Cerebral PalsyNCT02167022
Intense Physiotherapies to Improve Function in Young Children With Cerebral Palsy
A study testing Physical and occupational therapy in people with Cerebral Palsy, run by University of Arizona at 4 sites in United States.
Trial summary
Cerebral palsy (CP) is a non-progressive disorder caused by an insult or injury to the brain when the brain is most rapidly developing and which results in some motor dysfunction. Read more about this study
- Who is it for?
- Ages 12 to 36 months
- Conditions the study is for
- Cerebral Palsy
- What is being tested?
- Physical and occupational therapy
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 90 participants
- Where
- 4 sitesUnited States
- Study dates
- 30 Sep 2014to 31 Jul 2021
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 36 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age: 12 to 36 months of age (The diagnosis of CP is often uncertain under the age of 12 months. The cutoff at 36 months is to have a population of young children when the brain is most "plastic" and most susceptible to reorganization).
- Diagnosis: Diagnosis of spastic CP confirmed by a pediatric neurologist or pediatric rehabilitation specialist.
- Etiology: The insult to the central nervous system that caused the motor dysfunction must have occurred during gestation or within one year after birth independent of gestational age.
- Disease severity level: Gross Motor Function Classification System (GMFCS) levels I, II and III.
Who cannot join
- Diagnosis: Diagnosis of CP secondary to neuronal migration.
- Co-morbidities: Medical conditions that may prevent the administration of rehabilitation therapies at the intensity required by the study, or that may compromise the study ability to maintain blindness, or that have a co-morbidity not typically associated with CP (i.e. cancer, cystic fibrosis).
- Co-interventions: Anticipated pharmacological intervention or procedure or participation in other studies that may interfere with this study.
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Nemours Children's Hospital
Orlando, Florida, 32827
Phoenix Children's Hospital
Phoenix, Arizona, 85006
United Cerebral Palsy-Central Arizona
Phoenix, Arizona, 85027
Tucson Medical Center
Tucson, Arizona, 85712
About this study
Cerebral palsy (CP) is a non-progressive disorder caused by an insult or injury to the brain when the brain is most rapidly developing and which results in some motor dysfunction. Causes for the injury to the brain are numerous and can occur prior to birth, during the birth process, or within the first few months following birth. The motor dysfunction can involve any or all four extremities but most often affects the legs, causing abnormal ambulation. The level of severity depends on the extent of the injury to the brain and can be mild to severe. In severe instances, the child is dependent on others for all his/her care.
There is no known cure for CP, but physical and occupational therapies are administered in an attempt to improve function. The frequency of these therapies varies from once a week (the standard of care in the Western Hemisphere) to five times a week (the standard of care in Asia and some Eastern European countries). The current understanding of brain plasticity offers a theoretical explanation to justify the more intense approach. Active repetitive motor skill-directed rehabilitation utilizes the plasticity of the brain and can restore some function. Intense active physiotherapy can stimulate non-injured but 'dormant' neurons and prevent their 'natural' degeneration in order for them to substitute for the function of injured neurons. It is the very young brain that is most likely to respond to this therapy.
Read more
The aim of this proposal is to evaluate the effect of administering both physical and occupational therapy five times each week for 12 weeks and compare it with the standard of care (SOC) approach of one time each week in children between the ages of 12 months and 36 months. This is the first randomized crossover trial to both enroll this young a population of children with cerebral palsy as well as to evaluate this approach from both the therapists and the parents perspectives. The number of children that this study will enroll is larger than in most CP studies. The children will be evaluated clinically with two validated instruments, one of which was designed specifically for children with CP and is administered and scored by certified therapists and the other which was designed for children with developmental disabilities and is scored by the child's care provider. A sub-set of children will have a special type of MRI to evaluate any changes in the neurological structure of the brain.
The Department of Pediatrics at the University of Arizona recently completed a collaborative study with the Neurologic Department at the Beijing Children's Hospital where the intense approach of five therapies per week is the SOC. The positive results prompted another investigation to determine if such an approach would be feasible in the United States. A compliance rate of 81% confirmed feasibility and the perception that parents who have a child diagnosed with CP will do whatever they can to improve their child's motor function.
Read the detailed description
Specific Aim #1: To compare the effectiveness of an intense physiotherapy program with the current standard of care in the management of children with spastic cerebral palsy.
Hypothesis #1: Children receiving a short cluster of intense therapies (5 times per week for 12 weeks) will show greater functional gains as determined by the Gross Motor Function Measure (GMFM-66) and the Pediatric Evaluation Disability Inventory (PEDI) than those receiving the same therapies once a week, the current standard of care.
Specific Aim #2: To determine if the functional gains by children with spastic cerebral palsy achieved with an intense physiotherapy program will continue to improve while receiving less intense standard weekly therapies for at least 36 weeks (9 months) following completion of the intense program.
Hypothesis #2: Motor skills gained after 12 weeks of intensely administered physiotherapies as determined by the GMFM and the PEDI will continue to improve at a rate greater than that seen in children receiving the same therapies once a week, the current standard of care.
Specific Aim #3: After receiving the same number of therapies at the end of the 48-week protocol, children receiving the intense series of therapies during the first 12-weeks will have made greater functional gains than those receiving them during the last 12-weeks.
Hypothesis #3: Children who receive intense physiotherapies at an earlier age make greater gains then those who receive the same therapies at a later age.
Specific Aim #4: To develop a clinical profile that will identify those children most likely to benefit from intensely administered physiotherapies.
Hypothesis #4: Clinical and radiologic characteristics play a major role in response to therapy.
Explorative Aim: To assess the correlation between clinical improvement from intense physiotherapies and anatomical changes using magnetic resonance imaging (MRI).
Hypothesis: The improvements from intense physiotherapies seen in the clinical assessments will be reflected by alterations of brain connectivity parameters available from neuroanatomical MRI and diffusion tensor imaging (DTI).
- Conditions
- Cerebral Palsy
- Being tested
- Physical and occupational therapy
- Keywords
- intense therapy, brain plasticity, Perception-Action approach
- Medical subject headings
- Cerebral Palsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Physical and occupational therapyOther
The type of physiotherapy to be administered is Perception-Action (P-A). The P-A (motor) intervention is a child-driven approach without the need for special equipment and considers the child and environment as a single unit.Treatment is a coupling of the child with the environment. The environment is changed to enable, enhance or stimulate movement. The action is initiated by the child with the therapist augmenting the child's environment using touch (tactile information) or light support (cushions, bolsters, and gentle touch or pressure) to assist and/or alter the child's active movement. Toys are used and positioned in different places to encourage movement of upper and lower extremities and the trunk; reaching, turning, sitting balance, crawling, walking.
| Study group | Role | What they receive |
|---|---|---|
| Intense Physiotherapy (Group 1) | Experimental | 30 minutes each of physical and occupational therapy each weekday for 12 weeks (intense physiotherapy) followed by the same therapies administered once a week for 36 weeks (the current standard of care). Receives: Other: Physical and occupational therapy |
| Delayed Intense Physiotherapy (Group 2) | Experimental | 30 minutes each of physical and occupational therapy once a week for 36 weeks (the current standard of care) followed by the same therapies administered each weekday for 12 weeks (intense physiotherapy). Receives: Other: Physical and occupational therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Gross Motor Function Measure-66 (GMFM-66)
The primary aim is to assess function. The evaluation instruments we chose are standard in CP research and include an assessment by a certified therapist of Gross Motor Function Measure-66 (GMFM-66) and a parental assessment of the child's functional abilities in the home environment (PEDI-FS). For the primary and secondary outcomes, these endpoints will be assessed at baseline and at 12 weeks as well as every 12 weeks for the next 36 weeks. The evaluation at 48 weeks after the completion of the intense schedule allows an estimation of the persistence of change.
The GMFM-66 is a criterion referenced validated instrument that has been shown to detect change in gross motor function (performance) of children with CP as a result of various interventions. The 66 test items have been grouped into five dimensions: (1) lying and rolling; (2) crawling and kneeling; (3) sitting, standing and walking; (4) running, and (5) jumping.
Measured: Change from Baseline at 12 weeks, 24 weeks, 36 weeks, and 48 weeks
Secondary outcomes (1)
Change in Pediatric Evaluation of Disability Inventory-Functional Skills (PEDI-FS)
The primary aim is to assess function. The evaluation instruments we chose are standard in CP research and include an assessment by a certified therapist of Gross Motor Function Measure-66 (GMFM-66) and a parental assessment of the child's functional abilities in the home environment (PEDI-FS). For the primary and secondary outcomes, these endpoints will be assessed at baseline and at 12 weeks as well as every 12 weeks for the next 36 weeks. The evaluation at 48 weeks after the completion of the intense schedule allows an estimation of the persistence of change.
The PEDI-FS is an instrument developed specifically for children from 6 months through 7 years of age who have some type of a disability. It is a child's primary care provider's assessment of what skills the child is able to perform. The skills are grouped into three domains: (1) self-care, (2) mobility, and (3) social function.
Measured: Change from Baseline at 12 weeks, 24 weeks, 36 weeks, and 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jun 2014 (estimated)
- Study start30 Sep 2014
- Primary completion31 Jul 2021
- Study completion31 Jul 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cerebral PalsyStudies of the same condition, open and under way.
- More trials run by University of ArizonaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 90 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Arizona
- Sponsor type
- University, hospital or other
- Collaborators
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), Tucson Medical Center, Phoenix Children's Hospital, United Cerebral Palsy-Central Arizona, Nemours Children's Hospital
- Responsible party
- Sponsor
- Investigators
- Burris R Duncan, MD, Principal investigator, University of Arizona
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02167022
- Sponsor's protocol ID
- 1401195212
- Other IDs
- 1R01HD079498-01
- Status last verified
- Jan 2022
- First submitted
- 12 Jun 2014
- Last updated
- 4 Jan 2022
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 4 Jan 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intense Physiotherapies to Improve Function in Young Children With Cerebral Palsy
- ClinicalTrials.gov ID
- NCT02167022
- Acronym / sponsor ID
- 1401195212
- Conditions
- Cerebral Palsy
- Keywords
- intense therapy, brain plasticity, Perception-Action approach
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 90 participants (actual)
- Lead sponsor
- University of ArizonaUniversity, hospital or other
- Collaborators
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), Tucson Medical Center, Phoenix Children's Hospital, United Cerebral Palsy-Central Arizona, Nemours Children's Hospital
- Study start
- 30 Sep 2014 (actual)
- Primary completion
- 31 Jul 2021 (actual)
- Study completion
- 31 Jul 2021 (actual)
- First posted
- 18 Jun 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Cerebral palsy (CP) is a non-progressive disorder caused by an insult or injury to the brain when the brain is most rapidly developing and which results in some motor dysfunction. Causes for the injury to the brain are numerous and can occur prior to birth, during the birth process, or within the first few months following birth. The motor dysfunction can involve any or all four extremities but most often affects the legs, causing abnormal ambulation. The level of severity depends on the extent of the injury to the brain and can be mild to severe. In severe instances, the child is dependent on others for all his/her care.
There is no known cure for CP, but physical and occupational therapies are administered in an attempt to improve function. The frequency of these therapies varies from once a week (the standard of care in the Western Hemisphere) to five times a week (the standard of care in Asia and some Eastern European countries). The current understanding of brain plasticity offers a theoretical explanation to justify the more intense approach. Active repetitive motor skill-directed rehabilitation utilizes the plasticity of the brain and can restore some function. Intense active physiotherapy can stimulate non-injured but 'dormant' neurons and prevent their 'natural' degeneration in order for them to substitute for the function of injured neurons. It is the very young brain that is most likely to respond to this therapy.
The aim of this proposal is to evaluate the effect of administering both physical and occupational therapy five times each week for 12 weeks and compare it with the standard of care (SOC) approach of one time each week in children between the ages of 12 months and 36 months. This is the first randomized crossover trial to both enroll this young a population of children with cerebral palsy as well as to evaluate this approach from both the therapists and the parents perspectives. The number of children that this study will enroll is larger than in most CP studies. The children will be evaluated clinically with two validated instruments, one of which was designed specifically for children with CP and is administered and scored by certified therapists and the other which was designed for children with developmental disabilities and is scored by the child's care provider. A sub-set of children will have a special type of MRI to evaluate any changes in the neurological structure of the brain.
The Department of Pediatrics at the University of Arizona recently completed a collaborative study with the Neurologic Department at the Beijing Children's Hospital where the intense approach of five therapies per week is the SOC. The positive results prompted another investigation to determine if such an approach would be feasible in the United States. A compliance rate of 81% confirmed feasibility and the perception that parents who have a child diagnosed with CP will do whatever they can to improve their child's motor function.
Detailed description
Specific Aim #1: To compare the effectiveness of an intense physiotherapy program with the current standard of care in the management of children with spastic cerebral palsy.
Hypothesis #1: Children receiving a short cluster of intense therapies (5 times per week for 12 weeks) will show greater functional gains as determined by the Gross Motor Function Measure (GMFM-66) and the Pediatric Evaluation Disability Inventory (PEDI) than those receiving the same therapies once a week, the current standard of care.
Specific Aim #2: To determine if the functional gains by children with spastic cerebral palsy achieved with an intense physiotherapy program will continue to improve while receiving less intense standard weekly therapies for at least 36 weeks (9 months) following completion of the intense program.
Hypothesis #2: Motor skills gained after 12 weeks of intensely administered physiotherapies as determined by the GMFM and the PEDI will continue to improve at a rate greater than that seen in children receiving the same therapies once a week, the current standard of care.
Specific Aim #3: After receiving the same number of therapies at the end of the 48-week protocol, children receiving the intense series of therapies during the first 12-weeks will have made greater functional gains than those receiving them during the last 12-weeks.
Hypothesis #3: Children who receive intense physiotherapies at an earlier age make greater gains then those who receive the same therapies at a later age.
Specific Aim #4: To develop a clinical profile that will identify those children most likely to benefit from intensely administered physiotherapies.
Hypothesis #4: Clinical and radiologic characteristics play a major role in response to therapy.
Explorative Aim: To assess the correlation between clinical improvement from intense physiotherapies and anatomical changes using magnetic resonance imaging (MRI).
Hypothesis: The improvements from intense physiotherapies seen in the clinical assessments will be reflected by alterations of brain connectivity parameters available from neuroanatomical MRI and diffusion tensor imaging (DTI).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Intense Physiotherapy (Group 1) | Experimental | Other: Physical and occupational therapy30 minutes each of physical and occupational therapy each weekday for 12 weeks (intense physiotherapy) followed by the same therapies administered once a week for 36 weeks (the current standard of care). |
| Delayed Intense Physiotherapy (Group 2) | Experimental | Other: Physical and occupational therapy30 minutes each of physical and occupational therapy once a week for 36 weeks (the current standard of care) followed by the same therapies administered each weekday for 12 weeks (intense physiotherapy). |
Who may qualify
- Ages
- 12 to 36 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Age: 12 to 36 months of age (The diagnosis of CP is often uncertain under the age of 12 months. The cutoff at 36 months is to have a population of young children when the brain is most "plastic" and most susceptible to reorganization).
- Diagnosis: Diagnosis of spastic CP confirmed by a pediatric neurologist or pediatric rehabilitation specialist.
- Etiology: The insult to the central nervous system that caused the motor dysfunction must have occurred during gestation or within one year after birth independent of gestational age.
- Disease severity level: Gross Motor Function Classification System (GMFCS) levels I, II and III.
Key exclusion criteria (3 listed)
- Diagnosis: Diagnosis of CP secondary to neuronal migration.
- Co-morbidities: Medical conditions that may prevent the administration of rehabilitation therapies at the intensity required by the study, or that may compromise the study ability to maintain blindness, or that have a co-morbidity not typically associated with CP (i.e. cancer, cystic fibrosis).
- Co-interventions: Anticipated pharmacological intervention or procedure or participation in other studies that may interfere with this study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Physical and occupational therapyThe type of physiotherapy to be administered is Perception-Action (P-A). The P-A (motor) intervention is a child-driven approach without the need for special equipment and considers the child and environment as a single unit.Treatment is a coupling of the child with the environment. The environment is changed to enable, enhance or stimulate movement. The action is initiated by the child with the therapist augmenting the child's environment using touch (tactile information) or light support (cushions, bolsters, and gentle touch or pressure) to assist and/or alter the child's active movement. Toys are used and positioned in different places to encourage movement of upper and lower extremities and the trunk; reaching, turning, sitting balance, crawling, walking. | Other | Delayed Intense Physiotherapy (Group 2), Intense Physiotherapy (Group 1) |
Primary outcome
Change in Gross Motor Function Measure-66 (GMFM-66)
Time frame: Change from Baseline at 12 weeks, 24 weeks, 36 weeks, and 48 weeks
The primary aim is to assess function. The evaluation instruments we chose are standard in CP research and include an assessment by a certified therapist of Gross Motor Function Measure-66 (GMFM-66) and a parental assessment of the child's functional abilities in the home environment (PEDI-FS). For the primary and secondary outcomes, these endpoints will be assessed at baseline and at 12 weeks as well as every 12 weeks for the next 36 weeks. The evaluation at 48 weeks after the completion of the intense schedule allows an estimation of the persistence of change. The GMFM-66 is a criterion referenced validated instrument that has been shown to detect change in gross motor function (performance) of children with CP as a result of various interventions. The 66 test items have been grouped into five dimensions: (1) lying and rolling; (2) crawling and kneeling; (3) sitting, standing and walking; (4) running, and (5) jumping.
1 secondary outcome
Change in Pediatric Evaluation of Disability Inventory-Functional Skills (PEDI-FS)
Time frame: Change from Baseline at 12 weeks, 24 weeks, 36 weeks, and 48 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jan 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Burris R Duncan, MD principal investigator
University of Arizona
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Physical and occupational therapy in people with Cerebral Palsy, run by University of Arizona at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Jan 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Gross Motor Function Measure-66 (GMFM-66) (time frame: Change from Baseline at 12 weeks, 24 weeks, 36 weeks, and 48 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Jan 2022.