First pediatric patient in the world enrolled in PIONEER clinical trial for kidney glomerular disease at IU School of Medicine

INDIANAPOLIS — Indiana University School of Medicine researchers are leading a first-of-its-kind clinical trial to combat immunoglobulin A (IgA) nephropathy, an autoimmune kidney condition that inflames and can permanently damage the kidneys, in children.  The clinical site at Riley Children’s Health enrolled the world’s first pediatric participant into the international trial, marking a significant step for the treatment of chronic autoimmune disease that causes kidney failure and has limited treatment options.

This globally conducted, multi-site pediatric trial, PIONEER, is being led at IU and Riley Children’s Health by Myda Khalid, MD, an associate professor of clinical pediatrics and the associate vice chair of clinical trials at Riley. Last month, Khalid’s team enrolled the world’s first pediatric patient in this study, one of the first trials in IgA nephropathy to include children, is paving the way for a novel therapeutic option that could ultimately prevent or delay kidney failure.

"Without pediatric clinical trials, children must wait until they reach adulthood to get access to IgA treatments," Khalid said. "A 12-year-old child would have to wait six years before being able to access cutting edge therapies approved only in adults. That would mean six years of risk of permanent kidney function decline before accessing new therapies."

This trial is a basket trial, meaning it includes participants with several different but related diseases, for children and adults with glomerular disease, is one of trials in autoimmune kidney disease. One of the conditions targeted in this trial is IgA nephropathy and vasculitis, as well as minimal change disease, focal segmental glomerulosclerosis and membranous nephropathy. In IgA nephropathy, the body begins to react to IgA with a slight abnormality in its structure. As a result, there is trapping of this abnormal IgA in the kidney tissue and resulting inflammation and kidney damage. Patients with the condition may first present with dark brown urine due to kidney inflammation. With previous IgA nephropathy research mainly focused on adults, the PIONEER trial is helping shape guidelines for the pediatric population.

Weekly injections will be used to test the safety of the treatment, with the goal of securing FDA approval if they demonstrate reduced protein levels in participants’ urine and improved kidney function.

With an increase in studies on the condition in recent years, physicians have started to recognize that IgA nephropathy leads to kidney failure for many patients, including children. This outcome leaves pediatric patients especially vulnerable as they have not had any specifically developed treatment for this condition.

Historically, the only treatment option for IgA nephropathy has been a prednisone steroid prescription, reserved for adults and children who are on the brink of kidney failure. Physicians cautiously use or avoid giving prednisone to children due to significant risks of long-term side effects like stunted growth, impaired bone density, delayed puberty, increased infection susceptibility and behavioral changes.

"When I started my training about 18 years ago and tried to look up literature, there was nothing there," Khalid said. "Now, there is so much hope that we will be able to stamp out the disease. That is extraordinarily exciting. That's how you know you’re changing the face of medicine."

To offer a new treatment option for patients with IgA nephropathy and other forms of nephrotic syndrome in children and adults, participants will attend monthly clinic visits for blood draws and monitoring. Vera Therapeutics is the sponsor of the PIONEER clinical trial.

"We are becoming a destination for pediatric kidney care, and I would love to see us as the best place in the country, if not the world," Khalid said. "I think we can do that."

Visit the Pediatric Nephrology website to submit questions or to learn more.

About the Indiana University School of Medicine

The IU School of Medicine is the largest medical school in the U.S. and is annually ranked among the top medical schools in the nation by U.S. News & World Report. The school offers high-quality medical education, access to leading medical research and rich campus life in nine Indiana cities, including rural and urban locations consistently recognized for livability. According to the Blue Ridge Institute for Medical Research, the IU School of Medicine ranks No. 15 in 2025 National Institutes of Health funding among all public medical schools in the country.

Writer: Salem Lucas, slucas2@iu.edu

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