2017_LBL_NIS_01

• Research type

  Research Study

• Full title

  Cerebrotendinous Xanthomatosis Registry: Long term Non-Interventional Follow-up of Safety and Effectiveness of Chenodeoxycholic Acid Leadiant.

• IRAS ID

  297469

• Contact name

  Karolina Stepien

• Contact email

  Karolina.stepien@srft.nhs.uk

• Sponsor organisation

  Leadiant Biosciences Ltd.

• Duration of Study in the UK

  5 years, 0 months, 1 days

• Research summary

  This is a patient registry project which aims to provide information about the long-term effectiveness and safety of CDCA in the CTX patient population. The design is
  observational, non-interventional, follow-up of CTX patients treated with
  Chenodeoxycholic Acid Leadiant. Data will be collected from patients treated with
  Chenodeoxycholic Leadiant at centres in Europe during routine clinical practice. The
  study design is non-interventional with no randomization or blinding. No additional
  follow-up or tests will be specified. Data collection will be prospective, with some
  retrospective data retrieval from patient records where necessary.

• REC name

  South West - Central Bristol Research Ethics Committee

• REC reference

  21/SW/0091

• Date of REC Opinion

  25 Aug 2021

• REC opinion

  Further Information Favourable Opinion