Trial Outcomes & Findings for Effect of Dysport Injections on Energy Expenditure and Walking Efficiency in Children With Cerebral Palsy (NCT NCT03469999)

NCT ID: NCT03469999

Last Updated: 2022-06-01

Results Overview

Energy expenditure (oxygen consumption (VO2)) will be measured during the 6 minute walk test using a portable metabolic cart. The participants will use a 10 meter walkway to walk back and forth during the 6 minutes to coverage as much distance as possible. During this time they will be wearing a portable spirometric device that collects the oxygen consumption (VO2) per minute.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 12 participants
• Primary outcome timeframe: Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection
• Results posted on: 2022-06-01

Participant Flow

Participant milestones

Measure	Dysport Injectable Product All participants will participate in baseline data collection of energy expenditure, gait analysis, and lower limb spasticity assessment. All participants will receive single event multi level chemoneurolysis with Dysport and will have repeat data collection at 4 weeks and 12 weeks post injection. Selected dose of medication will be determined by affected muscle(s), severity of spasticity, and the patient's body weight. We will follow the recommended total Dysport dose of 10-15 units/kg per limb, not to exceed 15 units/kg for unilateral lower limb, 30 units/kg for bilateral lower limb, or a total of 1000 units, whichever is lower in a given session.
Overall Study STARTED	12
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Measure

Dysport Injectable Product All participants will participate in baseline data collection of energy expenditure, gait analysis, and lower limb spasticity assessment. All participants will receive single event multi level chemoneurolysis with Dysport and will have repeat data collection at 4 weeks and 12 weeks post injection. Selected dose of medication will be determined by affected muscle(s), severity of spasticity, and the patient's body weight. We will follow the recommended total Dysport dose of 10-15 units/kg per limb, not to exceed 15 units/kg for unilateral lower limb, 30 units/kg for bilateral lower limb, or a total of 1000 units, whichever is lower in a given session.

Overall Study Lost to Follow-up

2

Baseline Characteristics

Effect of Dysport Injections on Energy Expenditure and Walking Efficiency in Children With Cerebral Palsy

Baseline characteristics by cohort

Measure	Dysport Injectable Product n=12 Participants All participants will participate in baseline data collection of energy expenditure, gait analysis, and lower limb spasticity assessment. All participants will receive single event multi level chemoneurolysis with Dysport and will have repeat data collection at 4 weeks and 12 weeks post injection. Selected dose of medication will be determined by affected muscle(s), severity of spasticity, and the patient's body weight. We will follow the recommended total Dysport dose of 10-15 units/kg per limb, not to exceed 15 units/kg for unilateral lower limb, 30 units/kg for bilateral lower limb, or a total of 1000 units, whichever is lower in a given session.
Age, Continuous	11.5 years STANDARD_DEVIATION 3.4 • n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	12 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	12 Participants n=5 Participants
Region of Enrollment United States	12 participants n=5 Participants
Gross Motor Function Classification System (GMFCS) Diagnosis GMFCS I (Children walk at home, school, outdoors and in the community)	2 Participants n=5 Participants
Gross Motor Function Classification System (GMFCS) Diagnosis GMFCS II (Children walk in most settings and climb stairs holding onto a railing)	7 Participants n=5 Participants
Gross Motor Function Classification System (GMFCS) Diagnosis GMFCS III (Children walk using a hand-held mobility device in most indoor settings)	2 Participants n=5 Participants
Gross Motor Function Classification System (GMFCS) Diagnosis Unknown (not part of analysis)	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection

Population: All participants who received Dysport and had VO2 level measured at baseline, week 4 and week 12.

Energy expenditure (oxygen consumption (VO2)) will be measured during the 6 minute walk test using a portable metabolic cart. The participants will use a 10 meter walkway to walk back and forth during the 6 minutes to coverage as much distance as possible. During this time they will be wearing a portable spirometric device that collects the oxygen consumption (VO2) per minute.

Outcome measures

Measure	Dysport Injectable Product n=11 Participants All participants will participate in baseline data collection of energy expenditure, gait analysis, and lower limb spasticity assessment. All participants will receive single event multi level chemoneurolysis with Dysport and will have repeat data collection at 4 weeks and 12 weeks post injection. Selected dose of medication will be determined by affected muscle(s), severity of spasticity, and the patient's body weight. We will follow the recommended total Dysport dose of 10-15 units/kg per limb, not to exceed 15 units/kg for unilateral lower limb, 30 units/kg for bilateral lower limb, or a total of 1000 units, whichever is lower in a given session.
Oxygen Consumption (ml/kg/Min) Baseline (pre-injection)	14.82 ml/kg/min Interval 12.76 to 16.88
Oxygen Consumption (ml/kg/Min) 4 Week	16.75 ml/kg/min Interval 13.53 to 19.96
Oxygen Consumption (ml/kg/Min) 12 Week	28.77 ml/kg/min Interval 9.33 to 48.21

SECONDARY outcome

Timeframe: Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection

Population: Only includes participants who received Dysport and had gait analyzed at baseline, week 4 and week 12.

Gait analysis will be measured by the GaitMat system while the child walks on the mat back and forth for five trials.

Outcome measures

Measure	Dysport Injectable Product n=11 Participants All participants will participate in baseline data collection of energy expenditure, gait analysis, and lower limb spasticity assessment. All participants will receive single event multi level chemoneurolysis with Dysport and will have repeat data collection at 4 weeks and 12 weeks post injection. Selected dose of medication will be determined by affected muscle(s), severity of spasticity, and the patient's body weight. We will follow the recommended total Dysport dose of 10-15 units/kg per limb, not to exceed 15 units/kg for unilateral lower limb, 30 units/kg for bilateral lower limb, or a total of 1000 units, whichever is lower in a given session.
Gait Velocity (cm/s) Baseline (pre-injection)	84.86 cm/sec Interval 67.9 to 101.81
Gait Velocity (cm/s) 4 weeks	92.84 cm/sec Interval 80.04 to 105.06
Gait Velocity (cm/s) 12 weeks	87.11 cm/sec Interval 66.76 to 197.47

SECONDARY outcome

Timeframe: Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection

Population: Only includes participants who received Dysport and completed the MAS at baseline, week 4 and week 12.

The Modified Ashworth Scale (MAS) measures muscle spasticity during passive stretching. Scores are on a 6-point scale and include 0, 1, 1+, 2, 3, and 4. Scores range from 0 (better outcome), which indicates no increase in muscle tone/muscle spasticity to a score of 4 (worse outcome), which indicates a rigid muscle or maximum spasticity. Each subscale is independently scored and not combined to create a composite score. When calculating the mean score, 1+ is equivalent to a value of 1.5.

Outcome measures

Measure	Dysport Injectable Product n=11 Participants All participants will participate in baseline data collection of energy expenditure, gait analysis, and lower limb spasticity assessment. All participants will receive single event multi level chemoneurolysis with Dysport and will have repeat data collection at 4 weeks and 12 weeks post injection. Selected dose of medication will be determined by affected muscle(s), severity of spasticity, and the patient's body weight. We will follow the recommended total Dysport dose of 10-15 units/kg per limb, not to exceed 15 units/kg for unilateral lower limb, 30 units/kg for bilateral lower limb, or a total of 1000 units, whichever is lower in a given session.
Muscle Spasticity Using MAS MAS R Hip Adduction Baseline	1.71 score on a scale Interval 1.26 to 2.17
Muscle Spasticity Using MAS MAS R Hip Adduction 4 week	1.29 score on a scale Interval 0.83 to 1.74
Muscle Spasticity Using MAS MAS R Hip Adduction 12 week	1.17 score on a scale Interval 0.38 to 1.96
Muscle Spasticity Using MAS MAS L Hip Adduction Baseline	1.71 score on a scale Interval 1.26 to 2.17
Muscle Spasticity Using MAS MAS L Hip Adduction 4 week	1.14 score on a scale Interval 0.79 to 1.49
Muscle Spasticity Using MAS MAS L Hip Adduction 12 week	1.17 score on a scale Interval 0.38 to 1.96
Muscle Spasticity Using MAS MAS R Knee Flexion Baseline	2.11 score on a scale Interval 1.65 to 2.57
Muscle Spasticity Using MAS MAS R knee Flexion 4 week	1.44 score on a scale Interval 1.04 to 1.85
Muscle Spasticity Using MAS MAS R Knee Flexion 12 week	1.89 score on a scale Interval 1.43 to 2.35
Muscle Spasticity Using MAS MAS L Knee Flexion Baseline	1.89 score on a scale Interval 1.43 to 2.35
Muscle Spasticity Using MAS MAS L Knee Flexion 4 week	1.78 score on a scale Interval 1.27 to 2.29
Muscle Spasticity Using MAS MAS L Knee Flexion 12 week	1.78 score on a scale Interval 1.14 to 2.42
Muscle Spasticity Using MAS MAS R Dorsiflexion Baseline	2.33 score on a scale Interval 1.95 to 2.72
Muscle Spasticity Using MAS MAS R Dorsiflexion 4 week	1.89 score on a scale Interval 1.63 to 2.15
Muscle Spasticity Using MAS MAS R Dorsiflexion 12 week	2.38 score on a scale Interval 1.94 to 2.81
Muscle Spasticity Using MAS MAS L Dorsiflexion Baseline	2.11 score on a scale Interval 1.3 to 2.92
Muscle Spasticity Using MAS MAS L Dorsiflexion 4 week	1.89 score on a scale Interval 1.43 to 2.35
Muscle Spasticity Using MAS MAS L dorsiflexion 12 week	2.63 score on a scale Interval 1.86 to 3.39

SECONDARY outcome

Timeframe: Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection

Population: Data was not collected for any of the participants.

The Modified Tardieu Scale (MTS) measures muscle spasticity during a passive stretch at both slow and fast speeds. Scores range from 0, which indicates no resistance throughout the course of the passive movement (better outcome), to 5, which indicates that the joint is immovable (worse outcome). The first measure determines the maximum range of motion of a target muscle group in degrees and the second measure determines the angle where muscle resistance is felt during a rapid velocity stretch, recorded in degrees. The total score is calculated by subtracting measure one from measure two. A greater difference between measures indicates less muscle spasticity. The Modified Tardieu Scale will be performed in the hamstrings, gastrocnemius, and soleus muscles.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection

Population: Data was not collected for any of the participants.

The Cerebral Palsy Quality of Life (CP QOL) Questionnaire consists of 66 items ranging from 1 to 9 with a lower score indicating a higher quality of life. The total score will be calculated as well as an individual score for the seven subscale domains: Social well-being and acceptance, functioning, Participation and physical health, Emotional well-being, Access to services, pain and feeling about disability, and family health. Scoring involves 2 steps. First, items are transformed to a scale with a possible range of 0-100. The scores are recoded as follows 1 to 0, 2 to 12.5, 3 to 25, 4 to 37.5, 5 to 50, 6 to 6.2, 7 to 75, 8 to 87.5, 9 to 100. Then the algebraic mean of item values is computed from the composite score for each domain. Once rescored, the final score ranges from 0 to 100, with 0 indicating a worse outcome.

Outcome measures

Outcome data not reported

Adverse Events

Dysport Injectable Product

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Heakyung Kim

Columbia University Irving Medical Center

Phone: 212-305-9416

Email: hk2641@cumc.columbia.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place