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| ID | Type | Description | Link |
|---|---|---|---|
| 2012-005055-17 | EudraCT Number |
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The purpose of this study is to determine whether injections of Botulinum toxin type A into muscles of the leg(s) or of leg(s) and one arm are safe in treating children/adolescents (age 2-17 years) long-term with increased muscle tension/uncontrollable muscle stiffness (spasticity) due to cerebral palsy.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 16-20 Units per kg body weight incobotulinumtoxinA | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| IncobotulinumtoxinA (16-20 Units per kg body weight) | Drug | Active ingredient: Clostridium Botulinum neurotoxin type A free from complexing proteins. Solution for injection prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl); Total dose per injection cycle: up to 500 units; Mode of administration: intramuscular injection into spastic muscles. |
| Measure | Description | Time Frame |
|---|---|---|
| Occurrence of Treatment Emergent Adverse Events (TEAEs) Overall and Per Injection Cycle | TEAEs are events observed from the time point of first injection until end of study visit (Week 50-66). Values reported here refer to the number of participants affected. | From the timepoint of first injection up to end of study visit (Week 50-66) |
| Occurrence of Treatment Emergent Adverse Events of Special Interest (TEAESI) Overall and Per Injection Cycle | TEAEs occurring after treatment that were thought to possibly indicate toxin spread throughout the trial conduct are defined as TEAESI. Values reported here refer to the number of participants affected. | From the timepoint of first injection until end of study visit (Week 50-66) |
| Occurrence of Treatment-emergent Serious Adverse Events (TESAEs) Overall and Per Injection Cycle | TESAEs are events observed from the time point of first injection until end of study visit (Week 50-66). Values reported here refer to the number of participants affected. | From the timepoint of first injection until end of study visit (Week 50-66) |
| Measure | Description | Time Frame |
|---|---|---|
| Investigator's Global Assessment of Tolerability at Day 99 (Week 14) of Each Injection Cycle | The investigator's global assessment of tolerability was assessed on a 4-point ordinal scale where 1 = very good, 2 = good, 3 = moderate, and 4 = poor. Results for Day 99 (Week 14) of 4th injection cycles were collected at the end of study visit. | Day 99 (Week 14) of 1st, 2nd, 3rd and 4th injection cycle |
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Inclusion Criteria:
Main clinical inclusion criteria for completers of study MRZ60201_3070_1:
Unilateral treatment of LL spasticity with 8 U/kg BW NT 201 (maximum of 200 U) into pes equinus and need for additional 8 U/kg BW NT 201 (maximum of 200 U) for treatment of clinical pattern flexed knee or adducted thigh (ipsilateral) or bilateral treatment of LL spasticity with 8 U/kg BW NT 201 (maximum of 200 U) into pes equinus on each side.
No treatment of other clinical patterns is allowed.
Main clinical inclusion criteria for subjects who did not participate in MRZ60201_3070_1:
Female or male subject of 2 to 17 years age (inclusive).
Uni- or bilateral CP with clinical need for BoNT injection to treat limb spasticity.
AS score ≥ 2 in plantar flexors (at least unilaterally).
Clinical need according to the clinical judgment of the investigator in one out of four treatment combinations:
Exclusion Criteria:
Exclusion Criteria for subjects who completed MRZ60201_3070_1:
Exclusion Criteria for subjects who did not participate in MRZ60201_3070_1:
Fixed contracture defined as severe restriction of the range of joint movement on passive stretch in the target clinical pattern(s) or predominant forms of muscle hypertonia other than spasticity (e.g., dystonia) in the target limb(s).
Surgery in the pes equinus on side(s) intended to treat with BoNT injections within 12 months prior to Screening Visit (V1), within the screening period or planned for the time of participation in this study.
Hip flexion requiring BoNT injection.
Limitation of hip abduction to less than 40° or pre-diagnosed migrational percentage greater than 30.
Vaccination within 2 weeks prior to Screening Visit (V1) and/or within the screening period.
Non-resolved fractures of the treated limb.
Ventilator dependency.
Severe neurological diagnosis and comorbidity outside the spectrum of cerebral palsy.
Pure dyskinetic CP or mixed CP with predominantly dyskinetic movements.
Treatment with BoNT (other than study drug in this study) for any body region within 14 weeks prior to Screening Visit (V1), within the screening period and/or intended to be administered during the study period.
Treatment with phenol or alcohol of any muscle within 6 months prior to Screening Visit (V1), within the screening period, and/or intended to be administered during the study period.
Treatment with
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| Name | Affiliation | Role |
|---|---|---|
| Merz Medical Expert | Merz Pharmaceuticals GmbH | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Merz Investigational Site #043036 | Vienna | 1100 | Austria | |||
| Merz Investigational Site #420029 |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36136523 | Derived | Berweck S, Banach M, Gaebler-Spira D, Chambers HG, Schroeder AS, Geister TL, Althaus M, Hanschmann A, Vacchelli M, Bonfert MV, Heinen F, Dabrowski E. Safety Profile and Lack of Immunogenicity of IncobotulinumtoxinA in Pediatric Spasticity and Sialorrhea: A Pooled Analysis. Toxins (Basel). 2022 Aug 25;14(9):585. doi: 10.3390/toxins14090585. | |
| 34957963 |
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A total of 391 participants were screened, of which 370 participants were enrolled and treated. Of these, 124 participants were recruited from the lead-in study (62, 29 and 33 participants from the IncobotulinumtoxinA high, mid and low dose group respectively of study MRZ60201_3070_1 [2012-005054-30]) and 246 participants were newly recruited.
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| ID | Title | Description |
|---|---|---|
| FG000 | 16 - 20 U/kg Body Weight IncobotulinumtoxinA (Xeomin) | Participants received total doses of 16 to 20 unit per kilogram (U/kg) body weight of IncobotulinumtoxinA (Xeomin) with a maximum of 400 to 500 units per injection treatment via intramuscular injection into spastic muscles on Day 1 of 4 treatment cycles (12 to 16 weeks treatment per each cycle). The higher dose could only be administered to participants with gross motor function classification system expanded and revised (GMFCS-E&R) levels I to III. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Changes in AS Score of Left and Right Plantar Flexors (PF) From Baseline to All Other Visits, From Day 1 of Each Injection Cycle to Day 29 (Week 4), Day 57 (Week 8, 1st Injection Cycle Only) and Day 99 (Week 14) of the Respective Injection Cycle | The Ashworth Scale (AS) is a well-known and commonly used scale in clinical trials with spasticity. In spastic muscles the resistance to passive movement is assessed. It is a 5-point scale that ranges from 0 (= no increase in tone) to 4 (=limb rigid in flexion or extension). For participants with bilateral pes equinus, the body side for efficacy analysis that is, "primary body side" was decided by investigator at screening and was kept throughout the entire study. V3 = Week 4 of 1st Injection Cycle; V4 = Week 8 of 1st Injection Cycle; V5 = Day 1 of 2nd Injection Cycle; V6= Week 4 of 2nd Injection Cycle; V7 = Day 1 of 3rd Injection Cycle; V8 = Week 4 of 3rd Injection Cycle; V9 = Day 1 of 4th Injection Cycle; V10 = Week 4 of 4th Injection Cycle; V11= Week 14th of 4th Injection Cycle = end of study visit. | Baseline (Day 1, Visit [V] 2) to all other visits (V3, V4, V5, V6, V7, V8, V9, V10, and V11); From Day 1 of Each Injection Cycle to Day 29 (Week 4), Day 57 (Week 8, 1st Injection Cycle only) and Day 99 (Week 14) of the respective Injection Cycle |
| Investigator's, Child's/Adolescent's, and Parent's/Caregiver's Global Impression of Change Scale (GICS) at Day 29 (Week 4) of Each Injection Cycle | The GICS are global outcomes to assess the impression of change due to treatment. GICS were assessed by the investigator, by the participant (if feasible) and by parents'/caregiver (if applicable). GICS is 7-Point Likert Scale ranging from +3 (very much improved function) to -3 (very much worse function). | Day 29 (Week 4) of 1st, 2nd, 3rd and 4th injection cycle |
| Investigator's Global Impression of Change of Plantar Flexor Spasticity Scale (GICS-PF) of Left and Right PF at Day 29 (Week 4) of Each Injection Cycle | The GICS are global outcomes to assess the impression of change due to treatment. GICS were assessed by the investigator, by the participant (if feasible) and by parents'/caregiver (if applicable). GICS is a 7-Point Likert Scale ranging from +3 (very much improved function) to -3 (very much worse function). For participants with bilateral pes equinus, the body side for efficacy analysis that is "primary body side" was decided by investigator at screening and was kept throughout the entire study. | Day 29 (Week 4) of 1st, 2nd, 3rd and 4th injection cycle |
| Changes in Modified Tardieu Scale (MTS) of Left and Right PF From Baseline to All Other Visits, From Day 1 of Each Injection Cycle (IC) to Day 29 (Week 4), Day 57 (Week 8, 1st IC Only) and Day 99 (Week 14) of the Respective Injection Cycle | The MTS assesses spastic muscle tone by subtraction of two angles measured at different conditions of passive muscle stretch. R2 is the angle of passive range of motion with a passive movement at slow speed. R1 is the angle where a "catch-and-release" or clonus can be triggered at the fastest possible speed. Score values represent the measured (R2-R1) difference, that is, the dynamic tone component of the examined muscle(s). Decreases of (R2-R1) represent reductions in the dynamic component of spasticity, that is, improvement of dynamic muscle spasticity. V3 = Week 4 of 1st IC; V4 = Week 8 of 1st IC; V5 = Day 1 of 2nd IC; V6 = Week 4 of 2nd IC; V7 = Day 1 of 3rd IC; V8 = Week 4 of 3rd IC; V9 = Day 1 of 4th IC; V10 = Week 4 of 4th IC; V11 = Week 14 of 4th IC = end of study visit. | Baseline (Day 1, Visit [V] 2) to all other visits (V3, V4, V5, V6, V7, V8, V9, V10, and V11); From Day 1 of Each IC to Day 29 (Week 4), Day 57 (Week 8, 1st IC cycle only) and Day 99 (Week 14) of the respective IC |
| Change in Scores of Pain Intensity (From Participants) and Frequency (From Parent/Caregiver) From Baseline to All Visits, From Day 1 of Each IC to Day 29 (Week 4), Day 57 (Week 8, 1st IC Cycle Only) and Day 99 (Week 14) of Respective Injection | The questionnaire on pain caused by Spasticity (QPS) is a participant-reported outcome for children and adolescents (2-17 years) with cerebral palsy on spasticity-related pain. Pain intensity (from participants) and pain frequency (from parent/caregiver) to be assessed with QPS. The QPS total score for pain intensity ranges from 0 ('No Hurt') to 10 ('Hurt Worst'). The QPS total score for the observed pain frequency ranges from 0 (Never) to 4 (Always). V3 = Week 4 of 1st IC; V4 = Week 8 of 1st IC; V5= Day 1 of 2nd IC; V6 = Week 4 of 2nd IC; V7 = Day 1 of 3rd IC; V8 = Week 4 of 3rd IC; V9 = Day 1 of 4th IC; V10 = Week 4 of 4th IC; V11 = Week 14 of 4th IC = end of study visit. | Baseline (Day 1, Visit [V] 2) to all other visits (V3, V4, V5, V6, V7, V8, V9, V10, and V11); From Day 1 of Each IC to Day 29 (Week 4), Day 57 (Week 8, 1st IC cycle only) and Day 99 (Week 14) of the respective IC |
| Changes in Gross Motor Function Measure (GMFM)-66 Score From Baseline to All Injection Visits and End of Study | The GMFM-66 is a standardized observational 66-item instrument designed and validated to measure change in gross motor function over time in participants with cerebral palsy. Score values represent the total GMFM-66 score. Total GMFM scores range from 0 (worst) to 100 (best). | Baseline to Day 1 of 2nd (V5), 3rd (V7), 4th (V9) IC and End of study (Week 44-68) (V11) |
| Brno |
| 65691 |
| Czechia |
| Merz Investigational Site #420028 | Olomouc | 77520 | Czechia |
| Merz Investigational Site #372001 | Tallinn | 13419 | Estonia |
| Merz Investigational Site #372002 | Tartu | 51014 | Estonia |
| Merz Investigational Site #049328 | Bochum | 44791 | Germany |
| Merz Investigational Site #049327 | Munich | 80337 | Germany |
| Merz Investigational Site #049329 | Münster | 48149 | Germany |
| Merz Investigational Site #049326 | Vogtareuth | 83569 | Germany |
| Merz Investigational Site #972003 | Jerusalem | 91240 | Israel |
| Merz Investigational Site #972001 | Tel Aviv | 6423906 | Israel |
| Merz Investigational Site #972002 | Tel Aviv | 6423906 | Israel |
| Merz Investigational Site #048089 | Bialystok | 15-274 | Poland |
| Merz Investigational Site #048063 | Gdansk | 80-389 | Poland |
| Merz Investigational Site #048059 | Krakow | 30-539 | Poland |
| Merz Investigational Site #048084 | Lublin | 20-828 | Poland |
| Merz Investigational Site #048072 | Luboń | 62-030 | Poland |
| Merz Investigational Site #048075 | Sandomierz | 27-600 | Poland |
| Merz Investigational Site #048061 | Warsaw | 02-315 | Poland |
| Merz Investigational Site #040003 | Bucharest | 041408 | Romania |
| Merz Investigational Site #040001 | Bucharest | 041914 | Romania |
| Merz Investigational Site #040002 | Iași | 700309 | Romania |
| Merz Investigational Site #007014 | Kazan' | 420097 | Russia |
| Merz Investigational Site #007015 | Khabarovsk | 680038 | Russia |
| Merz Investigational Site #007018 | Novosibirsk | 630091 | Russia |
| Merz Investigational Site #007017 | Saint Petersburg | 194100 | Russia |
| Merz Investigational Site #007013 | Smolensk | 214029 | Russia |
| Merz Investigational Site #007019 | Stavropol | 355029 | Russia |
| Merz Investigational Site #421003 | Banská Bystrica | 97409 | Slovakia |
| Merz Investigational Site #421008 | Bratislava | 82108 | Slovakia |
| Merz Investigational Site #421006 | Krompachy | 05342 | Slovakia |
| Merz Investigational Site #421004 | Levoča | 05401 | Slovakia |
| Merz Investigational Site #082019 | Goyang | 410-773 | South Korea |
| Merz Investigational Site #082021 | Incheon | 400-711 | South Korea |
| Merz Investigational Site #082018 | Seongnam-si | 463-712 | South Korea |
| Merz Investigational Site #082020 | Seoul | 135-710 | South Korea |
| Merz Investigational Site #090005 | Elâzığ | 23119 | Turkey (Türkiye) |
| Merz Investigational Site #090003 | Izmir | 35100 | Turkey (Türkiye) |
| Merz Investigational Site #090002 | İzmit | 41380 | Turkey (Türkiye) |
| Merz Investigational Site #380001 | Dnipropetrovsk | 49027 | Ukraine |
| Merz Investigational Site #380005 | Kharkiv | 61068 | Ukraine |
| Merz Investigational Site #380002 | Kiev | 04209 | Ukraine |
| Merz Investigational Site #380003 | Odesa | 65012 | Ukraine |
| Kanovsky P, Heinen F, Schroeder AS, Chambers HG, Dabrowski E, Geister TL, Hanschmann A, Martinez-Torres FJ, Pulte I, Banach M, Gaebler-Spira D. Safety and efficacy of repeat long-term incobotulinumtoxinA treatment for lower limb or combined upper/lower limb spasticity in children with cerebral palsy. J Pediatr Rehabil Med. 2022;15(1):113-127. doi: 10.3233/PRM-210041. |
| COMPLETED |
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| NOT COMPLETED |
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The safety evaluation set (SES) was the subset of all participants treated with investigational product (IP) at least once.
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| ID | Title | Description |
|---|---|---|
| BG000 | 16 - 20 U/kg Body Weight IncobotulinumtoxinA (Xeomin) | Participants received total doses of 16 to 20 unit per kilogram (U/kg) body weight of IncobotulinumtoxinA (Xeomin) with a maximum of 400 to 500 units per injection treatment via intramuscular injection into spastic muscles on Day 1 of 4 treatment cycles (12 to 16 weeks treatment per each cycle). The higher dose could only be administered to participants with GMFCS-E&R levels I to III. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Age, Customized | Count of Participants | Participants |
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| Sex: Female, Male | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Occurrence of Treatment Emergent Adverse Events (TEAEs) Overall and Per Injection Cycle | TEAEs are events observed from the time point of first injection until end of study visit (Week 50-66). Values reported here refer to the number of participants affected. | The SES was the subset of all participants treated with IP at least once. | Posted | Number | participants | From the timepoint of first injection up to end of study visit (Week 50-66) |
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| Primary | Occurrence of Treatment Emergent Adverse Events of Special Interest (TEAESI) Overall and Per Injection Cycle | TEAEs occurring after treatment that were thought to possibly indicate toxin spread throughout the trial conduct are defined as TEAESI. Values reported here refer to the number of participants affected. | The SES was the subset of all participants treated with IP at least once. | Posted | Number | participants | From the timepoint of first injection until end of study visit (Week 50-66) |
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| Primary | Occurrence of Treatment-emergent Serious Adverse Events (TESAEs) Overall and Per Injection Cycle | TESAEs are events observed from the time point of first injection until end of study visit (Week 50-66). Values reported here refer to the number of participants affected. | The SES was the subset of all participants treated with IP at least once. | Posted | Number | participants | From the timepoint of first injection until end of study visit (Week 50-66) |
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| Secondary | Investigator's Global Assessment of Tolerability at Day 99 (Week 14) of Each Injection Cycle | The investigator's global assessment of tolerability was assessed on a 4-point ordinal scale where 1 = very good, 2 = good, 3 = moderate, and 4 = poor. Results for Day 99 (Week 14) of 4th injection cycles were collected at the end of study visit. | The SES was the subset of all participants treated with IP at least once. | Posted | Number | participants | Day 99 (Week 14) of 1st, 2nd, 3rd and 4th injection cycle |
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| Secondary | Changes in AS Score of Left and Right Plantar Flexors (PF) From Baseline to All Other Visits, From Day 1 of Each Injection Cycle to Day 29 (Week 4), Day 57 (Week 8, 1st Injection Cycle Only) and Day 99 (Week 14) of the Respective Injection Cycle | The Ashworth Scale (AS) is a well-known and commonly used scale in clinical trials with spasticity. In spastic muscles the resistance to passive movement is assessed. It is a 5-point scale that ranges from 0 (= no increase in tone) to 4 (=limb rigid in flexion or extension). For participants with bilateral pes equinus, the body side for efficacy analysis that is, "primary body side" was decided by investigator at screening and was kept throughout the entire study. V3 = Week 4 of 1st Injection Cycle; V4 = Week 8 of 1st Injection Cycle; V5 = Day 1 of 2nd Injection Cycle; V6= Week 4 of 2nd Injection Cycle; V7 = Day 1 of 3rd Injection Cycle; V8 = Week 4 of 3rd Injection Cycle; V9 = Day 1 of 4th Injection Cycle; V10 = Week 4 of 4th Injection Cycle; V11= Week 14th of 4th Injection Cycle = end of study visit. | The full analysis set (FAS) was the subset of participants in the SES for whom at least a baseline value (Day 1 of the first cycle, Visit 2) of AS score of PF was available. For participants from lead-in study at least one post-baseline value was available. | Posted | Mean | Standard Deviation | units on a scale | Baseline (Day 1, Visit [V] 2) to all other visits (V3, V4, V5, V6, V7, V8, V9, V10, and V11); From Day 1 of Each Injection Cycle to Day 29 (Week 4), Day 57 (Week 8, 1st Injection Cycle only) and Day 99 (Week 14) of the respective Injection Cycle |
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| Secondary | Investigator's, Child's/Adolescent's, and Parent's/Caregiver's Global Impression of Change Scale (GICS) at Day 29 (Week 4) of Each Injection Cycle | The GICS are global outcomes to assess the impression of change due to treatment. GICS were assessed by the investigator, by the participant (if feasible) and by parents'/caregiver (if applicable). GICS is 7-Point Likert Scale ranging from +3 (very much improved function) to -3 (very much worse function). | The FAS was the subset of participants in the SES for whom at least a baseline value (Day 1 of the first cycle, Visit 2) of AS score of PF was available. For participants from lead-in study at least one post-baseline value was available. | Posted | Mean | Standard Deviation | units on a scale | Day 29 (Week 4) of 1st, 2nd, 3rd and 4th injection cycle |
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| Secondary | Investigator's Global Impression of Change of Plantar Flexor Spasticity Scale (GICS-PF) of Left and Right PF at Day 29 (Week 4) of Each Injection Cycle | The GICS are global outcomes to assess the impression of change due to treatment. GICS were assessed by the investigator, by the participant (if feasible) and by parents'/caregiver (if applicable). GICS is a 7-Point Likert Scale ranging from +3 (very much improved function) to -3 (very much worse function). For participants with bilateral pes equinus, the body side for efficacy analysis that is "primary body side" was decided by investigator at screening and was kept throughout the entire study. | The FAS was the subset of participants in the SES for whom at least a baseline value (Day 1 of the first cycle, Visit 2) of AS score of PF was available. For participants from lead-in study at least one post-baseline value was available. | Posted | Mean | Standard Deviation | units on a scale | Day 29 (Week 4) of 1st, 2nd, 3rd and 4th injection cycle |
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| Secondary | Changes in Modified Tardieu Scale (MTS) of Left and Right PF From Baseline to All Other Visits, From Day 1 of Each Injection Cycle (IC) to Day 29 (Week 4), Day 57 (Week 8, 1st IC Only) and Day 99 (Week 14) of the Respective Injection Cycle | The MTS assesses spastic muscle tone by subtraction of two angles measured at different conditions of passive muscle stretch. R2 is the angle of passive range of motion with a passive movement at slow speed. R1 is the angle where a "catch-and-release" or clonus can be triggered at the fastest possible speed. Score values represent the measured (R2-R1) difference, that is, the dynamic tone component of the examined muscle(s). Decreases of (R2-R1) represent reductions in the dynamic component of spasticity, that is, improvement of dynamic muscle spasticity. V3 = Week 4 of 1st IC; V4 = Week 8 of 1st IC; V5 = Day 1 of 2nd IC; V6 = Week 4 of 2nd IC; V7 = Day 1 of 3rd IC; V8 = Week 4 of 3rd IC; V9 = Day 1 of 4th IC; V10 = Week 4 of 4th IC; V11 = Week 14 of 4th IC = end of study visit. | The FAS was the subset of participants in the SES for whom at least a baseline value (Day 1 of the first cycle, Visit 2) of AS score of PF was available. For participants from lead-in study at least one post-baseline value was available. | Posted | Mean | Standard Deviation | Degrees | Baseline (Day 1, Visit [V] 2) to all other visits (V3, V4, V5, V6, V7, V8, V9, V10, and V11); From Day 1 of Each IC to Day 29 (Week 4), Day 57 (Week 8, 1st IC cycle only) and Day 99 (Week 14) of the respective IC |
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| Secondary | Change in Scores of Pain Intensity (From Participants) and Frequency (From Parent/Caregiver) From Baseline to All Visits, From Day 1 of Each IC to Day 29 (Week 4), Day 57 (Week 8, 1st IC Cycle Only) and Day 99 (Week 14) of Respective Injection | The questionnaire on pain caused by Spasticity (QPS) is a participant-reported outcome for children and adolescents (2-17 years) with cerebral palsy on spasticity-related pain. Pain intensity (from participants) and pain frequency (from parent/caregiver) to be assessed with QPS. The QPS total score for pain intensity ranges from 0 ('No Hurt') to 10 ('Hurt Worst'). The QPS total score for the observed pain frequency ranges from 0 (Never) to 4 (Always). V3 = Week 4 of 1st IC; V4 = Week 8 of 1st IC; V5= Day 1 of 2nd IC; V6 = Week 4 of 2nd IC; V7 = Day 1 of 3rd IC; V8 = Week 4 of 3rd IC; V9 = Day 1 of 4th IC; V10 = Week 4 of 4th IC; V11 = Week 14 of 4th IC = end of study visit. | The FAS was the subset of participants in the SES for whom at least a baseline value (Day 1 of the first cycle, Visit 2) of AS score of PF was available. For participants from lead-in study at least one post-baseline value was available. | Posted | Mean | Standard Deviation | units on a scale | Baseline (Day 1, Visit [V] 2) to all other visits (V3, V4, V5, V6, V7, V8, V9, V10, and V11); From Day 1 of Each IC to Day 29 (Week 4), Day 57 (Week 8, 1st IC cycle only) and Day 99 (Week 14) of the respective IC |
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| Secondary | Changes in Gross Motor Function Measure (GMFM)-66 Score From Baseline to All Injection Visits and End of Study | The GMFM-66 is a standardized observational 66-item instrument designed and validated to measure change in gross motor function over time in participants with cerebral palsy. Score values represent the total GMFM-66 score. Total GMFM scores range from 0 (worst) to 100 (best). | The FAS was the subset of participants in the SES for whom at least a baseline value (Day 1 of the first cycle, Visit 2) of AS score of PF was available. For participants from lead-in study at least one post-baseline value was available. Therefore all participants from lead-in study were included in FAS. | Posted | Mean | Standard Deviation | units on a scale | Baseline to Day 1 of 2nd (V5), 3rd (V7), 4th (V9) IC and End of study (Week 44-68) (V11) |
|
|
From the time point of first injection up to end of study visit (Week 50-66)
Adverse Events were collected systematically at each visit by the investigator.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 16 - 20 U/kg Body Weight IncobotulinumtoxinA (Xeomin) | Participants received total doses of 16 to 20 unit per kilogram (U/kg) body weight of IncobotulinumtoxinA (Xeomin) with a maximum of 400 to 500 units per injection treatment via intramuscular injection into spastic muscles on Day 1 of 4 treatment cycles (12 to 16 weeks treatment per each cycle). The higher dose could only be administered to participants with GMFCS-E&R levels I to III. | 0 | 370 | 16 | 370 | 20 | 370 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Tenotomy | Surgical and medical procedures | MedDRA (19.0) | Systematic Assessment |
| |
| Cast application | Surgical and medical procedures | MedDRA (19.0) | Systematic Assessment |
| |
| Arrhythmia | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Epilepsy | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Clonic convulsion | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Febrile convulsion | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Adenoiditis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Adenovirus infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Croup infectious | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Enteritis infectious | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Respiratory tract infection viral | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Hemagglutinin Type 1 and Neuraminidase Type 1 (H1N1) influenza | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nasopharyngitis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
|
Publication of study information usually requires agreement with the sponsor. In case of justified doubts by the sponsor, the INVESTIGATOR will consider these doubts in the publication as long as the scientific neutrality is not affected.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Public Disclosure Manager | Merz Pharmaceuticals GmbH | +49 69 1503 1 | clinicaltrials@merz.de |
| ID | Term |
|---|---|
| C545476 | incobotulinumtoxinA |
| D019274 | Botulinum Toxins, Type A |
| ID | Term |
|---|---|
| D001905 | Botulinum Toxins |
| D008666 | Metalloendopeptidases |
| D010450 | Endopeptidases |
| D010447 | Peptide Hydrolases |
| D006867 | Hydrolases |
| D004798 | Enzymes |
| D045762 | Enzymes and Coenzymes |
| D045726 | Metalloproteases |
| D001426 | Bacterial Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D001427 | Bacterial Toxins |
| D014118 | Toxins, Biological |
| D001685 | Biological Factors |
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