Advancing care for children with IgA nephropathy

For years, Myda Khalid, MD, a pediatric nephrologist at Riley Children’s Health and nationally recognized leader in pediatric glomerular disease treatment and research, has watched children with IgA nephropathy face a devastating reality: limited treatment options, harsh side effects and an uncertain future.

Pediatric IgA nephropathy remains an area of substantial unmet clinical need. While multiple disease-targeted therapies have emerged for adults in recent years, treatment options for children have largely relied on glucocorticoids—despite limited long-term efficacy data and a well-documented burden of toxicity. “Children historically have not had access to disease-specific therapies," says Khalid. “We have relied on prednisone despite its variable efficacy and significant side effect profile.”

A new clinical trial evaluating a novel IgA nephropathy therapy in both adult and pediatric populations represents a meaningful step toward closing this long-standing evidence gap, marked by the first pediatric participant enrolled at Riley Children’s.

A first-of-its-kind opportunity for children

IgA nephropathy is a progressive glomerular disease that may advance silently over time, ultimately leading to kidney failure, dialysis, or transplant. In pediatric patients, the stakes are particularly high: disease progression can result in decades of morbidity and lifelong dependence on renal replacement therapies. Yet, until recently, most clinical trials have excluded children.

The clinical trial, which enrolls both children and adults, is among the first to evaluate newer IgA nephropathy therapies in a pediatric population. For pediatric nephrology, the study represents an important evolution—from extrapolating adult outcomes to generating age-specific evidence that can inform clinical decision-making earlier in the disease course.

Many pediatric participants entering the study had undergone prolonged steroid exposure, often over several years, with cumulative side effects. “For many of these patients, standard therapies had reached their limits,” Khalid notes. “The availability of a trial investigating a therapy with the potential for improved tolerability and renal protection was a significant development.”

First in the world

Strong cross-functional collaboration accelerated activation of the trial. Riley Hospital for Children became the first site globally to enroll a pediatric participant, reflecting the institution’s readiness to execute complex glomerular disease studies efficiently.

“Complete credit goes to the systems and the personnel involved in getting a clinical trial running,” Khalid says. Rapid site activation was achieved through close alignment of regulatory, pharmacy, laboratory, and research operations and enabled timely enrollment. The study moved from initiation to enrollment in approximately six months, an uncommon timeline in nephrology research and a marker of growing institutional capacity to support early-phase and translational trials.

Driven by long-term outcomes

For Khalid, this research is deeply personal. “I have seen children lose their kidney function to this disease,” she says. “It is devastating for the family and for the child.”

A clear clinical objective fuels her commitment to clinical trials and drug development. Preventing kidney failure and sparing children from lifelong consequences is the goal behind every study, including work conducted through Riley Children’s integrated glomerular disease research program, which aligns coordinated patient visits with biomarker discovery and therapeutic development.

Looking ahead

This IgA nephropathy trial reflects Riley Children’s Health’s expanding role in pediatric glomerular disease research and contributes to a growing national focus on early, evidence-based intervention. The study is part of a broader effort to accelerate access to effective therapies through close collaboration among clinicians, researchers, industry partners and regulatory bodies. With additional pediatric trials on the horizon, Riley Children's continues to position itself as a leader in pediatric glomerular disease research and nephrology care.

“For clinicians and families seeking access to emerging therapies in glomerular disease, we want Riley Children’s to be recognized as a place where that work is happening,” Khalid says.

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