Overcoming Barriers to Pediatric Glomerular Disease Clinical Trials

Children with glomerular diseases have historically had fewer therapies developed and tested on their behalf than adults, leaving pediatric nephrologists to extrapolate safety and efficacy from adult data. As targeted agents move through adult pipelines, pediatric glomerular disease clinical trials are beginning to catch up.

Myda Khalid, MD, of Riley Hospital for Children at Indiana University, and Scott Wenderfer, MD, PhD, of BC Children’s Hospital and the University of British Columbia, co-chairs of the International Society of Glomerular Disease (ISGD) GD-Kids working group, spoke with HCPLive about the obstacles and how the field is addressing them.¹

What Has Held Back Pediatric Glomerular Disease Trials?

Wenderfer pointed to a longstanding tendency to protect children from research, which effectively barred them from trials. As a result, pediatric nephrology practice has largely relied on adapting adult safety and efficacy findings.

“Now rather than protecting them from research, we’re advocating for them to be involved with research,” Wenderfer said. He noted trial participants receive closer follow-up and more safeguards than in routine care, and drugs can work well and safely in children even when they are less effective or less safe in adults.

Khalid said the trials simply were not available until recently. Regulators, scientists, physicians, and patient organizations have since pushed for pediatric inclusion, and the number of pediatric trials is rising.

Wenderfer, whose work centers on lupus nephritis, said the lack of trials has limited the evidence behind pediatric consensus treatment plans. Khalid, who runs a glomerular disease research clinic at Riley, recalled a young patient who regularly asked when she would receive a medicine specific to her disease.

Which Assumptions About Pediatric Trial Participation Proved Wrong?

Drawing on her IgA nephropathy trial experience, Khalid described several assumptions disproven in her clinic. Parents were not unwilling to enroll their children once trust was established.

“I realized it was important for me as their physician and for us as a community to teach people about the disease, to teach them about the science, to bring them to a place of trust in the physician team,” Khalid said.

Distance has not been a barrier, as most of her trial patients drive about three hours to reach her center. Vulnerability indices such as income and household size did not predict participation, and retention has been strong.

Families value consistent coordinators, facilities, and physicians, which creates a routine and individualized care. Khalid said she once expected trials to feel like a burden, but many families told her they were grateful to be part of something bigger.

Wenderfer highlighted pediatric-specific requirements, including child assent, reassent as children mature, and transition to consent at the age of majority. Institutional review boards also apply added protections for children as a vulnerable population.

How Can Pediatric Nephrologists Get Involved in Glomerular Disease Trials?

Wenderfer said some trials now enroll children and adults together from the start. Others begin pediatric enrollment only after an adult trial succeeds, often starting with older adolescents and moving to younger age groups after data safety monitoring board review.

Khalid leads the PIONEER basket trial at Riley, which enrolls both children and adults with glomerular diseases.² Wenderfer said pediatric nephrologists can contribute beyond serving as site investigators, including protocol development, data safety monitoring boards, and industry roles.

Promoting equitable trial access in underrepresented regions is one of four core GD-Kids goals.¹ Wenderfer noted the working group includes members from North America, Europe, Japan, Hong Kong, and India, with plans to expand into South America and Africa.¹

Khalid said some newer agents may eventually allow clinicians to offer patients the prospect of disease elimination. Wenderfer said he hopes GD-Kids will draw more colleagues into clinical trial research.

References:

1. International Society of Glomerular Disease. International Society of Glomerular Disease launches GD-Kids to advance pediatric glomerular medicine worldwide. Published October 15, 2026. Accessed November 14, 2026. https://www.is-gd.org/en/news-from-isgd/a-global-effort-to-move-pediatric-glomerular-medicine-forward

2. Lucas S. First pediatric patient in the world enrolled in PIONEER clinical trial for kidney glomerular disease at IU School of Medicine. Indiana University School of Medicine. Published April 30, 2026. Accessed November 14, 2026. https://medicine.iu.edu/news/2026/04/kidney-glomerular-disease-trial