For a long time, children with kidney diseases that filter the blood through glomeruli were left to inherit the answers meant for adults, borrowing dosages and hopes from a different body and a different stage of life. Two pediatric nephrologists now describe a turning point, in which the old reflex to shield children from research is giving way to a conviction that protection and participation can coexist. Their clinics suggest that families, far from being too burdened by distance, income, or household size to take part, have been willing partners once trust is patiently built. The question
Pediatric Glomerular Disease Trials Are Catching Up, Clinicians Say, as Myths About Family Participation Fall
Rather than protecting them from research, we're advocating for them to be involved
Mark
Why does it matter that children were left out of these trials?
Mimi
Because without trials, pediatric nephrologists have had to borrow adult findings on safety and efficacy. Wenderfer says that limited the evidence behind pediatric consensus treatment plans.
Mark
And the old thinking was protective?
Mimi
Yes. A tendency to protect children from research effectively barred them. He says the field now advocates for inclusion, partly because trial participants get closer follow-up and more safeguards than routine care.
Luke
Worth noting that's his view of the safeguards. Nothing here quantifies how outcomes compare.
Mark
What surprised Khalid about families?
Mimi
Parents enrolled once trust was built. Most of her trial patients drive about three hours, and income and household size didn't predict participation.
Luke
That's one clinic's experience, in IgA nephropathy trials. No enrollment or retention numbers are given, just that retention has been strong.
Mark
What does trust look like in practice?
Mimi
Teaching families the science and the disease, and keeping the same coordinators, facilities, and physicians.
Mark
Where is this heading?
Mimi
Trials enrolling children and adults together, the PIONEER basket trial at Riley, and GD-Kids expanding toward South America and Africa.
Luke
Khalid says newer agents may eventually offer disease elimination. That's a hope, not a result yet.
El Pulso
- Children with glomerular diseases have waited years for medicines tested on their own bodies, while pediatric care has leaned on adult evidence.
- A long-held instinct to protect children from research kept them out of trials, and that caution is now being challenged.
- Clinicians are pushing to enroll children alongside adults, with new basket trials such as PIONEER opening doors at Riley Hospital.
- Assumptions that distant or low-income families cannot participate are being overturned by strong enrollment and retention.
- The GD-Kids working group is racing to extend equitable trial access into South America and Africa, where participation remains uncertain.
For a long time, children with kidney diseases that filter the blood through glomeruli were left to inherit the answers meant for adults, borrowing dosages and hopes from a different body and a different stage of life. Two pediatric nephrologists now describe a turning point, in which the old reflex to shield children from research is giving way to a conviction that protection and participation can coexist. Their clinics suggest that families, far from being too burdened by distance, income, or household size to take part, have been willing partners once trust is patiently built. The question that remains is whether the trials now beginning to include children will reach the families who have so far been left out.
Most of Myda Khalid's trial patients at Riley Hospital for Children in Indiana drive about three hours to reach her clinic, and many of them keep coming back. Her experience, along with that of Scott Wenderfer at BC Children's Hospital, challenges much of what the field once assumed about families and research.
For years, children with glomerular kidney diseases had far fewer tested therapies than adults, so pediatric doctors relied on extrapolating from adult data. Wenderfer traced part of that gap to a habit of shielding children from research, which in practice left them out. That stance has now reversed, and the field advocates for children's participation, since trial participants often receive closer follow-up and more safeguards than in routine care. Khalid also noted that the trials simply were not available until recently, and that regulators, scientists, and patient groups have since pushed for inclusion.
The absence of evidence has real costs. Khalid described a young patient who regularly asked when she would get a medicine made for her disease. From her IgA nephropathy trial work, Khalid said her clinic has found that parents are willing to enroll their children once trust exists, and that trust grows through teaching families about the disease and building a relationship with the care team. Distance, income, and household size did not predict participation, and families have stayed in studies, valuing consistent coordinators, facilities, and physicians. Many told her they were grateful to be part of something larger.
Pediatric research carries its own safeguards, including assent from the child, reassent as the child matures, and consent at the age of majority, along with review board protections. Some trials now enroll children and adults together, while others start with older adolescents. Khalid leads the PIONEER basket trial at Riley, and nephrologists can contribute well beyond site work, through protocol design, safety monitoring, and industry roles.
GD-Kids, launched in October 2026 by the International Society of Glomerular Disease, aims to bring equitable access to underrepresented regions and plans to expand into South America and Africa. Khalid hopes newer agents may one day let clinicians offer the prospect of eliminating disease, and Wenderfer hopes the group will draw more colleagues into trial research.
Most of Myda Khalid's trial patients at Riley Hospital for Children in Indiana drive about three hours to reach her clinic. Many of them are children with glomerular disease, and they keep coming back. That fact runs against what the field long assumed about families and research, and it is one of several assumptions she and a colleague say have been disproven.
Khalid and Scott Wenderfer, MD, PhD, of BC Children's Hospital and the University of British Columbia, spoke with HCPLive about why pediatric glomerular disease trials have lagged. The two co-chair the GD-Kids working group of the International Society of Glomerular Disease. Historically, children with these kidney diseases have had fewer therapies developed and tested for them than adults. Pediatric nephrologists have had to extrapolate safety and efficacy from adult data. As targeted agents move through adult pipelines, the pediatric trials are starting to catch up.
Wenderfer traced part of the gap to a longstanding instinct to shield children from research, which in practice kept them out of trials. He said the stance has reversed: the field now advocates for children to take part. He noted that trial participants get closer follow-up and more safeguards than they would in routine care, and that a drug can work well and safely in children even when it is less effective or less safe in adults. Khalid offered a simpler explanation: the trials were not available until recently. Regulators, scientists, physicians, and patient organizations have since pushed for pediatric inclusion, and the number of trials is rising.
The missing evidence has consequences. Wenderfer, whose work centers on lupus nephritis, said the shortage of trials has limited the evidence behind pediatric consensus treatment plans. Khalid, who runs a glomerular disease research clinic at Riley, described a young patient who regularly asked when she would get a medicine made for her disease.
From her IgA nephropathy trial experience, Khalid listed what her clinic has shown to be wrong. Parents were not unwilling to enroll their children once trust existed. She said building that trust meant teaching families about the disease and the science and bringing them into a relationship with the physician team. Distance has not been a barrier. Vulnerability indices such as income and household size did not predict participation, and retention has been strong. Families, she said, value having the same coordinators, facilities, and physicians, which creates a routine and individualized care. She once expected trials to feel like a burden, but many families told her they were grateful to be part of something bigger.
Wenderfer pointed to requirements specific to pediatric research: assent from the child, reassent as the child matures, and a transition to consent at the age of majority. Institutional review boards also add protections for children as a vulnerable population.
On how nephrologists can get involved, Wenderfer said some trials now enroll children and adults together from the start. Others open to children only after an adult trial succeeds, often beginning with older adolescents and moving to younger groups after review by a data safety monitoring board. Khalid leads the PIONEER basket trial at Riley, which enrolls children and adults with glomerular diseases; Indiana University School of Medicine reported in April 2026 that the world's first pediatric patient had been enrolled. Wenderfer said pediatric nephrologists can contribute beyond serving as site investigators, including in protocol development, data safety monitoring boards, and industry roles.
GD-Kids, launched by the International Society of Glomerular Disease in October 2026, lists equitable trial access in underrepresented regions among its four core goals. Its members come from North America, Europe, Japan, Hong Kong, and India, with plans to expand into South America and Africa. Khalid said some newer agents may eventually let clinicians offer patients the prospect of disease elimination, and Wenderfer said he hopes the group will draw more colleagues into trial research.
Citas Notables
Now rather than protecting them from research, we're advocating for them to be involved with research.— Scott Wenderfer, MD, PhD
It was important to teach people about the disease and the science and bring them to a place of trust in the physician team.— Myda Khalid, MD (paraphrased)
Quieres la nota completa?
Lee el original en HCPLive ↗