Data from observational studies and clinical trials, which depend on volunteer participants, form the foundation of regulatory authorities’ decisions to approve new therapies for broader use.
Encoded carefully designs clinical studies to assess safety and efficacy of its investigational precision gene therapies for the treatment of pediatric CNS disorders.
People considering enrollment in an Encoded study may evaluate their eligibility by speaking first with their physician.
A first-in-human investigational trial in the US designed to evaluate safety and efficacy of ETX101 in infants and young children with SCN1A+ Dravet syndrome
ENDEAVOR is a two-part Phase 1/2 dose escalation study of ETX101 in infants and young children aged 6 months to <3 years. In Part 1, up to two doses of ETX101 will be evaluated, with the primary aims of assessing safety and preliminary efficacy of ETX101. ENDEAVOR Part 2 is planned following positive outcomes in Part 1. ETX101 has received FDA clearance for clinical trials, and ENDEAVOR is expected to begin in the first half of 2024.
ETX101 has been granted Orphan Drug Designation and Rare Pediatric Disease Designation by the US Food and Drug Administration and Orphan Designation by the European Medicines Agency.
For more information about ENDEAVOR (NCT05419492), visit clinicaltrials.gov.
A first-in-human investigational trial in Australia designed to evaluate safety and efficacy of ETX101 in young children with SCN1A+ Dravet syndrome
WAYFINDER is a Phase 1/2 dose escalation study of ETX101 in children aged 3 to <7 years with SCN1A+ Dravet syndrome. Up to two doses of ETX101 will be evaluated, with the primary aims of assessing safety and preliminary efficacy of ETX101. WAYFINDER has received approval from the Australian Therapeutic Goods Administration (TGA) and is expected to begin in the first half of 2024.
For more information about WAYFINDER (NCT06112275), visit clinicaltrials.gov.
An observational study of infants and children with SCN1A+ Dravet syndrome
The ENVISION prospective natural history study was designed to further define the seizure, neurodevelopmental, motor and behavioral manifestations of SCN1A+ Dravet syndrome in children aged 6 to 60 months with SCN1A variants. The study examined these characteristics for up to 18 months using standardized assessments and also explored the impact of the disease on parents/caregivers and healthcare resource utilization (HCRU). ENVISION provided a robust data set to advance understanding of the early evolution of DS and to support the clinical development of potential therapies, including ETX101.
For more information about ENVISION (NCT04537832), visit clinicaltrials.gov.
More information on Clinical Studies
A clinical study is medical research involving people. There are two types of clinical studies: observational studies and clinical trials.
In an observational study, researchers observe participants on their current treatment plan and monitor health outcomes and other measures. No gene therapy or other investigational treatments are provided.
The other main type of clinical study is a clinical trial. Clinical trials test and evaluate new medical treatments or procedures in human volunteers. They are typically conducted after preliminary safety and efficacy is shown in preclinical (non-human) studies, and are the primary way that clinical researchers and regulatory agencies evaluate whether a potential new treatment or intervention is safe and effective in people. Clinical trials are governed by the FDA in the United States and by similar regulatory agencies in other countries.
Below is an overview of the traditional stages of trials for a therapy in development. However, this is just a guide: With innovative trial designs for serious and rare diseases for which there is a clear unmet medical need, phases sometimes can be combined to make the evaluation process more efficient.
Combining phases through expedited regulatory pathways can help accelerate the assessment and approval process for therapies that meet certain criteria while still maintaining safety.
Visit these websites for more information on the process of clinical studies:
We would like to thank the healthcare professionals; medical, scientific and other experts; regulatory officials; and the patient community for their collaboration to design robust and meaningful clinical studies. We also are grateful to all study participants, as well as their family networks and healthcare teams, whose support enables Encoded’s clinical research to progress. Thank you!