By Maxine Bookbinder

December 6, 2019 | Final steps are underway to launch the first completely decentralized, virtual clinical study trial with the ultimate goal of giving patients worldwide equal access to clinical trial research.

In a collaboration between The Janssen Pharmaceutical Companies of Johnson & Johnson and PRA Heath Sciences, the study will compare canagliflozin, commonly known as INVOKANA, in a Phase IIIB double-blind study to track quality of life improvements for patients with heart failure. This will also be the first virtual study testing new applications for a drug.

The Canagliflozin: Impact on Health Status, Quality of Life and Functional Status in Heart Failure (CHIEF-HF) trial will launch in early 2020 and is an effort to make trials more patient-centric, accessible, cheaper, and efficient. All contact with the participants will be conducted through smart phones and wearable devices.

“This transformation is long overdue,” says Kent Thoelke, Executive Vice President & Chief Scientific Officer, PRA Health Sciences. He adds that patients who participate in research receive better care. But, due to multiple meetings with doctors, work and family obligations, and transportation difficulties, among other obstacles, only 5% of eligible patients actually participate in clinical trials.

“We must bring trials to patients,” says Thoelke. “PRA believes we must do a better job in the industry to democratize and increase access to clinical research and care options.”

The collaboration is an effort by both companies to move closer to trial decentralization. Thoelke says PRA Health Sciences, who he describes as an industry leader in virtual trials, wanted to partner with Pharma companies also working towards virtual trials. Janssen’s study for INVOKANA fit the criteria. “I am thrilled that the first foray into a siteless study is with an innovative partner like Janssen,” says Thoelke. “It’s a nice opportunity for both of us.”

Clinical trials are undoubtedly a crucial component of medical research and the development of new drugs. But, if people cannot access trials, and costs and time are prohibitive, then the value of trials is depleted. CHIEF-HF is designed to change that. A patient can download the study app to get more trial information, to register, and even to sign an agreement online. Drugs and participation directions are then shipped to the patient’s home.

“Twenty years ago, patients loaded up with blood, urine, and all sorts of tests,” says Paul Burton, Vice President, Cardiovascular and Metabolism Medical Affairs at The Janssen Pharmaceutical Companies. “This inefficiency kept getting carbon-copied into the next trial protocol that companies did. We all talk about being more patient-friendly. Patients want to be diagnosed quickly, given information that’s understandable but not dummied down, have sufficient information to make the right treatment choices, and access to the best medicine. They want to be respected and heard and to feel like they’re really participating. CHIEF gives them the opportunity to do this.”

Doctors at ten large U.S. medical facilities are helping to recruit 2,000 patients. The Phase I double-blind study will last three months. Patients will receive wearable devices, provided by a third party manufacturer, such as Fitbit, Apple, or Garmin, that will track daily steps, VO2 and heart rates. However, for this initial launch, only daily steps will be analyzed. “Step counts track quality of life changes,” says Burton. “We will compare steps at the start and end of the study. We want patients to not just feel better but have a better quality of life.”

Patients will need a smart phone to download PRA’s mobile app, which will include the Kansas City Cardiomyopathy Questionnaire (KCCQ) to be completed monthly during the three-month Registrational Trial. However, Burton emphasizes that the study’s aim is to provide sufficient support and therefore no patient will be turned away. “We just want to ensure patients can access and participate,” says Thoelke.

All data will be sent directly to Janssen for analysis and application of the drug tested. At the completion of Phase I, data will be disclosed to participants, allowing them to see how they did individually and how both groups performed. Patients in the placebo group who wish to change to the canagliflozin group can then do so.

Burton says that by working collaboratively with patients like this, researchers can track patient behaviors and treatment choices. “This is a powerful part of the test. It’s a way to say to the patient: you were in this study, this is the group you were in, this is how you did, and give patients a choice of what they want to do. It’s giving power and autonomy back to patients.”

The Janssen team wanted its initial siteless clinical study to involve an already well-established and tested drug; canagliflozin has excellent efficacy and a good safety profile. Despite its decentralization, the test still requires a team of at least 100 people to ensure patient-friendly recruitment, safety, oversight, and data analysis.

PRA Health Sciences provided the trial architectural support, including the mobile platform, app development, and connections for all connectable devices. It also customized the trial’s app and created a design that was user-friendly and appropriate for different patients at varying levels of technical abilities. “We anticipate patients will have different levels of tech experience,” says Thoelke. “We look at the patient population, characteristics, and provide a mobile platform that is most appropriate. Regardless of age, we find that the ability to interact with the platform is quite good. We expect limited challenges but have a support center just in case.”

Although the initial study is in the U.S., Burton hopes to eventually expand siteless studies worldwide. “We absolutely understand that digital platforms will become a fundamental part of healthcare. Our mission is transformational innovation to take on diseases, and to give meaningful treatments to people.”

The advantages of siteless clinical trials include increased participation, increased participant diversity, decreased trial time and cost, and shorter time to market. They are best for out-patients with chronic illnesses who do not need direct physician interaction.

This study will show that hybrid trials can move to fully virtual, says Thoelke. “This is the beginning of democratization of trials to patients. This will show patients and Pharma it is absolutely achievable. The impact to the industry and of bringing drugs to market is huge. This is just the beginning, but I hope to see much more in the future.”