Clinical trial NCT02166801: Small Step Intervention for Infants With Cerebral Palsy and Other Neurodevelopmental Disorders
Official title: A Small-step Program for Development of New Treatment Principles for Children With Cerebral Palsy and Other Neurodevelopmental Disorders
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Gross Motor Development Delay
Cerebral Palsy
Keywords: Cerebral palsy
Children
Early Intervention
Communication
Gross motor function
Hand function
Lead sponsor: Karolinska Institutet
Sponsor class: OTHER
Collaborators: name: The Swedish Research Council
First posted: 2014-06-18
Enrollment: 39
Sex: ALL
Minimum age (years): 4
Maximum age (years): 10
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Children with cerebral palsy (CP) have life-long motor disorders and are typically subjected to extensive treatment throughout childhood. Despite this there is a lack of evidence supporting the effectiveness of treatment aiming at improving motor function and activity in daily life. The primary area of interest of this research programme is to determine the effectiveness of an early intervention program in children younger than 12 months of age who are at risk of developing CP. A randomised control trial is planned, addressing hand use, mobility and communication in a home-based program. New treatment principles based on recent knowledge of brain plasticity will be employed.

The overarching goal of this research programme is to develop and evaluate new intervention principles for children with neurodevelopmental disorders based upon theories of early learning induced brain plasticity. Our overall aims can be formulated as follows:

To evaluate the effects of an early intervention programme on the overall development in children with risk of developing cerebral palsy and other neurodevelopmental disorders. The program includes intensive intervention towards the foci: hand use, mobility and communication in a home based program

The hypothesis is that the design of the Small-Step-Program intervention, with clear foci on specific areas of development during different time periods and conducted in the child's home environment, will facilitate development and be more effective than usual care.

The second hypothesis is that children learn what they practice, meaning that children will have a more rapid development within the focus of each specific step in the training, when compared to the, for the time being, untrained steps.

The third hypothesis is that children's ability to learn within the different steps of the intervention programme will be influenced by the specific characteristics of any underlying brain pathology.

The fourth hypothesis is that parents in the study group will be less stressed and can better cope with their child's situation than parents to children receiving usual care. Thus, the tools provided within the Small-Step-Program intervention, like education, supervision and feedback of how to practice communication and task performance will make parents more able to cope with the child's delayed development.

Detailed description: Rational for subject selection: Signs of CP may be visible already during the first months, but according to recommendations the diagnosis of CP should typically not be made until 2-4 years of age. The reason is that the individual developmental trajectories of muscle tone, postural control and motor function vary considerably during the first two years. A child with a late development may recover and atypical signs may disappear, and opposite, CP may develop although there have been no early signs. Thus, there is a dilemma in that it is too late to wait with early intervention until the CP diagnosis has been made. In this project we are addressing this problem by developing criteria for infants with high risk to develop CP based on early signs of atypical motor development and on perinatal risk factors. That means that we will include both infants who will get and infants who will not get the CP diagnosis. Children who are not meeting the criteria of CP at 2 years of age will be excluded from the analysis. The investigators assume that the intervention will influence the course of development and help the children to function at a higher level, but not that the intervention will "cure" CP and prevent later diagnosis. Infants with increased risk to develop CP will be recruited in this project.

The study group will be recruited from infants exposed to increased perinatal risk factors, such as preterm birth, hypoxia, infections, retinopathy of prematurity (ROP) chronic lung disease and heart insufficiency, small for gestational age, hypoxic ischemic insult and morphological brain abnormalities. These children commonly participate in a clinical follow up programme at the children's hospital with regular examinations at 3months. The high-risk preterm infants that at follow up examinations exhibit a delayed psycho-motor development or display clinical signs of abnormal neurology will be recruited. Recruitment will also include children below 8 months, demonstrating delayed psycho-motor development or clinical signs of abnormal neurology referred to the physiotherapy department from child neurologists at the hospital.

Randomisation The aim is to include 40 children in the study group with neurodevelopmental disorder to reach sufficient power in the study. This number is high but the investigators expect that about 50% will not fulfil the diagnose criteria at 2 years of age based on previous experience from intervention studies. The aim is to include 20 children with CP, which is a reasonable approximation of study population size to reach sufficient power. The children will be allocated to group by block randomisation. The intervention group (n=20) will take part in a newly developed Small-Step-Program. The comparison group (n=20) will continue their ordinary follow up programme. Stratification will be used to control for gestational age (preterm\<37 and term\>37 week). Children in the Small-Step-Program" will be further randomised to start with the mobility step or hand use step.

Rational for second randomisation Our hypothesis is that the trained step will improve, while the non-trained step will result in minor changes and that children in the control group will have a continuous developmental pace. The randomised steps are hand use and mobility.

The children in both groups will be assessed at four occasions, at start (first examination), after 12 weeks (second examination) and after 30 weeks (third examination) as well as at 2 years of age (fourth examination). For children in the study group there will be 3 additional times for assessments in order to investigate the second hypothesis. Parents in both groups will be asked to fill in questionnaires' at all four examinations. Both parents will be asked to fill out the questionnaires.

Eligibility criteria: Inclusion Criteria:

will be based on delayed psychomotor development, as assessed by Alberta Motor Infants Scale, using -2SD as a cut-off point. Neurological signs will be investigated by Hammersmith Infant Neurological examination (HINE) together with a clinical neurological examinations. Magnetic resonance imaging (MR) will be used if available to support the risk of development of CP, only MR done for clinical purpose will be used. The risk factors suggested for inclusion in the intervention program will be confirmed by a child neurologist.

Exclusion Criteria:

is unstable health, uncontrolled epilepsy, progressive disorder, diagnosis with a specific syndrome. Parent's communication language should be Swedish or English with satisfactory skills in either language for simple conversation. If the children show a catch-up in development at the second assessment (after 12 weeks) and show no sign of neurodevelopmental disorder the program will be terminated

Primary outcomes:
measure: Peabody Developmental Motor Scales (PDMS-2) is used to measure change over time
timeFrame: Small Step group: at baseline, 6w, 12w,18w, 24w, 30w and 2 years. In the usual care group: at baseline, 12 w, 30w and at 2 years.
Secondary outcomes:
measure: Alberta Infant Motor Scale (AIMS) is used to measure change over time
timeFrame: Small Step group: at baseline, 6w, 12w,18w, 24w, 30w and 2 years. In the usual care group: at baseline, 12 w, 30w and at 2 years.
measure: Hand Assessment for Infants, (HAI) is used to measure change over time
timeFrame: Small Step group: at baseline, 6w, 12w,18w, 24w, 30w and 2 years. In the usual care group: at baseline, 12 w, 30w and at 2 years.
measure: Gross Motor Function Measure (GMFM-66) is used to measure change over time
timeFrame: Small Step group: at baseline, 6w, 12w,18w, 24w, 30w and 2 years. In the usual care group: at baseline, 12 w, 30w and at 2 years.
measure: Hammersmith Infant Neurological Examination (HINE) is used to measure change over time
timeFrame: Small Step group: at baseline, 6w, 12w,18w, 24w, 30w and 2 years. In the usual care group: at baseline, 12 w, 30w and at 2 years.
measure: Bayley Scales of Infant Development (BSID-III) is used to measure change over time
timeFrame: Two occacions in both groups: 30w and 2 years.
measure: Responsive Augmentative and Alternative Communication Style Scale (RAACS) is used to measure change over time
timeFrame: Small Step group: at baseline, 12w,18w, 30w and 2 years. In the usual care group: at baseline, 12 w, 30w and at 2 years.
measure: Swedish Parenthood Stress Questionnaire (SPSQ) is used to measure change over time
timeFrame: Both groups: baseline, 12w, 30w and at 2 years
measure: The Hospital Anxiety and Depression Scale (HADS) is used to measure change over time
timeFrame: Both groups: baseline, 12w, 30w and at 2 years
measure: The Working Model of the Child Interview, (WMCI) is used to measure change over time
timeFrame: Both groups: 30w and 2 years
measure: Swedish Early Communicative Development Inventory, SECDI is used to measure change over time
timeFrame: Both groups: 30w and 2 years
measure: Parent-Child Early Relational Assessment, PC-ERA is used to measure change over time
timeFrame: Both groups: 30w and 2 years
measure: Pediatric Evaluation of the Disability Inventory (PEDI) is used to measure change over time
timeFrame: Both groups: 30w and 2 years
measure: Parent Development Interview, PDI-R is used to measure change over time
timeFrame: 2 occacions in both groups, after intervention and at 2 year follow up
Other outcomes:
measure: Semi structured interview
timeFrame: After interventionprogram, 30w, in the Small step group
measure: Structural magnetic resonance imaging (MRI)
timeFrame: One time when the child is approximately 10-24 months
Locations:
city: Stockholm
country: Sweden
facility: Astrid Lindgren Children Hospital
Study officials:
References:
citation: Eliasson AC, Holmstrom L, Aarne P, Nakeva von Mentzer C, Weiland AL, Sjostrand L, Forssberg H, Tedroff K, Lowing K. Efficacy of the small step program in a randomised controlled trial for infants below age 12 months with clinical signs of CP; a study protocol. BMC Pediatr. 2016 Nov 3;16(1):175. doi: 10.1186/s12887-016-0711-x.