ICON was approached by a large pharmaceutical company to conduct a fully decentralised placebo-controlled trial, focused on demonstrating that the drug reduced symptom burden in patients with heart failure. ICON randomly assigned 476 patients in the trial to drug or placebo.

This short-term trial was initiated two weeks before a US national COVID-19 lockdown and therefore presented several logistical and organisational challenges to the study staff. One significant challenge was a result of the minimal patient pool being recruited through hospital systems, that were already strained due to the pandemic.

The study’s fully decentralised novel design required staff to ensure the regulatory agencies, IRB and IEC were aligned with the study plan and consenting process and this involved meetings to review and discuss the approach. The design also dictated that patients needed their own smartphone technology, which increased the need for patient support and coordination.

These challenges, together with a short study deadline, drove the study team to develop
innovative methods for the set-up and management of this symptom-focused trial.

ICON leveraged an integrated health network to gain access, identify and enrol patients. The study team assessed medical claims data and serious adverse events (SAEs) to ensure patient safety.

The team engaged the FDA, IRB and EC in the early stages of the trial and on an ongoing basis to ensure the quality and compliance of the data was at the correct standard and to gain approval of the primary endpoint ePRO Kansas City Cardiomyopathy Questionnaire (KCCQ) and secondary endpoint based on data from the wearable devices.

To connect the range of services and deliver an integrated data solution for this study, The ICON Digital Platform — first generation, developed on an innovative data architecture, was deployed. This platform enabled the study team to streamline the process of conducting EMR data tokenisation, eConsent and eCOA/ePRO.

ICON’s Concierge Services were put in place to support the patient at various touchpoints throughout the clinical trial process including set-up, scheduling, eConsent facilitation, compliance check-ins, ePRO support, treatment completion check-in and retrieval of study-based materials.

The primary outcome was a 12 week change in KCCQ TSS.

Investigators also noted greater diversity compared to traditional chronic heart failure trials, previously a longstanding limitation in trials of this indication. The study team were successful in enrolling a higher number of women (52%) and non-Caucasian people (17%) with a wide age range of 22-91 years of age with a median of 64 years.

Concierge Services proved to be an effective strategy to engage patients remotely and delivered positive results such as: 98% confirmed eligibility, with 0% of participants having taken prohibited medication post-enrolment, and patient compliance was very high with >97% ePRO compliance, 95% eDiary compliance and 80% medication compliance.

This study was the first fully decentralised clinical trial with positive results, published in a prestigious publication – Nature.

The successful delivery of this study demonstrates that a novel trial design can improve the way in which clinical studies are conducted and deliver compliance and diversity.