The trial is being conducted at the SGS CPU, with SGS providing a full scope of services that includes regulatory support, the recruitment of healthy volunteers, project management, site monitoring, pharmacovigilance, medical monitoring, data management, Secure Data Office (handling of unblinding data), medical writing, PK and statistical analysis.
“Here at SGS, we are delighted to be conducting this Phase 1 trial. The study medication that we are testing is incredibly innovative and may have important implications in the treatment of genetic disorders,” said Jonathan van Gucht, Clinical Research Physician at SGS.
AAV gene therapy uses a modified, harmless virus known as adeno-associated virus (AAV) to introduce a specific gene into a patient’s cells, although in this healthy volunteer study the AAV is “empty” and does not contain a gene.
Operationally speaking, the SGS CPU team must manage a unique set of qualifications for study subjects. For example, study volunteers must be AAV8 antibody negative, meaning there is no presence of AAV8 antibodies in their bloodstream. Finding this type of volunteer is the first step, because some volunteers already have these antibodies as a result of ordinary exposure to viruses during one’s lifetime. While SGS CPU has a large database of volunteers from which to recruit, additional efforts include the creation of an AAV8-negative database that accelerates its ability to recruit volunteers for the study.
Once administered, humans develop antibodies against the AAV8 vector, which neutralize the effect of any future administrations of this specific gene therapy. By administering ImmTOR in the trial, the Selecta/AskBio team will attempt to prevent the generation of these antibodies to allow redosing of the gene therapy within the patient. If successful, this study could be a significant first step towards overcoming a common obstacle associated with gene therapy.
The day of (and the day before) the study medications are administered, a series of specific steps must be performed sequentially within a strict time interval. Furthermore, the well-being of the volunteer is regularly monitored by checking multiple safety parameters.
The SGS team is providing its expertise and assistance to the study amidst strict healthy volunteer requirements and tight timelines, which have been exacerbated by the ongoing COVID-19 pandemic. Together, with our experienced and well-trained study staff, we anticipate bringing this study to a successful conclusion.
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