Dose-response study of efficacy and safety of Botulinum toxin type A to treat spasticity of the leg(s) in cerebral palsy

• Conditions: ower limb spasticity due to cerebral palsy; MedDRA version: 16.1Level: LLTClassification code 10058977Term: Spastic paresisSystem Organ Class: 100000004852; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2012-005054-30-Outside-EU/EEA
• Lead Sponsor: Merz Pharmaceuticals GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2014-02-04
• Last Update Posted: 10 February 2014

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 300

Inclusion Criteria

1. Female or male subject of 2 to 17 years of age (inclusive).
2. Uni- or bilateral cerebral palsy with clinical need for uni- or bilateral LL injections with BoNT for the treatment of spasticity.
3. Ashworth Scale [AS] score =2 in plantar flexors (at least unilaterally).
4. Clinical need for a total dose of 16 U/kg BW NT 201 (maximum of 400 U) for the treatment of LL spasticity according to the clinical judgment of the investigator.

Are the trial subjects under 18? yes
Number of subjects for this age range: 300
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Fixed contracture defined as severe restriction of the range of joint movement on passive stretch or predominant forms of muscle hypertonia other than spasticity (e.g., dystonia) in the target limb(s).
2. Surgery on pes equinus on side(s) intended to be treated with BoNT injections in this study within 12 months prior to Screening Visit (V1), in the screening period or planned for the time of participation in this study.
3. Hip flexion requiring BoNT injection.
4. Limitation of hip abduction to less than 40° or pre-diagnosed migrational percentage greater than 30.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The purpose of this study is to determine whether injections of Botulinum toxin type A into muscles of the leg(s) are effective in treating children/adolescents (age 2-17 years) with increased muscle tension/uncontrollable muscle stiffness (spasticity) due to cerebral palsy.<br>;Secondary Objective: Not applicable;Primary end point(s): Change from baseline (Day 1) in the AS score of plantar flexors (for subjects with bilateral pes equinus body side to be decided by investigator at screening and to be kept throughout the entire study) at Day 29 (Week 4) of the 1st injection cycle.<br><br>Co-primary efficacy variable (to fulfil the PMC, for US regulatory authorities only):<br>• Investigator’s Global Impression of Change of Plantar Flexor Spasticity Scale [GICS-PF] (for subjects with bilateral treatment on same body side as chosen for the primary efficacy variable) at Day 29 (Week 4) of the 1st injection cycle.<br>;Timepoint(s) of evaluation of this end point: week 4 of 1st injection cycle<br>