The Impact of Dosing Parameters on Motor Skill Acquisition and Retention in Bilateral Cerebral Palsy (BCP)

Study Overview
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Brief Summary
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A recent systematic review found that therapeutic interventions that apply principles of motor learning with intense practice improve functional upper extremity movement in children with unilateral CP. Evidence of efficacy for any treatment approach aimed at improving motor function in bilateral CP (the most prevalent form) is lacking. Preliminary investigation suggests that intensive (90 hours) goal-directed, task-specific training provided in a 3-week day camp format can improve functional movement of both the upper (UE) and lower extremity (LE) and postural control in children with BCP. To date, HABIT-ILE has only been provided in a day camp setting over several weeks. Implementing the dosing schedule of this promising intensive approach in a hospital setting requires innovative resource allocation (space and staff); thus, examining alternative delivery models is imperative. The purpose of this study is to conduct a multi-center randomized control trial (RCT) to determine whether 90 hours of HABIT-ILE improves functional motor skills, activity and motivation in children with BCP when dosed in a camp format at 6-hours/day, 5 days/week for three weeks and 6-hours/day, one day/week for 15 weeks.

Detailed Description
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Cerebral Palsy (CP) is the leading cause of childhood motor impairment with 1 in 323 American children affected. The resulting impaired motor function in individuals with CP limits their ability to independently perform many functional activities, and participate in academic, social, and leisure activities. Children with CP are referred to physical, occupational, and speech therapy at varying intensities (1-3x week) for years, depending on individual needs. While these therapeutic services are offered regularly, the clinical efficacy and optimal dosing parameters are being challenged. In a meta-analysis of all available therapeutic interventions for CP, just 5 had strong enough evidence to recommend implementation into clinical practice for children with unilateral CP (Novak et al. 2013) and no therapies had sufficient evidence of efficacy for treatment of upper (UE) or lower extremities (LE) in children with bilateral CP (BCP). The key features of interventions with proven evidence of clinical efficacy include: a motor learning approach involving active movement, an environment to shape desired movements, and a much higher treatment intensity than provided in weekly therapies. Despite BCP being the most prevalent subtype, the majority of the research evaluating the clinical efficacy and dosing parameters of therapeutic interventions has been determined in children with UCP with upper extremity approaches such as Constraint Induced Movement Therapy (CIMT) and Hand-Arm Bimanual Intensive Therapy (HABIT) demonstrating efficacy. One promising therapy gaining interest and support for children with BCP is Hand-Arm Bimanual Intensive Therapy Including the Lower Extremities (HABIT-ILE). HABIT-ILE aims to improve upper and lower movement control by engaging the UE and LE during combined activities for many hours every day for 3 weeks. Preliminary investigation has demonstrated that children with BCP participating in a 90-hour HABIT-ILE day camp achieve improvement within the body function and structure, activity and participation levels of the International Classification of Functioning, Disability and Health (ICF). While intensive therapies, such as HABIT-ILE, are typically provided in a day camp setting over several weeks, implementing the dosing schedule of this promising intensive approach in a hospital setting requires innovative resource allocation (space and staff). In order to establish the efficacy of this approach for children with BCP, it is essential to further examine the efficacy of the 3-week HABIT-ILE camp format of delivery in a larger, multisite trial, in addition to examining whether an alternative model of intensity and resource allocation can demonstrate similar efficacy. The overall aim of the proposed work is to determine the effectiveness of two different dosing schedules of an intensive therapeutic intervention that applies motor learning to upper and lower extremity movement and postural control to improve functional motor skills, activity, and self-mastery in children with BCP. The specific aims are: Specific Aim 1: To determine if there are significant gains and retention in functional motor skills, activity, and self-mastery in children with BCP receiving 90 hours of HABIT-ILE dosed in a camp format for 6-hours per day, 5 days per week for three weeks and for 6-hours a day, one day per week for 15 weeks. Specific Aim 2: To determine whether the degree of involvement, age and self-mastery predict the magnitude and maintenance of change seen in children with BCP receiving 90 hours of HABIT-ILE dosed in a camp format for 6-hours per day, 5 days per week for three weeks and for 6-hours a day, one day per week for 15 weeks.

Official Title
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The Impact of Dosing Parameters on Motor Skill Acquisition and Retention in Bilateral Cerebral Palsy (BCP)

Conditions
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Cerebral Palsy

Intervention / Treatment
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* Other: HABIT-ILE


Participation Criteria
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Eligibility Criteria
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Need to live within commuting distance of Columbia University (NY, NY), Shriners Hospitals for Children-Greenville (Greenville, SC) or Shriners Hospitals for Children-Portland (Portland, OR) Inclusion Criteria: Diagnosis of Bilateral Cerebral Palsy Gross Motor Function Classification System (GMFCS) levels II-IV Able to get up from a chair with standby assist Able to sit on a mat table with upper extremity support Ability to understand directions Ability to walk 10 steps with/without assistive device and standby assist Ability to understand and speak english Willing to restrict Botox/Dysport use for 3 months prior to each of the 4 full assessments Willing to be randomized to either camp format Exclusion Criteria: Traumatic Brain Injury Predominate Dystonia Predominate Athetosis Orthopedic surgery in the last year or plans during the study period

Ages Eligible for Study
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Minimum Age: 5 Years
Maximum Age: 17 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: Parallel Assignment
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 3-Week Camp<br>Subject will participate in a HABIT-ILE camp format for 6-hours/day, 5 days/week for three weeks. | Other: HABIT-ILE<br>* Goal-directed, task-specific training for both the upper (UE) and lower extremity (LE) and postural control.<br>|
| Experimental: 15-Week Camp<br>Subject will participate in a HABIT-ILE camp format for 6-hours/day, one day/week for 15 weeks. | Other: HABIT-ILE<br>* Goal-directed, task-specific training for both the upper (UE) and lower extremity (LE) and postural control.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Gross Motor Function Measure (GMFM) | Objective assessment of crawling, kneeling, standing, walking, running, jumping.The GMFM is scored on a 4-point scale ranging from does not initiate (0) to completes (3). Each dimension is scored independently providing a measure of functional motor ability within the dimension. | 3 Assessments: Baseline will be just before the start of camp. Two end of study evaluations: one the week following completion of camp and one at 6-months followup | 
| Change in Jebsen-Taylor Test of Hand Function | Objective test of hand function for activities of daily living. Scoring is based on time to perform the individual tasks and times are compared to normative tables with assessment by age and gender. | 3 Assessments: Baseline will be just before the start of camp. Two end of study evaluations: one the week following completion of camp and one at 6-months followup | 
| Change in Pediatric Balance Scale | Assessment of Standing Balance. THE PBS is a 14 item performance based balance test which is scored on a 5-point scale ranging from a low of 0 to a maximum of 4, with a maximum score of 56. | 3 Assessments: Baseline will be just before the start of camp. Two end of study evaluations: one the week following completion of camp and one at 6-months followup | 
| Change in Trunk Control Measurement Scale | The TCMS assesses trunk control during both static and dynamic sitting balance. Dynamic sitting is further divided into selective movement control and dynamic reaching. Higher scores indicate greater balance. | 3 Assessments: Baseline will be just before the start of camp. Two end of study evaluations: one the week following completion of camp and one at 6-months followup | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Box and Blocks | Grasping, transporting and releasing blocks. The test is performed three times for each hand and the mean number of blocks transferred in a minute from the trials will be used to assess gains in manual dexterity. | 3 Assessments: Baseline will be just before the start of camp. Two end of study evaluations: one the week following completion of camp and one at 6-months followup | 
| Change in 2-minute walk | The total distance walked in 2 minutes will be used to assess changes in mobility. | 3 Assessments: Baseline will be just before the start of camp. Two end of study evaluations: one the week following completion of camp and one at 6-months followup | 
| Change in ABILOCO | The ABILOCO-kids is a 10-item parent-report measure of locomotor ability for children with lower limb impairments. The items represent continuous progression in difficulty of the locomotor activity. | 3 Assessments: Baseline will be just before the start of camp. Two end of study evaluations: one the week following completion of camp and one at 6-months followup | 
| Change in ABILHAND | The ABILHAND-kids is a 21-item parent-report measure of bimanual activity for children with CP. The bimanual activities demonstrate continuous progression in difficulty. Higher scores indicate greater ability. | 3 Assessments: Baseline will be just before the start of camp. Two end of study evaluations: one the week following completion of camp and one at 6-months followup | 
| Change in Pediatric Evaluation of Disability Inventory (PEDI-CAT) | Parent questionnaire to assess both capability and performance of functional activities in self-care, mobility, and social function. Each of the three scales will be assessed separately to determine whether specific functional activity areas change with intervention. Higher scores indicate a higher functional level. | 3 Assessments: Baseline will be just before the start of camp. Two end of study evaluations: one the week following completion of camp and one at 6-months followup | 
| Change in Dimensions of Mastery Questionnaire | The 45-item parent-report questionnaire assesses two elements of mastery motivation: instrumental (persistence at object-related tasks, social activities with adults, social interactions with peers, and gross motor tasks) and expressive (behavioral indicators of positive affect during or following tasks and negative reaction to failure). Subscale scores will be used to determine whether motivation impacts gains in functional motor skills and whether gains in functional motor skills impact the magnitude of motivation. | 3 Assessments: Baseline will be just before the start of camp. Two end of study evaluations: one the week following completion of camp and one at 6-months followup | 
| Change in Canadian Occupational Performance Measure (COPM) | Semi-structured interview that enables and open dialogue between the patient/parent on issues of importance to the patient/parent. The COPM is an individualized measure designed to detect change in a child's self-reported occupational performance over time. Occupations are classified into three categories: self-care, productivity and leisure. | One assessment at Baseline will be used to define goals. Two end of study evaluations (EOS) one the week after completion of camp and a 6-month followup | 
| Change in Functional and quantitative muscle strength | Measurement of muscle strength.Quantitative strength of the abductors, knee extensors, and ankle plantarflexors will be measured with a hand-held dynamometer. Functional assessments of standing heel rise, sit to stand and lateral steps ups will assess functional strength. Higher numbers indicate greater strength. | 3 Assessments: Baseline will be just before the start of camp. Two end of study evaluations: one the week following completion of camp and one at 6-months followup |