cTTP PRO instrument validation study_Protocol 281701

• Research type

  Research Study

• Full title

  Prospective psychometric evaluation study of a patient-reported outcomes (PRO) instrument for congenital thrombotic thrombocytopenic purpura (cTTP, Upshaw-Schulman Syndrome [USS], hereditary thrombotic thrombocytopenic purpura [hTTP])

• IRAS ID

  240562

• Contact name

  Marie Scully

• Contact email

  m.scully@ucl.ac.uk

• Sponsor organisation

  Shire

• Clinicaltrials.gov Identifier

  N/A, N/A

• Duration of Study in the UK

  0 years, 6 months, 1 days

• Research summary

  The overall aim of this study is to assess reliability and to validate the psychometric properties of this newly developed cTTP-specific health-related quality of life (HRQL) instrument.
  Primary objectives:
   To assess the item performance, factor structure, and psychometric properties of the newly developed congenital thrombotic thrombocytopenic purpura (cTTP)-specific patient-reported outcome (PRO) instrument
   To identify potential subscales
   To develop a provisional scoring algorithm
   To modify the conceptual framework of the cTTP PRO instrument
   To identify plans for further revision and refinement
  Secondary objective: to determine whether the cTTP-specific PRO instrument recently developed for adults is appropriate for use in adolescents (ages 12 to 17 years) with cTTP.

• REC name

  East Midlands - Derby Research Ethics Committee

• REC reference

  18/EM/0163

• Date of REC Opinion

  25 Sep 2018

• REC opinion

  Further Information Favourable Opinion