New DCRI Registry Could Provide Path for Pediatric Migraine Drug Development

March 5, 2020 – A recently published paper details the development and design of the FDA-funded Pediatric Migraine Registry, which has enrolled around 200 patients between the ages of 4 and 17.

A new registry established by the DCRI seeks to answer critical questions for pediatric patients who experience migraines, while providing a potential pathway for future drug development to help these patients.

A recent paper in Headache: The Journal of Head and Face Pain, authored by the DCRI’s Christoph Hornik, MD, PhD, MPH, and colleagues, details the development and design of the Pediatric Migraine Registry. The FDA-funded registry progressed from concept to first patient enrolled in about six months, and is currently implemented at 20 sites in 15 states across the U.S. The registry met its initial goal to enroll 200 children between the ages of 4 and 17 in the span of 13 months, and creators of the registry say they are planning for potential international expansion.

Hornik said the team chose to focus on pediatric migraine because of the critical need in this area. About 5 percent of children are diagnosed with migraine by age 10, and this number only continues to increase as children progress through adolescence. However, evidence supporting treatments for pediatric migraine remains limited. In alignment with the FDA's Framework for Real-World Evidence Program, Hornik and colleagues began to work on a solution to this problem in the form of a real-world data from a registry. The team leaned on the registry’s co-primary investigator and steering committee co-chair, Amy Gelfand, MD, from University of California, San Francisco, as a subject matter expert in pediatric migraine. Combining Gelfand’s clinical expertise with the DCRI’s experience in developing and overseeing registries contributed to the success of the project’s implementation.

The DCRI's Christoph Hornik, MD, PhD, MPH, principal investigator for the Pediatric Migraine Registry, discusses the goals of the project with Monica Lemmon, MD, a child neurologist at Duke.

“Not only was the study team dedicated to launching the registry, but we are also fortunate to have engaged patients, families, and sites,” Hornik said. “Because of their commitment to advancing the science in this area and finding potential treatments, we were able to advance quickly through the process as we worked to get the registry off the ground.”

Patients enrolled in the registry will be followed for up to a year at three-month intervals, and the registry will include clinical data collected at study sites, data reported by patients and their parents in “headache diaries” through a mobile health app, and biological samples. The data collected are regulatory compliant and provide the infrastructure to support future drug development for pediatric migraine. Participating sites are encouraged to propose research projects that could leverage registry data. In addition, registry leaders are focusing on site training to prepare sites for potential involvement in clinical trials.

“The registry is able to generate the type of data that sponsors, investigators, and others may need to inform their future clinical trials in the space,” Hornik said. “It’s also an ideal platform to act as a springboard for other projects associated with pediatric migraine.”

The registry was developed in collaboration with DCRI Research Together, a group that helped engage patients and their families to ensure that the registry’s goals were aligned with their needs. Investigators also solicited feedback from the International Children’s Advisory Network, an organization committed to empowering pediatric patients, and Miles for Migraine, a nonprofit dedicated to improving quality of life for people with migraine and their families. “A highly collaborative approach with broad stakeholder engagement at all stages of the registry development was a key to our operational success,” the authors write.

Other DCRI contributors to the registry and the design paper include Danny Benjamin, MD, PhD, MPH; Michael Cohen-Wolkowiez, MD, PhD; and Amanda Utevsky (formerly of the DCRI), who helped lead the registry during the development phase.