What is the study?
This 15-month observational study aims to further understand the role of the faulty huntingtin protein in disease progression. There is no drug treatment in this study, as the goal is to understand the natural progression of HD. This study will include up to 100 participants with early manifest (Stage I and II) HD at up to 17 sites in Canada, Germany, the United Kingdom and the United States. For all patients who complete the HD Natural History study, an open-label extension study with the option of receiving RG6042 (no placebo control) is planned, pending eligibility, approval by Authorities and Ethics Committees/Institutional Review Boards and if data support the continued development of RG6042.
How are sites selected?
A variety of factors influence site selection, including assessments on experience with HD studies, clinic infrastructure capacity to run the study as well as usual site activities, ability to operationalise the study as quickly and completely as possible, patient population, and geographic location.
Expected HD Natural History Study sites in the UK
Birmingham – University Hospitals Birmingham
Cardiff – University Hospital of Wales
London – University College London
Manchester – University of Manchester, St. Mary’s Hospital
What about the next clinical trial for RG 6042?
We expect the phase 2b/3a trial of RG6042 (formerly IONIS Httrx) to being in early 2019 and will provide further details as we have them.