A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome
This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.
CAMBRIDGE, Mass.–(BUSINESS WIRE)– Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced that the first participant has been dosed in the HELIOS study, a Phase 2 clinical trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO]) for the treatment of Wolfram syndrome (WS).
Tim Barrett update for Winter TreatWolfram newsletter
Dear friends and colleagues,
I hope everyone is keeping OK and managed to enjoy at least some good weather this Summer.
We are getting to the end of the TREATWOLFRAM trial, and the last participants will be having their last visits this October. We then hope to collect all outstanding data from study sites by the end of the year. The Clinical Trials Unit will then ‘lock’ the database so that our statistician can do the analysis. We expect to be told whether the treatment is effective or not, by the end of March or April 2025. As soon as I am told the results, I will write a report to our funder, the UK Medical Research Council; and I will of course share results through Wolfram Syndrome UK. Our trials unit team, Amy Lamb, Lewis James, Victoria Homer and Darren Barton, are working hard to make sure all the information that participants have kindly provided, will be ready to be analysed when the trial ends in October 2024.
Tim Barrett update for Winter TreatWolfram newsletter
Dear friends and colleagues,
I hope everyone is keeping OK and are keeping warm enough in this cold weather. Our research teams have kept busy and I would like to use this letter to keep you updated on our progress.
The TREATWOLFRAM trial is now in its final year. It has been a long journey to get here, navigating medicine manufacture problems, BREXIT, Covid, and supply issues. Thank you to all the participants who have supported the trial with their involvement. Our trials unit team, Amy Lamb, Lewis James Victoria Homer and Darren Barton, are working hard to make sure all the information that participants have kindly supplied, will be ready to be analysed when the trial ends in October 2024. There has been an update from the UK Medicines Regulator (MHRA) to remind patients and the public about the safety precautions we all need to follow for people taking sodium valproate. Anyone female under 55 years who may be taking sodium valproate, needs to have a safety check with their doctor each year. It is important that sodium valproate should not be taken by women who may be pregnant, as there is a risk of significant harm to the baby. There is helpful information on this UK Government website: https://www.gov.uk/guidance/valproate-use-by-women-and-girls.
The Snow Foundation is a collective voice for Wolfram syndrome patients, working towards a cure for Wolfram syndrome and developing novel therapies for diabetes, vision loss, hearing loss and neurodegeneration.
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