MRI Techniques in TSC v1.0

• Research type

  Research Study

• Full title

  MRI Techniques in patients with Tuberous Sclerosis Complex

• IRAS ID

  344415

• Contact name

  Emily Redpath

• Contact email

  emily.redpath@stgeorges.nhs.uk

• Sponsor organisation

  St Georges University Hospitals NHS Foundation Trust

• Duration of Study in the UK

  9 years, 11 months, 31 days

• Research summary

  This study will enable the development of new magnetic resonance (MR) imaging protocols for scanning patients who have a rare disease called Tuberous Sclerosis Complex (TSC) and particularly those who have developed benign tumours in the kidneys called angio-myo-lipoma (AMLs). AMLs tend to grow in size and vascularity over time, which increases the risk that they will spontaneously haemorrhage. This can be very dangerous and even life-threatening for patients. A drug called Everolimus was introduced to standard care for patients with large AMLs in 2016. In clinical trials (Bissler et al. Lancet 2013), Everolimus was shown to shrink AMLs and reduce the risk of bleeding.
  We care for a large cohort of these patients at St George’s NHS Foundation Trust and Brighton University Hospitals, many of whom are being treated with Everolimus. These patients have scans at regular intervals, and doctors measure the size of the AMLs on these scans to assess whether each patient is responding to treatment. However, size is not a sufficient response criterion for all patients - some patients’ AMLs do not shrink, but doctors suspect that the treatment may still be working. Furthermore, many patients who have TSC experience learning difficulties and can find typical MRI scans very uncomfortable as they tend to be lengthy, require breath-holds and may require contrast injection. In this study, new MR techniques for motion-robustness, acceleration, and quantitation will be assessed with the aim of producing a new, objective measure of response to Everolimus treatment in scans which are comfortable for patients.

• REC name

  Wales REC 5

• REC reference

  25/WA/0057

• Date of REC Opinion

  27 Feb 2025

• REC opinion

  Favourable Opinion