Clinical trial NCT01251380: Dysport® Pediatric Lower Limb Spasticity Follow-on Study
Official title: A Phase III, Prospective, Multicentre, Open Label, Extension Study Assessing the Long Term Safety and Efficacy of Repeated Treatment With DYSPORT® Used in the Treatment of Lower Limb Spasticity in Children With Dynamic Equinus Foot Deformity Due to Cerebral Palsy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Cerebral Palsy
Muscle Spasticity
Children
Lead sponsor: Ipsen
Sponsor class: INDUSTRY
First posted: 2010-12-01
Enrollment: 216
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this research study was to determine the long term safety and efficacy of repeated treatments with Dysport® used in the treatment of lower limb spasticity in children with dynamic equinus foot deformity due to cerebral palsy.


Eligibility criteria: Inclusion Criteria:

Subjects were eligible for participation in the study if they met the following criteria:

1. Completion of the double blind study (Study 141) up to the Week 12, Week 16, Week 22 or Week 28 follow up visit.
2. Without any major protocol deviations and/or any ongoing adverse events (AEs), either of which, in the opinion of the Investigator would pose an unacceptable risk to the subject were he/she to continue receiving treatment in this open label extension study.
3. Written informed consent obtained from the child's parent(s)/guardian(s) for this study, and assent from the child when and where applicable.

Exclusion Criteria:

Subjects were excluded from entering the study for the following reasons:

1. Major limitation in the passive range of motion at the ankle, as defined by maximum ankle dorsiflexion measured by the angle of arrest (XV1) at slow speed \<80° (TS angle) in the most affected leg to be injected.
2. Unwillingness or inability to comply with the protocol.
3. Current need for surgery for spasticity of the gastrocnemius-soleus complex (GSC) and/or hamstring muscles (and/or tendons) in the most affected leg to be injected.
4. Treatment with any drug that interferes either directly or indirectly with neuromuscular function (e.g. aminoglycoside antibiotics) or neuroblocking agents used during surgery (e.g. curare) within the last 30 days prior to study medication or a planned treatment with such drugs.
5. Be pregnant and/or lactating.
6. Female subjects, not willing to use contraceptive measures throughout the course of the study if post pubertal and sexually active.
7. An infection at the injection site(s).
8. Planned treatment with any new investigational drug or device during the study period.

Primary outcomes:
measure: Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Reported in the Double Blind (DB) + Open Label (OL) Period.
timeFrame: From baseline (Day 1) until end of study (Week 40) of Cycle 1 and up to Week 28 of Cycles 2 to 4.
Secondary outcomes:
measure: Mean Change From Baseline (in the DB Study) in the MAS Score in the GSC Assessed at the Ankle Joint of the (Most) Affected Lower Limb
timeFrame: DB baseline; Weeks 4 and 12 of Treatment Cycles 1 to 3; Week 4 of Treatment Cycle 4
measure: Mean Change From Baseline (Prior to the First Injection Cycle in the Hamstrings) in the MAS Score in the Knee Flexors Assessed at the Knee Joint of the (Most) Affected Lower Limb
timeFrame: Baseline and Weeks 4 and 12 of Treatment Cycles 1 to 3; Baseline and Week 4 of Treatment Cycle 4.
measure: Mean Change From Baseline (Prior to the First Injection Cycle in Upper Limb Muscle Groups) in the Mean MAS Score for All Injected Upper Limb Muscle Groups From Treatment Cycle 2 Onwards
timeFrame: Baseline and Weeks 4 and 12 of Treatment Cycles 2 and 3.
measure: Mean Physician's Global Assessment (PGA) Score
timeFrame: Weeks 4 and 12 of Treatment Cycles 1 to 3; Week 4 of Treatment Cycle 4.
measure: Mean Goal Attainment Scale (GAS) Score
timeFrame: Weeks 4 and 12 of Treatment Cycles 1 to 3; Week 4 of Treatment Cycle 4
measure: Mean Change From DB Baseline in Angle of Arrest (XV1) Derived From the Tardieu Scale (TS), in the GSC Assessed at the Ankle Joint of the (Most) Affected Lower Limb
timeFrame: DB baseline and Weeks 4 and 12 of Treatment Cycles 1 to 3; Week 4 of Treatment Cycle 4
measure: Mean Change From DB Baseline in Angle of Catch (XV3) Derived From the TS, in the GSC Assessed at the Ankle Joint of the (Most) Affected Lower Limb
timeFrame: DB baseline and Weeks 4 and 12 of Treatment Cycles 1 to 3; Week 4 of Treatment Cycle 4
measure: Mean Change From DB Baseline in Spasticity Angle (X) Derived From the TS, in the GSC Assessed at the Ankle Joint of the (Most) Affected Lower Limb
timeFrame: DB Baseline and Weeks 4 and 12 of Treatment Cycles 1 to 3; Week 4 of Treatment Cycle 4
measure: Mean Change From DB Baseline in Spasticity Grade (Y) Derived From the TS, in the GSC Assessed at the Ankle Joint of the (Most) Affected Lower Limb
timeFrame: DB Baseline and Weeks 4 and 12 of Treatment Cycles 1 to 3; Week 4 of Treatment Cycle 4
measure: Mean Change From Baseline (Prior to the First Injection Cycle in the Hamstrings) in XV1 Derived From the TS, in the Knee Flexors Assessed at the Knee Joint of the (Most) Affected Lower Limb
timeFrame: Baseline and Weeks 4 and 12 of Treatment Cycles 1 to 3; Baseline and Week 4 of Treatment Cycle 4
measure: Mean Change From Baseline (Prior to the First Injection Cycle in the Hamstrings) in XV3 Derived From the TS, in the Knee Flexors Assessed at the Knee Joint of the (Most) Affected Lower Limb
timeFrame: Baseline and Weeks 4 and 12 of Treatment Cycles 1 to 3; Baseline and Week 4 of Treatment Cycle 4
measure: Mean Change From Baseline (Prior to the First Injection Cycle in the Hamstrings) in X Derived From the TS, in the Knee Flexors Assessed at the Knee Joint of the (Most) Affected Lower Limb
timeFrame: Baseline and Weeks 4 and 12 of Treatment Cycles 1 to 3; Baseline and Week 4 of Treatment Cycle 4
measure: Mean Change From Baseline (Prior to the First Injection Cycle in the Hamstrings) in Y Derived From the TS, in the Knee Flexors Assessed at the Knee Joint of the (Most) Affected Lower Limb
timeFrame: Baseline and Weeks 4 and 12 of Treatment Cycles 1 to 3; Baseline and Week 4 of Treatment Cycle 4
measure: Mean Change From DB Baseline in the Observational Gait Scale (OGS) Total Score of the (Most) Affected Leg
timeFrame: DB Baseline and Weeks 4 and 12 of Treatment Cycles 1 to 3; Week 4 of Treatment Cycle 4
measure: Mean Change From DB Baseline in the PedsQL Score (CP Module Scores) at Each Study Visit Except Week 4
timeFrame: Baseline and Week 12
measure: Mean Change From DB Baseline in the PedsQL Score (Generic Core Scores) at Each Study Visit Except Week 4
timeFrame: Baseline and Week 12
Other outcomes:
Locations:
city: Aurora
country: United States
facility: The Children's Hospital
state: Colorado
city: Chicago
country: United States
facility: Rehabilitation Institute of Chicago
state: Illinois
city: New Orleans
country: United States
facility: Children's Hospital New Orleans
state: Louisiana
city: Detroit
country: United States
facility: Children's Hospital of Michigan
state: Michigan
city: Saint Paul
country: United States
facility: Gillette Children's Speciality Healthcare
state: Minnesota
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Portland
country: United States
facility: Shriner's Hospital for Children
state: Oregon
city: Dallas
country: United States
facility: Texas Scottish Rite - Hospital for Children
state: Texas
city: Punta Arenas
country: Chile
facility: Club De Leones Cruz Del Sur Rehabilitation Corporation Punta Arenas
city: Santiago
country: Chile
facility: Dr Roberto Del Rio Hospital
city: Santiago
country: Chile
facility: Neurorehabilitation Laboratory, Pontifical Catholic University
city: Besancon
country: France
facility: CHU Jean Minjoz
city: Celaya
country: Mexico
facility: Hospital San José Celaya
city: Mexico City
country: Mexico
facility: Centro de Rehabilitacion Infantil
city: Queretaro
country: Mexico
facility: Centro de Rehabilitacion Integral de Queretaro (CRIQ)
city: Gdansk
country: Poland
facility: Non-public Healthcare Unit at the Association for Disabled People KROK PO KROKU
city: Lodz
country: Poland
facility: B i L- Specjalistyczne Centrum Medyczne
city: Poznan
country: Poland
facility: Non-public Healthcare Unit - Grunwaldzka Clinic
city: Wiazowna
country: Poland
facility: Non-public Healthcare Unit Mazovian Neurorehabilitatio
city: Ankara
country: Turkey
facility: Ghulane Military Medical Academy and School of Medicine
city: Ankara
country: Turkey
facility: Ibn-i-Sina Hospital
city: Ankara
country: Turkey
facility: Yildirim Beyazit Training and Research Hospital
city: Istanbul
country: Turkey
facility: GATA Haydarpasa Training Hospital
city: Istanbul
country: Turkey
facility: Istanbul Fizik Tedavi Rehabilitasyon Egitim ve Arastirma Hastanesi
city: Istanbul
country: Turkey
facility: Istanbul University Medical School
city: Izmir
country: Turkey
facility: Dokuz Eylül University Medical Faculty
city: Izmit
country: Turkey
facility: Kocaeli University Medical Faculty
Study officials:
affiliation: Ipsen
name: Ipsen Study Director
role: STUDY_DIRECTOR
References: