Clinical trial NCT00261131: Efficacy and Functional Outcomes of Botulinum Toxin A Injections to Hamstrings in Flexed Knee Gait in Cerebral Palsy
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Spastic Diplegic Cerebral Palsy
Keywords: cerebral palsy
spasticity
diplegia
Lead sponsor: Shriners Hospitals for Children
Sponsor class: OTHER
Collaborators: name: University of Virginia
name: Washington University School of Medicine
First posted: 2005-12-02
Enrollment: 250
Sex: ALL
Minimum age (years): 3
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: 3DGA Kinematics
measure: Passive Range of Motion
measure: Spasticity Measurement with AShworth and Tardieu Scales
measure: Muscle Strength and Control
Secondary outcomes:
measure: Gross Motor Function Measurement (GMFM)
measure: Pediatric Outcomes Data Collection Instrument (PODCI)
measure: Gillette Functional Assessment Questionnaire (GFAQ)
measure: 6-Minute Walk Test
measure: Goal Attainment Scale (GAS)
Other outcomes:
Locations:
Study officials:
affiliation: Shriners Hospitals for Children
name: Philip E. Gates, MD
role: PRINCIPAL_INVESTIGATOR
References: