Exploring Polycystic Kidney Disease Clinical Trials and New Treatments, Upcoming Webinar Hosted by Xtalks

In this free webinar, gain insight into the main challenges in patient recruitment for autosomal dominant polycystic kidney disease (ADPKD) clinical trials. Attendees will learn about the clinical presentations of patients with ADPKD. The featured speakers will share insights into the future of ADPKD treatment and potential new therapies.

TORONTO, Nov. 11, 2025 /PRNewswire/ -- This webinar will explore polycystic kidney disease (PKD) with a focus on autosomal dominant PKD (ADPKD), including clinical presentations, considerations for conducting clinical trials in this population and the future of ADPKD treatment.

In the United States, approximately 600,000 people have PKD¹, making it the fourth leading cause of kidney failure, and there is no cure presently available.² Men and women are equally at risk for this inherited disorder, and about 90% of PKD cases are ADPKD, meaning only one copy of the abnormal gene, either PKD1 or PKD2, is needed to cause the disease. Globally, ADPKD affects approximately 12 million people³ and is the most common cause of genetic kidney failure. Autosomal recessive PKD, or ARPKD, means that both parents must have and pass down the PKHD1 mutation to their child; ARPKD is extremely rare and only occurs in about one in 25,000 people.

Since 2014, genetic testing for ADPKD has improved in accuracy, availability and affordability, leading to higher usage; additional genes outside of PKD1 and PKD2 have also been associated with this disorder, and significant interest in improved outcomes for patients persists. There has been a noticeable increase in clinical trial activity related to potential new therapies for ADPKD over the last several years; tolvaptan was approved in 2015 and 2018 by Health Canada, the European Medicines Agency (EMA) and the US Food and Drug Administration, respectively, and there are more drugs in development for ADPKD now than ever before.⁴

The recently released Kidney Disease: Improving Global Outcome guidelines for ADPKD highlight the need for improved biomarkers of ADPKD progression and have led to focused research in the development of serum proteomics-based outcome prediction models.⁵ Beyond proteomics, another growing body of research surrounds ciliopathies, or primary cilia dysfunction. As ciliopathies lead to a wide range of disorders, the most common of which is ADPKD, and primary cilia are critical for the regulation of cellular metabolism, glutamine utilization via asparagine synthetase (ASNS) is another potential area of interest as a target for therapy.

The featured speakers will highlight the current landscape for ADPKD, factors to consider for clinical trials and what the future may hold for possible new therapies. They will consider the novel challenges and opportunities that these evolving questions present in addressing the urgent need for increasing the number of safe and effective treatments for people living with ADPKD.

Register for this webinar to learn how polycystic kidney disease clinical trials are shaping the future of ADPKD treatment.

Join Dr. Sayna Norouzi, Associate Professor of Medicine, Founder & Director of both the Polycystic Kidney Disease Clinic and the Glomerular Disease Clinic at Loma Linda University Medical Center; and experts from the PPD clinical research business of Thermo Fisher Scientific, Dr. Hannah Troutman, Nephrologist and Associate Medical Director; and Sarah Dixon Stump (moderator), MS, Project Oversight Senior Director, for the live webinar on Thursday, December 4, 2025, at 11am EST (5pm CET/EU-Central).

For more information, or to register for this event, visit Exploring Polycystic Kidney Disease Clinical Trials and New Treatments.

References:

1. Mahboob M, Rout P, Leslie SW, et al. Autosomal Dominant Polycystic Kidney Disease. [Updated 2024 Mar 20]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2025 Jan-. Available from: https://www.ncbi.nlm.nih.gov/sites/books/NBK532934
2. Polycystic kidney disease (PKD) - Symptoms, causes, treatment | National Kidney Foundation
3. KDIGO 2025 Clinical Practice Guideline for the Evaluation, Management, and Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD), Devuyst, Olivier et al. Kidney International, Volume 107, Issue 2, S1 - S239. KDIGO 2025 Clinical Practice Guideline for the Evaluation, Management, and Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) - Kidney International
4. Ostroff C, Perrone RD, Czerwiec FS. Current Challenges and Perspectives on Developing a Clinical Trial Design for ADPKD. Clin J Am Soc Nephrol. 2022 Oct;17(10):1559-1562. doi: 10.2215/CJN.05360522. Epub 2022 Aug 23. PMID: 35998976; PMCID: PMC9528266. Current Challenges and Perspectives on Developing a Clinical Trial Design for ADPKD - PMC
5. Aydogan Balaban, H.Ö., Arjune, S., Grundmann, F. et al. Developing serum proteomics based prediction models of disease progression in ADPKD. Nat Commun 16, 6646 (2025). https://doi.org/10.1038/s41467-025-61887-8

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