This is an exploratory, randomized, double-blind, placebo-controlled, parallel group, multicenter, proof of concept study (Phase 2a), evaluating orally administered GLPG2737 for a double-blind (DB) treatment period of 52 weeks and 4 weeks of follow up as well as an open-label extension (OLE) treatment period of 52 weeks and 4 weeks of follow-up, in participants with rapidly progressing ADPKD.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
66
Cliniques Universitaires St. Luc (UCL)
Brussels, Belgium
UZ Leuven
Leuven, Belgium
DB Period: Mean Percent Change From MRI Baseline in htTKV
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).
Time frame: MRI Baseline up to Week 52
DB Period: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
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.
Time frame: From first dose to Week 56
DB Period: Mean Change From Baseline in eGFR
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).
Time frame: Baseline up to Week 52
DB Period: Area Under the Plasma Concentration-Time Curve During a Dosing Interval (AUCtau) of GLPG2737 and Its Metabolite
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Fakultni nemocnice u sv. Anny v Brne
Brno, Czechia
Fakultni nemocnice Hradec Kralove
Hradec Králové, Czechia
Vseobecna fakultni nemocnice v Praze
Prague, Czechia
Uniklinikum Dresden
Dresden, Germany
IRCSS Ospedale San Raffaele
Milan, Italy
Azienda Ospedaliera Universitaria Federico II
Napoli, Italy
Uni Campania L. Vanvitelli
Napoli, Italy
Fondazione Salvatore Maugeri IRCCS
Pavia, Italy
...and 10 more locations
AUC0-tau described the area under the curve limited to the end of a dosing interval. The metabolite of GLPG2737 is M4.
Time frame: Predose (within 30 minutes prior to dosing), 1, 1.5, 2, 3, 4, 5, 6, 7 hours post dose through Week 52
DB Period: Maximum Observed Plasma Concentration (Cmax) of GLPG2737 and Its Metabolite
Cmax is the maximum observed plasma concentration of the drug. The metabolite of GLPG2737 is M4.
Time frame: Predose (within 30 minutes prior to dosing), 1, 1.5, 2, 3, 4, 5, 6, 7 hours post dose through Week 52