Dravet Syndrome Foundation Spain continues to support research and the participation of patients living in Spain in any study that addresses the discovery of new therapeutic options and the improvement of the quality of life of these people. Thus, Encoded Therapeutics, in close collaboration with the Foundation, will initiate ENVISION, a study of the natural history of Dravet syndrome, in Spain which will provide information on the evolution of the disease in order to propose appropriate therapeutic options.
ENVISION is an observational study of the natural history being carried out by Encoded with the aim of gathering information as broadly as possible on the impact of living with Dravet syndrome on children and their families over time. It is designed to further define, among other things, seizure, neurodevelopmental, motor and behavioural characteristics in children aged 6 to 60 months with Dravet syndrome and a SCN1A mutation. The study will examine these characteristics over two years, and will also explore the impact of the disease on parents/caregivers, as well as health resource utilisation. More on ENVISION can be found HERE.
When we heard the news that ENVISION would be conducted in the US, Australia and the UK, we didn’t hesitate to move mountains to bring the study to Spain. After many months of conversations with Encoded and Spanish hospitals, we are happy to announce that the Dravet Syndrome Foundation Spain has supported the start of ENVISION in Spain!
Two Spanish hospitals – Hospital de la Santa Creu i Sant Pau in Barcelona and Hospital Universitario y Politécnico La Fe in Valencia – and a number of patients already pre-screened will participate in ENVISION, and Encoded has made a waiting list available so that the centres can identify new families interested in participating in the study should additional places become available in the future. If you are interested in participating in the study, you or your doctors can contact the centres directly for more information.
As you know, Encoded is developing a potential gene regulatory therapy called ETX101. Dr Salvador Rico, Encoded’s Chief Medical Officer, told us about this potential therapy in his webinar for families which you can watch again HERE. The data collected in ENVISION will be used to inform the future ETX101 gene therapy clinical trial, called ENDEAVOR, which will test whether ETX101 is effective in eliminating or significantly reducing the seizure and non-seizure related symptoms of Dravet syndrome SCN1A+, thereby improving the quality of life of both children living with the condition and their families.
ENDEAVOR is scheduled to begin in the United States at the end of this year, and we hope that it will be available in Spain soon thereafter.
Although participation in ENVISION does not guarantee inclusion in ENDEAVOR, some participants in the natural history study may be able to access the potential gene regulation therapy trials. In such cases, the natural history information previously collected could be used to better understand the effect of the potential therapy on patients.
We would like to thank Encoded for its commitment to patients and families around the world, and especially in Spain, by allowing public hospitals and Spanish patients to be part of the ETX101 programme, which has the potential to be the first single-administration gene therapy that could potentially correct the underlying cause of Dravet syndrome. We also thank the participating centres and doctors for the important work they do in the search for efficient therapies for Dravet syndrome, as well as for their commitment to the work of the Dravet Syndrome Foundation Spain.
One dream, one goal.
