Challenges and strategies with international recruitment in early phase gene therapy trials
Molecular Genetics and Metabolism
; 138(2), 2023.
Article
in English
| EMBASE | ID: covidwho-2244788
ABSTRACT
The Children's NIHR Clinical Research Facility at Royal Manchester Children's Hospital has been involved in numerous early phase gene therapy trials for diseases such as GM1 gangliosidosis, Gaucher disease, MPSIIIA and MPSII. These trials have necessitated international recruitment which brings challenges for both site and families. In addition, we also actively recruited participants during the Covid-19 global pandemic, amplifying these challenges. A typical patient journey on one of these trials would involve being approached soon after diagnosis due to the rapid progression of these diseases and the need for early intervention. The family would then relocate to the UK with relatively short notice and commence an intensive period of screening involving a lot of extensive information for them to retain and invasive procedures for the patient. Some of these families will speak no English at all which is an additional barrier to managing the parental anxiety and expectations of the trial and its outcome. Once eligibility is confirmed the families are then faced with an extended stay in the UK without the support of their extended family/community. This impacts parent's employment and other siblings who may or may not be with them and who may also be affected by the same disease. Following administration of the gene therapy, participants then commence intensive follow up often associated with immunosuppressants. Close working with the local clinicians is essential for patient safety and trial integrity. Good engagement with families once they have returned to their home country is vital in obtaining continuing trial data and ensuring retention and compliance with attending future visits. Follow up visits are essential for safety and efficacy data for the progression of gene therapy trials. Travel restrictions brought about by the covid 19 pandemic exacerbated these challenges but with good communication and engagement we have mostly overcome them.
immunosuppressive agent; anxiety; child; clinical research; conference abstract; controlled study; coronavirus disease 2019; drug safety; drug therapy; early intervention; eligibility; employment; expectation; extended family; follow up; Gaucher disease; gene therapy; GM1 gangliosidosis; human; Hunter syndrome; invasive procedure; pandemic; patient safety; Sanfilippo syndrome; sibling; travel restriction
Full text:
Available
Collection:
Databases of international organizations
Database:
EMBASE
Language:
English
Journal:
Molecular Genetics and Metabolism
Year:
2023
Document Type:
Article
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