Recognizing National Kidney Month by Breaking Down Barriers to Improve Clinical Trials for Glomerular Diseases
To raise awareness about kidney diseases during National Kidney Month, we’d like to focus on rare renal diseases and the importance of studying not only adults but the pediatric population as well. Several rare glomerular diseases1 can impact the kidney’s important cleansing function. Tiny clusters of looping blood vessels, called glomeruli, sit within each kidney’s approximately 1 million nephrons. Similar to other kidney diseases, glomerular diseases often progress silently and remain undetected until the disease becomes more advanced. Once diagnosed, treatment options have historically been limited, but recent clinical trials and drug approvals have been encouraging.
Two of our specialists, Barbara Gillespie, MD, MMS, FASN, Vice President and Therapeutic Head of Nephrology, and Cindy Jackson, DO, FAAP, Vice President and General Manager, Rare Diseases and Pediatrics, recently spoke about their work in glomerular diseases as part of the nephrology community and commented on a recently published editorial in Kidney International that listed Dr. Gillespie as a co-author.
Connecting in an international clinical trial collaborative
In November 2018, NephCure Kidney International formed an international clinical trial collaborative that was supported by the FDA and the Kidney Health Initiative. Of the 90 stakeholders invited, two from our organization, Dr. Gillespie (a nephrologist) co-chaired the clinician’s workgroup; Dr. Jackson (a pediatrician) participated in a workgroup focused on pediatric inclusion in these rare renal disease trials. From this workgroup, Dr. Jackson later was connected to the American Society of Pediatric Nephrology Therapeutics workgroup and now actively participates in their site readiness workstream.
The editorial article, titled “Innovating and invigorating the clinical trial infrastructure for glomerular diseases,” highlighted the collaborative’s efforts to address the hurdles associated with clinical research in glomerular diseases.
One key issue cited was the lack of glomerular disease clinicians; out of approximately 11,000 practicing nephrologists, only 350 are conducting clinical trials. In addition, community physicians have not always referred eligible patients for trials for various reasons; this exacerbates patient recruitment efforts, which remains a common challenge in any rare disease clinical trial.
Promoting glomerular disease studies in the pediatric population
To help curb the progression of the disease as soon as possible, glomerular disease clinical trials must consider the ongoing need to also study pediatric populations.
“Renal disease doesn’t just affect adults,” explained Dr. Jackson. “In fact, around 40% of these glomerular diseases present in childhood. If you can affect a positive change in renal function when a patient is young, you have a better chance of stopping the damage. As I learn from the nephrologists I have worked with, ‘it’s all about preserving nephrons,’ and in the case of children, the earlier, the better.”
Dr. Gillespie, an adjunct professor who cares for adult patients at the University of North Carolina in addition to her work at Labcorp, agrees.
“As an adult nephrologist, it’s tough to see a patient that has been a pediatric nephrology patient for years,” said Dr. Gillespie. “These children are often put on steroid treatments that have many side effects, which can also impact their emotional wellbeing.
When I work with sponsors studying kidney disease, I’ve learned that it’s important to ask about their pediatric investigation plan (PIP). Drugs being developed with Orphan Disease Designations aren’t mandated to have a PIP by the FDA, so it’s important for our team to promote the need to study the pediatric population as well.”
Looking ahead to grow glomerular disease research
Both Dr. Gillespie and Dr. Jackson feel hopeful about the future of and interest in drug development for rare renal diseases. They mentioned that in 2015, there were no Phase II/III trials in Immunoglobulin A Nephropathy (IgAN) and only two in Focal Segmental Glomerulosclerosis (FSGS), but just four years later, there was a dramatic change with 11 Phase II and III trials in FSGS and 13 in IgAN. As of the editorial’s publication date in January 2021, there were 20 clinical trials in primary glomerular diseases; four of these trials included a pediatric population.
We have also been part of the rapid growth in nephrology trials.
“We have expanded our footprint in nephrology trials to include the Asia Pacific region and formed a partnership with an academic research organization (ARO) in Australia,” said Dr. Gillespie. “Our sponsors have also benefited from Labcorp data to help better understand the current patient populations and ensure a protocol’s exclusion/inclusion criteria are appropriate.”
The current momentum in advancing glomerular disease therapeutics is promising, but ongoing success will require a concerted and collaborative effort between pharmaceutical companies, renal pathologists, nephrologists and regulatory agencies to improve clinical trial design and bolster trial recruitment. Labcorp will continue to contribute to the nephrology community and advocate for running rare disease trials that also include the pediatric population.