A mid-sized biopharmaceutical sponsor was conducting a phase 3 study on CDKL5 deficiency disorder, a rare genetic seizure disorder in children. The paediatric study spanned 36 sites constituting 102 patients. The sponsor utilised FIRECREST to centralise the tracking of training and information distribution in one of their trial platforms, specifically with the goal to understand and reduce the burden on patients and caregivers. The result increased visit efficiency by implementing a substantial training platform and accessible patient education models.

The sponsor was concerned about site adherence to protocols. For each of the geographically dispersed sites, the sponsor required detailed, substantial protocol training and the ability to track training compliance in real time and with instant access to completion records. It was also important to provide the ability to revisit training as needed given the rarity of disease, the paediatric patients and the time lapse between visits.

The team also needed ready, flexible access to pertinent information and resources in order to reduce risk potential and maximise the positive patient/caregiver experience. Additional information required for patient/caregiver included thorough patient education and assent modules. Given the nature of the paediatric study, it was important to ensure an accessible, efficient and stress-free environment, including during patient visits.

The FIRECREST team identified those procedures that are most open to risk and need the most attention while taking the specifics of the trial type and sensitive patient cohort into account. They implemented protocol overview training designed to ensure that all sites can access content in clear, easily understood topics in addition to protocol overview amendment trainings.

To facilitate consistent site staff preparedness, the FIRECREST production team created a comprehensive visit-by-visit guide for the study staff. Through the guide, staff could see what needed to be accomplished for each visit, including specific procedures that needed to be carried out for that visit (including details) along with instructions for the patient/caregiver before next visit.

For easy access and timely completion, key documentation was migrated to the portal. The ClinROs and PROs on the portal included the Anxiety Depression and Mood Scale (ADAMS), Caregiver Global Impression of Change Scales and Clinical Global Impression of Improvement. TrialDrive was also made available on the portal for the targeted distribution of documents to specific roles, countries or sites. For patients, education and assent modules were made readily available at all site locations via iPad.

The sponsor was ultimately able to minimise the study burden for the paediatric patients and their caregivers. Patients, caregivers and the PI were satisfied with the level and quality of patient education which builds trust in the study and strengthens the relationship with the PI. For the sponsor, there were strong online training completion figures by the site staff which was easily accessible for reporting at study, site and individual levels. Similarly, the added ability to access sponsor-owned training material, vendor training, IM recordings and recorded procedures proved valuable to site staff in the execution of visits.

The sponsor also saw widespread use of the tailored visit-by-visit guide which was a useful tool due to the time lapse between visits. The sponsor team benefitted from a dedicated FIRECREST project manager that acted as a consistent point of contact for the study team during the deployment of amendments and any other changes. Overall, the successful implementation led to the positive perception of FIRECREST as a one stop shop for all training support and communication around the study for all stakeholders.