Riley Children’s Health first in U.S. to randomize patient in pediatric CKD trial

Therapeutic options to slow pediatric CKD progression remain limited. While many patients are maximized on ACE (angiotensin-converting enzyme) inhibitors to reduce intraglomerular pressure, hypotension can limit tolerability in some children. SGLT2 inhibitors, which are already FDA-approved in adult populations and for children with diabetes, may offer a complementary mechanism to help further reduce progression of CKD. The EMPA-KIDNEY Kids trial represents one of the first major pediatric nephrology studies evaluating SGLT2 inhibitors in children with chronic kidney disease without diabetes, a major advancement in a field with historically limited treatment options. This signals an important shift from supportive management to disease-modifying care, with the goal of delaying interventions like dialysis and kidney transplantation. Riley is actively enrolling eligible pediatric patients with CKD into the study.

Neha Pottanat, MD, FASN is leading the study at Riley Children’s as site principal investigator, alongside co-investigators Myda Khalid, MD and David Hains, MD. Also serving as the director of Riley Children’s Chronic Pediatric Dialysis program, Dr. Pottanat sees the profound effects that kidney disease has on children and their families as it progresses, from the burden of constant disease management to physical growth impairment, frequent hospitalizations, and psychosocial impacts. “When I talk with patients and families about this new treatment option, it gives them hope—whether they’re learning about their child’s condition for the first time or we’ve spent years discussing the possibility of dialysis or transplant,” says Dr. Pottanat. “Being able to offer something that may help delay those interventions and give children more time before dialysis or transplant is huge.”

Riley’s rapid activation of the study is the result of high-functioning pediatric clinical research infrastructure, led by nephrology research staff and including collaboration across Institutional Review Board (IRB), investigational drug services, regulatory teams, and translational research laboratories. Dr. Pottanat credited the milestone to the dedicated work of Kiran Naqvi, Jennifer Mullett and the broader research team, along with strong institutional support from division leadership committed to expanding pediatric nephrology clinical trial infrastructure and accelerating access to innovative therapies for children.

Notably, as the first active site in this trial in the US, the team is helping establish early operational workflows and clinical implementation strategies that may inform participating sites across the study network. “Because this is among the first true clinical trials in pediatric chronic kidney disease, it’s creating opportunities to establish best practices for future pediatric kidney studies and demonstrating the importance of research designed specifically for children,” says Jennifer Mullett, BSN RN, pediatric nephrology research coordinator. “Riley and IU School of Medicine have built a collaborative environment that helps move that work forward.”

This milestone reinforces Riley’s growing position as a destination center for complex pediatric kidney disease and clinical trials, with interest extending beyond Indiana. “We’re seeing families travel long distances to participate because they recognize the value of access to novel therapies and highly specialized pediatric kidney care,” says Kiran Naqvi, nephrology research coordinator at Riley Children’s. “For many, this trial represents hope during a very difficult stage of disease.” With an expanding nephrology research portfolio, Riley Children’s combines advanced pediatric subspecialty care with an integrated research ecosystem capable of rapidly translating emerging therapies into clinical trial opportunities for children.

Learn more about Riley Children’s Health pediatric nephrology program, research and clinical breakthroughs in the annual report.