The EHDN is closely monitoring recent developments in two ongoing clinical trials: the VIBRANT trial sponsored by Novartis and the AMT-130 trial sponsored by Uniqure. Both trials are endorsed by EHDN and represent distinct pharmacological approaches towards the shared goal of lowering expression of the Huntingtin protein in patients with HD. The safety concerns that have arisen appear to be unrelated to one another and are currently being analysed. While this is a sobering moment on the road towards the development of effective interventions against the devastations of HD, these setbacks are not unexpected and are also a reflection of the robust trial activity in the field. It is also a reminder of the risks that participants in such trials are taking to advance our cause as well as the importance and appropriate application of intense surveillance of participant safety. Continued follow up of already-dosed participants in these trials is critical.
The EHDN is committed to continuously improving clinical trials and thus it is our obligation to learn as much as possible from these developments. We look forward to the analyses of results by the trial sponsors along with the wider HD research community.