Questionnaire Follow-Up study

• Research type

  Research Study

• Full title

  Questionnaire Follow-Up Study (Q Follow-Up study*)

• IRAS ID

  262550

• Contact name

  Lakshminarayan Ranganath

• Contact email

  lrang@liv.ac.uk

• Sponsor organisation

  Royal Liverpool University Hospital

• Clinicaltrials.gov Identifier

  N/A, N/A

• Duration of Study in the UK

  1 years, 0 months, 1 days

• Research summary

  The Questionnaire Follow-Up Study will consist of patients who completed 4 years in the SONIA 2 study at the UK site. They will be sent a brief questionnaire to understand the state of their health since leaving the study.

  Summary of results
  Study drug nitisinone was not made available at end of SONIA 2 clinical trial. Access to nitisinone post-study was difficult and limited despite proper planning. Pain increased in those patients on nitisinone in SONIA 2 after SONIA 2 study cpmpletion and nitisinone withdrawal. In those who accessed nitisinone post-SONIA 2, pain decreased promptly without weeks. Response rate in the questionnaire follow-up study was reasonable considering that it was a postal questionnaire tarns-national study.

• REC name

  East Midlands - Derby Research Ethics Committee

• REC reference

  19/EM/0204

• Date of REC Opinion

  10 Jun 2019

• REC opinion

  Favourable Opinion