We were delighted to learn recently that the very first patient has now been dosed in the Phase 3 KHENERFIN clinical trial evaluating sonlicromanol for people with m.3243A>G‑related mitochondrial disease.
This study represents a significant step forward in understanding the safety and effectiveness of sonlicromanol as a potential treatment for this currently untreatable condition. Crucially, the trial is now recruiting participants in the UK, offering members of our patient community the opportunity to take part should they wish.
The randomised, placebo-controlled study aims to enrol up to 220 patients aged 18 years and older across Europe, the UK and the US. It will assess whether sonlicromanol can meaningfully improve symptoms that matter most to patients, including fatigue and physical function, over a 52‑week treatment period.
We’ve been following the development of sonlicromanol closely, and this milestone marks another step forward in the quest for effective treatments for what is a progressive and debilitating condition. It’s a step that will bring renewed hope to patients and families who are affected by the disease.
Clinical trials play a crucial role in transforming scientific possibility into real‑world treatments for conditions like mitochondrial disease. Studies like KHENERFIN give us hope – hope that through research, collaboration and courage, we can move ever closer to effective treatments and, one day, a cure.
Find out more about the KHENERFIN study and all other current UK trials and research studies.