Kidney Week 2025 Annual Meeting-Upping Our Game: Advances in Clinical Trials for Pediatric Kidney Diseases

Video Transcription

Video Summary

The session “Upping Our Game: Advances in Clinical Trials for Pediatric Kidney Diseases” highlighted the expanding push to include children in kidney disease clinical trials, addressing a long history of off-label prescribing in pediatric nephrology. Moderators emphasized new opportunities, including an upcoming global pediatric study of SGLT2 inhibitors to assess whether adult benefits and safety translate to children.<br /><br />A parent advocate, Anne Rohall-Andrade, shared her daughter Conley’s story following twin-to-twin transfusion syndrome leading to hypoplastic kidneys and eventual transplant. She stressed that pediatric kidney disease stories are universal across diagnoses, and described how clinical trials (IROC and VIRTUOS) can improve care and reduce invasive monitoring. Survey feedback from families showed major gaps: most were offered only one treatment option, few received emotional support or resources, and many did not understand what clinical trials are—underscoring the need for education, trust, and patient-centered support.<br /><br />An FDA presentation reviewed pediatric drug development standards, key laws (BPCA and PREA), and the challenge that orphan-drug exemptions limit required pediatric studies. The FDA emphasized innovative approaches for rare diseases: adaptive designs, external controls, master protocols, and pediatric extrapolation using adult data (including Bayesian borrowing).<br /><br />Dr. Cal Robinson detailed barriers to pediatric trials (rare outcomes, dispersed patients, limited infrastructure) and explained Bayesian adaptive designs, precision-medicine enrichment, N-of-1 trials, and “target trial emulation” using real-world data to improve causal inference when RCTs are infeasible.<br /><br />Adult nephrologist Rona Smith argued for earlier and deeper adult–pediatric collaboration to avoid decade-long delays (e.g., ACE inhibitors, SGLT2 inhibitors), promote age-inclusive trials, and align endpoints and regulatory strategies. The panel discussed practical obstacles—especially complex consent forms, transition gaps between pediatric and adult systems, and the urgency to recruit quickly before widespread off-label adoption undermines trials.

Asset Subtitle

Moderator(s): William Smoyer, Howard Trachtman

Presentation(s):

Introduction - William Smoyer
A Parent's Request to Increase Treatment Options for Children with Kidney Diseases - Anne Rohall-Andrade
Strategies in the Development and Evaluation of Treatments for Children with Rare Diseases - Lynne Yao
Novel Study Designs to Test Treatment Effectiveness - Cal Robinson
Opportunities for Collaboration with Adult Nephrologists in Clinical Trials for Children - Rona Smith

Note: Continuing education credits are not being offered for this session.

Meta Tag

Date 11/9/2025

Pathway 1 Pediatric Nephrology

Pathway 2 Glomerular Diseases

Session ID 504462

Keywords

pediatric kidney disease

pediatric nephrology

clinical trials

off-label prescribing

SGLT2 inhibitors

global pediatric study

kidney transplant

twin-to-twin transfusion syndrome

hypoplastic kidneys

patient advocacy

family education and support

FDA pediatric drug development

BPCA

PREA

Bayesian adaptive trial design