Dysport® Pediatric Lower Limb Spasticity Study

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.

Study Overview

• Status: Completed
• Conditions: Cerebral Palsy; Children; Muscle Spasticity
• Intervention / Treatment: Biological: Botulinum toxin type A; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 241
• Phase: Phase 3

Contacts and Locations

Study Locations

• Chile
  • Punta Arenas, Chile
    • Club De Leones Cruz Del Sur Rehabilitation Corporation, Punta Arenas
  • Santiago, Chile
    • Dr Roberto Del Rio Hospital
  • Santiago, Chile
    • Neurorehabilitation Laboratory, Pontifical Catholic University
• France
  • Besancon, France
    • CHU Jean Minjoz
• Mexico
  • Celaya, Mexico
    • Hospital San José Celaya
  • Mexico City, Mexico
    • Centro de Rehabilitacion Infantil
  • Queretaro, Mexico
    • Centro de Rehabilitacion Integral de Queretaro (CRIQ)
  • San Luis Potosi, Mexico
    • Hospital Central Dr Ignacio Morones Prieto
• Poland
  • Gdansk, Poland
    • Non-public Healthcare Unit at the Association for Disabled People KROK PO KROKU
  • Lodz, Poland
    • B i L- Specjalistyczne Centrum Medyczne
  • Poznan, Poland
    • Non-public Healthcare Unit - Grunwaldzka Clinic
  • Wiazowna, Poland
    • Non-public Healthcare Unit Mazovian Neurorehabilitatio
• Turkey
  • Ankara, Turkey
    • Ghulane Military Medical Academy and School of Medicine
  • Ankara, Turkey
    • Ibn-i-Sina Hospital
  • Ankara, Turkey
    • Yildirim Beyazit Training and Research Hospital
  • Istanbul, Turkey
    • GATA Haydarpasa Training Hospital
  • Istanbul, Turkey
    • Istanbul Fizik Tedavi Rehabilitasyon Egitim ve Arastirma Hastanesi
  • Istanbul, Turkey
    • Istanbul Fizik Tedavi Rehabilitasyon
  • Istanbul, Turkey
    • Istanbul University Medical School
  • Izmir, Turkey
    • Dokuz Eylul University Medical Faculty
  • Izmit, Turkey
    • Kocaeli University Medical Faculty
• United States
  • Colorado
    • Aurora, Colorado, United States, 80045
      • The Children's Hospital
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Rehabilitation Institute of Chicago
  • Louisiana
    • New Orleans, Louisiana, United States, 70118
      • Children's Hospital New Orleans
  • Michigan
    • Detroit, Michigan, United States, 48202
      • Children's Hospital of Michigan
  • Minnesota
    • Saint Paul, Minnesota, United States, 55101
      • Gillette Children's Speciality Healthcare
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center
  • Oregon
    • Portland, Oregon, United States, 97210
      • Shriner's Hospital for Children
  • Texas
    • Dallas, Texas, United States, 75219
      • Texas Scottish Rite - Hospital for Children

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 17 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• 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

Exclusion Criteria:

• Fixed contracture
• Previous phenol, alcohol injection or surgical intervention
• Other neurological / neuromuscular disorder
• Severe athetoid or dystonic movements

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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.	Biological: Botulinum toxin type A; I.M. (in the muscle) injection on day 1 of a single treatment cycle.; Other Names: AbobotulinumtoxinA (Dysport®)
Experimental: Dysport 15 U/Kg; 15 U/Kg per lower limb. Either one or both lower limbs can be treated. Total volume injected, 2ml per leg.	Biological: Botulinum toxin type A; I.M. (in the muscle) injection on day 1 of a single treatment cycle.; Other Names: AbobotulinumtoxinA (Dysport®)
Placebo Comparator: Placebo; Total volume to be injected per lower limb - 2ml. Either one or both lower limbs can be treated.	Drug: Placebo; I.M. injection on day 1 of a single treatment cycle.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Secondary Outcome Measures

Outcome Measure	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).	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

Collaborators and Investigators

• Sponsor: Ipsen

Study record dates

Study Major Dates

• Study Start: July 1, 2011
• Primary Completion (Actual): February 1, 2014
• Study Completion (Actual): June 1, 2014

Study Registration Dates

• First Submitted: November 25, 2010
• First Submitted That Met QC Criteria: November 25, 2010
• First Posted (Estimate): November 29, 2010

Study Record Updates

• Last Update Posted (Actual): September 28, 2022
• Last Update Submitted That Met QC Criteria: September 15, 2022
• Last Verified: September 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Brain Damage, Chronic; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Manifestations; Muscle Hypertonia; Cerebral Palsy; Muscle Spasticity; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Peripheral Nervous System Agents; Cholinergic Agents; Membrane Transport Modulators; Acetylcholine Release Inhibitors; Neuromuscular Agents; Botulinum Toxins; Botulinum Toxins, Type A; abobotulinumtoxinA
• Other Study ID Numbers: Y-55-52120-141; 2009-017709-12 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes

IPD Plan Description

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.

• IPD Sharing 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.
• IPD Sharing Access Criteria: Further details on Ipsen's sharing criteria, eligible studies and process for sharing are available here (https://vivli.org/members/ourmembers/).