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Dr. Timothy Barrett’s Update | University of Birmingham, United Kingdom

Treat Wolfram

Dr. Timothy Barrett’s Update | University of Birmingham, United Kingdom

Dear friends and colleagues,

I hope everyone is keeping OK. I have 3 items to update you on.

Firstly, the TREATWOLFRAM trial is continuing to progress well. We had an independent Data Monitoring Committee meeting in January. This is chaired by Professor Karen Morrison, an honorary consultant in adult neurology, who previously led the highly specialised service for adults with Wolfram syndrome. The Data Monitoring Committee reviewed the unblinded data. I am pleased to say that they had no safety concerns, and were happy for the trial to continue to completion. Following this meeting, our Trial Steering Committee met, chaired by Professor Marc Peschanski, Director of INSERM, a large research institute outside Paris. We discussed the trial progress so far, and plans for acting on the results at the end of the trial. The last participant will complete the trial at the end of October 2024. We will then have 2-3 months to collect any outstanding data from study sites. The Clinical Trials Unit… read in full here.

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Professor Timothy G Barrett

University of Birmingham, United Kingdom

UPDATE ON TREATWOLFRAM CLINICAL TRIAL

Dear friends and colleagues, to recap, this is a trial of sodium valproate given by mouth, for 3 years, to try and slow down the progress of vision loss in Wolfram. It involves 4 countries in Europe and is comparing the effects of sodium valproate to placebo. The trial recruited its first patient in January 2019. We currently have 12 recruits at our children’s hospital site in Birmingham, looked after by my colleague Dr Renuka Dias. We had a pause due to Coronavirus, but I am very pleased to say that Dr Ben Wright has just recruited our first adult patient at the adult site in Birmingham. With lots of help from our international colleagues, we now have all the regulatory approvals in place in each country. We spoke with Dr Gema Esteban in Spain this week, and she will recruit her first patient in October. We hope to complete recruitment in June 2021.

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