A Phase 2 Trial of the Safety and Efficacy of Bardoxolone Methyl in Patients With Rare Chronic Kidney Diseases - PHOENIX

NCT03366337 | Completed Phase 2 Results FDA Drug

• 1 other identifier: 402-C-1702
• Study Type: interventional
• Target: 103
• Locations: 1 country
• Sites: 30

Brief Summary

This multi-center, open-label Phase 2 trial will study the safety, tolerability, and efficacy of bardoxolone methyl in qualified patients with the following rare chronic kidney diseases (CKD): CKD associated with type 1 diabetes (T1D), IgA nephropathy (IgAN), focal segmental glomerulosclerosis (FSGS), and autosomal dominant polycystic kidney disease (ADPKD). Patients will be enrolled in disease specific cohorts within the trial, and effectiveness of bardoxolone methyl in treating CKD will be assessed separately by cohort for each rare CKD. All patients in the study will follow the same visit and assessment schedule. Following randomization on Day 1, patients will be scheduled to be assessed during treatment at Weeks 1, 2, 4, 6, 8, and 12, and by telephone contact on Days 3, 10, 21, 31, 38, and 45. Patients will also be scheduled to be assessed at an in-person follow-up visit at Week 16, four weeks after the end of treatment.

Conditions

IgA Nephropathy; CKD Associated With Type 1 Diabetes; Focal Segmental Glomerulosclerosis; Autosomal Dominant Polycystic Kidney

Keywords

IgA Nephropathy; CKD associated with type 1 diabetes; Focal segmental glomerulosclerosis; Autosomal dominant polycystic kidney disease; IgAN; FSGS; ADPKD; Bardoxolone methyl; CDDO-ME; RTA 402

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 12

  To assess the change in eGFR from baseline to week 12. eGFR is a measure of kidney function assessed through blood/serum. Higher eGFRs represent better/improved kidney function. Lower eGFRs represent poorer/decreased kidney function.

  12 weeks after participant receives the first dose

Study Arms (4)

Bardoxolone Methyl - ADPKD

EXPERIMENTAL

Participants with autosomal polycystic kidney disease (ADPKD) will receive bardoxolone methyl capsules at a starting dose of 5 mg and titrate up to a maximal dose of 20 mg (participants with UACR less than or equal to 300 mg/g) or 30 mg (participants with UACR greater than 300 mg/g) daily.

Drug: Bardoxolone methyl capsules

Bardoxolone Methyl - IgAN

EXPERIMENTAL

Participants with IgA nephropathy (IgAN) will receive bardoxolone methyl capsules at a starting dose of 5 mg and titrate up to a maximal dose of 20 mg (participants with UACR less than or equal to 300 mg/g) or 30 mg (participants with UACR greater than 300 mg/g) daily.

Drug: Bardoxolone methyl capsules

Bardoxolone Methyl - T1D

EXPERIMENTAL

Participants with Type 1 diabetes (T1D) will receive bardoxolone methyl capsules at a starting dose of 5 mg and titrate up to a maximal dose of 20 mg (participants with UACR less than or equal to 300 mg/g) or 30 mg (participants with UACR greater than 300 mg/g) daily.

Drug: Bardoxolone methyl capsules

Bardoxolone Methyl - FSGS

EXPERIMENTAL

Participants with focal segmental glomerulosclerosis (FSGS) will receive bardoxolone methyl capsules at a starting dose of 5 mg and titrate up to a maximal dose of 20 mg (participants with UACR less than or equal to 300 mg/g) or 30 mg (participants with UACR greater than 300 mg/g) daily.

Drug: Bardoxolone methyl capsules

Interventions

Bardoxolone methyl capsules DRUG

Bardoxolone 5 mg capsules

Also known as: RTA 402

Bardoxolone Methyl - ADPKD; Bardoxolone Methyl - FSGS; Bardoxolone Methyl - IgAN; Bardoxolone Methyl - T1D

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male and female patients 18 ≤ age ≤ 65 upon study consent;
• Screening eGFR (average of Screen A and Screen B eGFR values) ≥ 30 and ≤ 90 mL/min/1.73 m2. The two eGFR values collected at Screen A and Screen B visits used to determine eligibility must have a percent difference ≤ 25%;
• Albumin to creatinine ratio (ACR) ≤ 2500 mg/g at Screen B visit;
• If receiving an angiotensin-converting enzyme (ACE) inhibitor and/or an angiotensin II receptor blocker (ARB), patients should be prescribed the maximally tolerated labeled daily dose (MTLDD) for at least 6 weeks prior to the Screen A visit;
• For patients enrolling in T1D Cohort: Diagnosis of type 1 diabetes confirmed by fasting C-peptide level. Diagnosis must have been made ≤ 35 years of age; and prescribed stable dose of insulin to maintain adequate glucose control for at least 6 months prior to the Screen A visit;
• For patients enrolling in IgAN Cohort: Biopsy-confirmed IgA nephropathy;
• For patients enrolling in FSGS Cohort: Biopsy-confirmed FSGS that is not due to known secondary causes including morbid obesity, decreased renal mass, viral infections, drug-induced nephrotoxicity, or prior history of vasculitis;
• For patients enrolling in ADPKD Cohort: Genetic confirmation of PKD1 mutation;
• Adequate bone marrow reserve and organ function at the Screen A visit as follows: Hematologic: Absolute neutrophil count \> 1.5 x 109/L, platelets \> 100 x 109/L, hemoglobin (Hgb) ≥ 9 g/dL; and Hepatic: Total bilirubin (TBL) ≤ 1.5 times the upper limit of normal (ULN), alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 1.5 times ULN.

You may not qualify if:

• Kidney or any other solid organ transplant recipient or a planned transplant during the study;
• B-type natriuretic peptide (BNP) level \> 200 pg/mL at Screen A visit;
• Acute dialysis or acute kidney injury within 12 weeks prior to Screen A visit or during Screening;
• Serum albumin \< 3 g/dL at Screen A visit;
• Systemic immunosuppression for more than 2 weeks, cumulatively, within the 12 weeks prior to randomization or anticipated need for immunosuppression during the study;
• For patients enrolling in IgAN Cohort: Systemic manifestations of Henoch-Schonlein purpura within 1 year prior to Screen A visit; or have used belimumab, eculizumab, or rituximab within 6 months prior to Screen A visit;
• For patients enrolling in ADPKD Cohort: Receiving tolvaptan;
• Cerebrovascular event (stroke, transient ischemic attack) or aneurysm within 6 months prior to Screen A visit or during Screening;
• History of clinically significant left-sided heart disease and/or clinically significant cardiac disease;
• Uncontrolled systemic hypertension;
• Systolic BP \< 90 mm Hg at Screen A visit after a period of rest;
• History of malignancy within 2 years prior to Screen A visit, with the exception of localized skin or cervical carcinomas;
• Uncontrolled diabetes (HbA1c \> 10.0%) at Screen A visit;
• Untreated or uncontrolled active bacterial, fungal, or viral infection;
• Participation in other interventional clinical studies within 30 days prior to Day 1;

Sponsors & Collaborators

• Biogen: lead

Study Sites (30)

University of Alabama Birmingham

Birmingham, Alabama, 35294, United States

Location

Phoenician Centers for Research & Innovation (PCRI)

Phoenix, Arizona, 85021, United States

Location

University of California Los Angeles

Los Angeles, California, 90095, United States

Location

Denver Nephrology

Denver, Colorado, 80230, United States

Location

Gulfcoast Endocrine and Diabetes Center

Clearwater, Florida, 33756, United States

Location

South Florida Research Institute

Lauderdale Lakes, Florida, 33313, United States

Location

Coastal Nephrology Associates

Port Charlotte, Florida, 33952, United States

Location

Kidney Care Augusta

Augusta, Georgia, 30909, United States

Location

Boise Kidney & Hypertension, PLLC

Caldwell, Idaho, 83605, United States

Location

Boise Kidney & Hypertension, PLLC

Meridian, Idaho, 83642, United States

Location

Research By Design

Evergreen Park, Illinois, 60805, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Four Rivers Clinical Research, Inc

Paducah, Kentucky, 42003, United States

Location

Northwest Louisiana Nephrology

Shreveport, Louisiana, 71101, United States

Location

Tufts Medical Center - Division of Nephrology Tufts Medical Center

Boston, Massachusetts, 02110, United States

Location

Clinical Research Consultants, LLC

Kansas City, Missouri, 64111, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Columbia University Medical Center - Nephrology

New York, New York, 10032, United States

Location

Jacobi Medical Center

The Bronx, New York, 10461, United States

Location

Physician's East Endocrine Research

Greenville, North Carolina, 27834, United States

Location

Remington-Davis Clinical Research

Columbus, Ohio, 43215, United States

Location

Northeast Clinical Research Center, LLC

Bethlehem, Pennsylvania, 18017, United States

Location

The Warren Alpert School of Brown University

Providence, Rhode Island, 02903, United States

Location

Nephrology Associates

Chattanooga, Tennessee, 37404, United States

Location

Research Management, Inc.

Austin, Texas, 78751, United States

Location

Renal Disease Research Intitute

Dallas, Texas, 75235, United States

Location

Renal Associates, PA

San Antonio, Texas, 78215, United States

Location

Advanced Clinical Research

West Jordan, Utah, 84088, United States

Location

Mendez Center for Clinical Research LLC

Fairfax Station, Virginia, 22039, United States

Location

Larry Stonesifer, M.D.

Federal Way, Washington, 98003, United States

Location

MeSH Terms

Conditions

Glomerulonephritis, IGA; Glomerulosclerosis, Focal Segmental; Polycystic Kidney, Autosomal Dominant

Interventions

bardoxolone methyl

Condition Hierarchy (Ancestors)

Glomerulonephritis; Nephritis; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Autoimmune Diseases; Immune System Diseases; Polycystic Kidney Diseases; Kidney Diseases, Cystic; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Ciliopathies; Genetic Diseases, Inborn

Results Point of Contact

• Title: US Biogen Clinical Trial Center
• Organization: Biogen

clinicaltrials@biogen.com

866-633-4636

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 4, 2017
• First Posted: December 8, 2017
• Study Start: December 26, 2017
• Primary Completion: January 2, 2019
• Study Completion: January 29, 2019
• Last Updated: June 3, 2025
• Results First Posted: January 18, 2022

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will share

Locations

US (30)