Ignite Creation Date:
2024-05-06 @ 6:55 PM
Last Modification Date:
2024-10-26 @ 2:57 PM
Study NCT ID:
NCT05835674
Status:
RECRUITING
Last Update Posted:
2023-04-28
First Post:
2023-03-31
Brief Title:
CP-EDIT Cerebral Palsy - Early Diagnosis and Intervention Trial
Sponsor:
Rigshospitalet Denmark
Organization:
Rigshospitalet Denmark
Study Overview
Official Title:
Multi-center Prospective Cohort Study CP-EDIT Cerebral Palsy - Early Diagnosis and Intervention Trial
Status:
RECRUITING
Status Verified Date:
2023-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
CP-EDIT
Brief Summary:
Background Early diagnosis of cerebral palsy is important as intervention becomes possible at a time where neuroplasticity is at the highest Current mean age at diagnosis is 13 months in Denmark Recent research has documented that implementation of an early-diagnosis set-up can lower diagnostic age of cerebral palsy The aim of the current study is to show that the response to the early intervention program added to standard care is superior to standard care alone in a Danish multi-site setting in children from both a newborn and infant detectable risk pathway
Methods The current study CP-EDIT Early Diagnosis and Intervention Trial with the GO-PLAY intervention included Goal Oriented ParentaL supported home ActivitY program aims at testing feasibility of an early diagnosis and intervention set-up in four paediatric centers In a prospective cohort study design we will consecutively include a total of 500 infants We will systematically collect data at inclusion and follow a subset of participants with definite cerebral palsy or high risk of cerebral palsy until they are two years of age The focus is on eight areas related to implementation and the perspective of the families Early MRI early genetic testing implementation of the General Movements Assessment method early prediction of cerebral palsy comparative analysis of the Hand Assessment for Infants method and evaluation by Hammersmith Infant Neurological Examination MRI and the General Movements method analysis of the GO-PLAY early intervention parental perspective of early intervention and parental perspective of having an early diagnosis
Discussion Early screening for CP is increasingly possible and an interim diagnosis of high risk of CP is recommended but not currently used in our clinical care There is a need to accelerate identification in mild or ambiguous cases to facilitate appropriate therapy early The majority of studies on early diagnosis focus on identifying CP in infants below five months corrected age Little is known about early diagnosis in the 50 of all CP cases that are discernible later in infancy which is also addressed in this study The study aims at improving care of patients with cerebral palsy even before they have the diagnosis established
Methods The current study CP-EDIT Early Diagnosis and Intervention Trial with the GO-PLAY intervention included Goal Oriented ParentaL supported home ActivitY program aims at testing feasibility of an early diagnosis and intervention set-up in four paediatric centers In a prospective cohort study design we will consecutively include a total of 500 infants We will systematically collect data at inclusion and follow a subset of participants with definite cerebral palsy or high risk of cerebral palsy until they are two years of age The focus is on eight areas related to implementation and the perspective of the families Early MRI early genetic testing implementation of the General Movements Assessment method early prediction of cerebral palsy comparative analysis of the Hand Assessment for Infants method and evaluation by Hammersmith Infant Neurological Examination MRI and the General Movements method analysis of the GO-PLAY early intervention parental perspective of early intervention and parental perspective of having an early diagnosis
Discussion Early screening for CP is increasingly possible and an interim diagnosis of high risk of CP is recommended but not currently used in our clinical care There is a need to accelerate identification in mild or ambiguous cases to facilitate appropriate therapy early The majority of studies on early diagnosis focus on identifying CP in infants below five months corrected age Little is known about early diagnosis in the 50 of all CP cases that are discernible later in infancy which is also addressed in this study The study aims at improving care of patients with cerebral palsy even before they have the diagnosis established
Detailed Description:
Cerebral palsy CP is the most common cause of lifelong motor impairment in children and is defined by the Surveillance of Cerebral Palsy in Europe SCPE as a group of permanent but not unchanging disorders of movement andor posture and of motor function which are due to a non-progressive interference lesion or abnormality of the developingimmature brain An early diagnosis is important in order to start relevant intervention when neuroplasticity is highest Research suggests that early intervention programs have a positive influence on cognitive and motor outcomes and is a parental request
In 2004 the Danish Cerebral Palsy Follow-up Program CPOP was introduced with the aim of establishing a standardized follow-up program The CPOP guarantees that children with CP are followed annually and aims to document monitor and improve the quality of health care for children with CP in Denmark A previous Danish nationwide study from birth years 1995-2003 found that the median corrected diagnostic age of Danish children with CP was 11 months based on first mention in the patient journal In an novel analysis we have found that for children with CP in birth years 2010-2013 the diagnostic age was 13 months based on first registered diagnosis The best predictive tools for early diagnosis of CP has been found to be the Prechtl Qualitative Assessment of General Movements GMA Hammersmith Infant Neurological Examination HINE and cerebral MRI These tools can be used to diagnose CP before the corrected age of five months The method Hand Assessment for Infants HAI has in recent publications been found promising to complement these tests but little evidence on HAI has been published Feasibility of an early-diagnosis set-up of a systematic approach with neurological assessment GMAHINE and neuroimaging has been evaluated in several studies but not yet in Denmark and not in a setting where both the newborn- and infant-detectable risk pathways were included Neonates may have obvious risk factors at birthin the neonatal period but in the present study infants with findings suggesting CP emerging in the first year of life are also included
For more than 80 of children with CP neuroimaging performed with MRI is abnormal It has been recommended for many years to use MRI while diagnosing CP A review from 2007 showed that 86 of the MRIs performed on children with CP were abnormal and for the majority gave clues to the pathogenesis of CP In a previous Australian study of children with CP specific MRI patterns varied depending on parity gestational age level of neonatal care Apgar score and time to established respiration Diagnostic MRI of newborns and infants with suspected brain damage are today based on structural images with the focus on identifying major structural brain pathology such as periventricular leukomalacia or white matter damage However advanced research MRI-sequences can be sensitive to detect brain damage and may provide important biological information about myelination microstructure and connectivity of the white matter fibre tracts as well as the metabolic profile including markers of neuronal integrity and glial markers of the brain tissue MRI will be offered earlier than regular clinical practice for participants in this research project and for a subset of patients repeat MRI will be possible
Many gene variants may play a role in the development of CP Previous research with genetic testing has shown that genetic aetiology while often seen in children with no previous gestational or neonatal risk factor is not limited to this group nor those with other neurodevelopmental comorbidities such as epilepsy or intellectual disability One study has suggested an association between CP and genes that hinder early brain development and also predispose to susceptibility to environmental risk factors The cause for the various CP types including the most prevalent spastic subtype and the rare ataxic or dyskinetic CP are different In some cases the cause is obvious such as asphyxia during labour cerebral bleeding or infection but in many cases the cause is unknown However in an increasing number of patients it is now possible to identify the underlying cause since it has become clear that several treatable metabolic and genetic diseases such as dopa-responsive dystonia may be misdiagnosed as CP Identifying the cause makes it possible to provide genetic counselling and direct specific treatment eg with nutrient-specific diets or medications which can interrupt disease progression and hinder further injury In the current study extensive genetic testing with whole genome sequencing will be performed in participants with definite CP or high risk of CP
Receiving an early diagnosis of CP or high risk of CP is a parent priority and both parents and care providers agree that early access to interventions is important for the child Parents of children with CP may experience high stress levels depression and chronic sorrow symptoms Intensive home-based approaches addressing cognitive and motor improvement increasingly involve parents as treatment providers supervised by therapists This can lead to both a positive parental outcome such as motivation knowledge and increased competences as well as negative outcomes because parents may be overwhelmed by the burden of responsibility and the feeling of insufficiency and lack of confidence
The current study CP-EDIT Early Diagnosis and Intervention Trial with the GO-PLAY intervention included Goal Oriented ParentaL supported home ActivitY program that will be registered separately at ClinTrials aims at testing feasibility of an early diagnosis and intervention set-up in four neuropaediatric centers in Denmark with focus on eight areas related to implementation and the perspective of the families Early MRI early genetic testing implementation of the GMA method early prediction of CP comparative analysis of the HAI and HINE MRI and GMA methods analysis of the GO-PLAY early intervention and parental perspective of intervention and early diagnosis
In 2004 the Danish Cerebral Palsy Follow-up Program CPOP was introduced with the aim of establishing a standardized follow-up program The CPOP guarantees that children with CP are followed annually and aims to document monitor and improve the quality of health care for children with CP in Denmark A previous Danish nationwide study from birth years 1995-2003 found that the median corrected diagnostic age of Danish children with CP was 11 months based on first mention in the patient journal In an novel analysis we have found that for children with CP in birth years 2010-2013 the diagnostic age was 13 months based on first registered diagnosis The best predictive tools for early diagnosis of CP has been found to be the Prechtl Qualitative Assessment of General Movements GMA Hammersmith Infant Neurological Examination HINE and cerebral MRI These tools can be used to diagnose CP before the corrected age of five months The method Hand Assessment for Infants HAI has in recent publications been found promising to complement these tests but little evidence on HAI has been published Feasibility of an early-diagnosis set-up of a systematic approach with neurological assessment GMAHINE and neuroimaging has been evaluated in several studies but not yet in Denmark and not in a setting where both the newborn- and infant-detectable risk pathways were included Neonates may have obvious risk factors at birthin the neonatal period but in the present study infants with findings suggesting CP emerging in the first year of life are also included
For more than 80 of children with CP neuroimaging performed with MRI is abnormal It has been recommended for many years to use MRI while diagnosing CP A review from 2007 showed that 86 of the MRIs performed on children with CP were abnormal and for the majority gave clues to the pathogenesis of CP In a previous Australian study of children with CP specific MRI patterns varied depending on parity gestational age level of neonatal care Apgar score and time to established respiration Diagnostic MRI of newborns and infants with suspected brain damage are today based on structural images with the focus on identifying major structural brain pathology such as periventricular leukomalacia or white matter damage However advanced research MRI-sequences can be sensitive to detect brain damage and may provide important biological information about myelination microstructure and connectivity of the white matter fibre tracts as well as the metabolic profile including markers of neuronal integrity and glial markers of the brain tissue MRI will be offered earlier than regular clinical practice for participants in this research project and for a subset of patients repeat MRI will be possible
Many gene variants may play a role in the development of CP Previous research with genetic testing has shown that genetic aetiology while often seen in children with no previous gestational or neonatal risk factor is not limited to this group nor those with other neurodevelopmental comorbidities such as epilepsy or intellectual disability One study has suggested an association between CP and genes that hinder early brain development and also predispose to susceptibility to environmental risk factors The cause for the various CP types including the most prevalent spastic subtype and the rare ataxic or dyskinetic CP are different In some cases the cause is obvious such as asphyxia during labour cerebral bleeding or infection but in many cases the cause is unknown However in an increasing number of patients it is now possible to identify the underlying cause since it has become clear that several treatable metabolic and genetic diseases such as dopa-responsive dystonia may be misdiagnosed as CP Identifying the cause makes it possible to provide genetic counselling and direct specific treatment eg with nutrient-specific diets or medications which can interrupt disease progression and hinder further injury In the current study extensive genetic testing with whole genome sequencing will be performed in participants with definite CP or high risk of CP
Receiving an early diagnosis of CP or high risk of CP is a parent priority and both parents and care providers agree that early access to interventions is important for the child Parents of children with CP may experience high stress levels depression and chronic sorrow symptoms Intensive home-based approaches addressing cognitive and motor improvement increasingly involve parents as treatment providers supervised by therapists This can lead to both a positive parental outcome such as motivation knowledge and increased competences as well as negative outcomes because parents may be overwhelmed by the burden of responsibility and the feeling of insufficiency and lack of confidence
The current study CP-EDIT Early Diagnosis and Intervention Trial with the GO-PLAY intervention included Goal Oriented ParentaL supported home ActivitY program that will be registered separately at ClinTrials aims at testing feasibility of an early diagnosis and intervention set-up in four neuropaediatric centers in Denmark with focus on eight areas related to implementation and the perspective of the families Early MRI early genetic testing implementation of the GMA method early prediction of CP comparative analysis of the HAI and HINE MRI and GMA methods analysis of the GO-PLAY early intervention and parental perspective of intervention and early diagnosis
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None