Lower Limb Spasticity Due to Cerebral PalsyPhase 3NCT01893411
Dose-response Study of Efficacy and Safety of Botulinum Toxin Type A to Treat Spasticity of the Leg(s) in Cerebral Palsy
A Phase 3 study testing IncobotulinumtoxinA (16 Units per kg body weight), IncobotulinumtoxinA (12 Units per kg body weight) and IncobotulinumtoxinA (4 Units per kg body weight) in people with Lower Limb Spasticity Due to Cerebral Palsy, run by Merz Pharmaceuticals GmbH at 53 sites in 14 countries.
Trial summary
The purpose of this study is to determine whether injections of Botulinum toxin type A into muscles of the leg(s) are effective in treating children/adolescents (age 2-17 years) with increased muscle tension/uncontrollable muscle stiffness (spasticity) due to cerebral palsy. Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Lower Limb Spasticity Due to Cerebral Palsy
- What is being tested?
- IncobotulinumtoxinA (16 Units per kg body weight), IncobotulinumtoxinA (12 Units per kg body weight) and IncobotulinumtoxinA (4 Units per kg body weight)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 311 participants
- Where
- 53 sitesin 14 countries
- Study dates
- Jun 2013to May 2016
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Female or male subject of 2 to 17 years of age (inclusive).
- Uni- or bilateral cerebral palsy with clinical need for uni- or bilateral LL injections with BoNT for the treatment of spasticity.
- Ashworth Scale [AS] score ≥2 in plantar flexors (at least unilaterally).
- Clinical need for a total dose of 16 U/kg BW NT 201 (maximum of 400 U) for the treatment of LL spasticity according to the clinical judgment of the investigator.
Who cannot join
- Fixed contracture defined as severe restriction of the range of joint movement on passive stretch or predominant forms of muscle hypertonia other than spasticity (e.g., dystonia) in the target limb(s).
- Surgery on pes equinus on side(s) intended to be treated with BoNT injections in this study within 12 months prior to Screening Visit (V1), in the screening period or planned for the time of participation in this study.
- Hip flexion requiring BoNT injection.
Trial locations and contacts
53 sites in 14 countries.
53 sites in 14 countries
Poland7 sites
Merz Investigational Site #048089
Bialystok, 15-274
Merz Investigational Site #048063
Gdansk, 80-389
Merz Investigational Site #048059
Krakow, 30-539
Merz Investigational Site #048084
Lublin, 20-828
Merz Investigational Site #048072
Luboń, 62-030
Merz Investigational Site #048075
Sandomierz, 27-600
Merz Investigational Site #048061
Warsaw, 02-315
Russia6 sites
Merz Investigational Site #007014
Kazan', 420097
Merz Investigational Site #007015
Khabarovsk, 680038
Merz Investigational Site #007018
Novosibirsk, 630091
Merz Investigational Site #007017
Saint Petersburg, 194100
Merz Investigational Site #007013
Smolensk, 214029
Merz Investigational Site #007019
Stavropol, 355029
Germany5 sites
Merz Investigational Site #049328
Bochum, 44791
Merz Investigational Site #049330
Marburg, 35043
Merz Investigational Site #049327
Munich, 80337
Merz Investigational Site #049329
Münster, 48149
Merz Investigational Site #049326
Vogtareuth, 83569
France4 sites
Merz Investigational Site #033056
Amiens, 80054
Merz Investigational Site #033052
Bron, 69677
Merz Investigational Site #033054
La Tronche, 38700
Merz Investigational Site #033055
Palavas-les-Flots, 34250
Slovakia4 sites
Merz Investigational Site #421003
Banská Bystrica, 97409
Merz Investigational Site #421008
Bratislava, 82108
Merz Investigational Site #421006
Krompachy, 05342
Merz Investigational Site #421004
Levoča, 05401
South Korea4 sites
Merz Investigational Site #082019
Goyang, 410-773
Merz Investigational Site #082021
Incheon, 400-711
Merz Investigational Site #082018
Seongnam-si, 463-712
Merz Investigational Site #082020
Seoul, 135-710
Spain4 sites
Merz Investigational Site #034031
Granada, 18013
Merz Investigational Site #034032
Manresa, 08243
Merz Investigational Site #034030
Seville, 41013
Merz Investigational Site #034026
Seville, 41071
Ukraine4 sites
Merz Investigational Site #380001
Dnipropetrovsk, 49027
Merz Investigational Site #380005
Kharkiv, 61068
Merz Investigational Site #380002
Kiev, 04209
Merz Investigational Site #380003
Odesa, 65012
Israel3 sites
Merz Investigational Site #972003
Jerusalem, 91240
Merz Investigational Site #972001
Tel Aviv, 6423906
Merz Investigational Site #972002
Tel Aviv, 6423906
Romania3 sites
Merz Investigational Site #040003
Bucharest, 041408
Merz Investigational Site #040001
Bucharest, 041914
Merz Investigational Site #040002
Iași, 700309
Turkey (Türkiye)3 sites
Merz Investigational Site #090005
Elâzığ, 23119
Merz Investigational Site #090003
Izmir, 35100
Merz Investigational Site #090002
İzmit, 41380
Austria2 sites
Merz Investigational Site #043037
Graz, 8036
Merz Investigational Site #043036
Vienna, 1100
Czechia2 sites
Merz Investigational Site #420029
Brno, 65691
Merz Investigational Site #420028
Olomouc, 77520
Estonia2 sites
Merz Investigational Site #372001
Tallinn, 13419
Merz Investigational Site #372002
Tartu, 51014
About this study
Official title: Prospective, Multicenter, Randomized, Double-blind, Parallel-group, Dose-response Study of Three Doses Xeomin® (incobotulinumtoxinA, NT 201) for the Treatment of Lower Limb Spasticity in Children and Adolescents (Age 2 - 17 Years) With Cerebral Palsy
The purpose of this study is to determine whether injections of Botulinum toxin type A into muscles of the leg(s) are effective in treating children/adolescents (age 2-17 years) with increased muscle tension/uncontrollable muscle stiffness (spasticity) due to cerebral palsy.
- Being tested
- IncobotulinumtoxinA (16 Units per kg body weight), IncobotulinumtoxinA (12 Units per kg body weight), IncobotulinumtoxinA (4 Units per kg body weight)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IncobotulinumtoxinA (16 Units per kg body weight)Drug
Also called Xeomin, NT 201, Botulinum toxin type A (150 kiloDalton), free from complexing proteins
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 volume 8.0 mL; 400 units; Mode of administration: intramuscular injection into spastic muscles.
IncobotulinumtoxinA (12 Units per kg body weight)Drug
Also called Xeomin, NT 201, Botulinum toxin type A (150 kiloDalton), free from complexing proteins
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 volume 8.0 mL; 300 units; Mode of administration: intramuscular injection into spastic muscles.
IncobotulinumtoxinA (4 Units per kg body weight)Drug
Also called Xeomin, NT 201, Botulinum toxin type A (150 kiloDalton), free from complexing proteins
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 volume 8.0 mL; 100 units; Mode of administration: intramuscular injection into spastic muscles.
| Study group | Role | What they receive |
|---|---|---|
| 16 Units per kg body weight incobotulinumtoxinA (Xeomin) | Experimental | Not described Receives: Drug: IncobotulinumtoxinA (16 Units per kg body weight) |
| 12 Units per kg body weight incobotulinumtoxinA (Xeomin) | Experimental | Not described Receives: Drug: IncobotulinumtoxinA (12 Units per kg body weight) |
| 4 Units per kg body weight incobotulinumtoxinA (Xeomin) | Experimental | Not described Receives: Drug: IncobotulinumtoxinA (4 Units per kg body weight) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in the Ashworth Scale (AS) Score of Plantar Flexors of the Primary Body Side at Day 29 (Week 4) of the First Injection Cycle (1st IC)
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 primary efficacy analysis i.e. "primary body side" was decided by investigator at screening and was kept throughout the entire study. For participants with unilateral treatment, the treated body side was kept throughout the entire study.
Values represent least square (LS) mean differences between baseline and Week 4 resulting from MMRM (Mixed Model Repeated Measurement) models comparing high versus low and in a second step mid versus low dose groups, respectively. Values for the low group may differ slightly depending on the comparison and are therefore provided separately for each comparison.
Measured: Baseline, Week 4
Co-primary Variable: Investigator's Global Impression of Change of Plantar Flexor Spasticity Scale (GICS-PF) of the Primary Body Side at Day 29 (Week 4) of the First Injection Cycle
This variable is classified as co-primary to satisfy a Food and Drug Administration (FDA) request. The GICS-PF scale is a 7-Point Likert Scale for the assessment of the functional change due to treatment of plantar flexor spasticity only. Ranges from +3 (very much improved function) to -3 (very much worse function). For participants with bilateral pes equinus, the body side for primary efficacy analysis i.e. "primary body side" was decided by investigator at screening and was kept throughout the entire study. For participants with unilateral treatment, the treated body side was kept throughout the entire study.
Values represent least square (LS) mean differences between baseline and Week 4 resulting from ANCOVA models comparing high versus low and in a second step mid versus low dose groups, respectively. Values for the low group may differ slightly depending on the comparison and are therefore provided separately for each comparison.
Measured: Baseline, Week 4
Secondary outcomes (17)
Change From Baseline in the AS Score of Plantar Flexors of the Nonprimary Body Side in Participants With Bilateral Treatment at Day 29 (Week 4) of the First (1st) and Second Injection Cycle (2nd IC)
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).
Values represent least square (LS) mean differences between baseline and the respective week (w) resulting from MMRM (Mixed Model Repeated Measurement) models comparing high versus low and in a second step mid versus low dose groups, respectively. Values for the low group may differ slightly depending on the comparison and are therefore provided separately for each comparison.
Measured: Baseline, Week 4 of 1st IC and Week 16-40 of 2nd IC
Change From Baseline in the AS Score of Plantar Flexors of the Primary Body Side at Day 29 (Week 4) of the Second 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 primary efficacy analysis i.e. "primary body side" was decided by investigator at screening and was kept throughout the entire study. For participants with unilateral treatment, the treated body side was kept throughout the entire study.
Values represent least square (LS) mean differences between baseline and Week 16-40 resulting from MMRM (Mixed Model Repeated Measurement) models comparing high versus low and in a second step mid versus low dose groups, respectively. Values for the low group may differ slightly depending on the comparison and are therefore provided separately for each comparison.
Measured: Baseline to Week 4 of 2nd IC (Week 16-40)
Changes From Baseline in AS Score of Plantar Flexors of the Primary Body Side at Day 57 (Week 8) and Day 85 (Week 12) of the First and of the Second 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 primary efficacy analysis i.e. "primary body side" was decided by investigator at screening and was kept throughout the entire study. For participants with unilateral treatment, the treated body side was kept throughout the entire study.
Values represent least square (LS) mean differences between baseline and the respective week (w) resulting from MMRM (Mixed Model Repeated Measurement) models comparing high versus low and in a second step mid versus low dose groups, respectively. Values for the low group may differ slightly depending on the comparison and are therefore provided separately for each comparison.
Measured: Baseline to Week 8 and 12 of 1st IC and 2nd IC (Week 20-44 and 24-48)
Changes From Baseline in AS Score of Knee Flexors or Thigh Adductors in Participants With Unilateral Treatment at Day 29 (Week 4) of the First and of the Second 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).
Values represent least square (LS) mean differences between baseline and the respective week (w) resulting from MMRM (Mixed Model Repeated Measurement) models comparing high versus low and in a second step mid versus low dose groups, respectively. Values for the low group may differ slightly depending on the comparison and are therefore provided separately for each comparison.
KF = Knee Flexors; TA = Thigh Adductors; w = week.
Measured: Baseline to Week 4 of 1st IC and 2nd IC (Week 16-40)
Changes From Baseline in Modified Tardieu Scale [MTS] of Plantar Flexors of Primary Body Side at Day 29 (Week 4), Day 57 (Week 8), and Day 85 (Week 12) of the First and of the Second Injection Cycle
The Modified Tardieu Scale (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, i.e. the dynamic tone component of the examined muscle(s). Decreases of (R2-R1) represent reductions in the dynamic component of spasticity, i.e. improvement of dynamic muscle spasticity.
Values represent least square (LS) mean differences between baseline and the respective week (w) resulting from ANCOVA models comparing high versus low and in a second step mid versus low dose groups, respectively. Values for the low group may differ slightly depending on the model used for comparison and are therefore provided separately for each comparison.
Measured: Baseline to Week 4, 8, and 12 of 1st IC and 2nd IC (Week 16-40, 20-44 and 24-48)
Investigator's, Child's/Adolescent's, and Parent's/Caregiver's Global Impression of Change Scale [GICS] at Day 29 (Week 4) of the First and Second Injection Cycle
The Global Impression of Change Scales (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 are 7-Point Likert Scales ranging from +3 (very much improved function) to -3 (very much worse function).
Values represent least square (LS) mean differences between baseline and the respective week (w) resulting from MMRM (Mixed Model Repeated Measurement) models comparing high versus low and in a second step mid versus low dose groups, respectively. Values for the low group may differ slightly depending on the comparison and are therefore provided separately for each comparison.
Measured: Baseline to Week 4 of 1st IC and 2nd IC (Week 16-40)
Investigator's Global Impression of Change of GICS-Plantar-Flexor of Primary Body Side at Day 29 (Week 4) of the First and Second 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 are 7-Point Likert Scales ranging from +3 (very much improved function) to -3 (very much worse function). For participants with bilateral pes equinus, the body side for primary efficacy analysis i.e. "primary body side" was decided by investigator at screening and was kept throughout the entire study. For participants with unilateral treatment, the treated body side was kept throughout the entire study.
Values represent least square (LS) mean differences between baseline and the respective week (w) resulting from ANCOVA models comparing high versus low and in a second step mid versus low dose groups, respectively. Values for the low group may differ slightly depending on the comparison and are therefore provided separately for each comparison.
Measured: Baseline to Week 4 of 1st IC and 2nd IC (Week 16-40)
Changes From Baseline in Gross Motor Function Measure [GMFM]-66 Score at the End of First Injection Cycle and at the End of Study Visit
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).
Values represent least square (LS) mean differences between baseline and the respective week (w) resulting from ANCOVA models comparing high versus low and in a second step mid versus low dose groups, respectively. Values for the low group may differ slightly depending on the comparison and are therefore provided separately for each comparison.
Measured: Baseline to Week 12-36 of 1st IC and 2nd IC (End of study = Week 24-72)
Change in Scores of Pain Intensity (From Participants) and Pain Frequency (From Parent/Caregiver) to All Post Baseline Visits of the First and of the Second Injection Cycle
The QPS is a patient-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 'Questionnaire on Pain caused by Spasticity [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).
Values represent least square (LS) mean differences between baseline and the respective week (w) resulting from ANCOVA models comparing high versus low and in a second step mid versus low dose groups, respectively. Values for the low group may differ slightly depending on the comparison and are therefore provided separately for each comparison.
Measured: Baseline to Week 4, 8, and 12 of 1st IC and 2nd IC (Week 16-40, 20-44 and 24-48)
Time to Reinjection for Each of the Three Dose Groups for the First and Second Injection Cycle
Measured: Baseline up to Week 24-72
Occurrence of Treatment Emergent Adverse Events (TEAEs) Overall and Per Injection Cycle
Treatment-emergent Adverse Events (TEAEs) are events observed from the time point of first injection until end of study visit (week 24-72). Values reported here refer to the number of participants affected.
Measured: Up to End of study visit (Week 24-72)
Occurrence of Participants With TEAEs of Special Interest (TEAESIs) Overall and Per Injection Cycle
Adverse Events (AE's) occurring after treatment that were thought to possibly indicate toxin spread throughout the trial conduct are defined as AE's of Special Interests. Values reported here refer to the number of participants affected.
Measured: Up to End of study visit (Week 24-72)
Occurrence of Serious TEAEs (TESAEs) Overall and Per Injection Cycle
Treatment-emergent Serious Adverse Events (TESAEs) are events observed from the time point of first injection until end of study visit (week 24-72). Values reported here refer to the number of participants affected.
Measured: Up to End of study visit (Week 24-72)
Occurrence of TEAEs Related to Treatment as Assessed by the Investigator Overall and Per Injection Cycle
Treatment-emergent Adverse Events (TEAEs) are events observed from the time point of first injection until end of study visit (week 24-72). Values reported here refer to the number of participants affected.
Measured: Up to End of study visit (Week 24-72)
Occurrence of TEAEs by Worst Intensity Overall and Per Injection Cycle
Treatment-emergent Adverse Events (TEAEs) are events observed from the time point of first injection until end of study visit (week 24-72). Values reported here refer to the number of participants affected.
Measured: Up to End of study visit (Week 24-72)
Occurrence of TEAEs by Final Outcome Overall and Per Injection Cycle
Treatment-emergent Adverse Events (TEAEs) are events observed from the time point of first injection until end of study visit (week 24-72). Values reported here refer to the number of participants affected.
Measured: Up to End of study visit (Week 24-72)
Occurrence of TEAEs Leading to Discontinuation Overall and Per Injection Cycle
Treatment-emergent Adverse Events (TEASs) are events observed from the time point of first injection until end of study visit (week 24-72). Values reported here refer to the number of participants affected.
Measured: Up to End of study visit (Week 24-72)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jul 2013 (estimated)
- Study startJun 2013
- Primary completionAug 2015
- Study completionMay 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Lower Limb Spasticity Due to Cerebral PalsyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 311 participants (actual)
Sponsor and investigators
- Lead sponsor
- Merz Pharmaceuticals GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Merz Medical Expert, Study director, Merz Pharmaceuticals GmbH
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01893411
- Sponsor's protocol ID
- MRZ60201_3070_1
- Other IDs
- 2012-005054-30
- Status last verified
- Aug 2017
- First submitted
- 2 Jul 2013
- Last updated
- 5 Aug 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Heinen F, Kanovsky P, Schroeder AS, Chambers HG, Dabrowski E, Geister TL, Hanschmann A, Martinez-Torres FJ, Pulte I, Banach M, Gaebler-Spira D. IncobotulinumtoxinA for the treatment of lower-limb spasticity in children and adolescents with cerebral palsy: A phase 3 study. J Pediatr Rehabil Med. 2021;14(2):183-197. doi: 10.3233/PRM-210040.
PubMed 34092664Berweck 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.
PubMed 36136523
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective, Multicenter, Randomized, Double-blind, Parallel-group, Dose-response Study of Three Doses Xeomin® (incobotulinumtoxinA, NT 201) for the Treatment of Lower Limb Spasticity in Children and Adolescents (Age 2 - 17 Years) With Cerebral Palsy
- ClinicalTrials.gov ID
- NCT01893411
- Acronym / sponsor ID
- MRZ60201_3070_1
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 311 participants (actual)
- Lead sponsor
- Merz Pharmaceuticals GmbHIndustry (pharma, biotech, devices)
- Study start
- Jun 2013
- Primary completion
- Aug 2015 (actual)
- Study completion
- May 2016 (actual)
- First posted
- 9 Jul 2013 (estimated)
- Results
- Posted 12 May 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine whether injections of Botulinum toxin type A into muscles of the leg(s) are effective in treating children/adolescents (age 2-17 years) with increased muscle tension/uncontrollable muscle stiffness (spasticity) due to cerebral palsy.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 16 Units per kg body weight incobotulinumtoxinA (Xeomin) | Experimental | Drug: IncobotulinumtoxinA (16 Units per kg body weight) |
| 12 Units per kg body weight incobotulinumtoxinA (Xeomin) | Experimental | Drug: IncobotulinumtoxinA (12 Units per kg body weight) |
| 4 Units per kg body weight incobotulinumtoxinA (Xeomin) | Experimental | Drug: IncobotulinumtoxinA (4 Units per kg body weight) |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Female or male subject of 2 to 17 years of age (inclusive).
- Uni- or bilateral cerebral palsy with clinical need for uni- or bilateral LL injections with BoNT for the treatment of spasticity.
- Ashworth Scale [AS] score ≥2 in plantar flexors (at least unilaterally).
- Clinical need for a total dose of 16 U/kg BW NT 201 (maximum of 400 U) for the treatment of LL spasticity according to the clinical judgment of the investigator.
Key exclusion criteria (3 listed)
- Fixed contracture defined as severe restriction of the range of joint movement on passive stretch or predominant forms of muscle hypertonia other than spasticity (e.g., dystonia) in the target limb(s).
- Surgery on pes equinus on side(s) intended to be treated with BoNT injections in this study within 12 months prior to Screening Visit (V1), in the screening period or planned for the time of participation in this study.
- Hip flexion requiring BoNT injection.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| IncobotulinumtoxinA (16 Units per kg body weight)Also: Xeomin, NT 201, Botulinum toxin type A (150 kiloDalton), free from complexing proteinsActive 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 volume 8.0 mL; 400 units; Mode of administration: intramuscular injection into spastic muscles. | Drug | 16 Units per kg body weight incobotulinumtoxinA (Xeomin) |
| IncobotulinumtoxinA (12 Units per kg body weight)Also: Xeomin, NT 201, Botulinum toxin type A (150 kiloDalton), free from complexing proteinsActive 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 volume 8.0 mL; 300 units; Mode of administration: intramuscular injection into spastic muscles. | Drug | 12 Units per kg body weight incobotulinumtoxinA (Xeomin) |
| IncobotulinumtoxinA (4 Units per kg body weight)Also: Xeomin, NT 201, Botulinum toxin type A (150 kiloDalton), free from complexing proteinsActive 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 volume 8.0 mL; 100 units; Mode of administration: intramuscular injection into spastic muscles. | Drug | 4 Units per kg body weight incobotulinumtoxinA (Xeomin) |
Primary outcome
Change From Baseline in the Ashworth Scale (AS) Score of Plantar Flexors of the Primary Body Side at Day 29 (Week 4) of the First Injection Cycle (1st IC)
Time frame: Baseline, Week 4
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 primary efficacy analysis i.e. "primary body side" was decided by investigator at screening and was kept throughout the entire study. For participants with unilateral treatment, the treated body side was kept throughout the entire study. Values represent least square (LS) mean differences between baseline and Week 4 resulting from MMRM (Mixed Model Repeated Measurement) models comparing high versus low and in a second step mid versus low dose groups, respectively. Values for the low group may differ slightly depending on the comparison and are therefore provided separately for each comparison.
Co-primary Variable: Investigator's Global Impression of Change of Plantar Flexor Spasticity Scale (GICS-PF) of the Primary Body Side at Day 29 (Week 4) of the First Injection Cycle
Time frame: Baseline, Week 4
This variable is classified as co-primary to satisfy a Food and Drug Administration (FDA) request. The GICS-PF scale is a 7-Point Likert Scale for the assessment of the functional change due to treatment of plantar flexor spasticity only. Ranges from +3 (very much improved function) to -3 (very much worse function). For participants with bilateral pes equinus, the body side for primary efficacy analysis i.e. "primary body side" was decided by investigator at screening and was kept throughout the entire study. For participants with unilateral treatment, the treated body side was kept throughout the entire study. Values represent least square (LS) mean differences between baseline and Week 4 resulting from ANCOVA models comparing high versus low and in a second step mid versus low dose groups, respectively. Values for the low group may differ slightly depending on the comparison and are therefore provided separately for each comparison.
17 secondary outcomes
Change From Baseline in the AS Score of Plantar Flexors of the Nonprimary Body Side in Participants With Bilateral Treatment at Day 29 (Week 4) of the First (1st) and Second Injection Cycle (2nd IC)
Time frame: Baseline, Week 4 of 1st IC and Week 16-40 of 2nd IC
Change From Baseline in the AS Score of Plantar Flexors of the Primary Body Side at Day 29 (Week 4) of the Second Injection Cycle
Time frame: Baseline to Week 4 of 2nd IC (Week 16-40)
Changes From Baseline in AS Score of Plantar Flexors of the Primary Body Side at Day 57 (Week 8) and Day 85 (Week 12) of the First and of the Second Injection Cycle
Time frame: Baseline to Week 8 and 12 of 1st IC and 2nd IC (Week 20-44 and 24-48)
Changes From Baseline in AS Score of Knee Flexors or Thigh Adductors in Participants With Unilateral Treatment at Day 29 (Week 4) of the First and of the Second Injection Cycle
Time frame: Baseline to Week 4 of 1st IC and 2nd IC (Week 16-40)
Changes From Baseline in Modified Tardieu Scale [MTS] of Plantar Flexors of Primary Body Side at Day 29 (Week 4), Day 57 (Week 8), and Day 85 (Week 12) of the First and of the Second Injection Cycle
Time frame: Baseline to Week 4, 8, and 12 of 1st IC and 2nd IC (Week 16-40, 20-44 and 24-48)
Investigator's, Child's/Adolescent's, and Parent's/Caregiver's Global Impression of Change Scale [GICS] at Day 29 (Week 4) of the First and Second Injection Cycle
Time frame: Baseline to Week 4 of 1st IC and 2nd IC (Week 16-40)
Investigator's Global Impression of Change of GICS-Plantar-Flexor of Primary Body Side at Day 29 (Week 4) of the First and Second Injection Cycle
Time frame: Baseline to Week 4 of 1st IC and 2nd IC (Week 16-40)
Changes From Baseline in Gross Motor Function Measure [GMFM]-66 Score at the End of First Injection Cycle and at the End of Study Visit
Time frame: Baseline to Week 12-36 of 1st IC and 2nd IC (End of study = Week 24-72)
Change in Scores of Pain Intensity (From Participants) and Pain Frequency (From Parent/Caregiver) to All Post Baseline Visits of the First and of the Second Injection Cycle
Time frame: Baseline to Week 4, 8, and 12 of 1st IC and 2nd IC (Week 16-40, 20-44 and 24-48)
Time to Reinjection for Each of the Three Dose Groups for the First and Second Injection Cycle
Time frame: Baseline up to Week 24-72
Occurrence of Treatment Emergent Adverse Events (TEAEs) Overall and Per Injection Cycle
Time frame: Up to End of study visit (Week 24-72)
Occurrence of Participants With TEAEs of Special Interest (TEAESIs) Overall and Per Injection Cycle
Time frame: Up to End of study visit (Week 24-72)
Occurrence of Serious TEAEs (TESAEs) Overall and Per Injection Cycle
Time frame: Up to End of study visit (Week 24-72)
Occurrence of TEAEs Related to Treatment as Assessed by the Investigator Overall and Per Injection Cycle
Time frame: Up to End of study visit (Week 24-72)
Occurrence of TEAEs by Worst Intensity Overall and Per Injection Cycle
Time frame: Up to End of study visit (Week 24-72)
Occurrence of TEAEs by Final Outcome Overall and Per Injection Cycle
Time frame: Up to End of study visit (Week 24-72)
Occurrence of TEAEs Leading to Discontinuation Overall and Per Injection Cycle
Time frame: Up to End of study visit (Week 24-72)
Results and safety
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Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Aug 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 34092664Heinen F, Kanovsky P, Schroeder AS, Chambers HG, Dabrowski E, Geister TL, Hanschmann A, Martinez-Torres FJ, Pulte I, Banach M, Gaebler-Spira D. IncobotulinumtoxinA for the treatment of lower-limb spasticity in children and adolescents with cerebral palsy: A phase 3 study. J Pediatr Rehabil Med. 2021;14(2):183-197. doi: 10.3233/PRM-210040.
- Derived from this study: PubMed 36136523Berweck 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.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Merz Medical Expert study director
Merz Pharmaceuticals GmbH
53 sites in 14 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing IncobotulinumtoxinA (16 Units per kg body weight), IncobotulinumtoxinA (12 Units per kg body weight) and IncobotulinumtoxinA (4 Units per kg body weight) in people with Lower Limb Spasticity Due to Cerebral Palsy, run by Merz Pharmaceuticals GmbH at 53 sites in 14 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 53 sites in Poland, Russia, Germany, France, Slovakia and 9 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 12 May 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in the Ashworth Scale (AS) Score of Plantar Flexors of the Primary Body Side at Day 29 (Week 4) of the First Injection Cycle (1st IC) (time frame: Baseline, Week 4). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01893411, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Aug 2021.