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

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/47518
• EU PAS number:
  EUPAS45205
• Study ID:
  47518
• Official title and acronym:
  Delineating a full phenotype of health, neurodevelopment and adaptive functioning in children and young adults with Fetal Valproate Spectrum Disorder.
• DARWIN EU® study:
  No
• Study countries:
  Australia

  Ireland

  New Zealand

  United Kingdom
• Study description:
  Exposure in the womb to the antiseizure medication sodium valproate (VPA) is associated with an increased risk of altered fetal development leading to a range of congenital anomalies and neurodevelopmental difficulties (Fetal Valproate Spectrum Disorder (ICD-11 LD2F.03). There is limited data available regarding the health and neurodevelopment of children and young people with Fetal Valproate Spectrum Disorder (FVSD). In younger and school age children information is extrapolated from studies investigating the risks associated with VPA exposure, but studies into older children and young people with fetal VPA exposure are extremely limited. Further, there is almost no documented evidence about health and neurodevelopmental outcomes for teenagers and young adults with the condition, particularly from the second decade of life onwards. This project aims to ascertain parent views regarding the health and neurodevelopmental difficulties experienced by children and young people with FVSD as they get older. This is a preliminary study and utilises both standardised and validated questionnaires, and a set of bespoke questions specific to later outcomes that have been developed through expert collaboration and engagement with key charities who support families who have a child with FVSD. While the findings will themselves provide key insights into the wider phenotype of FVSD in older children and young adults, the results will also aid the design of future, more detailed clinical studies regarding health and neurodevelopment in older children and young adults with FVSD, which in turn, will provide the required information to enable optimal diagnosis and intervention through clinical services.
• Study status:
  Ongoing

Research institutions and networks

Institutions

University of Manchester

United Kingdom

Study timelines

Date when funding contract was signed

Planned: 01/04/2019

Actual: 01/04/2019

Study start date

Planned: 14/02/2022

Actual: 18/02/2022

Data analysis start date

Planned: 01/06/2022

Actual: 01/09/2022

Date of final study report

Planned: 19/12/2022

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

IMI ConcePTION, Research/Clinical Salary

Study protocol

Initial protocol

EUPAS45205-45352

Updated protocol

ConcePTION WP2 Demonstration 3. Protocol Version 2. 24.01.22

Regulatory

• Was the study required by a regulatory body?:
  No
• Is the study required by a Risk Management Plan (RMP)?:
  Not applicable