Cerebral PalsyPhase 3NCT01249417
Dysport® Pediatric Lower Limb Spasticity Study
A Phase 3 study testing Botulinum toxin type A in people with Cerebral Palsy and Muscle Spasticity, run by Ipsen at 29 sites in 6 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Cerebral Palsy, Muscle Spasticity, Children
- What is being tested?
- Botulinum toxin type A
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 241 participants
- Where
- 29 sitesin 6 countries
- Study dates
- Jul 2011to Jun 2014
- 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
- Children aged 2 to 17 years with cerebral palsy
- Equinus foot position
- Ambulatory
- Intensity of muscle tone equal to or greater than 2 in affected lower limb, as measured on the Modified Ashworth Scale
Who cannot join
- Fixed contracture
- Previous phenol, alcohol injection or surgical intervention
- Other neurological / neuromuscular disorder
- Severe athetoid or dystonic movements
Trial locations and contacts
29 sites in 6 countries.
29 sites in 6 countries
Turkey (Türkiye)9 sites
Ghulane Military Medical Academy and School of Medicine
Ankara
Ibn-i-Sina Hospital
Ankara
Yildirim Beyazit Training and Research Hospital
Ankara
GATA Haydarpasa Training Hospital
Istanbul
Istanbul Fizik Tedavi Rehabilitasyon Egitim ve Arastirma Hastanesi
Istanbul
Istanbul Fizik Tedavi Rehabilitasyon
Istanbul
Istanbul University Medical School
Istanbul
Dokuz Eylül University Medical Faculty
Izmir
Kocaeli University Medical Faculty
İzmit
United States8 sites
The Children's Hospital
Aurora, Colorado, 80045
Rehabilitation Institute of Chicago
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Texas Scottish Rite - Hospital for Children
Dallas, Texas, 75219
Children's Hospital of Michigan
Detroit, Michigan, 48202
Children's Hospital New Orleans
New Orleans, Louisiana, 70118
Shriner's Hospital for Children
Portland, Oregon, 97210
Gillette Children's Speciality Healthcare
Saint Paul, Minnesota, 55101
Mexico4 sites
Hospital San José Celaya
Celaya
Centro de Rehabilitacion Infantil
Mexico City
Centro de Rehabilitacion Integral de Queretaro (CRIQ)
Querétaro
Hospital Central Dr Ignacio Morones Prieto
San Luis Potosí City
Poland4 sites
Non-public Healthcare Unit at the Association for Disabled People KROK PO KROKU
Gdansk
B i L- Specjalistyczne Centrum Medyczne
Lodz
Non-public Healthcare Unit - Grunwaldzka Clinic
Poznan
Non-public Healthcare Unit Mazovian Neurorehabilitatio
Wiązowna
Chile3 sites
Club De Leones Cruz Del Sur Rehabilitation Corporation, Punta Arenas
Punta Arenas
Dr Roberto Del Rio Hospital
Santiago
Neurorehabilitation Laboratory, Pontifical Catholic University
Santiago
About this study
Official title: A Phase III, Multicentre, Double Blind, Prospective, Randomised, Placebo Controlled Study Assessing the Efficacy and Safety of DYSPORT® Used in the Treatment of Lower Limb Spasticity in Children With Dynamic Equinus Foot Deformity Due to Cerebral Palsy
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.
- Conditions
- Cerebral Palsy, Muscle Spasticity, Children
- Being tested
- Botulinum toxin type A, Placebo
- Medical subject headings
- Cerebral Palsy, Muscle Spasticity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Botulinum toxin type ABiological
Also called AbobotulinumtoxinA (Dysport®)
I.M. (in the muscle) injection on day 1 of a single treatment cycle.
PlaceboDrug
I.M. injection on day 1 of a single treatment cycle.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Biological: Botulinum toxin type A |
| 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. Receives: Biological: Botulinum toxin type A |
| Placebo | Placebo comparator | Total volume to be injected per lower limb - 2ml. Either one or both lower limbs can be treated. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: Change from baseline to Week 4
Secondary outcomes (2)
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).
Measured: 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).
Measured: Week 4
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Nov 2010 (estimated)
- Study startJul 2011
- Primary completionFeb 2014
- Study completionJun 2014
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.
- Other trials for Cerebral PalsyStudies of the same condition, open and under way.
- More trials run by IpsenThe sponsor's other registered studies.
- 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
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 241 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ipsen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ipsen Study Director, Study director, Ipsen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01249417
- Sponsor's protocol ID
- Y-55-52120-141
- Other IDs
- 2009-017709-12, 2009
- Status last verified
- Sep 2022
- First submitted
- 25 Nov 2010
- Last updated
- 28 Sep 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- 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.
Study at a glance
From the registry record, last updated by the sponsor on 28 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase III, Multicentre, Double Blind, Prospective, Randomised, Placebo Controlled Study Assessing the Efficacy and Safety of DYSPORT® Used in the Treatment of Lower Limb Spasticity in Children With Dynamic Equinus Foot Deformity Due to Cerebral Palsy
- ClinicalTrials.gov ID
- NCT01249417
- Acronym / sponsor ID
- Y-55-52120-141
- Conditions
- Cerebral Palsy, Muscle Spasticity, Children
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 241 participants (actual)
- Lead sponsor
- IpsenIndustry (pharma, biotech, devices)
- Study start
- Jul 2011
- Primary completion
- Feb 2014 (actual)
- Study completion
- Jun 2014 (actual)
- First posted
- 29 Nov 2010 (estimated)
- Results
- Posted 3 Feb 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 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Dysport 10 U/Kg | Experimental | Biological: Botulinum toxin type A10 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 | Biological: Botulinum toxin type A15 U/Kg per lower limb. Either one or both lower limbs can be treated. Total volume injected, 2ml per leg. |
| Placebo | Placebo comparator | Drug: PlaceboTotal volume to be injected per lower limb - 2ml. Either one or both lower limbs can be treated. |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Children aged 2 to 17 years with cerebral palsy
- Equinus foot position
- Ambulatory
- Intensity of muscle tone equal to or greater than 2 in affected lower limb, as measured on the Modified Ashworth Scale
Key exclusion criteria (4 listed)
- Fixed contracture
- Previous phenol, alcohol injection or surgical intervention
- Other neurological / neuromuscular disorder
- Severe athetoid or dystonic movements
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 |
|---|---|---|
| Botulinum toxin type AAlso: AbobotulinumtoxinA (Dysport®)I.M. (in the muscle) injection on day 1 of a single treatment cycle. | Biological | Dysport 10 U/Kg, Dysport 15 U/Kg |
| PlaceboI.M. injection on day 1 of a single treatment cycle. | Drug | Placebo |
Primary outcome
Change in MAS Score in the Gastrocnemius-soleus Complex (GSC) at the Ankle Joint of the (Most) Affected Lower Limb
Time frame: Change from baseline to Week 4
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).
2 secondary outcomes
Physician's Global Assessment (PGA) of the Treatment Response.
Time frame: Week 4
Goal Attainment Scale (GAS) Score
Time frame: Week 4
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Sep 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: 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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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.
Ipsen Study Director study director
Ipsen
29 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Botulinum toxin type A in people with Cerebral Palsy and Muscle Spasticity, run by Ipsen at 29 sites in 6 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 4 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 28 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 29 sites in Turkey (Türkiye), United States, Mexico, Poland, Chile and 1 more country. 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. Some participants in this study may receive a placebo. 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 3 Feb 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 in MAS Score in the Gastrocnemius-soleus Complex (GSC) at the Ankle Joint of the (Most) Affected Lower Limb (time frame: Change from baseline to Week 4). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 28 Sep 2022.