Scott Wenderfer, MD, PhD, is Division Head of Nephrology at BC Children's Hospital Research Institute (BCCHR) and Clinical Professor in the Department of Pediatrics at the University of British Columbia.

Scott Wenderfer, MD, PhD

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)

 about the obstacles and how the field is addressing them.¹

Why Children Have Been Left Out of Glomerular Disease Trials

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.

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

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

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Myda Khalid, MD, and Scott Wenderfer, MD, PhD, discuss barriers to pediatric glomerular disease clinical trials and the myths they have seen disproven.

Overcoming Barriers to Pediatric Glomerular Disease Clinical Trials

Pediatric glomerular disease clinical trials have long trailed adult research, leaving pediatric nephrologists with few trained investigators, limited infrastructure, and little formal guidance on how to start. The 

 formed the GD-Kids working group in 2026 to help close this gap.¹

At the inaugural ISGD Clinical Trials Summit in Galveston, Texas, co-chairs 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, spoke with 

 about why the group was needed and how it is training the next generation of investigators.

Training the Next Generation of Pediatric Glomerular Disease Trialists | GD-Kids

Khalid said new glomerular disease therapies developed for adults are now beginning to expand into children. GD-Kids gives physicians, sponsors, patients, and others in the space a shared platform to improve how pediatric trials are run.

Why Do Pediatric Nephrologists Need a Dedicated Glomerular Disease Trials Group?

Wenderfer described GD-Kids as a home for pediatric nephrologists interested in glomerular disease. He said pediatric investigators needed a society to advocate for their priorities, and ISGD’s emphasis on pediatric trials drew him to the working group.

Khalid noted the group’s international makeup quickly became one of its strengths. She said she has already learned from colleagues in Asia and Europe and hopes to draw on other regions as the group grows.

ISGD has outlined 4 core goals for GD-Kids: building global pediatric trial capacity, strengthening collaboration across institutions and regions, promoting equitable trial access in underrepresented regions, and developing shared best practices for trial design, start-up, and execution.¹ Initial priorities include a global survey of experienced investigators and sites to identify barriers and needed resources.¹

Efforts such as the PIONEER pediatric gateway initiative have similarly sought earlier integration of children into glomerular disease drug development.²

How Are Clinical Trial Boot Camps Preparing Pediatric Investigators?

GD-Kids grew out of ISGD’s first Clinical Trials Bootcamp, held during the 2025 European Society for Paediatric Nephrology (ESPN) meeting in Athens, Greece.¹ Khalid said the program targeted fellows, trainees, and junior faculty. It introduced basic clinical trial concepts, common challenges, and strategies to overcome them while building a community of early-career investigators.

A second boot camp followed in Miami in partnership with the Pediatric Nephrology Research Consortium, at a meeting focused on trainees. Khalid and Wenderfer both participated in a more recent boot camp in Bordeaux, France, and a combined ISGD and ESPN boot camp is planned for Florence, Italy, next year.

Wenderfer noted many ESPN juniors return to the boot camps year after year. This allows faculty to build on prior sessions and help attendees grow more comfortable in the glomerular disease space over time.

Khalid recalled starting clinical trial work 8 to 10 years ago, when only a handful of pediatric nephrologists were running trials.

“I remember asking people, ‘Hey, does anybody know how to do these? Is there a booklet? Do I know where to start?’ And in fact, I couldn’t find an answer,” Khalid said.

She said the boot camps could help the field compile a formal curriculum and a faculty network newcomers can turn to.

Wenderfer said many clinicians want to participate in research and give their patients access to innovative therapies but do not know where to begin. He added fellows in particular struggle to find clinical trial exposure during academic training, and the boot camps offer an early entry point.

Gipson DS, Tarnoff J, Lee L, et al. A pediatric gateway initiative for glomerular disease: introducing PIONEER. Kidney Int. 2021;99(3):515-518. doi:10.1016/j.kint.2020.11.013

Myda Khalid, MD, and Scott Wenderfer, MD, PhD, explain how ISGD’s GD-Kids group and its clinical trial boot camps aim to build pediatric glomerular disease research capacity.

Scott Wenderfer, MD, PhD

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