Trial Outcomes & Findings for A Phase 2 Trial of the Safety and Efficacy of Bardoxolone Methyl in Patients With Rare Chronic Kidney Diseases - PHOENIX (NCT NCT03366337)

Last Updated: 2025-06-03

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

103 participants

Primary outcome timeframe

12 weeks after participant receives the first dose

Results posted on

2025-06-03

Participant Flow

Participant milestones

Participant milestones
Measure	Bardoxolone Methyl - ADPKD Participants with autosomal polycystic kidney disease (ADPKD) who received 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. Bardoxolone methyl capsules: Bardoxolone 5 mg capsules	Bardoxolone Methyl - IgAN Participants with IgA nephropathy (IgAN) who received 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. Bardoxolone methyl capsules: Bardoxolone 5 mg capsules	Bardoxolone Methyl - T1D Participants with Type 1 diabetes (T1D) who received 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. Bardoxolone methyl capsules: Bardoxolone 5 mg capsules	Bardoxolone Methyl - FSGS Participants with focal segmental glomerulosclerosis (FSGS) who received 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. Bardoxolone methyl capsules: Bardoxolone 5 mg capsules
Overall Study STARTED	31	26	28	18
Overall Study COMPLETED	28	26	26	17
Overall Study NOT COMPLETED	3	0	2	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Bardoxolone Methyl - ADPKD Participants with autosomal polycystic kidney disease (ADPKD) who received 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. Bardoxolone methyl capsules: Bardoxolone 5 mg capsules	Bardoxolone Methyl - IgAN Participants with IgA nephropathy (IgAN) who received 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. Bardoxolone methyl capsules: Bardoxolone 5 mg capsules	Bardoxolone Methyl - T1D Participants with Type 1 diabetes (T1D) who received 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. Bardoxolone methyl capsules: Bardoxolone 5 mg capsules	Bardoxolone Methyl - FSGS Participants with focal segmental glomerulosclerosis (FSGS) who received 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. Bardoxolone methyl capsules: Bardoxolone 5 mg capsules
Overall Study Lost to Follow-up	1	0	1	0
Overall Study Withdrawal by Subject	2	0	1	1

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Bardoxolone Methyl - ADPKD n=31 Participants Participants with autosomal polycystic kidney disease (ADPKD) who received 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. Bardoxolone methyl capsules: Bardoxolone 5 mg capsules	Bardoxolone Methyl - IgAN n=26 Participants Participants with IgA nephropathy (IgAN) who received 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. Bardoxolone methyl capsules: Bardoxolone 5 mg capsules	Bardoxolone Methyl - T1D n=28 Participants Participants with Type 1 diabetes (T1D) who received 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. Bardoxolone methyl capsules: Bardoxolone 5 mg capsules	Bardoxolone Methyl - FSGS n=18 Participants Participants with focal segmental glomerulosclerosis (FSGS) who received 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. Bardoxolone methyl capsules: Bardoxolone 5 mg capsules	Total n=103 Participants Total of all reporting groups
Age, Continuous	47.4 years STANDARD_DEVIATION 9.49 • n=5 Participants	48.5 years STANDARD_DEVIATION 9.53 • n=7 Participants	49 years STANDARD_DEVIATION 9.91 • n=5 Participants	48.6 years STANDARD_DEVIATION 12.79 • n=4 Participants	48.3 years STANDARD_DEVIATION 10.12 • n=21 Participants
Sex: Female, Male Female	21 Participants n=5 Participants	11 Participants n=7 Participants	21 Participants n=5 Participants	10 Participants n=4 Participants	63 Participants n=21 Participants
Sex: Female, Male Male	10 Participants n=5 Participants	15 Participants n=7 Participants	7 Participants n=5 Participants	8 Participants n=4 Participants	40 Participants n=21 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants	1 Participants n=4 Participants	8 Participants n=21 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	28 Participants n=5 Participants	24 Participants n=7 Participants	26 Participants n=5 Participants	17 Participants n=4 Participants	95 Participants n=21 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants	2 Participants n=21 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	4 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants	6 Participants n=21 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Black or African American	4 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	5 Participants n=4 Participants	10 Participants n=21 Participants
Race (NIH/OMB) White	25 Participants n=5 Participants	22 Participants n=7 Participants	27 Participants n=5 Participants	11 Participants n=4 Participants	85 Participants n=21 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Baseline estimated glomerular filtration rate (eGFR)	47.69 mL/min/1.73 m^2 STANDARD_DEVIATION 13.630 • n=5 Participants	46.19 mL/min/1.73 m^2 STANDARD_DEVIATION 12.576 • n=7 Participants	67.52 mL/min/1.73 m^2 STANDARD_DEVIATION 17.059 • n=5 Participants	51.66 mL/min/1.73 m^2 STANDARD_DEVIATION 18.144 • n=4 Participants	53.39 mL/min/1.73 m^2 STANDARD_DEVIATION 17.427 • n=21 Participants
Baseline urine albumin-to-creatinine ratio (UACR)	44.40 mg/g n=5 Participants	104.03 mg/g n=7 Participants	30.88 mg/g n=5 Participants	184.30 mg/g n=4 Participants	63.95 mg/g n=21 Participants

PRIMARY outcome

Timeframe: 12 weeks after participant receives the first dose

Population: Intent-to-treat population (all enrolled patients)

Outcome measures

Outcome measures
Measure	Bardoxolone Methyl - ADPKD n=31 Participants Participants with autosomal polycystic kidney disease (ADPKD) who received 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. Bardoxolone methyl capsules: Bardoxolone 5 mg capsules	Bardoxolone Methyl - IgAN n=26 Participants Participants with IgA nephropathy (IgAN) who received 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. Bardoxolone methyl capsules: Bardoxolone 5 mg capsules	Bardoxolone Methyl - T1D n=28 Participants Participants with Type 1 diabetes (T1D) who received 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. Bardoxolone methyl capsules: Bardoxolone 5 mg capsules	Bardoxolone Methyl - FSGS n=18 Participants Participants with focal segmental glomerulosclerosis (FSGS) who received 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. Bardoxolone methyl capsules: Bardoxolone 5 mg capsules
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 12	9.31 mL/min/1.73 m^2 Standard Error 1.3743	8.00 mL/min/1.73 m^2 Standard Error 1.5700	5.46 mL/min/1.73 m^2 Standard Error 2.2792	7.83 mL/min/1.73 m^2 Standard Error 2.216

Adverse Events

Bardoxolone Methyl - ADPKD

Serious events: 2 serious events

Other events: 30 other events

Deaths: 0 deaths

Bardoxolone Methyl - IgAN

Serious events: 0 serious events

Other events: 22 other events

Deaths: 0 deaths

Bardoxolone Methyl - T1D

Serious events: 0 serious events

Other events: 25 other events

Deaths: 0 deaths

Bardoxolone Methyl - FSGS

Serious events: 1 serious events

Other events: 16 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Bardoxolone Methyl - ADPKD n=31 participants at risk Participants with autosomal polycystic kidney disease (ADPKD) who received 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. Bardoxolone methyl capsules: Bardoxolone 5 mg capsules	Bardoxolone Methyl - IgAN n=26 participants at risk Participants with IgA nephropathy (IgAN) who received 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. Bardoxolone methyl capsules: Bardoxolone 5 mg capsules	Bardoxolone Methyl - T1D n=28 participants at risk Participants with Type 1 diabetes (T1D) who received 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. Bardoxolone methyl capsules: Bardoxolone 5 mg capsules	Bardoxolone Methyl - FSGS n=18 participants at risk Participants with focal segmental glomerulosclerosis (FSGS) who received 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. Bardoxolone methyl capsules: Bardoxolone 5 mg capsules
Hepatobiliary disorders Cholecystitis	0.00% 0/31 • 16 weeks After the first dose, documentation of adverse events was to continue until 30 days (+/- 3 days) following administration of the final dose of study medication, regardless of the relationship of the adverse event to study drug.	0.00% 0/26 • 16 weeks After the first dose, documentation of adverse events was to continue until 30 days (+/- 3 days) following administration of the final dose of study medication, regardless of the relationship of the adverse event to study drug.	0.00% 0/28 • 16 weeks After the first dose, documentation of adverse events was to continue until 30 days (+/- 3 days) following administration of the final dose of study medication, regardless of the relationship of the adverse event to study drug.	5.6% 1/18 • 16 weeks After the first dose, documentation of adverse events was to continue until 30 days (+/- 3 days) following administration of the final dose of study medication, regardless of the relationship of the adverse event to study drug.
Infections and infestations Histoplasmosis disseminated	3.2% 1/31 • 16 weeks After the first dose, documentation of adverse events was to continue until 30 days (+/- 3 days) following administration of the final dose of study medication, regardless of the relationship of the adverse event to study drug.	0.00% 0/26 • 16 weeks After the first dose, documentation of adverse events was to continue until 30 days (+/- 3 days) following administration of the final dose of study medication, regardless of the relationship of the adverse event to study drug.	0.00% 0/28 • 16 weeks After the first dose, documentation of adverse events was to continue until 30 days (+/- 3 days) following administration of the final dose of study medication, regardless of the relationship of the adverse event to study drug.	0.00% 0/18 • 16 weeks After the first dose, documentation of adverse events was to continue until 30 days (+/- 3 days) following administration of the final dose of study medication, regardless of the relationship of the adverse event to study drug.
Infections and infestations Sepsis	3.2% 1/31 • 16 weeks After the first dose, documentation of adverse events was to continue until 30 days (+/- 3 days) following administration of the final dose of study medication, regardless of the relationship of the adverse event to study drug.	0.00% 0/26 • 16 weeks After the first dose, documentation of adverse events was to continue until 30 days (+/- 3 days) following administration of the final dose of study medication, regardless of the relationship of the adverse event to study drug.	0.00% 0/28 • 16 weeks After the first dose, documentation of adverse events was to continue until 30 days (+/- 3 days) following administration of the final dose of study medication, regardless of the relationship of the adverse event to study drug.	0.00% 0/18 • 16 weeks After the first dose, documentation of adverse events was to continue until 30 days (+/- 3 days) following administration of the final dose of study medication, regardless of the relationship of the adverse event to study drug.
Renal and urinary disorders Renal failure acute	3.2% 1/31 • 16 weeks After the first dose, documentation of adverse events was to continue until 30 days (+/- 3 days) following administration of the final dose of study medication, regardless of the relationship of the adverse event to study drug.	0.00% 0/26 • 16 weeks After the first dose, documentation of adverse events was to continue until 30 days (+/- 3 days) following administration of the final dose of study medication, regardless of the relationship of the adverse event to study drug.	0.00% 0/28 • 16 weeks After the first dose, documentation of adverse events was to continue until 30 days (+/- 3 days) following administration of the final dose of study medication, regardless of the relationship of the adverse event to study drug.	0.00% 0/18 • 16 weeks After the first dose, documentation of adverse events was to continue until 30 days (+/- 3 days) following administration of the final dose of study medication, regardless of the relationship of the adverse event to study drug.
Vascular disorders Hypertensive crisis	3.2% 1/31 • 16 weeks After the first dose, documentation of adverse events was to continue until 30 days (+/- 3 days) following administration of the final dose of study medication, regardless of the relationship of the adverse event to study drug.	0.00% 0/26 • 16 weeks After the first dose, documentation of adverse events was to continue until 30 days (+/- 3 days) following administration of the final dose of study medication, regardless of the relationship of the adverse event to study drug.	0.00% 0/28 • 16 weeks After the first dose, documentation of adverse events was to continue until 30 days (+/- 3 days) following administration of the final dose of study medication, regardless of the relationship of the adverse event to study drug.	0.00% 0/18 • 16 weeks After the first dose, documentation of adverse events was to continue until 30 days (+/- 3 days) following administration of the final dose of study medication, regardless of the relationship of the adverse event to study drug.

Other adverse events

Additional Information

US Biogen Clinical Trial Center

Biogen

Phone: 866-633-4636

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Our agreement is subject to confidentiality but generally the PI can publish, for noncommercial purposes only, results and methods of the trial, but no other Sponsor Confidential Information. PI must give Sponsor no less than 60 days to review any manuscript for a proposed publication and must delay publication for up to an additional 90 days thereafter if Sponsor needs to file any patent application to protect any of Sponsor's intellectual property contained in the proposed publication.
Publication restrictions are in place

Restriction type: OTHER