Efficacy and Functional Outcomes of Botulinum Toxin A Injections to Hamstrings in Flexed Knee Gait in Cerebral Palsy

Phase 3

Completed

• Conditions: Spastic Diplegic Cerebral Palsy

• Registration Number: NCT00261131
• Lead Sponsor: Shriners Hospitals for Children

Brief Summary

The study proposes to determine if injections of BTX-A to the hamstring muscles result in measurable physiologic changes not observed with normal saline injections in children with spastic diplegic cerebral palsy who walk with a flexed-knee gait pattern.

Detailed Description

This study proposes to: (1) to determine if injections of BTX-A to the hamstring muscles result in measurable physiologic (body structure and body function) changes not observed with normal saline injections, in children with spastic diplegic CP who walk with a flexed-knee gait pattern; (2) to assess whether physiologic changes translate into functional (activity and participation) improvements; (3) to evaluate family's perception of change in function, activity, participation, and quality of life.

This study will demonstrate if BTX-A injected into overactive hamstring muscles of children with spastic cerebral palsy (CP) and a flexed-knee walking pattern has measurable effects across the spectrum of dimensions of disablement of the International Classification of Functioning, Disability, and Health (ICF). It is a multi-center, prospective, randomized, double-blind trial comparing results of injection of BTX- A and placebo saline in controls, into overactive hamstring muscles, using multiple outcomes measures. Temporal-spatial gait parameters, instrumented 3DGA kinematics, passive ROM, spasticity measurement with both Ashworth and Tardieu scales, muscle strength/control, Gross Motor Function Measurement (GMFM), Pediatric Outcomes Data Collection Instrument (PODCI), Gillette Functional Assessment Questionnaire (FAQ), 6-Minute Walk Test, and Goal Attainment Scale (GAS), assessing all ICF domains, will be collected at baseline and at 1 month, 3 months, and 6 months post-injection. Power analysis demonstrates the need to randomize 250 children (125 with BTX-A, 125 with saline), allowing for attrition. Nine participating hospitals will contribute patients.

Study Timeline

• Study First Submitted: 2005-11-30
• Study First Submitted QC: 2005-11-30
• Study First Posted: 2005-12-02
• Study Start: 2006-03
• Study Completion: 2007-12
• Status Verified: 2009-02
• Last Update Submitted: 2009-02-04
• Last Update Posted: 2009-02-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 250

Inclusion Criteria

1. Spastic cerebral palsy;
2. Bilateral lower extremity involvement;
3. Spasticity present in the hamstrings;
4. Flexed knee gait >15 degrees by observation (OGS);
5. Age between 3 and 18 years old; (
6. GMFCS Level I to Level IV; (
7. Able to walk a minimum of 4 complete steps without resting with and without braces/shoes a minimum of 3 times;
8. Able to follow simple commands;
9. Cooperative with physicians and therapist;
10. Able to tolerate application of equipment to the skin.

Exclusion Criteria

1. Rhizotomy surgery within the last 1 year;
2. Lower extremity surgical procedures (soft tissue or bony) within the past 1 year;
3. Currently implanted and operating Intrathecal Baclofen Pump (oral baclofen OK);
4. Lower extremity BTX-A injections within the past 6 months;
5. Multilevel BTX-A injections;
6. Gait trainer reliance;
7. Serial casting 3 months prior to or during study protocol.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
3DGA Kinematics
Passive Range of Motion
Spasticity Measurement with AShworth and Tardieu Scales
Muscle Strength and Control

Secondary Outcome Measures

Name	Time	Method
Goal Attainment Scale (GAS)
Gross Motor Function Measurement (GMFM)
Pediatric Outcomes Data Collection Instrument (PODCI)
Gillette Functional Assessment Questionnaire (GFAQ)
6-Minute Walk Test