Tirzepatide trial in polycystic kidney disease begins enrolling 126 adults
The first trial of tirzepatide in polycystic kidney disease will test whether the drug changes kidney volume, but results are not expected until 2029.
A phase 2 trial testing tirzepatide in adults with autosomal dominant polycystic kidney disease (ADPKD) has begun randomizing participants, according to its registry entry [1]. The record describes it as the first trial of tirzepatide in this condition [1].
The study, registered as NCT06582875, plans to enroll 126 adults with ADPKD and a body mass index of at least 27 [1]. Participants are assigned at random to tirzepatide, titrated to 5 milligrams weekly, or to placebo, and are treated for 12 months [1]. Tirzepatide is sold as Mounjaro for type 2 diabetes and Zepbound for obesity; it is not approved to treat kidney disease, so this trial tests an investigational use [1].
The primary outcome is change in height-adjusted total kidney volume measured by MRI, which the trial describes as the standard marker of ADPKD progression [1]. Researchers will also measure a set of mechanistic markers: adiponectin, leptin, interleukin-6, TNF-alpha, high-sensitivity C-reactive protein and 8-isoprostane [1].
The trial is supported by the National Institute of Diabetes and Digestive and Kidney Diseases and led by the University of Colorado Denver, with Washington University and Mayo Clinic listed as collaborators [1]. People with diabetes and people who use tolvaptan cannot take part [1]. No results have been posted, and the primary completion date is listed as June 2029 [1].
Why it matters for patients
In ADPKD, total kidney volume grows over time, and height-adjusted total kidney volume is the standard measure researchers use to track that growth [1]. The trial is designed to find out whether a drug used mainly for weight loss and diabetes changes that measure over a year, and whether it shifts markers of inflammation and metabolism [1].
The design has limits that affect how the results will apply. With 126 participants, the study may only detect a large effect on kidney volume, and 12 months is a short window for a disease that progresses over decades [1]. Because diabetes is an exclusion, the findings will not directly describe people who have both ADPKD and type 2 diabetes, a group that is commonly prescribed GLP-1 and related drugs [1].
Because tolvaptan use is an exclusion, the trial will not show how tirzepatide performs alongside that treatment, and anyone already taking tolvaptan will not be represented in the results [1]. What tirzepatide does to kidney cyst growth, kidney function or kidney-related side effects in this population has not been reported [1].
For patients weighing whether to keep taking a GLP-1 or dual-agonist drug while living with ADPKD, the trial will not provide an answer for several years, and it is not set up to answer questions about kidney function or pain, only kidney volume and the listed laboratory markers [1].
What happens next
The trial is enrolling at the University of Colorado Denver, Washington University and Mayo Clinic [1]. Primary completion is listed as June 2029, and the registry shows no results posted [1]. How many participants have been randomized so far, and whether more sites will be added, has not been disclosed [1]. How the trial handles participants who need to start diabetes treatment or tolvaptan during the 12 months has not been reported [1].
Sources
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