Clinical trial NCT03264339: The Small Step Program - Early Intervention for Children With High Risk of Developing Cerebral Palsy
Official title: The Small Step Program - Early Intervention for Children With High Risk of Developing Cerebral Palsy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Cerebral Palsy
Keywords: Infant
Hand/Growth & Development
Motor activity
Exercise
Communication
Lead sponsor: St. Olavs Hospital
Sponsor class: OTHER
Collaborators: name: Norwegian University of Science and Technology
First posted: 2017-08-29
Enrollment: 12
Sex: ALL
Minimum age (years): 3
Maximum age (years): 8
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Single-subject research design (SSRD) will be used with each participant serving as his/her own control.
Primary purpose: TREATMENT

Brief summary: Children with cerebral palsy (CP) have life-long motor disorders caused by brain injuries which occur around birth. These children go through extensive treatment during childhood, but the treatment has generally been started late due to late diagnosis (median age 15 months). New recommendations state that the clinical diagnosis "high risk of CP" should be given before 6 months corrected age, in order to be able to intervene as early as possible, and have the best possibilities to prevent or limit the adverse neurodevelopmental consequences of brain injuries occurring around birth. Thus, there is a great need to develop evidence-based early interventions for children at high risk of developing cerebral palsy.

The Small Step program is developed at Karolinska Institutet (KI) in Stockholm, Sweden and is based upon theories of brain plasticity induced by early learning. The efficacy of the program is presently being investigated at KI. Preliminary results indicate a large individual variation in response to the program among the participating children.

The aim of the present study is therefore to explore individual responses to the Small Step Program in infants at risk of developing cerebral palsy.

The main hypothesis is that infant characteristics, such as severity of brain pathology, are associated with differential response to the program. Also, infants with absent fidgety movements and children with sporadic fidgety movements are believed to respond differently to the program.

Detailed description: The efficacy of the Small Step program is presently being investigated through a randomized controlled trial (NCT02166801) at Karolinska Institutet (KI) in Stockholm, Sweden. In the original project proposal for the current study, the plan was to contribute with inclusion of children from St. Olavs Hospital to the randomized controlled study initiated at KI. This project proposal has previously been approved by the Regional Ethical Committee (REK) for Medical Research in Mid-Norway (2016/1366). However, the researchers at KI have now completed the inclusion of participants for the randomized controlled study, and their preliminary results indicate that there is a large individual variation among the included participants regarding effect of the Small Step program. In agreement with the researchers at KI, it was therefore decided to change the design of the current study using Single Subject Research Design, a study design that is more appropriate to investigate individual response to treatment. The change in study design has been approved by REK (2016/1366-10).

Participation in this study is based on informed consent from the parents, and the child and the family participating in the project will be offered a potentially effective early intervention program that they would otherwise not be able to access.

The Small Step intervention program has three alternating treatment foci (B, C, and D) divided into five different steps, each lasting for 6 weeks (in total 30 weeks). The three treatment foci are Communication, Hand function and Mobility/gross motor function. The Hand function and Mobility steps will be conducted during two time-periods and the order will be randomised. Communication will have one intervention period during step III. The training will be conducted in the children's home by the parents on a daily basis under weekly supervision by the therapist responsible for each specific step of the intervention. General principles for the small step program are: a) collaborative goal-setting; b) promote infant's self-initiated actions; c) use of enriched home environment, d) intensity and repetition.

Before and after the intervention, there is a baseline phase with no treatment (A) and a withdrawal phase with no treatment (A), resulting in the following design: A-B-C-D-B-C-A. In addition, there is a follow-up phase when the children are 2 years of age.

Eligibility criteria: Inclusion Criteria:

* increased perinatal risk factors, such as preterm birth, hypoxic ischemic encephalopathy (asphyxia), and morphological brain abnormalities. These "at-risk" children are included in the regular clinical follow-up program at St. Olavs University Hospital comprising a standard examination at 3 months of age (for preterm born children: 3 months post term, so-called corrected age). At this examination, infants who exhibit two or more of the following additional risk factors will be eligible for study participation:

  * Neurological signs assessed with Hammersmith Infant Neurological Examination (HINE) with a cut-off score of \< 57 (20).
  * Delayed psycho-motor development measured with Alberta Infant Motor Scale (AIMS) using 2SD as the cut-off.
  * Absent or sporadic fidgety movements assessed with the General Movements Assessment (GMA)
  * Pathological findings on cerebral imaging (magnetic resonance imaging-MRI/cerebral ultrasound) done in regular clinical practice.

Exclusion Criteria:

* unstable medical condition
* progressive disorders
* diagnosis with a specific syndrome.
* neither parent is fluent in Norwegian or English. Satisfactory skills in either language are required for participation in data collection and the coaching and education program.

Primary outcomes:
measure: Peabody Developmental Motor Scales, second edition (PDMS-2)
timeFrame: up to 2 years
Secondary outcomes:
measure: Hand Assessment for Infants (HAI)
timeFrame: up to 2 years
measure: Alberta Infant Motor Scale (AIMS)
timeFrame: up to 2 years
measure: Gross Motor Function Measure (GMFM-66)
timeFrame: up to 2 years
measure: Parent-Child Early Relational Assessment (PC-ERA)
timeFrame: up to 2 years
measure: Treatment Expectancy and Credibility Survey (TCS) - modification
timeFrame: up to 2 years
Other outcomes:
measure: Hammersmith Infant Neurological Examination (HINE)
timeFrame: at baseline, after the intervention at 30 weeks and at follow-up up at 2 years
measure: The Working Model of the Child Interview (WMCI)
timeFrame: at baseline, after the intervention at 30 weeks and at follow-up up at 2 years
measure: The Ages & Stages Questionnaire: Social and Emotional (ASQ:SE)
timeFrame: at baseline, after the intervention at 30 weeks and at follow-up up at 2 years
measure: Norwegian Parenting Stress Index (PSI)
timeFrame: at baseline, after the intervention at 30 weeks and at follow-up up at 2 years
measure: The Hospital Anxiety and Depression Scale (HADS)
timeFrame: at baseline, after the intervention at 30 weeks and at follow-up up at 2 years
measure: Pediatric Evaluation of the Disability Inventory (PEDI)
timeFrame: at 2 years
measure: Bayley Scales of Infant Development (BSID-III)
timeFrame: at 2 years
measure: Norwegian Communicative Development Inventory (NCDI)
timeFrame: at 2 years
measure: Structural magnetic resonance imaging (MRI)/cerebral ultrasound
timeFrame: before 2 years corrected age
measure: General Movements Assessment (GMA)
timeFrame: at 3 months corrected age
Locations:
city: Trondheim
country: Norway
facility: St Olavs Hospital
Study officials:
affiliation: St Olavs Hospital, Children's Clinic
name: Elisabeth Selvaag, md
role: STUDY_DIRECTOR
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.