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

Study Overview
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Brief Summary
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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
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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.

Official Title
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Efficacy and Functional Outcomes of Botulinum Toxin A Injections to Hamstrings in Flexed Knee Gait in Cerebral Palsy: A Double-Blind, Randomized, Placebo-Controlled Trial

Conditions
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Spastic Diplegic Cerebral Palsy

Intervention / Treatment
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* Drug: Botulinum Toxin A


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Spastic cerebral palsy; Bilateral lower extremity involvement; Spasticity present in the hamstrings; Flexed knee gait >15 degrees by observation (OGS); Age between 3 and 18 years old; ( GMFCS Level I to Level IV; ( Able to walk a minimum of 4 complete steps without resting with and without braces/shoes a minimum of 3 times; Able to follow simple commands; Cooperative with physicians and therapist; Able to tolerate application of equipment to the skin. Exclusion Criteria: Rhizotomy surgery within the last 1 year; Lower extremity surgical procedures (soft tissue or bony) within the past 1 year; Currently implanted and operating Intrathecal Baclofen Pump (oral baclofen OK); Lower extremity BTX-A injections within the past 6 months; Multilevel BTX-A injections; Gait trainer reliance; Serial casting 3 months prior to or during study protocol.

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Double


Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: Botulinum Toxin A|nan|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 3DGA Kinematics |  |  | 
| Passive Range of Motion |  |  | 
| Spasticity Measurement with AShworth and Tardieu Scales |  |  | 
| Muscle Strength and Control |  |  | 

Secondary Outcome Measures

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

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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cerebral palsy, spasticity, diplegia