UK Clinical Trial update


March 2018

There had never been a clinical trial of a potential treatment for Rett Syndrome in the UK until the Sarizotan trial started at the Clinical Research Facility, King’s College Hospital in London last year.

The drug is being trialled for the treatment of breathing irregularities in Rett Syndrome. Many children and adults with Rett struggle to breathe properly throughout most of their waking hours which not only affects their day to day life but can also lead to life threatening complications.

There are currently no approved drugs to treat this or any other symptom of Rett.


One year on....

Since the trial was launched in April 2017, 25 patients have been identified and pre-screened by Reverse Rett. This has been a major effort, critical to the trial’s success.


Reverse Rett continue to support the trial by:

  • Funding a dedicated Clinical Trial Co-ordinator based at the trial site since Nov 2016
  • Recruiting patients from our UK Rett Syndrome Patient Registry for pre-screening
  • Pre-screening patients for subsequent referral to trial site
  • Working to support retention of patients on the trial through logistical support, making arrangements for travel, accommodation and meal expenses for participating patients and families


Looking after trial participants

Study days on the trial can be long, especially at the beginning. Many tests are needed, including eye tests, blood, urine tests and repeated ECGs and vitals.

Bloods can be particularly difficult to draw from patients with Rett Syndrome and there are study days when several blood draws are needed at different times in the day.

The team at the trial site do as much as they can to make sure the patient is as comfortable as possible. Reverse Rett have supported by providing a dedicated Vein Viewer device, Emla numbing cream, Tegaderm dressings and Newcastle collection kits to the trial site, all to care for participants in this vital trial. 


‘Working to support the families on the clinical trial with travel and accommodation arrangements over the last year has been rewarding. The patients and families are all different and have different requirements but they all have one thing in common, an absolute drive to bring about the best possible outcome for the person they love.

Some participants live relatively close to the trial site but others travel from far and wide and their arrangements can be much more extensive. Being in a clinical trial can be hard enough. We work with families to ensure their needs are met and that the difficulties implicit in taking part are reduced wherever possible.’

Andy Stevenson, Reverse Rett Co-Founder, Clinical Trial Logistical Lead


Register your child or loved one on the UK Rett Syndrome Patient Registry today.