week 16
Mean (SD)	3.4 (4.0)	4.5 (4.5)
P value	0.765
Goal-Total Score
Week 4
Mean (SD)	2.9 (2.9)	3.9 (6.3)
P value	0.945
Week 8
Mean (SD)	3.8 (4.6)	4.8 (7.5)
P value	0.878
Week 16
Mean (SD)	5.9 (5.6)	6.6 (6.6)
P value	0.925

: aboBoNTA = abobotulinumtoxinA; APT = all patients treated; GMFM = Gross Motor Function Measure; SD = standard deviation.
: Source: Study 701 Clinical Study Report.³⁰

Table 36Leeds Videographic Gait Assessment for Study 701 — APT Population

	AboBoNTA (N = 52)	Placebo (N = 52)
Initial Foot Contact
Week 4, N (%)
Heel strike	▬	▬
Flat foot	▬	▬
Toe strike	▬	▬
Mild toe	▬	▬
Marked toe	▬	▬
P value	▬
Week 16, N (%)
Heel strike	▬	▬
Flat foot	▬	▬
Toe strike	▬	▬
Mild toe	▬	▬
Marked toe	▬	▬
P value	▬
Leeds Videographic Assessment
At week 4
Degree of knee flexion,^a N (%)
Neutral/slightly flexed	▬	▬
Hyperextended	▬	▬
Marked knee flexion	▬	▬
P value	▬
Rocker-bottom foot,^a N (%)
Not present	▬	▬
Present	▬	▬
P value	▬
Hindfoot deformity,^a N (%)
Neutral	▬	▬
Occasionally neutral	▬	▬
Valgus	▬	▬
Varus	▬	▬
P value	▬
Use of walking aids,^b N (%)	▬	▬
At week 16
Degree of knee flexion,^a N (%)
Neutral/slightly flexed	▬	▬
Hyperextended	▬	▬
Marked knee flexion	▬	▬
P value	▬
Rocker-bottom foot,^a N (%)	▬	▬
Not present	▬	▬
Present	▬	▬
P value	▬
Hindfoot deformity,^a N (%)	▬	▬
Neutral	▬	▬
Occasionally neutral	▬	▬
Valgus	▬	▬
Varus	▬	▬
P value	▬
Use of walking aids,^b N (%)	▬	▬

: aboBoNTA = abobotulinumtoxinA; APT = all patients treated; GMFM = Gross Motor Function Measure.
: ▬▬▬▬▬
: ▬▬▬▬▬
: ▬▬▬▬▬
: Source: Study 701 Clinical Study Report.³⁰

Table 37Subjective Functional Assessment of Gait for Study 701 — APT Population

	AboBoNTA (N = 26)	Placebo (N = 26)
At Week 4
Parent assessment, N (%)
Good response	13 (50)	12 (46)
Minimal response	9 (35)	6 (23)
No response	4 (15)	8 (31)
P value	0.478
Investigator assessment, N (%)
Good response	15 (58)	11 (42)
Minimal response	6 (23)	5 (19)
No response	5 (19)	10 (39)
P value	0.171
At Week 8
Parent assessment, N (%)
Good response	15 (58)	10 (39)
Minimal response	5 (19)	8 (31)
No response	6 (23)	8 (31)
P value	0.238
Investigator assessment, N (%)
Good response	14 (54)	7 (27)
Minimal response	5 (19)	9 (35)
No response	7 (27)	10 (39)
P value	0.099
At Week 16
Parent assessment, N (%)
Good response	8 (31)	7 (27)
Minimal response	8 (31)	6 (23)
No response	10 (39)	13 (50)
P value	0.457
Investigator assessment, N (%)
Good response	8 (31)	8 (31)
Minimal response	6 (23)	4 (15)
No response	12 (46)	14 (54)
P value	0.673

: aboBoNTA = abobotulinumtoxinA; APT = all patients treated.
: Source: Study 701 Clinical Study Report.³⁰

Table 38Analysis of Number of Patients in Each Category of Change in Leeds Functional Mobility Questionnaire — Sitting and Standing Treatment Comparisons at Weeks 4 and 16, APT Population

	Treatment Comparison^a AboBoNTA Versus Placebo
	Odds Ratio (95% CI)	P Value
At Week 4
How difficult is it for your son/daughter to get up from the floor to standing?	▬	▬
How difficult is it for your son/daughter to sit on a normal chair without falling?	▬	▬
How difficult is it for your son/daughter to balance when bending forward?	▬	▬
How difficult is it for your son/daughter to balance when standing on one affected leg?	▬	▬
How difficult is it for your son/daughter to stand unaided without splints?	▬	▬
How difficult is it for you to relax your son/daughter’s leg muscles with stretching exercises?	▬	▬
At Week 16
How difficult is it for your son/daughter to get up from the floor to standing?	▬	▬
How difficult is it for your son/daughter to sit on a normal chair without falling?	▬	▬
How difficult is it for your son/daughter to balance when bending forward?	▬	▬
How difficult is it for your son/daughter to balance when standing on one affected leg?	▬	▬
How difficult is it for your son/daughter to stand unaided without splints?	▬	▬
How difficult is it for you to relax your son/daughter’s leg muscles with stretching exercises?	▬	▬

: aboBoNTA = abobotulinumtoxinA; APT = all patients treated; CI = confidence interval; LOCF = last observation carried forward.
: ▬▬▬▬▬
: ▬▬▬▬▬
: Source: Study 701 Clinical Study Report.³⁰

Table 39Analysis of Number of Patients in Each Category of Change in Leeds Functional Mobility Questionnaire — Mobility, Treatment Comparisons at Weeks 4 and 16 (APT Population)

	Treatment Comparison^a AboBoNTA Versus Placebo
	Odds Ratio (95% CI)	P Value
At Week 4
How difficult is it for your son/daughter to walk while carrying something?	▬	▬
How difficult is it for your son/daughter to get up stairs?	▬	▬
How difficult is it for your son/daughter to get down stairs?	▬	▬
How tired does your son/daughter become during walking?	▬	▬
How frequently does your son/daughter’s left foot turn in during walking?	▬	▬
At Week 16
How difficult is it for your son/daughter to walk unaided without splints?	▬	▬
How difficult is it for your son/daughter to walk while carrying something?	▬	▬
How difficult is it for your son/daughter to get up stairs?	▬	▬
How difficult is it for your son/daughter to get down stairs?	▬	▬
How tired does your son/daughter become during walking?	▬	▬
How frequently does your son/daughter’s left foot turn in during walking?	▬	▬
How frequently does your son/daughter’s right foot turn in during walking?	▬	▬

: aboBoNTA = abobotulinumtoxinA; CI = confidence interval; APT = all patients treated.
: ▬▬▬▬▬
: ▬▬▬▬▬
: Source: Study 701 Clinical Study Report.³⁰

Table 40Analysis of Number of Patients in Each Category of Change in Leeds Functional Mobility Questionnaire — Other Activities, Treatment Comparisons at Weeks 4 and 16 (APT Population)

	Treatment Comparison^a AboBoNTA Versus Placebo
	Odds Ratio (95% CI)	P Value
At Week 4
How much help does your son/daughter need to use the toilet?	▬	▬
How much help does your son/daughter need to get dressed?	▬	▬
How much help does your son/daughter need to get undressed?	▬	▬
How much help does your son/daughter need to get in the bath?	▬	▬
How much help does your son/daughter need to get out of the bath?	▬	▬
How difficult is it for your son/daughter to play while sitting on the floor with legs straight out in front?	▬	▬
How difficult is it for your son/daughter to play while sitting on the floor with legs in any position?	▬	▬
How difficult is it for your son/daughter to ride a tricycle or bicycle?	▬	▬
How difficult is it for your son/daughter to jump?	▬	▬
How difficult is it for your son/daughter to kick a ball?	▬	▬
How difficult is it for your son/daughter to walk to the side of the swimming pool?	▬	▬
How difficult is it for your son/daughter to swim a width of the swimming pool?	▬	▬
How difficult is it for your son/daughter to get on the school bus?	▬	▬
At Week 16
How much help does your son/daughter need to use the toilet?	▬	▬
How much help does your son/daughter need to get dressed?	▬	▬
How much help does your son/daughter need to get undressed?	▬	▬
How much help does your son/daughter need to get in the bath?	▬	▬
How much help does your son/daughter need to get out of the bath?	▬	▬
How difficult is it for your son/daughter to play while sitting on the floor with legs straight out in front?	▬	▬
How difficult is it for your son/daughter to play while sitting on the floor with legs in any position?	▬	▬
How difficult is it for your son/daughter to jump?	▬	▬
How difficult is it for your son/daughter to walk to the side of the swimming pool?	▬	▬
How difficult is it for your son/daughter to swim a width of the swimming pool?	▬	▬
How difficult is it for your son/daughter to get on the school bus?	▬	▬
How difficult is it for your son/daughter to get off the school bus?	▬	▬
How confident is your son/daughter while walking?	▬	▬

: aboBoNTA = abobotulinumtoxinA; CI = confidence interval; APT = all patients treated.
: ▬▬▬▬▬
: ▬▬▬▬▬
: Source: Study 701 Clinical Study Report.³⁰

Table 41Proportion of Patients Eligible for Re-Treatment (Dose per Leg) for Study 141 — ITT Population

Eligible for Re-Treatment at Visit, n (%)	AboBoNTA 10 U/kg/leg (N = 79)	AboBoNTA 15 U/kg/leg (N = 79)	Placebo (N = 77)
Total eligible for re-treatment	▬	▬	▬
At week 12	▬	▬	▬
At week 16	▬	▬	▬
At week 22	▬	▬	▬
At week 28	▬	▬	▬
After week 28^a	▬	▬	▬

: aboBoNTA = abobotulinumtoxinA; N = number of patients in group; U = unit.
: ▬▬▬▬▬
: ▬▬▬▬▬
: ▬▬▬▬▬
: Source: Study 141 Clinical Study Report.²²

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