TK0117

• Research type

  Research Study

• Full title

  NONINTERVENTIONAL CLINICAL STUDY TO CONFIRM ORIGINAL SOURCES AND VERIFY SOURCE DATA FOR UNTREATED PATIENT DATA COMPILED FROM TK2D PUBLICATIONS

• IRAS ID

  349007

• Contact name

  Roshni Vara

• Contact email

  Roshni.Vara@gstt.nhs.uk

• Sponsor organisation

  UCB

• Duration of Study in the UK

  0 years, 11 months, 26 days

• Research summary

  The study sponsor UCB is developing doxecitine and doxribtimine as a treatment for patients with the condition thymidine kinase 2 deficiency (TK2d) with an age of symptom onset on or below 12 years of age.

  In the majority of prior research, untreated TK2d patients have been used as the 'control group' in the survival analysis, versus patients treated with doxecitine and doxribtimine.

  The purpose of this study is to identify the untreated patients and review their medical records to perform Source Data Verification of the data used in prior publications in preparation for regulatory inspections.

• REC name

  West Midlands - Black Country Research Ethics Committee

• REC reference

  25/WM/0015

• Date of REC Opinion

  19 Mar 2025

• REC opinion

  Further Information Favourable Opinion