Buffalo clinical trial contributed to FDA approval for fast-acting insulin for children

BUFFALO, N.Y. — The approval on Dec. 19, 2019 by the Food and Drug Administration of a fast-acting insulin for children with diabetes mellitus came about with the help of researchers and providers at the University at Buffalo, UBMD Pediatrics and Oishei Children’s Hospital, as well as some highly committed Western New York families.

The Novo Nordisk clinical trial spanned 17 countries and ultimately included more than 700 children worldwide. It was led in Buffalo by Kathleen E. Bethin, MD, PhD, clinical professor in the Department of Pediatrics in the Jacobs School of Medicine and Biomedical Sciences at UB, a physician with UBMD Pediatrics and the Diabetes Center at UBMD Pediatrics and Oishei Children’s Hospital (OCH).

Lucy D. Mastrandrea, MD, PhD, associate professor and division chief, Endocrinology/Diabetes in the Department of Pediatrics at UB and medical director of the Diabetes Center, was co-investigator. The successful trial makes the fast-acting insulin, called Fiasp® (insulin aspart injection) 100 u/mL, which was approved for adult use in 2017, available for children as young as 2 years old.

Closer to natural insulin

“The goal in the diabetes field is to develop insulin analogs that behave more like natural insulin, which is rapid on, rapid off, meaning it’s quickly released, then quickly dissipates,” Mastrandrea explained. “Part of the reason this drug was developed was to have a better timeline of action that’s closer to natural insulin.”

Fiasp can be given up to 20 minutes after eating has begun, which the researchers say allows for more flexibility in attaining blood sugar control, especially important with children and adolescents. Teenage boys with Type 1 diabetes, for example, tend to do better if they eat frequently, not just at mealtimes. Patients also sometimes forget to take their pre-meal insulin.

“Kids in general don’t always eat what they think they’re going to eat,” said Bethin. “With the older insulin, parents needed to give it 30 to 60 minutes before eating. So if the child didn’t eat the healthy food, their parents might panic and give them candy instead.”

The FDA approval is important because, historically, there have been few clinical trials focused on pediatric patients. That’s changing now, but Mastrandrea noted that the field of pediatrics has a long history of having to prescribe for children drugs that were only tested in adults.

Excellent reputation for clinical trials

Novo Nordisk’s invitation to the Buffalo researchers to participate came about largely as a result of the UB team’s excellent reputation for conducting and completing clinical trials.

“The company chose us because we’ve done a fair number of studies and they know we provide excellent research support,” said Mastrandrea. “We have an excellent reputation for recruiting and retaining patients, a tribute to our team and our excellent research coordinators.”

This clinical trial was performed at 150 sites in 17 countries, so each was small and labor-intensive. In Buffalo, five children aged 13 to 17 participated at UBMD Pediatrics in the Conventus medical office building.

“Those numbers highlight how challenging it is to do really good clinical trials,” said Mastrandrea.

Clinical trials have to enroll a specific number of subjects in an ambitious timeline set by the pharmaceutical company. That can be challenging in any population, but especially in pediatrics.

“This was a big commitment for the families. The kids were in the study for almost a year. There were a lot of extra blood draws and more blood sugar checks than are typically required,” Bethin added. “One thing our team does a really good job of is to be flexible for families, to make their participation as convenient as possible.”

Clinical and Translational Science Institute support

Mastrandrea noted that key to the success of this clinical trial and others is the support of the Jacobs School and UB’s Clinical and Translational Science Institute (CTSI). Collaboration with OCH was also critical, she said.

“Having the support of the Department of Pediatrics and the medical school to do clinical research that will benefit our patients is paramount to our medical community,” she said. “And the resources that the CTSI has built, in terms of improved infrastructure for conducting clinical trials, really helps academic investigators be successful in recruiting and retaining patients.”

Having the Jacobs School, UBMD Pediatrics and OCH on one campus downtown delivers additional advantages, Mastrandrea added: “We have a wonderful collaboration with Kaleida. Our partnership with OCH allows us to easily tap into their resources and expertise. Our patients receive state-of-the-art care because of the research we do.”