A Phase 3 interventional study of Botulinum toxin type A in Upper Limb Spasticity (Altered Skeletal Muscle Performance) in Children, sponsored by Ipsen. Completed at 31 sites in 8 countries. Open to participants aged 2 Years to 17 Years. Per ClinicalTrials.gov, last updated 2022-09-28.
Sponsored by Ipsen · Phase 3, Interventional, and Treatment
The purpose of this study is to assess the efficacy and safety of multiple doses of Dysport used in the treatment of upper limb spasticity (altered skeletal muscle performance) in children with cerebral palsy (CP).
704 studies on the registry are indexed under Muscle Spasticity; 149 are open to participants now.
This study's enrollment of 212 is above the median of 36 across 525 interventional studies indexed under Muscle Spasticity.
Browse Muscle Spasticity studies →Ipsen is the lead sponsor of 282 studies on the registry; 16 are open to participants now.
Of its 24 completed or terminated interventional studies of FDA-regulated products, 19 (79%) have results posted.
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Exclusion Criteria:
Group A - Treatments 1, 2, 3 and 4: Dysport 16 Units (U)/kg in one upper extremity (the study limb).
Biological: Botulinum toxin type A
Group B - Treatments 1, 2, 3 and 4: Dysport 8 U/kg in one upper extremity (the study limb).
Biological: Botulinum toxin type A
Group C - Treatment 1: Dysport 2 U/kg in one upper extremity (the study limb). Group C - Treatments 2, 3 and 4: Dysport 8 or 16 U/kg in one upper extremity (the study limb).
Biological: Botulinum toxin type A
Subjects randomised to receive Dysport 2 U/kg, 8 U/kg or 16 U/kg administered intramuscularly in the study limb.
Also known as: AbobotulinumtoxinA (Dysport®)
Mean Change From Baseline to TC 1, Week 6 in MAS Score in the TC 1 PTMG
The MAS was used to assess muscle tone in the upper limb PTMG and consists of 6 grades: 0 (no increase in muscle tone), 1 (slight increase in muscle tone, manifested by a catch and release or by minimal resistance at the end of the ROM) when the affected part is moved in flexion or extension, 1+ (slight increase in muscle tone, manifested by a catch, followed by minimal resistance throughout the remainder (less than half) of the ROM), 2 (more marked increase in muscle tone), 3 (considerable increase in muscle tone) or 4 (affected part(s) rigid in flexion or extension). The original score '+1' was given a derived numeric score of '2' and the higher numeric scores were incremented by 1 so that the MAS score range was from 0 to 5 with higher scores indicating greater muscle tone. A negative change from baseline indicates a decrease in muscle tone.
Time frame: Baseline (TC 1, Day 1) and TC 1, Week 6.
Mean Physician's Global Assessment (PGA) Score at TC 1, Week 6
The PGA of treatment response was assessed by asking the investigator the following question: 'How would you rate the response to treatment in the subject's upper limb since the start of the study?'. Answers were on a 9-point rating scale (-4: markedly worse, -3: much worse, -2: worse, -1: slightly worse, 0: no change, +1: slightly improved, +2: improved, +3: much improved and +4: markedly improved). The mean scores for each treatment group at TC 1, Week 6 are presented.
Time frame: TC 1, Week 6.
Mean Goal Attainment Scale (GAS) Total Score at TC 1, Week 6
The GAS is a functional 5-point scale used to measure progress towards individual therapy goals. At start of each TC, 1 to 3 individual goals were defined for each subject by investigator and child's parents/guardians/caregivers prior to treatment. Outcome to reach each goal was rated on a 5-point scale ranging from -2 to +2 (-2: much less than expected outcome, -1: somewhat less than expected outcome, 0: expected outcome, +1: somewhat more than expected outcome, +2: much more than expected outcome). Higher score indicates a better outcome. A GAS T-score was calculated as: 50+(10∑_(i=1)\^n wi xi)/√(0.7∑_(i=1)\^n wi\^2 +0.3(∑_(i=1)\^n wi)\^2) where, xi = rating of ith goal post-baseline; wi = weight of ith goal, calculated as importance \* difficulty as defined at baseline; n = number of goals assessed at baseline and post-baseline. A GAS T-score of 50 indicates goals achieved as expected. Scores below 50 reflect under attainment of goals and scores above 50 reflect over attainment of goals.
Time frame: TC 1, Week 6.
Mean Change From Baseline to TC 1 Week 16 in MAS Score in the TC 1 PTMG
The MAS was used to assess muscle tone in the upper limb PTMG and consists of 6 grades: 0 (no increase in muscle tone), 1 (slight increase in muscle tone, manifested by a catch and release or by minimal resistance at the end of the ROM) when the affected part is moved in flexion or extension, 1+ (slight increase in muscle tone, manifested by a catch, followed by minimal resistance throughout the remainder (less than half) of the ROM), 2 (more marked increase in muscle tone), 3 (considerable increase in muscle tone) or 4 (affected part(s) rigid in flexion or extension). The original score '+1' was given a derived numeric score of '2' and the higher numeric scores were incremented by 1 so that the MAS score range was from 0 to 5 with higher scores indicating greater muscle tone. A negative change from baseline indicates a decrease in muscle tone.
Time frame: Baseline (TC 1, Day 1) and TC 1, Week 16.
Mean Change From Baseline to TC 1 Weeks 6 and 16 in MAS Score in the Elbow Flexors of the Study Limb
The MAS was used to assess muscle tone in the upper limb PTMG and consists of 6 grades: 0 (no increase in muscle tone), 1 (slight increase in muscle tone, manifested by a catch and release or by minimal resistance at the end of the ROM) when the affected part is moved in flexion or extension, 1+ (slight increase in muscle tone, manifested by a catch, followed by minimal resistance throughout the remainder (less than half) of the ROM), 2 (more marked increase in muscle tone), 3 (considerable increase in muscle tone) or 4 (affected part(s) rigid in flexion or extension). The original score '+1' was given a derived numeric score of '2' and the higher numeric scores were incremented by 1 so that the MAS score range was from 0 to 5 with higher scores indicating greater muscle tone. A negative change from baseline indicates a decrease in muscle tone. Data is presented for subjects injected in the elbow flexors.
Time frame: Baseline (TC 1, Day 1) and TC 1, Weeks 6 and 16.
Mean Change From Baseline to TC 1 Weeks 6 and 16 in MAS Score in the Wrist Flexors of the Study Limb
The MAS was used to assess muscle tone in the upper limb PTMG and consists of 6 grades: 0 (no increase in muscle tone), 1 (slight increase in muscle tone, manifested by a catch and release or by minimal resistance at the end of the ROM) when the affected part is moved in flexion or extension, 1+ (slight increase in muscle tone, manifested by a catch, followed by minimal resistance throughout the remainder (less than half) of the ROM), 2 (more marked increase in muscle tone), 3 (considerable increase in muscle tone) or 4 (affected part(s) rigid in flexion or extension). The original score '+1' was given a derived numeric score of '2' and the higher numeric scores were incremented by 1 so that the MAS score range was from 0 to 5 with higher scores indicating greater muscle tone. A negative change from baseline indicates a decrease in muscle tone. Data is presented for subjects injected in the wrist flexors.
Time frame: Baseline (TC 1, Day 1) and TC 1, Weeks 6 and 16.
Mean Change From Baseline to TC 1 Weeks 6 and 16 in MAS Score in the Finger Flexors of the Study Limb
The MAS was used to assess muscle tone in the upper limb PTMG and consists of 6 grades: 0 (no increase in muscle tone), 1 (slight increase in muscle tone, manifested by a catch and release or by minimal resistance at the end of the ROM) when the affected part is moved in flexion or extension, 1+ (slight increase in muscle tone, manifested by a catch, followed by minimal resistance throughout the remainder (less than half) of the ROM), 2 (more marked increase in muscle tone), 3 (considerable increase in muscle tone) or 4 (affected part(s) rigid in flexion or extension). The original score '+1' was given a derived numeric score of '2' and the higher numeric scores were incremented by 1 so that the MAS score range was from 0 to 5 with higher scores indicating greater muscle tone. A negative change from baseline indicates a decrease in muscle tone. Data is presented for subjects injected in the finger flexors.
Time frame: Baseline (TC 1, Day 1) and TC 1, Weeks 6 and 16.
Mean PGA Score at TC 1 Week 16
The PGA of treatment response was assessed by asking the investigator the following question: 'How would you rate the response to treatment in the subject's upper limb since the start of the study?'. Answers were on a 9-point rating scale (-4: markedly worse, -3: much worse, -2: worse, -1: slightly worse, 0: no change, +1: slightly improved, +2: improved, +3: much improved and +4: markedly improved). The mean scores for each treatment group at TC 1 Week 16 are presented.
Time frame: Baseline (TC 1, Day 1) and TC 1, Week 16.
Mean GAS Total Score at TC 1, Week 16
The GAS is a functional 5-point scale used to measure progress towards individual therapy goals. At start of each TC, 1 to 3 individual goals were defined for each subject by investigator and child's parents/guardians/caregivers prior to treatment. Outcome to reach each goal was rated on a 5-point scale ranging from -2 to +2 (-2: much less than expected outcome, -1: somewhat less than expected outcome, 0: expected outcome, +1: somewhat more than expected outcome, +2: much more than expected outcome). Higher score indicates a better outcome. A GAS T-score was calculated as: 50+(10∑_(i=1)\^n wi xi)/√(0.7∑_(i=1)\^n wi\^2 +0.3(∑_(i=1)\^n wi)\^2) where, xi = rating of ith goal post-baseline; wi = weight of ith goal, calculated as importance \* difficulty as defined at baseline; n = number of goals assessed at baseline and post-baseline. A GAS T-score of 50 indicates goals achieved as expected. Scores below 50 reflect under attainment of goals and scores above 50 reflect over attainment of goals.
Time frame: Baseline (TC 1, Day 1) and TC 1, Week 16.
Mean Change From Baseline to TC 1, Week 16 in the Paediatric Quality of Life (PedsQL) Scores
Parents/guardians completed questionnaires on their child's quality of life. The PedsQL parent inventory measured healthcare concepts for children/adolescents aged 2-18 years. The Generic Core Scales include physical, emotional, social and school aspects. The CP module was also completed. Scores were transformed on a scale from 0 to 100 with higher scores indicating a better quality of life. Mean changes from baseline to TC 1, Week 16 are presented for the General Core Scale and for the CP module. A positive change from baseline indicates an improvement in quality of life.
Time frame: Baseline (TC 1, Day 1) and TC 1, Week 16.
Male and female subjects aged between 2 and 17 years with upper limb spasticity due to cerebral palsy (CP) were recruited from April 2014 and the study completed in September 2018. Subjects could receive a maximum of 4 treatment cycles (TC) over a minimum of 1 year and maximum of 1 year and 9 months, with at least 16 weeks between each TC.
| Milestone | Dysport 2 U/kg | Dysport 8 U/kg | Dysport 16 U/kg |
|---|---|---|---|
| Started | 71 | 70 | 71 |
| Received treatment in tc 1 | 70 | 70 | 70 |
| Completed | 66 | 67 | 67 |
| Not completed | 5 | 3 | 4 |
| Withdrew: Adverse event | 2 | 0 | 0 |
| Withdrew: Consent withdrawn | 1 | 0 | 2 |
| Withdrew: Lost to follow-up | 1 | 0 | 0 |
| Withdrew: Other | 0 | 3 | 1 |
| Withdrew: Randomised but not treated | 1 | 0 | 1 |
| Milestone | Dysport 2 U/kg | Dysport 8 U/kg | Dysport 16 U/kg |
|---|---|---|---|
| Started | 0 | 88 | 90 |
| Completed | 0 | 83 | 87 |
| Not completed | 0 | 5 | 3 |
| Withdrew: Adverse event | 0 | 2 | 0 |
| Withdrew: Consent withdrawn | 0 | 1 | 1 |
| Withdrew: Other | 0 | 2 | 2 |
| Milestone | Dysport 2 U/kg | Dysport 8 U/kg | Dysport 16 U/kg |
|---|---|---|---|
| Started | 0 | 49 | 58 |
| Completed | 0 | 45 | 53 |
| Not completed | 0 | 4 | 5 |
| Withdrew: Adverse event | 0 | 1 | 0 |
| Withdrew: Consent withdrawn | 0 | 0 | 2 |
| Withdrew: Lost to follow-up | 0 | 1 | 0 |
| Withdrew: Other | 0 | 2 | 3 |
| Milestone | Dysport 2 U/kg | Dysport 8 U/kg | Dysport 16 U/kg |
|---|---|---|---|
| Started | 0 | 22 | 33 |
| Completed | 0 | 21 | 31 |
| Not completed | 0 | 1 | 2 |
| Withdrew: Consent withdrawn | 0 | 0 | 2 |
| Withdrew: Other | 0 | 1 | 0 |
The MAS was used to assess muscle tone in the upper limb PTMG and consists of 6 grades: 0 (no increase in muscle tone), 1 (slight increase in muscle tone, manifested by a catch and release or by minimal resistance at the end of the ROM) when the affected part is moved in flexion or extension, 1+ (slight increase in muscle tone, manifested by a catch, followed by minimal resistance throughout the remainder (less than half) of the ROM), 2 (more marked increase in muscle tone), 3 (considerable increase in muscle tone) or 4 (affected part(s) rigid in flexion or extension). The original score '+1' was given a derived numeric score of '2' and the higher numeric scores were incremented by 1 so that the MAS score range was from 0 to 5 with higher scores indicating greater muscle tone. A negative change from baseline indicates a decrease in muscle tone.
| score on a scale | Dysport 2 U/kg | Dysport 8 U/kg | Dysport 16 U/kg |
|---|---|---|---|
| Mean Change From Baseline to TC 1, Week 6 in MAS Score in the TC 1 PTMG | -1.5 ± 1.1 | -1.9 ± 1.0 | -2.2 ± 0.9 |
The PGA of treatment response was assessed by asking the investigator the following question: 'How would you rate the response to treatment in the subject's upper limb since the start of the study?'. Answers were on a 9-point rating scale (-4: markedly worse, -3: much worse, -2: worse, -1: slightly worse, 0: no change, +1: slightly improved, +2: improved, +3: much improved and +4: markedly improved). The mean scores for each treatment group at TC 1, Week 6 are presented.
| score on a scale | Dysport 2 U/kg | Dysport 8 U/kg | Dysport 16 U/kg |
|---|---|---|---|
| Mean Physician's Global Assessment (PGA) Score at TC 1, Week 6 | 1.7 ± 0.9 | 2.0 ± 0.9 | 2.0 ± 0.9 |
The GAS is a functional 5-point scale used to measure progress towards individual therapy goals. At start of each TC, 1 to 3 individual goals were defined for each subject by investigator and child's parents/guardians/caregivers prior to treatment. Outcome to reach each goal was rated on a 5-point scale ranging from -2 to +2 (-2: much less than expected outcome, -1: somewhat less than expected outcome, 0: expected outcome, +1: somewhat more than expected outcome, +2: much more than expected outcome). Higher score indicates a better outcome. A GAS T-score was calculated as: 50+(10∑_(i=1)\^n wi xi)/√(0.7∑_(i=1)\^n wi\^2 +0.3(∑_(i=1)\^n wi)\^2) where, xi = rating of ith goal post-baseline; wi = weight of ith goal, calculated as importance \* difficulty as defined at baseline; n = number of goals assessed at baseline and post-baseline. A GAS T-score of 50 indicates goals achieved as expected. Scores below 50 reflect under attainment of goals and scores above 50 reflect over attainment of goals.
| T-score | Dysport 2 U/kg | Dysport 8 U/kg | Dysport 16 U/kg |
|---|---|---|---|
| Mean Goal Attainment Scale (GAS) Total Score at TC 1, Week 6 | 51.3 ± 9.9 | 52.6 ± 10.1 | 52.0 ± 9.6 |
The MAS was used to assess muscle tone in the upper limb PTMG and consists of 6 grades: 0 (no increase in muscle tone), 1 (slight increase in muscle tone, manifested by a catch and release or by minimal resistance at the end of the ROM) when the affected part is moved in flexion or extension, 1+ (slight increase in muscle tone, manifested by a catch, followed by minimal resistance throughout the remainder (less than half) of the ROM), 2 (more marked increase in muscle tone), 3 (considerable increase in muscle tone) or 4 (affected part(s) rigid in flexion or extension). The original score '+1' was given a derived numeric score of '2' and the higher numeric scores were incremented by 1 so that the MAS score range was from 0 to 5 with higher scores indicating greater muscle tone. A negative change from baseline indicates a decrease in muscle tone.
| score on a scale | Dysport 2 U/kg | Dysport 8 U/kg | Dysport 16 U/kg |
|---|---|---|---|
| Mean Change From Baseline to TC 1 Week 16 in MAS Score in the TC 1 PTMG | -1.0 ± 1.0 | -1.4 ± 1.1 | -1.6 ± 1.2 |
The MAS was used to assess muscle tone in the upper limb PTMG and consists of 6 grades: 0 (no increase in muscle tone), 1 (slight increase in muscle tone, manifested by a catch and release or by minimal resistance at the end of the ROM) when the affected part is moved in flexion or extension, 1+ (slight increase in muscle tone, manifested by a catch, followed by minimal resistance throughout the remainder (less than half) of the ROM), 2 (more marked increase in muscle tone), 3 (considerable increase in muscle tone) or 4 (affected part(s) rigid in flexion or extension). The original score '+1' was given a derived numeric score of '2' and the higher numeric scores were incremented by 1 so that the MAS score range was from 0 to 5 with higher scores indicating greater muscle tone. A negative change from baseline indicates a decrease in muscle tone. Data is presented for subjects injected in the elbow flexors.
| score on a scale | Dysport 2 U/kg | Dysport 8 U/kg | Dysport 16 U/kg |
|---|---|---|---|
| Week 6 | -1.0 ± 1.1 | -1.7 ± 1.1 | -1.9 ± 1.2 |
| Week 16 | -0.6 ± 1.0 | -1.2 ± 1.2 | -1.3 ± 1.4 |
The MAS was used to assess muscle tone in the upper limb PTMG and consists of 6 grades: 0 (no increase in muscle tone), 1 (slight increase in muscle tone, manifested by a catch and release or by minimal resistance at the end of the ROM) when the affected part is moved in flexion or extension, 1+ (slight increase in muscle tone, manifested by a catch, followed by minimal resistance throughout the remainder (less than half) of the ROM), 2 (more marked increase in muscle tone), 3 (considerable increase in muscle tone) or 4 (affected part(s) rigid in flexion or extension). The original score '+1' was given a derived numeric score of '2' and the higher numeric scores were incremented by 1 so that the MAS score range was from 0 to 5 with higher scores indicating greater muscle tone. A negative change from baseline indicates a decrease in muscle tone. Data is presented for subjects injected in the wrist flexors.
| score on a scale | Dysport 2 U/kg | Dysport 8 U/kg | Dysport 16 U/kg |
|---|---|---|---|
| Week 6 | -1.3 ± 1.1 | -1.5 ± 1.2 | -1.7 ± 1.3 |
| Week 16 | -0.9 ± 1.2 | -1.0 ± 1.2 | -1.3 ± 1.2 |
The MAS was used to assess muscle tone in the upper limb PTMG and consists of 6 grades: 0 (no increase in muscle tone), 1 (slight increase in muscle tone, manifested by a catch and release or by minimal resistance at the end of the ROM) when the affected part is moved in flexion or extension, 1+ (slight increase in muscle tone, manifested by a catch, followed by minimal resistance throughout the remainder (less than half) of the ROM), 2 (more marked increase in muscle tone), 3 (considerable increase in muscle tone) or 4 (affected part(s) rigid in flexion or extension). The original score '+1' was given a derived numeric score of '2' and the higher numeric scores were incremented by 1 so that the MAS score range was from 0 to 5 with higher scores indicating greater muscle tone. A negative change from baseline indicates a decrease in muscle tone. Data is presented for subjects injected in the finger flexors.
| score on a scale | Dysport 2 U/kg | Dysport 8 U/kg | Dysport 16 U/kg |
|---|---|---|---|
| Week 6 | -0.8 ± 0.9 | -1.8 ± 1.1 | -1.9 ± 1.0 |
| Week 16 | -0.8 ± 1.3 | -1.3 ± 0.8 | -1.8 ± 1.0 |
The PGA of treatment response was assessed by asking the investigator the following question: 'How would you rate the response to treatment in the subject's upper limb since the start of the study?'. Answers were on a 9-point rating scale (-4: markedly worse, -3: much worse, -2: worse, -1: slightly worse, 0: no change, +1: slightly improved, +2: improved, +3: much improved and +4: markedly improved). The mean scores for each treatment group at TC 1 Week 16 are presented.
| score on a scale | Dysport 2 U/kg | Dysport 8 U/kg | Dysport 16 U/kg |
|---|---|---|---|
| Mean PGA Score at TC 1 Week 16 | 1.7 ± 1.0 | 1.6 ± 1.1 | 1.9 ± 1.2 |
The GAS is a functional 5-point scale used to measure progress towards individual therapy goals. At start of each TC, 1 to 3 individual goals were defined for each subject by investigator and child's parents/guardians/caregivers prior to treatment. Outcome to reach each goal was rated on a 5-point scale ranging from -2 to +2 (-2: much less than expected outcome, -1: somewhat less than expected outcome, 0: expected outcome, +1: somewhat more than expected outcome, +2: much more than expected outcome). Higher score indicates a better outcome. A GAS T-score was calculated as: 50+(10∑_(i=1)\^n wi xi)/√(0.7∑_(i=1)\^n wi\^2 +0.3(∑_(i=1)\^n wi)\^2) where, xi = rating of ith goal post-baseline; wi = weight of ith goal, calculated as importance \* difficulty as defined at baseline; n = number of goals assessed at baseline and post-baseline. A GAS T-score of 50 indicates goals achieved as expected. Scores below 50 reflect under attainment of goals and scores above 50 reflect over attainment of goals.
| T-score | Dysport 2 U/kg | Dysport 8 U/kg | Dysport 16 U/kg |
|---|---|---|---|
| Mean GAS Total Score at TC 1, Week 16 | 54.7 ± 9.8 | 54.2 ± 9.7 | 55.1 ± 10.1 |
Parents/guardians completed questionnaires on their child's quality of life. The PedsQL parent inventory measured healthcare concepts for children/adolescents aged 2-18 years. The Generic Core Scales include physical, emotional, social and school aspects. The CP module was also completed. Scores were transformed on a scale from 0 to 100 with higher scores indicating a better quality of life. Mean changes from baseline to TC 1, Week 16 are presented for the General Core Scale and for the CP module. A positive change from baseline indicates an improvement in quality of life.
| score on a scale | Dysport 2 U/kg | Dysport 8 U/kg | Dysport 16 U/kg |
|---|---|---|---|
| Generic Core Scale | 3.4 ± 9.7 | 3.4 ± 17.1 | 2.0 ± 12.0 |
| CP Module | 4.8 ± 16.8 | 2.1 ± 14.9 | 2.8 ± 16.2 |
Collected over Treatment emergent adverse events (TEAEs) were collected from the first injection of study treatment up to the end of TC 4 (up to 21 months).. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| TC1: Dysport 2 U/kg | 0/70 (0%) | 4/70 (5.7%) | 15/70 (21.4%) |
| TC 1: Dysport 8 U/kg | 0/70 (0%) | 2/70 (2.9%) | 23/70 (32.9%) |
| TC 1: Dysport 16 U/kg | 0/70 (0%) | 2/70 (2.9%) | 19/70 (27.1%) |
| TC 2: Dysport 8 U/kg | 0/88 (0%) | 6/88 (6.8%) | 18/88 (20.5%) |
| TC 2: Dysport 16 U/kg | 0/90 (0%) | 1/90 (1.1%) | 17/90 (18.9%) |
| TC 3: Dysport 8 U/kg | 0/49 (0%) | 3/49 (6.1%) | 14/49 (28.6%) |
| TC 3: Dysport 16 U/kg | 0/58 (0%) | 2/58 (3.4%) | 10/58 (17.2%) |
| TC 4: Dysport 8 U/kg | 0/22 (0%) | 2/22 (9.1%) | 14/22 (63.6%) |
| TC 4: Dysport 16 U/kg | 0/33 (0%) | 1/33 (3%) | 5/33 (15.2%) |
| Event | TC1: Dysport 2 U/kg | TC 1: Dysport 8 U/kg | TC 1: Dysport 16 U/kg | TC 2: Dysport 8 U/kg | TC 2: Dysport 16 U/kg | TC 3: Dysport 8 U/kg | TC 3: Dysport 16 U/kg | TC 4: Dysport 8 U/kg | TC 4: Dysport 16 U/kg |
|---|---|---|---|---|---|---|---|---|---|
| VomitingGastrointestinal disorders | 0/70 | 0/70 | 0/70 | 0/88 | 0/90 | 0/49 | 1/58 | 1/22 | 0/33 |
| Cerebrovascular accidentNervous system disorders | 0/70 | 0/70 | 0/70 | 0/88 | 0/90 | 0/49 | 0/58 | 1/22 | 0/33 |
| HeadacheNervous system disorders | 0/70 | 0/70 | 0/70 | 0/88 | 0/90 | 0/49 | 0/58 | 1/22 | 0/33 |
| Abdominal painGastrointestinal disorders | 0/70 | 0/70 | 0/70 | 0/88 | 0/90 | 0/49 | 0/58 | 1/22 | 0/33 |
| Electrocardiogram ST segment elevationInvestigations | 0/70 | 0/70 | 0/70 | 0/88 | 0/90 | 0/49 | 0/58 | 1/22 | 0/33 |
| DehydrationMetabolism and nutrition disorders | 0/70 | 0/70 | 0/70 | 0/88 | 0/90 | 0/49 | 0/58 | 1/22 | 0/33 |
| Scoliosis surgerySurgical and medical procedures | 0/70 | 0/70 | 0/70 | 0/88 | 0/90 | 0/49 | 0/58 | 1/22 | 0/33 |
| EpilepsyNervous system disorders | 0/70 | 0/70 | 1/70 | 0/88 | 0/90 | 0/49 | 2/58 | 0/22 | 0/33 |
| Hand fractureInjury, poisoning and procedural complications | 0/70 | 0/70 | 0/70 | 0/88 | 0/90 | 0/49 | 0/58 | 0/22 | 1/33 |
| SeizureNervous system disorders | 1/70 | 0/70 | 0/70 | 2/88 | 0/90 | 1/49 | 0/58 | 0/22 | 0/33 |
| Event | TC1: Dysport 2 U/kg | TC 1: Dysport 8 U/kg | TC 1: Dysport 16 U/kg | TC 2: Dysport 8 U/kg | TC 2: Dysport 16 U/kg | TC 3: Dysport 8 U/kg | TC 3: Dysport 16 U/kg | TC 4: Dysport 8 U/kg | TC 4: Dysport 16 U/kg |
|---|---|---|---|---|---|---|---|---|---|
| Viral upper respiratory tract infectionInfections and infestations | 10/70 | 6/70 | 6/70 | 7/88 | 6/90 | 6/49 | 5/58 | 0/22 | 3/33 |
| Upper respiratory tract infectionInfections and infestations | 5/70 | 6/70 | 8/70 | 5/88 | 4/90 | 0/49 | 0/58 | 1/22 | 0/33 |
| PharyngitisInfections and infestations | 6/70 | 3/70 | 4/70 | 0/88 | 0/90 | 4/49 | 2/58 | 0/22 | 0/33 |
| PyrexiaGeneral disorders | 2/70 | 4/70 | 2/70 | 7/88 | 1/90 | 0/49 | 0/58 | 1/22 | 0/33 |
| RhinorrhoeaRespiratory, thoracic and mediastinal disorders | 0/70 | 5/70 | 1/70 | 1/88 | 1/90 | 0/49 | 0/58 | 1/22 | 0/33 |
| VomitingGastrointestinal disorders | 0/70 | 0/70 | 0/70 | 0/88 | 0/90 | 3/49 | 2/58 | 0/22 | 0/33 |
| SinusitisInfections and infestations | 0/70 | 0/70 | 0/70 | 0/88 | 0/90 | 1/49 | 3/58 | 1/22 | 2/33 |
| Muscular weaknessMusculoskeletal and connective tissue disorders | 1/70 | 3/70 | 4/70 | 0/88 | 5/90 | 0/49 | 0/58 | 0/22 | 0/33 |
| HeadacheNervous system disorders | 0/70 | 4/70 | 2/70 | 0/88 | 0/90 | 0/49 | 0/58 | 0/22 | 0/33 |
| Ear infectionInfections and infestations | 0/70 | 0/70 | 0/70 | 0/88 | 0/90 | 0/49 | 0/58 | 1/22 | 1/33 |
Baseline characteristics are presented for the safety population, which consisted of all randomised subjects who received at least 1 injection of the study treatment.
| Age, Continuous(Years) | Dysport 2 U/kg | Dysport 8 U/kg | Dysport 16 U/kg | Total |
|---|---|---|---|---|
| Mean | 8.91 ± 4.55 | 8.97 ± 4.27 | 9.17 ± 4.30 | 9.02 ± 4.36 |
| Age, Customized(Participants) | Dysport 2 U/kg | Dysport 8 U/kg | Dysport 16 U/kg | Total |
|---|---|---|---|---|
| 2 - 9 Years | 40 | 40 | 40 | 120 |
| 10 - 17 Years | 30 | 30 | 30 | 90 |
| Sex: Female, Male(Participants) | Dysport 2 U/kg | Dysport 8 U/kg | Dysport 16 U/kg | Total |
|---|---|---|---|---|
| Female | 32 | 24 | 28 | 84 |
| Male | 38 | 46 | 42 | 126 |
| Race/Ethnicity, Customized(Participants) | Dysport 2 U/kg | Dysport 8 U/kg | Dysport 16 U/kg | Total |
|---|---|---|---|---|
| Asian | 2 | 1 | 0 | 3 |
| Black or African American | 7 | 6 | 3 | 16 |
| White | 49 | 54 | 54 | 157 |
| American Indian or Alaska Native | 0 | 1 | 0 | 1 |
| Multiple | 12 | 8 | 13 | 33 |
| Race/Ethnicity, Customized(Participants) | Dysport 2 U/kg | Dysport 8 U/kg | Dysport 16 U/kg | Total |
|---|---|---|---|---|
| Hispanic or Latino | 16 | 13 | 15 | 44 |
| Not Hispanic or Latino | 54 | 57 | 55 | 166 |
| BTX Status(Participants) | Dysport 2 U/kg | Dysport 8 U/kg | Dysport 16 U/kg | Total |
|---|---|---|---|---|
| BTX naïve | 25 | 23 | 24 | 72 |
| BTX non-naïve | 45 | 47 | 46 | 138 |
| Baseline MAS Score in the PTMG(score on a scale) | Dysport 2 U/kg | Dysport 8 U/kg | Dysport 16 U/kg | Total |
|---|---|---|---|---|
| Mean | 3.1 ± 0.3 | 3.1 ± 0.3 | 3.1 ± 0.5 | 3.1 ± 0.4 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: Yes — Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized, and study documents will be redacted to protect the privacy of study participants. Any requests should be submitted to www.vivli.org for assessment by an independent scientific review board.
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Ipsen