Two clinical investigators from the Jacobs School of Medicine and Biomedical Sciences at the University of Buffalo (UB) participated in a successful worldwide Novo Nordisk-sponsored clinical trial, confirming that a fast-acting insulin is safe for children with diabetes mellitus. The sponsor selected UB due to their experience, research support levels, and track record for recruiting and retaining patients, which ultimately is made possible by the hard work of the research teams, including the coordinators.
The Study Leads to FDA Approval
The Novo Nordisk-sponsored clinical trial spanned 17 countries and ultimately included more than 700 children nationwide. The study results contributed to the approval of the drug by the U.S. Food and Drug Administration (FDA) on Dec. 19, 2019.
Called the “onset 7” trial, Novo Nordisk sponsored this global trial to investigate the efficacy and safety of faster-acting insulin aspart (Fiasp®) compared to NovoRapid both in combination with insulin degludec in children and adolescents with type 1 diabetes. UB reports the study included 150 sites in 17 countries participating worldwide in the study. UB was able to recruit and treat five children ages 13 to 17 at the UBMD Pediatrics in the Conventus Medical Office building.
The approval was based on the data from this 26-week, Phase 3b, partially double-blind, basal-bolus, treat-to-target trial, which investigated the efficacy and safety of Fiasp compared with conventional insulin in 777 children with type 1 diabetes.
The approval can fit into the important category as historically there have been few clinical trials focused on pediatric patients. This is now changing as the field of pediatrics has a long history of having to prescribe for children drugs that were only tested in adults. Children matter!
Fiasp® is a fast-acting insulin approved for adult use in 2017. Thanks to the hard work of investigators and staff around the world (including the UB team) this treatment option is now available for children with diabetes. This drug , according to press releases from sponsor Novo Nordisk, is the first and only fast-acting mealtime insulin injection that does not have a pre-meal dosing recommendation. It is administered at the beginning of a meal or within 20 minutes after starting a meal.
Principal investigator Kathleen E. Bethin noted, “Kids in general don’t always eat what they think they’re going to eat.” She continued, “With 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 given them candy instead.”
UB Clinical Investigational Reputation
According to a news releasee from UB’s Jacob School of Medicine and Biomedical Sciences, Novo Nordisk invited the Buffalo researchers to participate largely as a result of the excellent reputation of the two lead clinical investigators there for conducting and completing clinical trials. The two leads are Lucy D. Mastrandrea, MD, PhD, Associate professor of pediatrics and division chief of endocrinology/diabetes and Kathleen E. Bethin, MD, PhD, professor of pediatrics.
Dr. Mastrandrea note,d “The company chose us because we’ve done a fair number of studies and they know we provide excellent research support.” The investigator continued, “We have an excellent reputation for recruiting and retaining patients, a tribute to our team and our research coordinators.”
Patient Engagement—a UB Specialty
Large clinical studies can be notoriously difficult to recruit sufficient numbers of participants. This can be especially the case in therapeutic areas involving certain demographics, such as pediatrics. UB has a strong reputation for not only recruiting and retaining but engaging with patients, and this study was no exception. One of UB’s investigators, Kathleen Bethin, MD, PhD, noted, “this was a big commitment for 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.” Professor Bethin continued, “One thing our team does a really good job of is to be flexible for families, to make their participation as convenient as possible.
Planning, Preparation & Collaboration Key at the Site & Beyond for Success
The support of the Jacobs School of Medicine and Biomedical Sciences as well as UB’s Clinical and Translational Science Institute (CTSI) in collaboration with Oishei Children’s Hospital (OCH) was key to the success of this and any trial and Buffalo understands the importance of a strong culture of collaboration to support complex global clinical trials. Dr. Mastrandrea noted, “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 emphasized, “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.”
Lucy D. Mastrandrea, MD, PhD, Associate professor of pediatrics and division chief of endocrinology/diabetes
Kathleen E. Bethin, MD, PhD, professor of pediatrics
Call to Action: Think about how UB has organized itself to be able to successfully contribute to a global clinical trial. Reputations for skilled, quality care not to mention study operational acumen and wherewithal to the intensive collaboration among university and local health care resources all form a vital local site network as a prerequisite for success.