Abstract: TH-PO0194
Scoping Review of Clinical Trial Outcomes in Patients with ADPKD
Session Information
- ADPKD and Cystic Kidney Disease - 1
October 22, 2026 | Location: Exhibit Hall A, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Genetic Diseases of the Kidneys
- 1201 Genetic Diseases of the Kidneys: Cystic (Monogenic)
Authors
- Dan, Yuet Ruh, Duke-NUS Medical School, Singapore, Singapore
- Chua, Eileen, Duke-NUS Medical School, Singapore, Singapore
- Poon, Ernest, Duke-NUS Medical School, Singapore, Singapore
- Kwek, Jia Liang, Singapore General Hospital, Singapore, Singapore
- Wang, Angela Yee-Moon, Duke-NUS Medical School, Singapore, Singapore
Background
Clinical trials in ADPKD have increased in recent years as more interventions become available for evaluation. Despite SONG-PKD standardisation efforts, significant heterogeneity exists in trial endpoints' definitions. This scoping review aims to present an updated descriptive analysis of outcomes used in clinical trials with ADPKD, focusing on kidney outcomes and patient-reported outcomes (PROs).
Methods
A systematic search was conducted on PubMed, WebofScience & ClinicalTrials.gov for randomized clinical trials, trial protocols & post-hoc trial analyses with ADPKD published between 2021 - November 2025, with screening of reference lists. Non-adult populations, non-randomised trials, observational & feasibility studies were excluded. Information on trial design, trial duration, types of intervention & trial outcomes were extracted. Outcomes were classified as kidney outcomes, PROs, cardiac outcomes, intervention-related outcomes & others. The outcomes' definition (eg. slope or rate of change) & methods of measurement were extracted.
Results
Overall, 97 clinical trials were included in the scoping review, with 87 outcome measures defined across 393 primary & secondary outcomes. Kidney outcomes included 15 kidney function & structure outcomes. Kidney function outcome was most commonly assessed using estimated glomerular filtration rate (eGFR) but defined in 3 different ways (absolute change: n=10, rate of change: n=8, % change in eGFR: n=1) & included 2 hard endpoints. Kidney structure was reported as 5 different outcomes, with total kidney volume (TKV) being the most commonly reported but reported in 3 different ways (absolute change: n=16, % change: n=8 & rate of change in TKV: n=4). PROs were reported in 10 studies & as primary outcomes in 4 studies. Pain & quality of life were assessed using 10 & 7 different questionnaires respectively. Generally, not all SONG-PKD core outcomes were reported & definitions of these core outcomes varied.
Conclusion
This scoping review observes substantial heterogeneity in kidney outcomes' definitions adopted in ADPKD trials, limiting comparisons of effectiveness of interventions across trials. PROs are also under-utilized despite SONG-PKD recommendations. The findings highlight the need for standardization of definition of kidney function, kidney volume and PROs as clinical trial outcomes in ADPKD in order for meaningful comparisons across trials.