Less than a year after enrolling its first patient, the MMRF’s Horizon One clinical trial already has important wins to celebrate: Not only is it pioneering important research for patients with relapsed and refractory multiple myeloma, but it’s enrolling a patient population that’s far more representative of the disease than other trials to date.
The Horizon Clinical Trials program, which is run by the MMRF’s Multiple Myeloma Research Consortium® (MMRC®), is innovative by design, starting with the fact that it’s aiming to optimize treatments for patients with significant unmet need. The first arm of the trial, Horizon One, is enrolling patients with relapsed and refractory multiple myeloma, while the forthcoming Horizon Two trial will enroll high-risk, newly diagnosed patients.
Unlike a traditional clinical trial, Horizon is what’s known as an adaptive platform clinical trial. This unique design allows investigators to test several treatments at the same time and has built-in flexibility to add or remove therapies in real time—all with the goal of generating data and insights at an accelerated pace.
Speed is especially important for the patients in Horizon One. While there are currently more than 15 FDA-approved drugs for the disease, the devastating reality is that most patients will relapse when the disease becomes resistant to a therapy. Others may eventually become refractory (unresponsive) to many kinds of treatment.
“This patient population needs more options, and they need them fast,” said the MMRF’s Chief Medical Officer Hearn Jay Cho, MD, PhD. “These patients and their providers still face complex questions about what treatments will be most effective and in what order. Horizon is designed to answer those critical questions that other types of trials can’t.”
Horizon is innovating in another way: by enrolling a study population that is representative of multiple myeloma patients across the United States. In doing so, the Horizon trial can generate new insights and answer questions that impact all patients.
Historically, clinical trials in multiple myeloma have not been as inclusive.
About 20 percent of multiple myeloma patients in the U.S. are Black, but those patients have been significantly underrepresented in clinical trials. In fact, one analysis of multiple myeloma trials conducted over a 13-year period found that only four percent of enrolled patients were Black.
The average age of a multiple myeloma diagnosis is 69, and two-thirds of people diagnosed with the disease are 65 years or older. But studies have found that these patients are also left out of research. One analysis found that patients over the age of 70 are half as likely to enroll in multiple myeloma clinical trials, and another found that nearly two-thirds of trial participants over a 16-year period were under the age of 65, not at all representative of the disease’s true age demographic.
But the MMRC’s Horizon trial is working to reach the broadest myeloma population—and succeeding.
As of now, more than 25 percent of patients enrolled in Horizon One identify as Black. Seventy-one percent of patients are 71 or older, with more than 20 percent between 81 and 90 years old, percentages that are rarely, if ever, seen in other myeloma trials.
Horizon One is also working to enroll across a mix of geographical locations, including in rural areas and at 25 community hospitals and clinics. In fact, the MMRC Horizon One trial recently opened the first-ever myeloma clinical trial in Flint, Michigan.
Meeting these impressive goals has required considerable collaboration that the MMRC—with its vast network of 13 major medical centers and researchers and a leadership team with decades of expertise and industry connections—is uniquely positioned to facilitate.
Working closely with the MMRC’s Health Equity Advisor Dr. Craig Cole of Karmanos Cancer Institute, the Horizon team has surveyed MMRC sites, collecting data and insights about why patients enroll and, importantly, why they don’t. Dr. Cole and the MMRC team have also brought all 13 MMRC sites together to share learnings and wins.
“Clinical trials have been exclusionary to older people, people of color, and people who live far from academic medical centers. We can enroll those populations by knowing why patients don’t consent to the clinical trial,” Dr. Cole said. “We’re making cultural change. The MMRC sites are sharing stories and ideas and thinking outside the box about how we can improve what we’re doing.”
Many of the trial’s wins, like the launch in Flint, came out of simply having intimate, one-on-one conversations out in the community.
“I did a church basement talk in Flint at an African American church, and I gave my awareness talk about what myeloma is,” Dr. Cole said. “And this little old lady said, ‘You need to bring clinical trials to us. I don’t want to hear about myeloma and all your fancy drugs that you give in Detroit.’ I took it back to the MMRC and said, ‘A little old lady told me to bring a clinical trial to Flint, and I think we really need to do that.’ And the MMRC leadership made that happen. Now, that little old lady can go down the street, get her bispecific, go home, and hang out with her grandchildren.”
Understanding that cures don’t come out of silos, the MMRF and MMRC have a long history of making their learnings and data available to the wider multiple myeloma field. Looking forward, the Horizon trial investigators plan to share their experiences and findings for other researchers, pharma, academic medicine, and others to learn from.
“We’re so proud to conduct trials like Horizon that will help us better understand the disease across a representative population and regardless of where someone lives,” said MMRF’s Senior Vice President of Clinical Research and Regulatory Affairs Katie Wozniak. “That’s how we’ll improve care and find cures for everyone diagnosed with multiple myeloma.”
