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Post-Trial Considerations for an Early Phase Optogenetic Trial in the Human Brain

Lookup NU author(s): Michael White, Professor Roger Whittaker



This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).


© 2022 White and Whittaker. For many clinical trials, the issue of post-trial access to research treatments is straightforward. Sponsors offer a range of follow-on studies, compassionate use programs or expanded access programs to allow participants to continue accessing beneficial experimental treatments. But there are times when this is not always the case and participants are required to stop beneficial treatments and return to standard care. Guidance states that post-trial access should be made available for “those participants who still need an intervention identified as beneficial”. This broad statement has allowed sponsors to make their own interpretation of when an intervention is still “needed” and when it is “beneficial”. As a result, there have been a number of situations where participants of clinical trials have been left afterwards with feelings of abandonment. Participants involved in studies with long-term, invasive treatments can be seen as being particularly vulnerable. Optogenetic technology has the potential to offer hope to people with neurological conditions, especially people who may not respond to current approved treatments. Optogenetics typically involves two components: a gene therapy medicinal product (GTMP) that induces long-term expression of light-reactive proteins within cells, and an active implantable device to stimulate the light-sensitised cells. Neither works without the other, hence for long-term patient benefit, both must remain active and may therefore require maintenance or replacement. With the potential life-long consequences of both components and the difficulty of accessing the brain, there is a need to reconsider post-trial guidelines and whether they are suitable to support early phase optogenetic trial participants. This paper considers the ethical and regulatory requirements in place for post-trial access and care in relation to optogenetic treatments of neurological conditions. We propose that a new perspective with wider responsibilities for sponsors is required when it comes to these types of novel therapies.

Publication metadata

Author(s): White M, Whittaker RG

Publication type: Review

Publication status: Published

Journal: Open Access Journal of Clinical Trials

Year: 2022

Volume: 14

Pages: 1-9

Online publication date: 02/02/2022

Acceptance date: 18/12/2021

ISSN (electronic): 1179-1519

Publisher: Dove Medical Press Ltd


DOI: 10.2147/OAJCT.S345482