With CMTA support of $354,826, researchers at the University College London (UCL) Queen Square Institute of Neurology in London, led by CMTA Strategy To Accelerate Research (CMTA-STAR) Advisory Board member Mary M. Reilly, MD, are completing the SENSE HSN1 clinical trial, which is investigating a potential treatment for hereditary sensory neuropathy type 1 (HSN1). HSN1 is caused by variants in the SPTLC1 or SPTLC2 genes and leads to progressive sensory loss and nerve damage.
The SENSE trial is also studying Magnetic Resonance Imaging (MRI) as a possible tool to track disease-related changes in muscle fat accumulation. If successful, this approach could provide a non-invasive outcome measure for future HSN1 trials. Learn more about Dr. Reilly’s work with MRI in CMT.
April 2026 Update
All participants have now completed their scheduled study visits, including final follow-up assessments. This marks the completion of the active participation phase of the trial. The study medication and procedures have been well tolerated overall, with no serious adverse events directly linked to the treatment or trial assessments.
The research team is now preparing the study data for final analysis. The statistical analysis plan has been finalized, and all clinical visits and assessments have been recorded, with data entry into the trial database ongoing. Biological samples collected during the study are being processed and shared with collaborating laboratories for specialized analysis. A detailed database clean-up is also underway to ensure all data are accurate and complete.
The next stage will involve completing the final data analysis. Once complete, the team will prepare a scientific manuscript and share the findings with participants and the full CMT community.
