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| ID | Type | Description | Link |
|---|---|---|---|
| 2009-017709-12 | EudraCT Number |
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The purpose of this research study is to determine whether Dysport® is effective in the treatment of increased stiffness of the calf muscles and to evaluate the safety of this treatment in children with Cerebral Palsy. In addition this study will also check whether Dysport® can lessen the pain caused by spasticity and improve the child's wellbeing.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Dysport 10 U/Kg | Experimental | 10 U/Kg per lower limb. Either one or both lower limbs can be treated. Total volume injected, 2ml per leg. |
|
| Dysport 15 U/Kg | Experimental | 15 U/Kg per lower limb. Either one or both lower limbs can be treated. Total volume injected, 2ml per leg. |
|
| Placebo | Placebo Comparator | Total volume to be injected per lower limb - 2ml. Either one or both lower limbs can be treated. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Botulinum toxin type A | Biological | I.M. (in the muscle) injection on day 1 of a single treatment cycle. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in MAS Score in the Gastrocnemius-soleus Complex (GSC) at the Ankle Joint of the (Most) Affected Lower Limb | The MAS is a 6-point scale which measures the intensity of muscle tone by measuring the resistance of the muscle to passive lengthening or stretching. Investigator will grade muscle tone in the GSC from 0 (no increase in tone) to 4 (affected parts rigid in flexion or extension). | Change from baseline to Week 4 |
| Measure | Description | Time Frame |
|---|---|---|
| Physician's Global Assessment (PGA) of the Treatment Response. | PGA Scale of the Treatment Response: Global assessment of treatment response assessed by asking the Investigator the following question: "how would you rate the response to treatment in the subject's lower limb(s) since the last injection?" Answers will be made 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, +4: markedly improved). |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Ipsen Study Director | Ipsen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Children's Hospital | Aurora | Colorado | 80045 | United States | ||
| Rehabilitation Institute of Chicago |
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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Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and EU or after the primary manuscript describing the results has been accepted for publication, whichever is later.
Further details on Ipsen's sharing criteria, eligible studies and process for sharing are available here (https://vivli.org/members/ourmembers/).
Screening details:
A total of 241 subjects were randomised to the study. The safety population included 239 subjects who were treated. Out of the 239 treated subjects, 4 subjects were excluded from the Intent-to-Treat (ITT) population because no MAS score was obtained at the baseline visit and/or at Week 4.
Each centre that participated in the study was expected to randomise between five and 25 subjects. It was planned to have at least two participating centres per country.
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| ID | Title | Description |
|---|---|---|
| FG000 | Dysport 10 U/Kg | 10 U/Kg per lower limb. Either one or both lower limbs can be treated. Total volume injected, 2ml per leg. Botulinum type A toxin (Dysport®): I.M. (in the muscle) injection on day 1 of a single treatment cycle. |
| FG001 | Dysport 15 U/Kg |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Randomised Population |
|
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| Placebo | Drug | I.M. injection on day 1 of a single treatment cycle. |
|
| Week 4 |
| Goal Attainment Scale (GAS) Score | GAS is a functional scale used to measure progress towards individual therapy goals. Individual goals defined for each subject by the physician, and the child's parents (caregiver) where applicable, prior to treatment. Post-baseline, the GAS for each goal rated using a defined scale (-2: Much less than expected outcome, -1: somewhat less than expected outcome, 0: expected outcome, 1: somewhat more than expected outcome, and 2: Much more than expected outcome). | Week 4 |
| Chicago |
| Illinois |
| 60611 |
| United States |
| Children's Hospital New Orleans | New Orleans | Louisiana | 70118 | United States |
| Children's Hospital of Michigan | Detroit | Michigan | 48202 | United States |
| Gillette Children's Speciality Healthcare | Saint Paul | Minnesota | 55101 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Shriner's Hospital for Children | Portland | Oregon | 97210 | United States |
| Texas Scottish Rite - Hospital for Children | Dallas | Texas | 75219 | United States |
| Club De Leones Cruz Del Sur Rehabilitation Corporation, Punta Arenas | Punta Arenas | Chile |
| Dr Roberto Del Rio Hospital | Santiago | Chile |
| Neurorehabilitation Laboratory, Pontifical Catholic University | Santiago | Chile |
| CHU Jean Minjoz | Besançon | France |
| Hospital San José Celaya | Celaya | Mexico |
| Centro de Rehabilitacion Infantil | Mexico City | Mexico |
| Centro de Rehabilitacion Integral de Queretaro (CRIQ) | Querétaro | Mexico |
| Hospital Central Dr Ignacio Morones Prieto | San Luis Potosí City | Mexico |
| Non-public Healthcare Unit at the Association for Disabled People KROK PO KROKU | Gdansk | Poland |
| B i L- Specjalistyczne Centrum Medyczne | Lodz | Poland |
| Non-public Healthcare Unit - Grunwaldzka Clinic | Poznan | Poland |
| Non-public Healthcare Unit Mazovian Neurorehabilitatio | Wiązowna | Poland |
| Ghulane Military Medical Academy and School of Medicine | Ankara | Turkey (Türkiye) |
| Ibn-i-Sina Hospital | Ankara | Turkey (Türkiye) |
| Yildirim Beyazit Training and Research Hospital | Ankara | Turkey (Türkiye) |
| GATA Haydarpasa Training Hospital | Istanbul | Turkey (Türkiye) |
| Istanbul Fizik Tedavi Rehabilitasyon Egitim ve Arastirma Hastanesi | Istanbul | Turkey (Türkiye) |
| Istanbul Fizik Tedavi Rehabilitasyon | Istanbul | Turkey (Türkiye) |
| Istanbul University Medical School | Istanbul | Turkey (Türkiye) |
| Dokuz Eylül University Medical Faculty | Izmir | Turkey (Türkiye) |
| Kocaeli University Medical Faculty | İzmit | Turkey (Türkiye) |
15 U/Kg per lower limb. Either one or both lower limbs can be treated. Total volume injected, 2ml per leg. Botulinum type A toxin (Dysport®): I.M. (in the muscle) injection on day 1 of a single treatment cycle. |
| FG002 | Placebo | Total volume to be injected per lower limb - 2ml. Either one or both lower limbs can be treated. Placebo: I.M. injection on day 1 of a single treatment cycle. |
| Treated | Constitutes the safety population. |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
| Treatment Phase - ITT Population |
|
|
Baseline characteristic values were based on the ITT population, defined as all randomised subjects who received at least one injection of study treatment and who had a MAS score in the GSC assessed both at baseline and at Week 4.
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| ID | Title | Description |
|---|---|---|
| BG000 | Dysport 10 U/Kg | Dysport 10 U/Kg intramuscular injection on either one or both lower limbs (2 ml per leg), single treatment. |
| BG001 | Dysport 15 U/Kg | Dysport 15 U/Kg intramuscular injection on either one or both lower limbs (2 ml per leg), single treatment. |
| BG002 | Placebo | Placebo intramuscular injection on either one or both lower limbs (2 ml per leg), single treatment. |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Age, Customized | Number | participants |
| ||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Number | participants |
| ||||||||||||||||
| Botulinum toxin Status | Number | participants |
| ||||||||||||||||
| Number of legs being treated | Number | participants |
| ||||||||||||||||
| Gross Motor Function Classification System (GMFCS) | Participants were staged according to the following GMFCS criteria: level 1 (walks without limitations (best outcome)), level 2 (walks with limitations) and level 3 (walks using a hand held mobility device (worst outcome)). Only participants classified as GMFCS levels 1 to 3 were eligible for entry into the study. | Number | participants |
| |||||||||||||||
| Height | Mean | Standard Deviation | cm |
| |||||||||||||||
| Weight | Mean | Standard Deviation | kg |
| |||||||||||||||
| Body Mass Index (BMI) | Mean | Standard Deviation | kg/m2 |
| |||||||||||||||
| Modified Ashworth Scale (MAS) score | The MAS is a 6-point scale which measures the intensity of muscle tone by measuring the resistance of the muscle to passive lengthening or stretching. The Investigator graded muscle tone in the gastrocnemius-soleus complex (GSC) from 0 (no increase in tone) to 4 (affected parts rigid in flexion or extension). | Mean | Standard Deviation | units on a scale |
|
| 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 | Change in MAS Score in the Gastrocnemius-soleus Complex (GSC) at the Ankle Joint of the (Most) Affected Lower Limb | The MAS is a 6-point scale which measures the intensity of muscle tone by measuring the resistance of the muscle to passive lengthening or stretching. Investigator will grade muscle tone in the GSC from 0 (no increase in tone) to 4 (affected parts rigid in flexion or extension). | ITT population, defined as all randomised subjects who received at least one injection of study treatment and who had a MAS score in the GSC assessed both at baseline and at Week 4. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | Change from baseline to Week 4 |
|
|
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| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Physician's Global Assessment (PGA) of the Treatment Response. | PGA Scale of the Treatment Response: Global assessment of treatment response assessed by asking the Investigator the following question: "how would you rate the response to treatment in the subject's lower limb(s) since the last injection?" Answers will be made 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, +4: markedly improved). | ITT population, defined as all randomised subjects who received at least one injection of study treatment and who had a MAS score in the GSC assessed both at baseline and at Week 4. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | Week 4 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Goal Attainment Scale (GAS) Score | GAS is a functional scale used to measure progress towards individual therapy goals. Individual goals defined for each subject by the physician, and the child's parents (caregiver) where applicable, prior to treatment. Post-baseline, the GAS for each goal rated using a defined scale (-2: Much less than expected outcome, -1: somewhat less than expected outcome, 0: expected outcome, 1: somewhat more than expected outcome, and 2: Much more than expected outcome). | ITT population, defined as all randomised subjects who received at least one injection of study treatment and who had a MAS score in the GSC assessed both at baseline and at Week 4. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | Week 4 |
|
Up to Week 28 of follow-up
The safety population, defined as all randomised subjects who received at least one injection of study treatment, was used for the analysis of adverse events (AEs).
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Dysport 10 U/Kg | Dysport 10 U/Kg intramuscular injection on either one or both lower limbs (2 ml per leg), single treatment. | 1 | 80 | 51 | 80 | ||
| EG001 | Dysport 15 U/Kg | Dysport 15 U/Kg intramuscular injection on either one or both lower limbs (2 ml per leg), single treatment. | 0 | 80 | 51 | 80 | ||
| EG002 | Placebo | Placebo intramuscular injection on either one or both lower limbs (2 ml per leg), single treatment. | 4 | 79 | 42 | 79 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Head injury | Injury, poisoning and procedural complications | MedDRA 17.0 | Systematic Assessment |
| |
| Upper limb fracture | Injury, poisoning and procedural complications | MedDRA 17.0 | Systematic Assessment |
| |
| Adenoidal hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA 17.0 | Systematic Assessment |
| |
| Gastroenteritis | Respiratory, thoracic and mediastinal disorders | MedDRA 17.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
| |
| Rotavirus infection | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Excoriation | Injury, poisoning and procedural complications | MedDRA 17.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 17.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 17.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 17.0 | Systematic Assessment |
| |
| Epilepsy | Nervous system disorders | MedDRA 17.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 17.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 17.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 17.0 | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 17.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
| |
| Upper respiratory tract infection bacterial | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
| |
| Respiratory tract infection viral | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
| |
| Varicella | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
| |
| Rhinorrhea | Respiratory, thoracic and mediastinal disorders | MedDRA 17.0 | Systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Director, Neurology | Ipsen | clinical.trials@ipsen.com | clinical.trials@ipsen.com |
| ID | Term |
|---|---|
| D002547 | Cerebral Palsy |
| D009128 | Muscle Spasticity |
| ID | Term |
|---|---|
| D001925 | Brain Damage, Chronic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009122 | Muscle Hypertonia |
| D020879 | Neuromuscular Manifestations |
| D009461 | Neurologic Manifestations |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| D019274 | Botulinum Toxins, Type A |
| C542869 | abobotulinumtoxinA |
| 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 |
Not provided
Not provided
| Withdrawal by Subject |
|
| Lost to Follow-up |
|
| Children (10-17 years) |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Caucasian/White |
|
| American Indian/Alaskan Native |
|
| Multiple |
|
| Non-naive |
|
| 2 Legs injected |
|
| GMFCS level 2 |
|
| GMFCS level 3 |
|
LS means for each treatment group and treatment comparisons, as well as the p-values were obtained from an ANCOVA on the change from baseline with treatment, baseline MAS score, age range at baseline, BTX status at baseline and centre as covariates. |
| ANCOVA |
| 0.0002 |
| Difference in LS means |
| -0.49 |
| 2-Sided |
| 95 |
| -0.75 |
| -0.23 |
| Superiority or Other (legacy) |
|
|
|
|
|
|