Trial Outcomes & Findings for A Study to Evaluate the Effects of GLPG2737 in Participants With Autosomal Dominant Polycystic Kidney Disease (ADPKD) (NCT NCT04578548)

Participants with autosomal dominant polycystic kidney disease (ADPKD) were enrolled. The study was conducted at 20 sites in 7 countries i.e. Belgium, Czech Republic, Germany, Italy, Netherlands, Poland, and Spain. Out of these 20 activated sites, 3 sites did not enroll any participants. 89 participants were screened, out of which 66 were randomized.

Participants who met protocol eligibility criteria were assigned to GLPG2737 or placebo in the double-blind (DB) treatment period and open-label extension (OLE) period. The study was prematurely terminated on 02 March 2023, due to the lack of efficacy of GLPG2737, making the expected benefit-risk balance negative.

SAS. Participants with MRI baseline were analyzed.

htTKV is used in participants with ADPKD disease to predict the onset of renal insufficiency. htTKV was calculated using TKV (in mL) obtained from MRI divided by height (in m). MRI Baseline: For MRI assessments, all non-missing values before the first study drug administration in the study +14 days (included) was considered as the primary baseline definition. Results were derived by mean of the individual slopes (i.e. using all MRI performed between baseline and Week 52).

An AE is any untoward medical occurrence in a participant administered a study drug, and which does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of the study drug, whether or not considered related to it. A serious adverse event (SAE) is defined as any untoward medical occurrence that results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results a congenital anomaly/birth defect or other medically important event. A TEAE was defined as an AE observed after starting administration of the study drug until 30 days after last DB dose or 1 day before OLE dose, whichever occurred first.

The eGFR is a test that measures level of kidney function and determines the stage of kidney disease. eGFR was based on CKD-EPI formula (2009) calculated from serum creatinine concentrations. Results were derived by mean of the individual slopes (i.e. using data between baseline and Week 52).

AUC0-tau described the area under the curve limited to the end of a dosing interval. The metabolite of GLPG2737 is M4.

Cmax is the maximum observed plasma concentration of the drug. The metabolite of GLPG2737 is M4.