Phase 3 N=216 Treatment

Dysport® Pediatric Lower Limb Spasticity Follow-on Study

Cerebral Palsy · Muscle Spasticity · Children

Bottom Line

View on ClinicalTrials.gov: NCT01251380 ↗

Enrolled (actual)

216

Serious AEs

3.3%

Results posted

Jun 2017

Primary outcome: Primary: Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Reported in the Double Blind (DB) + Open Label (OL) Period. — 177; 5; 91; 151 participants

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Botulinum toxin type A (Biological)
Age: Pediatric · 2+ yrs
Sex: All
Sponsor: Ipsen
Primary completion: Jan 2015

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Reported in the Double Blind (DB) + Open Label (OL) Period.	177; 5; 91; 151; 1; 29	—
SECONDARY 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	-1.0; -0.8; -1.1; -0.9; -1.0; -0.7	—
SECONDARY 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	-0.6; -0.8; -0.5; -0.3; -0.2; -0.6	—
SECONDARY 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	-1.2; -1.1; -1.6; -1.0; 1.0; -1.0	—
SECONDARY Mean Physician's Global Assessment (PGA) Score	1.5; 1.0; 1.5; 0.9; 1.4; 0.7	—
SECONDARY Mean Goal Attainment Scale (GAS) Score	50.6; 50.7; 51.2; 51.7; 48.3; 45.8	—
SECONDARY 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	2.6; 1.4; 1.4; 0.4; 0.5; -1.1	—
SECONDARY 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	12.2; 9.9; 13.3; 11.4; 12.7; 9.9	—
SECONDARY 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	-9.7; -8.5; -12.0; -10.9; -12.2; -11.0	—
SECONDARY 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	-0.4; -0.3; -0.4; -0.3; -0.4; -0.3	—
SECONDARY 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	3.2; 6.9; 3.0; 5.5; 3.8; 2.0	—
SECONDARY 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	10.1; 13.3; 8.2; 12.4; 11.9; 30.0	—
SECONDARY 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	-6.9; -6.5; -5.2; -6.9; -8.1; -28.0	—
SECONDARY 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	-0.1; -0.1; 0.0; -0.1; -0.1; -0.4	—
SECONDARY Mean Change From DB Baseline in the Observational Gait Scale (OGS) Total Score of the (Most) Affected Leg	1.0; 0.3; 1.4; 0.8; 1.4; 1.0	—
SECONDARY Mean Change From DB Baseline in the PedsQL Score (CP Module Scores) at Each Study Visit Except Week 4	6.9; 9.2; 14.4; 7.7; 8.9; 13.5	—
SECONDARY Mean Change From DB Baseline in the PedsQL Score (Generic Core Scores) at Each Study Visit Except Week 4	7.8; 6.1; 8.0; 4.1; 4.6; 8.9	—

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:

Completion of the double blind study (Study 141) up to the Week 12, Week 16, Week 22 or Week 28 follow up visit.
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.
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:

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.
Unwillingness or inability to comply with the protocol.
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.
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.
Be pregnant and/or lactating.
Female subjects, not willing to use contraceptive measures throughout the course of the study if post pubertal and sexually active.
An infection at the injection site(s).
Planned treatment with any new investigational drug or device during the study period.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01251380). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.