It has been years without a significant breakthrough in treatment for pediatric glomerular disease. Thankfully, that’s changing, according to Myda Khalid, MD, pediatric nephrologist at Riley Children’s Health and a nationally recognized leader in research and treatment of glomerular disease in children. Dr. Khalid and the pediatric nephrology division at Riley Children’s and Indiana University School of Medicine are contributors to groundbreaking initiatives in pediatric glomerular disease that will change the landscape of what is known about these conditions and how they are treated.
At Riley Children’s, Dr. Khalid leads several high-profile National Institutes of Health (NIH)- and industry-funded clinical trials, including HIKO-STEC and FIT4KID. She is a principal investigator for the Cure Glomerulonephropathy (CureGN) observational cohort study, and the Nephrotic Syndrome Study Network (Neptune), both supported by the NIH. Additionally, Riley Children’s is enrolling patients in APPEAR-C3G, a Phase 3 study to evaluate the safety and efficacy of iptacopan as a potential treatment for patients with C3G, a condition without current targeted therapies. Plans are underway to roll out additional trials for C3G by fall 2025.
Dr. Khalid also leads Indiana University’s participation in PARASOL—a collaborative international study that aims to define the relationships between short-term changes in kidney function and urine protein, with long-term kidney outcomes in order to support the use of proteinuria-based endpoints in clinical trials. This foundational project brings together the clinical, scientific and regulatory communities to accelerate research in focal segmental glomerulosclerosis (FSGS).
“It is critical for us to ensure clinical studies and trials include children—a group that remains understudied and unrepresented,” said Dr. Khalid, who serves on the board of the Pediatric Nephrology Research Consortium and is a leader in other national nephrology organizations. “With emerging focus on preventive medicine and a need to intervene early in the disease process to avoid long-term kidney damage, we must be able extend therapies to children that are now available for individuals over 18 years. To do this, we must aggressively expand the clinical research and clinical trial footprint in children.”
With Riley Children’s already a high-enrolling site for many of these pivotal trials, Dr. Khalid continues to build the pediatric health system’s reputation as a leading clinical interventional study site. She is currently working alongside fellow pediatric nephrologist and basic scientist Andrew L. Schwaderer, MD, to launch a biomarker discovery program at IU School of Medicine that will embed registry, biorepository, biomarker identification and clinical trials into a glomerular disease clinic. The program, called BRIDGE, will enhance the diagnosis and management of glomerular disease, as well as provide a solid foundation for the development and execution of novel clinical trials.
“We’ve got all the pieces at Riley—basic science labs, a robust clinical trial infrastructure and strong support from leadership—to make an impact in finding new treatments for these complex kidney diseases,” said Dr. Khalid. “With the programs we’re building and the resources already in place, I see Riley as a leader in cutting-edge research for glomerular diseases.”
It’s worth the effort, Dr. Khalid said, for the millions of children and adults with glomerular disease who face potential kidney failure.
“It’s devastating to tell parents there’s not much we can do for their child and to watch the children and their parents suffer the physical and mental anguish of a chronic illness that can be debilitating due to its side effects,” she said. “I want to be able to tell them something different in a year or two—that we have more answers and new medicines—that their child is going to be okay. We can get there.”