Reading back on the announcement 28/10/24 - .
“We have heard a clear message from a number of the trial investigators that they would
like to continue avicursen treatment for some of their patients beyond the formal
completion of the trial,” commented Percheron CEO, Dr James Garner. “The team has
worked closely with the sites and with external advisors to make this possible, and we
expect to be able to provide supply to patients who choose to continue, and whom
investigators feel may benefit from doing so, free of charge, for a period of time after
their participation in the trial concludes. We are grateful to the investigators, the patients
and their families for their continuing interest in avicursen.”
Such provision would be primarily on compassionate grounds rather than for the purpose of collecting additional data.
This has been on my mind since the trial readout, and I admit I’m not an expert in this area—it could just be wishful thinking on my part. However, I wanted to put this out there for anyone with more knowledge who might be able to confirm or challenge my assumptions about post-trial access. Here goes...
- Delayed onset benefits.... could avicursen take longer than 6 months to demonstrate efficacy, especially in stabilising or slowing progression. They may believe that longer exposure to avicurson could yield better outcomes than the initial six-month randomised phase?
- Could the treatment require extended time frames to show measurable benefits, particularly in muscular diseases such as DMD?
- Investigators may have seen anecdotal evidence or case-specific observations suggesting avicursen may benefit certain patients, even if the broader trial outcomes did not show statistical significance?
- While PUL scores may not have improved, there could be other endpoints (e.g., biomarkers, quality of life measures, or muscle inflammation reduction) that showed positive trends?
- Investigators might suspect subgroups of patients (e.g., those with specific genetic profiles) could benefit more significantly, even if the trial did not meet primary endpoints?
- The post-trial extension phase, during which all patients receive avicursen, could provide additional insights into its long-term effects?
- The transition to open-label use (where both participants and investigators know the treatment being given) could introduce biases but also highlight previously unseen trends?
I would like to hear others views on why this Post trial extension has been granted. To me there has to be some benefits to the boys for this request and why would the company agree to continue funding the trial if there were no benefits!
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