Autosomal dominant polycystic kidney disease MedDRA version: 20.0 Level: LLT Classification code 10036046 Term: Polycystic kidney, autosomal dominant System Organ Class: 100000004850
Conditions
Sponsors
Reata Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male and female patients 12 = age = 70 upon study consent; 2. Diagnosis of ADPKD: a. For adult (18 = age = 70) diagnosis of ADPKD by modified Pei-Ravine criteria: i. at least 3 cysts per kidney by sonography or at least 5 cysts by CT or MRI with family history of ADPKD; or ii. at least 10 cysts per kidney by any radiologic method and exclusion of other cystic kidney diseases if without family history; b. For adolescent (12 = age 1.5 x 10^9/L, platelets > 100 x 10^9/L, hemoglobin (Hgb) = 9 g/dL; b. Hepatic: Total bilirubin (TBL), alanine aminotransferase (ALT), and aspartate aminotransferase (AST) = the upper limit of normal (ULN); 7. Able to swallow capsules; 8. Willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures; 9. Evidence of a personally signed and dated informed consent/assent document indicating that the patient has been informed of all pertinent aspects of the study prior to initiation of any protocolmandated procedures. 10. Patients receiving an SGLT2 inhibitor must be on a stable dose for at least 4 weeks prior to the Screen A visit Are the trial subjects under 18? yes Number of subjects for this age range: 40 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 750 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 60
Exclusion criteria
Exclusion criteria: 1. Prior exposure to bardoxolone methyl; 2. Use of tolvaptan within 2 months prior to Screen A. Initiation of concomitant tolvaptan use during the study is not permitted; 3. History of administration of polycystic kidney disease-modifying agents (somatostatin analogues) within 2 months prior to the Screen A visit; 4. B-type natriuretic peptide (BNP) level > 200 pg/mL at Screen A visit; 5. Uncontrolled diabetes (HbA1c > 11.0%) at Screen A visit; 6. Serum albumin < 3 g/dL at Screen A visit; 7. History of intracranial aneurysms; 8. Kidney or any other solid organ transplant recipient or a planned transplant during the study; 9. Acute dialysis or acute kidney injury within 12 weeks prior to Screen A visit or during Screening; 10. History of clinically significant left-sided heart disease and/or clinically significant cardiac disease, including but not limited to any of the following: a. Clinically significant congenital or acquired valvular disease; b. Left ventricular ejection fraction < 40% (based on echocardiogram performed at Screen A visit or within 6 months prior to Day 1); c. Pericardial constriction (based on echocardiogram performed at Screen A visit or within 6 months prior to Day 1); d. Restrictive or congestive cardiomyopathy (based on echocardiogram performed at Screen A visit or within 6 months prior to Day 1); e. Symptomatic coronary disease (prior myocardial infarction, percutaneous coronary intervention, coronary artery bypass graft surgery, or angina); f. History of hospitalization for heart failure; g. Cardiac insufficiency, defined as New York Heart Association Class III or IV; h. History of untreated atrial fibrillation; i. History of unstable arrhythmias; 11. Systolic BP < 90 mm Hg at Screen A visit after a period of rest; 12. BMI < 18.5 kg/m^2 at the Screen A visit; 13. History of malignancy within 5 years prior to Screen A visit, with the exception of localized skin or cervical carcinomas; 14. Systemic immunosuppression for more than 2 weeks, cumulatively, within the 12 weeks prior to randomization or anticipated need for immunosuppression during the study; 15. Untreated or uncontrolled active bacterial, fungal, or viral infection; 16. Participation in other interventional clinical studies within 30 days prior to Day 1; 17. Unwilling to practice acceptable methods of birth control (both males who have partners of childbearing potential and females of childbearing potential) during Screening, while taking study drug, and for at least 30 days after the last dose of study drug is ingested; 18. Women who are pregnant or breastfeeding; 19. Known hypersensitivity to any component of the study drug; 20. Any abnormal laboratory level that, in the opinion of the investigator, would put the patient at risk by trial enrollment; 21. Patient is, in the opinion of the investigator, unable to comply with the requirements of the study protocol or is unsuitable for the study for any reason; 22. Coronavirus disease 2019 (COVID-19) pneumonia, related acute kidney injury, or related hospitalization within 6 months prior to Day 1.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: - To assess the off-treatment change from baseline in estimated glomerular filtration rate (eGFR) at Week 108. - To assess safety and tolerability of bardoxolone methyl. ;Secondary Objective: To assess the change from baseline in eGFR at Week 100.;Primary end point(s): Efficacy: Off-treatment change from baseline in eGFR at Week 108. Safety and Tolerability: Frequency, intensity, and relationship to study drug of AEs and SAEs, and change from baseline in the following assessments: vital sign measurements, 12-lead ECGs, clinical laboratory measurements, pediatric growth (height and weight), and sexual maturity using Tanner staging.;Timepoint(s) of evaluation of this end point: The primary efficacy endpoint will be analyzed after all enrolled patients have completed the study and the database has been locked. The primary safety endpoint will be assessed throughout the clinical trial. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Change from baseline in eGFR at Week 100.;Timepoint(s) of evaluation of this end point: The secondary efficacy endpoint will be analyzed after all enrolled patients have completed the study and the database has been locked. | — |
Countries
Australia, Belgium, Czechia, Czech Republic, Denmark, France, Germany, Hungary, Italy, Japan, Poland, Spain, United Kingdom, United States
Contacts
Public ContactClinical Program Operations
Reata Pharmaceuticals, Inc.
Outcome results
None listed