Efficacy And Safety Of Dysport In The Treatment Of Upper Limb Spasticity In Children
PUL
A Phase III, Multicentre, Double Blind, Prospective, Randomised, Controlled, Multiple Treatment Study Assessing Efficacy And Safety Of DYSPORT® Used In The Treatment Of Upper Limb Spasticity In Children
2 other identifiers
interventional
212
8 countries
31
Brief Summary
The purpose of this study is to assess the efficacy and safety of multiple doses of Dysport used in the treatment of upper limb spasticity (altered skeletal muscle performance) in children with cerebral palsy (CP).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Apr 2014
Typical duration for phase_3
31 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
April 1, 2014
CompletedFirst Submitted
Initial submission to the registry
April 3, 2014
CompletedFirst Posted
Study publicly available on registry
April 8, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 21, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
September 4, 2018
CompletedResults Posted
Study results publicly available
December 24, 2019
CompletedSeptember 28, 2022
September 1, 2022
3.5 years
April 3, 2014
October 25, 2019
September 15, 2022
Conditions
Outcome Measures
Primary Outcomes (1)
Mean Change From Baseline to TC 1, Week 6 in MAS Score in the TC 1 PTMG
The MAS was used to assess muscle tone in the upper limb PTMG and consists of 6 grades: 0 (no increase in muscle tone), 1 (slight increase in muscle tone, manifested by a catch and release or by minimal resistance at the end of the ROM) when the affected part is moved in flexion or extension, 1+ (slight increase in muscle tone, manifested by a catch, followed by minimal resistance throughout the remainder (less than half) of the ROM), 2 (more marked increase in muscle tone), 3 (considerable increase in muscle tone) or 4 (affected part(s) rigid in flexion or extension). The original score '+1' was given a derived numeric score of '2' and the higher numeric scores were incremented by 1 so that the MAS score range was from 0 to 5 with higher scores indicating greater muscle tone. A negative change from baseline indicates a decrease in muscle tone.
Baseline (TC 1, Day 1) and TC 1, Week 6.
Secondary Outcomes (2)
Mean Physician's Global Assessment (PGA) Score at TC 1, Week 6
TC 1, Week 6.
Mean Goal Attainment Scale (GAS) Total Score at TC 1, Week 6
TC 1, Week 6.
Other Outcomes (7)
Mean Change From Baseline to TC 1 Week 16 in MAS Score in the TC 1 PTMG
Baseline (TC 1, Day 1) and TC 1, Week 16.
Mean Change From Baseline to TC 1 Weeks 6 and 16 in MAS Score in the Elbow Flexors of the Study Limb
Baseline (TC 1, Day 1) and TC 1, Weeks 6 and 16.
Mean Change From Baseline to TC 1 Weeks 6 and 16 in MAS Score in the Wrist Flexors of the Study Limb
Baseline (TC 1, Day 1) and TC 1, Weeks 6 and 16.
- +4 more other outcomes
Study Arms (3)
Group A
EXPERIMENTALGroup A - Treatments 1, 2, 3 and 4: Dysport 16 Units (U)/kg in one upper extremity (the study limb).
Group B
EXPERIMENTALGroup B - Treatments 1, 2, 3 and 4: Dysport 8 U/kg in one upper extremity (the study limb).
Group C
EXPERIMENTALGroup C - Treatment 1: Dysport 2 U/kg in one upper extremity (the study limb). Group C - Treatments 2, 3 and 4: Dysport 8 or 16 U/kg in one upper extremity (the study limb).
Interventions
Subjects randomised to receive Dysport 2 U/kg, 8 U/kg or 16 U/kg administered intramuscularly in the study limb.
Eligibility Criteria
You may qualify if:
- Upper limb spasticity due to cerebral palsy
- Body weight 10 kg or over
- MAS score of 2 or more in affected elbow or wrist flexors
You may not qualify if:
- Fixed myocontracture
- Previous phenol or alcohol injection within 1 year
- Severe athetoid or dystonic movements
- Previous or planned surgery for spasticity in elbow or wrist flexors
- Neuromuscular disorders
- Previous Rhizotomy within 6 months
- Intrathecal baclofen within 30 days
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Ipsenlead
Study Sites (31)
Unknown Facility
Aurora, Colorado, United States
Unknown Facility
Washington D.C., District of Columbia, United States
Unknown Facility
New Orleans, Louisiana, United States
Unknown Facility
Royal Oak, Michigan, United States
Unknown Facility
Saint Paul, Minnesota, United States
Unknown Facility
Omaha, Nebraska, United States
Unknown Facility
Albuquerque, New Mexico, United States
Unknown Facility
New York, New York, United States
Unknown Facility
Columbus, Ohio, United States
Unknown Facility
Portland, Oregon, United States
Unknown Facility
Austin, Texas, United States
Unknown Facility
Dallas, Texas, United States
Unknown Facility
Brussels, Belgium
Unknown Facility
Brno, Czechia
Unknown Facility
Prague, Czechia
Unknown Facility
Beersheba, Israel
Unknown Facility
Jerusalem, Israel
Unknown Facility
Petah Tikva, Israel
Unknown Facility
Tel Aviv, Israel
Unknown Facility
Tel Litwinsky, Israel
Unknown Facility
La Paz, Baja California Sur, Mexico
Unknown Facility
Celaya, Mexico
Unknown Facility
Monterrey, Mexico
Unknown Facility
Gdansk, Poland
Unknown Facility
Poznan, Poland
Unknown Facility
Wiązowna, Poland
Unknown Facility
Barcelona, Spain
Unknown Facility
Terrassa, Spain
Unknown Facility
Istanbul, Turkey (Türkiye)
Unknown Facility
Izmir, Turkey (Türkiye)
Unknown Facility
Kocaeli, Turkey (Türkiye)
Related Publications (2)
Delgado MR, Tilton A, Carranza-Del Rio J, Dursun N, Bonikowski M, Aydin R, Maciag-Tymecka I, Oleszek J, Dabrowski E, Grandoulier AS, Picaut P; Dysport in PUL study group. Efficacy and safety of abobotulinumtoxinA for upper limb spasticity in children with cerebral palsy: a randomized repeat-treatment study. Dev Med Child Neurol. 2021 May;63(5):592-600. doi: 10.1111/dmcn.14733. Epub 2020 Nov 18.
PMID: 33206382DERIVEDShierk A, Jimenez-Moreno AC, Roberts H, Ackerman-Laufer S, Backer G, Bard-Pondarre R, Cekmece C, Pyrzanowska W, Vilain C, Delgado MR. Development of a Pediatric Goal-Centered Upper Limb Spasticity Home Exercise Therapy Program for Use in a Phase-III Trial of Abobotulinumtoxina (Dysport(R)). Phys Occup Ther Pediatr. 2019;39(2):124-135. doi: 10.1080/01942638.2018.1486346. Epub 2018 Sep 11.
PMID: 30204515DERIVED
MeSH Terms
Interventions
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Director
- Organization
- Ipsen
Study Officials
- STUDY DIRECTOR
Ipsen Medical Director
Ipsen
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 3, 2014
First Posted
April 8, 2014
Study Start
April 1, 2014
Primary Completion
September 21, 2017
Study Completion
September 4, 2018
Last Updated
September 28, 2022
Results First Posted
December 24, 2019
Record last verified: 2022-09
Data Sharing
- IPD Sharing
- Will share
- Time Frame
- 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.
- Access Criteria
- Further details on Ipsen's sharing criteria, eligible studies and process for sharing are available here (https://vivli.org/members/ourmembers/).
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.