Delineating a full phenotype of health, neurodevelopment and adaptive functioning in children and young adults with Fetal Valproate Spectrum Disorder.

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Disease epidemiology

  Other
• If ‘other’, further details on the scope of the study:
  Phenotyping Study

Non-interventional study

• Non-interventional study design:
  Cohort

  Cross-sectional

Study drug and medical condition

• Anatomical Therapeutic Chemical (ATC) code:
  (N03AG01) valproic acid

  valproic acid
• Medical condition to be studied:
  Foetal anticonvulsant syndrome
• Additional medical condition(s):
  Fetal Valproate Spectrum Disorder

Population studied

• Age groups:
  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)

  Adults (18 to < 46 years)
• Estimated number of subjects:
  114

Study design details

• Main study objective:
  To ascertain maternal views regarding the health, neurodevelopment and adaptive functioning of children and young adults with Fetal Valproate Spectrum Disorder in order to establish the phenotype of FVSD in older children and young adults.
• Outcomes:
  Parent reported cognitive development as measured by the Patient Reported Outcome Measurement Information System (PROMIS) Cognitive Function Item Bank (custom short-form). The following outcomes will also be explored: - Physical Health - Neurodevelopmental disorders - Service utilization - Academic Functioning & Educational Outcomes - Social Development - Emotional & Behavioral Development - Sensory Issues - Employment (16+ only) - Independence & Daily Living
• Data analysis plan:
  Data will be explored and described in terms of means, frequencies and percentages for the FVSD and comparison groups. This data will be inspected to develop a preliminary conceptualization of the FVSD phenotype. Differences within the FVSD group by age, sex and dose will also be explored. Appropriate statistical tests (e.g. t-test and Chi-square) will then be used to examine differences between the group with FVSD and the control group and, should large enough numbers be obtained, analysis adjusting for confounder and moderating and/or mediating factors will be undertaken (e.g. multiple linear and logistic regression analyses). Data from each age group will be pooled for analysis where appropriate and group differences will be explored using either total/mean scores (for scales measuring an underlying latent trait) or frequencies (for checklists of directly observable variables).