Cerebral Palsy
Conditions
Keywords
Delay in motor skill development
Brief summary
The purpose of this study is to determine the optimal treatment duration of a novel early mobility training program (dynamic supported mobility, DSM) between 6 to 24 weeks of treatment; and to evaluate the clinical futility of this intervention compared to current rehabilitation practice.
Detailed description
This study is a single-blind, randomized exploratory clinical trial with repeated assessments during a 24-week treatment phase and at three follow-up points over 12 months after treatment to track the developmental trajectory of participants' motor function. Gross motor ability will be compared to published percentile scores of motor function development in cerebral palsy (CP) to determine if the trajectory of predicted motor development is altered, and to outcomes of intensity-matched conventional treatment to determine if continued Phase III investigation is warranted.
Interventions
OTHERDynamic Supported Mobility
Dynamic weight support; Child-directed; No assistive devices, limited use of orthoses, no treadmill; Encourage high degree of error with reduced physical assistance; Encourage frequent variability in motor tasks (no redirection when moving from one activity to another); Physical therapist expertise is focused on designing a salient and challenging environment for the child's specific interests and ability level to encourage engagement, variability, challenge, and error experience, and on determining the appropriate amount of weight assistance
OTHERConventional Therapy
No or static weight support; Therapist-directed (therapist initiates); Traditional early gait training methods: use of assistive devices/orthoses and may use treadmill; Focus on producing typical movement patterns with extensive manual guidance/correction from therapist, prevention of falls; Therapy activities grouped into blocks of practice (i.e. repeated floor to stand practice followed by gait training); Physical therapist expertise is focused on designing and directing the specific practice activities each session, tailored to the individual child
Sponsors
Department of Health and Human Services
Children's Hospital of Philadelphia
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
12 Months to 36 Months
Healthy volunteers
No
Inclusion criteria
* 12-36 months of age * Gross motor function below the 10th percentile for age \[Bayley Scales of Infant and Toddler Development (BSID), BSID-III, corrected for gestational age, if applicable, under the age of two years\]. * Diagnosis of CP or neurological sign associated with CP (i.e. spasticity). * Ability to initiate pulling to stand at a surface \[Score of 1 on gross motor function measure (GMFM) Item 52\]. * Cognitive ability to follow one-step commands.
Exclusion criteria
* Secondary orthopedic, neuromuscular or cardiovascular condition unrelated to CP. * General muscle hypotonia, without other neurological signs associated with CP. * Independent walking ability (Score of 3 on GMFM Item 69 - Walks forward 10 steps). * At or above the 50th percentile of GMFCS Level I. * History of surgery or injury to the lower extremities in the past 6 months.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Gross Motor Function Measure (GMFM-66) During Treatment Phase | Baseline and 12 weeks | Computation of the GMFM-66 score involves statistical weighting of the raw item scores for difficulty. This score will also be used with the patient's age to determine Gross Motor Function Classification System (GMFCS) percentile rank. Scores range from 0 (no volitional movement) to 100 (gross motor function of an average 5 year old). Higher scores reflect better outcomes. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Postural Control | Baseline and 12 weeks | Early Clinical Assessment of Balance - a clinical test of balance and postural control developed for administration with young children. The minimum score is 0. The maximum score is 100. Higher scores reflect better postural control. |
| Change in Physical Activity | Weeks 0 and 12 | A wireless activity monitor will be provided to the caregiver, who will be instructed on use of the monitor at home, with a goal of recording 5 total hours of the child's free play in the following week. Amount and magnitude of physical activity will be calculated from the acceleration time series using signal processing software. |
| Change in Caregiver Satisfaction | Baseline and 12 weeks | Using the Canadian Occupational Performance Measure, one caregiver of each participant will rate their child's performance on the caregiver's self-identified goals, and then rate their own (caregiver's) satisfaction with the child's performance. Satisfaction is rated on a scale of 1-10, with higher ratings reflecting greater parent satisfaction. |
| Change in Child Engagement in Daily Life | Baseline and 12 weeks | One caregiver of each participant will complete the Child Engagement in Daily Life Measure to obtain a measure of the child's participation in play in daily life. Scaled scores range from 0-100. Higher scores reflect greater engagement in daily life. |
Countries
United States
Participant flow
Recruitment details
Participants were enrolled from January 2015 through January 2019. The primary sources of recruitment were the Cerebral Palsy, outpatient physical therapy and Neonatal Follow-up programs at our institution.
Pre-assignment details
Full baseline assessment not completed (n=1)
Participants by arm
| Arm | Count |
|---|---|
| Conventional Therapy The conventional treatment group will receive traditional, therapist-directed pediatric physical therapy. Therapy will focus on early gait training strategies and encouragement of normal movement patterns for walking and other age-appropriate movements, with manual guidance or correction of atypical movements from the therapist. This group may use assistive devices, orthoses, and may receive static body weight support for gait training. Therapy activities will be performed in blocks of practice, with the specific activities and level of therapist assistance tailored to each child.
Conventional Therapy: No or static weight support; Therapist-directed (therapist initiates); Traditional early gait training methods: use of assistive devices/orthoses and may use treadmill; Focus on producing typical movement patterns with extensive manual guidance/correction from therapist, prevention of falls; Therapy activities grouped into blocks of practice (i.e. repeated floor to stand practice followed by gait training); Physical therapist expertise is focused on designing and directing the specific practice activities each session, tailored to the individual child | 21 |
| Dynamic Supported Mobility Children will receive dynamic weight support during all DSM treatment time. The environment will be arranged to encourage active motor exploration, somewhat similar to a play gym for toddlers, to promote the motor variability, engagement, and error experiences that characterize the typical development of upright motor skills and walking. The floor area within 3 feet below either side of the overhead track for a distance of 20 feet (approximately 120 ft2 total) will be defined with colorful thin rubber interlocking mats and arranged with pediatric toys and activities, tailored to the child's interests and to encourage motor skills just beyond his/her current ability. The therapist will minimally assist the child as needed to perform the movements he/she initiates.
Dynamic Supported Mobility: Dynamic weight support; Child-directed; No assistive devices, limited use of orthoses, no treadmill; Encourage high degree of error with reduced physical assistance; Encourage frequent variability in motor tasks (no redirection when moving from one activity to another); Physical therapist expertise is focused on designing a salient and challenging environment for the child's specific interests and ability level to encourage engagement, variability, challenge, and error experience, and on determining the appropriate amount of weight assistance | 20 |
| Total | 41 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Follow-up Phase | Lost to Follow-up | 1 | 0 |
| Treatment Phase | Withdrawal by Subject | 2 | 2 |
Baseline characteristics
| Characteristic | Total | Conventional Therapy | Dynamic Supported Mobility |
|---|---|---|---|
| Age, Categorical <=18 years | 41 Participants | 21 Participants | 20 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Continuous | 21.8 months STANDARD_DEVIATION 6.3 | 23.2 months STANDARD_DEVIATION 7.2 | 20.4 months STANDARD_DEVIATION 5.1 |
| Anatomical typography Diplegia | 18 Participants | 10 Participants | 8 Participants |
| Anatomical typography Hemiplegia | 8 Participants | 5 Participants | 3 Participants |
| Anatomical typography Monoplegia | 1 Participants | 0 Participants | 1 Participants |
| Anatomical typography Quadriplegia | 10 Participants | 4 Participants | 6 Participants |
| Anatomical typography Triplegia | 3 Participants | 1 Participants | 2 Participants |
| Anatomical typography Unknown | 1 Participants | 1 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 4 Participants | 2 Participants | 2 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 36 Participants | 19 Participants | 17 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 1 Participants | 0 Participants | 1 Participants |
| Gross Motor Function Classification System (GMFCS) I | 5 Participants | 3 Participants | 2 Participants |
| Gross Motor Function Classification System (GMFCS) II | 17 Participants | 9 Participants | 8 Participants |
| Gross Motor Function Classification System (GMFCS) III | 10 Participants | 5 Participants | 5 Participants |
| Gross Motor Function Classification System (GMFCS) IV | 9 Participants | 4 Participants | 5 Participants |
| Gross Motor Function Classification System (GMFCS) V | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 2 Participants | 2 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 10 Participants | 3 Participants | 7 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 2 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) White | 27 Participants | 15 Participants | 12 Participants |
| Region of Enrollment United States | 41 Participants | 21 Participants | 20 Participants |
| Sex: Female, Male Female | 18 Participants | 10 Participants | 8 Participants |
| Sex: Female, Male Male | 23 Participants | 11 Participants | 12 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 21 | 0 / 20 |
| other Total, other adverse events | 3 / 21 | 1 / 20 |
| serious Total, serious adverse events | 0 / 21 | 0 / 20 |
Outcome results
Primary
Change in Gross Motor Function Measure (GMFM-66) During Treatment Phase
Computation of the GMFM-66 score involves statistical weighting of the raw item scores for difficulty. This score will also be used with the patient's age to determine Gross Motor Function Classification System (GMFCS) percentile rank. Scores range from 0 (no volitional movement) to 100 (gross motor function of an average 5 year old). Higher scores reflect better outcomes.
Time frame: Baseline and 12 weeks
Population: Participants with completed baseline and 12 week assessments
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Conventional Therapy | Change in Gross Motor Function Measure (GMFM-66) During Treatment Phase | 2.2 units on a scale | Standard Deviation 3.2 |
| Dynamic Supported Mobility | Change in Gross Motor Function Measure (GMFM-66) During Treatment Phase | 3.5 units on a scale | Standard Deviation 2.6 |
Secondary
Change in Caregiver Satisfaction
Using the Canadian Occupational Performance Measure, one caregiver of each participant will rate their child's performance on the caregiver's self-identified goals, and then rate their own (caregiver's) satisfaction with the child's performance. Satisfaction is rated on a scale of 1-10, with higher ratings reflecting greater parent satisfaction.
Time frame: Baseline and 12 weeks
Population: All participants with baseline and 12 week scores
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Conventional Therapy | Change in Caregiver Satisfaction | 1.5 COPM score units | Standard Deviation 2.7 |
| Dynamic Supported Mobility | Change in Caregiver Satisfaction | 2.5 COPM score units | Standard Deviation 2.8 |
Secondary
Change in Child Engagement in Daily Life
One caregiver of each participant will complete the Child Engagement in Daily Life Measure to obtain a measure of the child's participation in play in daily life. Scaled scores range from 0-100. Higher scores reflect greater engagement in daily life.
Time frame: Baseline and 12 weeks
Population: All participants with baseline and 12 week scores
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Conventional Therapy | Change in Child Engagement in Daily Life | 0.96 CEDL scaled score units | Standard Deviation 7.1 |
| Dynamic Supported Mobility | Change in Child Engagement in Daily Life | 3.8 CEDL scaled score units | Standard Deviation 6.1 |
Secondary
Change in Physical Activity
A wireless activity monitor will be provided to the caregiver, who will be instructed on use of the monitor at home, with a goal of recording 5 total hours of the child's free play in the following week. Amount and magnitude of physical activity will be calculated from the acceleration time series using signal processing software.
Time frame: Weeks 0 and 12
Population: All participants with evaluable data from using the wearable sensors at home at Times 0 and 12 weeks.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Conventional Therapy | Change in Physical Activity | 1.4 change in percent active time | Standard Deviation 8.9 |
| Dynamic Supported Mobility | Change in Physical Activity | -2.6 change in percent active time | Standard Deviation 11.5 |
Secondary
Change in Postural Control
Early Clinical Assessment of Balance - a clinical test of balance and postural control developed for administration with young children. The minimum score is 0. The maximum score is 100. Higher scores reflect better postural control.
Time frame: Baseline and 12 weeks
Population: All participants with baseline and 12 week scores
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Conventional Therapy | Change in Postural Control | 2.7 ECAB score units | Standard Deviation 4.7 |
| Dynamic Supported Mobility | Change in Postural Control | 4.3 ECAB score units | Standard Deviation 5.9 |