mdgroup recently supported a single ascending dose hybrid clinical trial, which investigated ways to correct the problem in protein transcription in the gene overexpression of Friedrich’s Ataxia, an autosomal-recessive genetic disease.
Due to the nature of the disease, which significantly impacts mobility, a large portion of the trial had to be conducted within participants’ homes. This was made more complicated by the fact that the participants were based all over the US and that some of the blood sampling required was challenging.
One of the key exploratory pharmacodynamic endpoints of the trial required human peripheral blood mononuclear cell (PBMC) isolation in order to learn more about the action of the investigational product. This is a complex process, typically performed in a laboratory setting by specially trained scientists. It was not possible to send whole blood samples to the laboratory for this process to be performed because the lab was far from many participants’ homes and there was a high probability of sample degradation during transit. To ensure the integrity of the sample and therefore the data quality, , the mobile HCPs needed to isolate the protein at the participants’ homes before sending it for further testing.
This level of expertise posed a big challenge, and only a few mobile health providers could offer a solution. We love an opportunity to innovate and jumped at the chance to facilitate the remarkable.
The client implemented and validated a technique to isolate and lyse PBMCs from the whole blood in the participants’ homes before the samples degraded. This two-and-a-half-hour, 15-step procedure required uncompromising attention to detail, and only the most experienced healthcare practitioners (HCPs) were up to the task.
Through our wholy owned HCP resourcing company, Seacole Health, we trained nine HCPs in different locations across the US to become experts in this process. They learned the procedure during an in-person training session, from experienced lab professionals who tested their samples and approved their training.
In addition to developing our nationwide subject matter expert team, our Navigator Project Coordinators arranged the transport of HCPs and the expensive amount of equipment required to perform this, and other protocol required assessments, to remote locations across the US, and oversaw the courier services that transported samples to the lab for assessment.
Due to the cohort recruitment on this project., we needed to accommodate up to six simultaneous remote visits, with real-time support from our in-house clinical managers. Our Navigator Project Coordinators organised this to be as time and cost-effective as possible by doing our best to limit HCPs to regional travel, while still providing quality PBMC extraction and exceptional patient care.
The Seacole HCP team expertly navigated the complexities of the trial, going above and beyond to make the participants feel comfortable at every stage of the trial.
Many of our HCPs remarked on what a transformative experience it was to work with individuals navigating the conditions of their rare disease and the impact of the patient/nurse relationship they formed during the trial.
Not only did this help the participants feel comfortable during the process, but it also allowed them to see the importance of their involvement in the study.
The client was happy with our safe, expert and cost-effective delivery of this trial. We completed the study with no patient dropouts and to the highest data quality – including the complex PBMC work. All of the PBMC samples collected from participants in their homes were deemed to be viable. The participant experience was positive and many have expressed interest in the subsequent multi-ascending dose trial, which is currently in progress.
In addition to our team of trained subject matter experts in PBMC isolation, we have internal expertise available to train future HCPs in this procedure, which permits us to run PBMC isolation remotely for other clients in the future. We look forward to running more studies involving PBMC isolation in the home soon.
Are you interested in how we can implement PBMC isolation in your hybrid or decentralised study? Get in touch.