The goal of the study was to compare and evaluate safety and efficacy of tesevatinib 50 milligrams (mg) versus placebo in participants with autosomal dominant polycystic kidney disease (ADPKD).
Safety and efficacy of 50 mg tesevatinib in comparison to placebo in participants with ADPKD was assessed. The primary purpose of this study was focused on evaluating the change from Baseline in height-adjusted total kidney volume (htTKV) as measured by magnetic resonance imaging (MRI) at Months 12, 18, and 24, and 30 days post-dose in participants with ADPKD treated with tesevatinib or placebo. If eligible for the study participation, participants were randomly assigned to either investigational treatment group or placebo group. Treatment group received 50 mg tesevatinib once daily for 24 months and control group received the placebo once daily for 24 months.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
80
Pharmaceutical form: Tablet; Route of administration: orally
Pharmaceutical form: Tablet (identical to tesevatinib); Route of administration: orally
University of California San Diego
La Jolla, California, United States
California Institute for Renal Research
Change From Baseline in Height Adjusted Total Kidney Volume (htTKV) at Month 12
htTKV was calculated using total kidney volume (in milliliters) obtained from magnetic resonance imaging (MRI) divided by height (in meters). Least square (LS) mean and standard error (SE) were estimated by using analysis of covariance (ANCOVA) model. Change from Baseline in htTKV at Month 12 was reported in this outcome measure.
Time frame: Baseline (Day 1), Month 12
Change From Baseline in Height Adjusted Total Kidney Volume at Month 18
htTKV was calculated using total kidney volume (in milliliters) obtained from MRI divided by height (in meters). LS mean and SE were estimated by using ANCOVA model. Change from Baseline in htTKV at Month 18 was reported in this outcome measure.
Time frame: Baseline (Day 1), Month 18
Change From Baseline in Height Adjusted Total Kidney Volume at Month 24
htTKV was calculated using total kidney volume (in milliliters) obtained from MRI divided by height (in meters). LS mean and SE were estimated by using ANCOVA model. Change from Baseline in htTKV at Month 24 was reported in this outcome measure.
Time frame: Baseline (Day 1), Month 24
Change From Baseline in Height Adjusted Total Kidney Volume at End of Study
htTKV was calculated using total kidney volume (in milliliters) obtained from MRI divided by height (in meters). LS mean and SE were estimated by using ANCOVA model. Change from Baseline in htTKV at end of study (i.e., up to 26 months) was reported in this outcome measure.
Time frame: Baseline (Day 1), End of study (anytime up to 26 months)
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
An adverse event (AE) was any untoward medical occurrence in a clinical study participant administered a medicinal product and which did not necessarily had to have a causal relationship with the treatment. TEAEs were AEs that developed or worsened or became serious from the first dose (Day 1) of the study drug until end of study.
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La Mesa, California, United States
University of California Los Angeles
Los Angeles, California, United States
University of California San Francisco
San Francisco, California, United States
University of Colorado Denver
Aurora, Colorado, United States
Yale Nephrology Clinical Research
New Haven, Connecticut, United States
Coastal Nephrology Associates Research Center, LLC
Port Charlotte, Florida, United States
Genesis Clinical Research
Tampa, Florida, United States
Emory University School of Medicine
Atlanta, Georgia, United States
University of Maryland
Baltimore, Maryland, United States
...and 9 more locations
Time frame: From Baseline (Day 1) up to 30 days post last dose of study drug (i.e., up to 26 months)
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Months 12, 18, 24 and End of Study
eGFR is a test for renal function. eGFR was calculated by using the 4-variable modification of diet in renal disease (MDRD-4) formula reported as milliliters per minute per 1.73 square meter (mL/min/1.73 m\^2). LS mean and SE were estimated using ANCOVA model.
Time frame: Baseline (Day 1), Months 12, 18, 24 and at end of study (i.e., anytime up to 26 months)