Novartis announced yesterday that dosing has been suspended for the VIBRANT-HD clinical trial.

According to Novartis, this decision was based on a recommendation from an independent Data Monitoring Committee (DMC) following a planned data review which revealed findings suggestive of branaplam-induced peripheral neuropathy in some participants. (Peripheral neuropathy results from injury to the nerves located outside of the brain and spinal cord.) The decision to temporarily suspend dosing was endorsed by the VIBRANT-HD steering committee.

The Ph2b VIBRANT-HD trial is the first study of branaplam in adults with HD, following pre-clinical and healthy volunteer studies and several years of clinical development in children with SMA (spinal muscular atrophy). The study was specifically designed with robust monitoring to detect early safety signals and allow us to make informed decisions for the trial. This included assessments which may be predictive of potential peripheral neuropathy, such as measurement of neurofilament light chain (NfL) in blood, neurological exams and nerve conduction studies, as well as symptom assessments. The recently reviewed data raised some concern of potential peripheral neuropathy in some participants.

The DMC did not recommend terminating the study at this time. Treatment with study drug has been temporarily suspended and participants will continue to have regular study assessments per the protocol. Neurological status will continue to be monitored; participants will receive neurological exams and nerve conduction studies along with other assessments to better understand these findings.

You can read more about the trial and news at HD Buzz.

"Although this news is disappointing, the data will be so important for guiding these and future drug programs trying to lower Huntingtin to slow HD. We need to find a dose that won't cause harm to brain cells before we can find out whether it helps brain cells combat HD," said Dr. Lauren Byrne, HDYO Board Member and Research Committee Co-Chair.

HDYO is here for anyone who needs support during this additional setback to Huntington's disease research. You are not alone!

We know that the past day has given a blow to the Huntington's disease community with two setbacks in clinical research studies. But we also know that the HD community is RESILIENT! As we all grieve and adapt, the strength and determination will continue to prevail," added Jenna Heilman, HDYO Executive Director.