Lucy D. Mastrandrea, MD, PhD, left, and Kathleen E. Bethin, MD, PhD, led the local arm of a successful worldwide clinical trial that confirmed that a fast-acting insulin is safe for children with diabetes mellitus.

Researchers Aid in Trial That Gets FDA OK for Kids’ Insulin

Published March 2, 2020

Kathleen E. Bethin, MD, PhD, and Lucy D. Mastrandrea, MD, PhD, were local investigators in a successful worldwide clinical trial that confirmed that a fast-acting insulin is safe for children with diabetes mellitus.

“The company chose us because we’ve done a fair number of studies and they know we provide excellent research support. We have an excellent reputation for recruiting and retaining patients, a tribute to our team and our excellent research coordinators.”
Associate professor of pediatrics and division chief of endocrinology/diabetes

Bethin, Mastrandrea Direct Local Operation

Bethin, clinical professor of pediatrics, led the local arm of the Novo Nordisk trial that spanned 17 countries and ultimately included more than 700 children worldwide. The approval by the Food and Drug Administration came on Dec. 19, 2019.

The fast-acting insulin, called Fiasp® — which was approved for adult use in 2017 — is now available for children with diabetes.

“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,” says Mastrandrea, co-investigator on the trial. “Part of the reason this drug was developed was to have a better timeline of action that’s closer to natural insulin.”

Fast-Acting Insulin Spurs Healthy Food Use

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,” Bethin says. “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.”

Reputation for Conducting, Completing Trials

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

Novo Nordisk’s invitation to the Buffalo researchers to participate came about largely as a result of the excellent reputation Bethin and Mastrandrea developed 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,” says Mastrandrea, associate professor of pediatrics and division chief of endocrinology/diabetes. “We have an excellent reputation for recruiting and retaining patients, a tribute to our team and our excellent research coordinators.”

150 Clinical Sites Utilized in 17 Countries

This clinical trial was performed at 150 sites in 17 countries.

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

In Buffalo, five children ages 13 to 17 participated at UBMD Pediatrics in the Conventus medical office building.

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 adds. “One thing our team does a really good job of is to be flexible for families, to make their participation as convenient as possible.”

Collaboration With Jacobs School, Partners Vital

Mastrandrea emphasizes the importance of the support of the Jacobs School of Medicine and Biomedical Sciences and UB’s Clinical and Translational Science Institute (CTSI), in collaboration with Oishei Children’s Hospital (OCH).

“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,” Mastrandrea says. “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. We also have a wonderful collaboration with Kaleida Health. 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.”