Trial Outcomes & Findings for A Trial of Bardoxolone Methyl in Patients With ADPKD - FALCON (NCT NCT03918447)

Last Updated: 2025-06-03

Results Overview

Estimated Glomerular filtration rate (eGFR) is a measure of kidney function assessed through blood/serum. eGFR was measured in milliliters per minute per 1.73 meters square (mL/min/1.73 m\^2). Higher eGFRs represent better/improved kidney function. Lower eGFRs represent poorer/decreased kidney function. Negative change from baseline in eGFR indicates worsened kidney function.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

667 participants

Primary outcome timeframe

Baseline, Week 108

Results posted on

2025-06-03

Participant Flow

Participants were enrolled at the investigative sites in the United States, Australia, Belgium, Czech Republic, France, Germany, Italy, Japan, Spain, and United Kingdom beginning on 29 May 2019. The study completion date was 8 August 2023.

A total of 667 participants were enrolled and randomized 1:1 to receive either bardoxolone methyl or placebo during the treatment period (up to Week 100) and continued to be assessed in the off-treatment period for 12 weeks (up to Week 112).

Participant milestones

Participant milestones
Measure	Bardoxolone Methyl During the treatment period, the participants received bardoxolone methyl capsules, once daily (QD) at a starting dose of 5 milligrams (mg), followed by dose-escalation to 10 mg at Week 2, and to 20 mg at Week 4. If the eligibility urine albumin to creatinine ratio (UACR) was \>300 milligrams per gram (mg/g), the dose was increased to 30 mg starting from Week 6 until Week 100. Participants continued to be assessed during the off-treatment period up to Week 112.	Placebo During the treatment period, participants received bardoxolone methyl matching-placebo capsules, orally, QD up to Week 100, with sham titration to maintain the blinding. Participants did not receive a bardoxolone methyl matching placebo capsule during the off-treatment period between Weeks 100 and 112.
Overall Study STARTED	334	333
Overall Study Safety Analysis Set	335	331
Overall Study COMPLETED	106	112
Overall Study NOT COMPLETED	228	221

Reasons for withdrawal

Reasons for withdrawal
Measure	Bardoxolone Methyl During the treatment period, the participants received bardoxolone methyl capsules, once daily (QD) at a starting dose of 5 milligrams (mg), followed by dose-escalation to 10 mg at Week 2, and to 20 mg at Week 4. If the eligibility urine albumin to creatinine ratio (UACR) was \>300 milligrams per gram (mg/g), the dose was increased to 30 mg starting from Week 6 until Week 100. Participants continued to be assessed during the off-treatment period up to Week 112.	Placebo During the treatment period, participants received bardoxolone methyl matching-placebo capsules, orally, QD up to Week 100, with sham titration to maintain the blinding. Participants did not receive a bardoxolone methyl matching placebo capsule during the off-treatment period between Weeks 100 and 112.
Overall Study Death	1	0
Overall Study Lost to Follow-up	13	6
Overall Study Withdrawal by Subject	19	17
Overall Study Study Terminated by Sponsor	195	197
Overall Study Reason not Specified	0	1

Baseline Characteristics

A Trial of Bardoxolone Methyl in Patients With ADPKD - FALCON

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Bardoxolone Methyl n=334 Participants During the treatment period, the participants received bardoxolone methyl capsules, QD at a starting dose of 5 mg, followed by dose-escalation to 10 mg at Week 2, and to 20 mg at Week 4. If the eligibility UACR was \>300 mg/g, the dose was increased to 30 mg starting from Week 6 until Week 100. Participants continued to be assessed during the off-treatment period up to Week 112.	Placebo n=333 Participants During the treatment period, participants received bardoxolone methyl matching-placebo capsules, orally, QD up to Week 100, with sham titration to maintain the blinding. Participants did not receive a bardoxolone methyl matching placebo capsule during the off-treatment period between Weeks 100 and 112.	Total n=667 Participants Total of all reporting groups
Age, Continuous	48.6 years STANDARD_DEVIATION 9.46 • n=5 Participants	48.3 years STANDARD_DEVIATION 9.58 • n=7 Participants	48.4 years STANDARD_DEVIATION 9.51 • n=5 Participants
Sex: Female, Male Female	188 Participants n=5 Participants	173 Participants n=7 Participants	361 Participants n=5 Participants
Sex: Female, Male Male	146 Participants n=5 Participants	160 Participants n=7 Participants	306 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	29 Participants n=5 Participants	35 Participants n=7 Participants	64 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	305 Participants n=5 Participants	298 Participants n=7 Participants	603 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race/Ethnicity, Customized Race · American Indian or Alaska Native	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race/Ethnicity, Customized Race · Asian	34 Participants n=5 Participants	45 Participants n=7 Participants	79 Participants n=5 Participants
Race/Ethnicity, Customized Race · Black or African American	23 Participants n=5 Participants	18 Participants n=7 Participants	41 Participants n=5 Participants
Race/Ethnicity, Customized Race · Native Hawaiian or Other Pacific Islander	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized Race · White	269 Participants n=5 Participants	261 Participants n=7 Participants	530 Participants n=5 Participants
Race/Ethnicity, Customized Race · Other	6 Participants n=5 Participants	8 Participants n=7 Participants	14 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Week 108

Population: ITT population included all enrolled participants categorized by their randomized treatment group (whether or not they received study drug). 'Overall number of participants analyzed' indicates the number of participants with data available for this outcome measure.

Outcome measures

Outcome measures
Measure	Bardoxolone Methyl n=70 Participants During the treatment period, the participants received bardoxolone methyl capsules, QD at a starting dose of 5 mg, followed by dose-escalation to 10 mg at Week 2, and to 20 mg at Week 4. If the eligibility UACR was \>300 mg/g, the dose was increased to 30 mg starting from Week 6 until Week 100. Participants continued to be assessed during the off-treatment period up to Week 112.	Placebo n=79 Participants During the treatment period, participants received bardoxolone methyl matching-placebo capsules, orally, QD up to Week 100, with sham titration to maintain the blinding. Participants did not receive a bardoxolone methyl matching placebo capsule during the off-treatment period between Weeks 100 and 112.
Off-treatment Period: Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 108	-4.59 mL/min/1.73 m^2 Standard Error 0.817	-5.56 mL/min/1.73 m^2 Standard Error 0.769

PRIMARY outcome

Timeframe: From first dose of the study drug up to end of follow-up (up to Week 112)

Population: Safety population included all enrolled participants who had received at least 1 dose of study drug. Participants who received 1 dose of bardoxolone methyl were classified in the bardoxolone methyl group. Participants who received at least 1 dose of placebo and no dose of bardoxolone methyl were classified in the placebo group.

AE:any untoward medical occurrence in a participant regardless of its causal relationship to study drug.AE can be any unfavorable \& unintended sign,symptom/disease temporally associated with use of study drug,whether considered to be study-drug related/not.This includes clinically significant abnormal laboratory test result,any newly occurring events/previous conditions that have increased in severity/frequency since administration of study drug. SAE:any AE that at any dose results in death,life-threatening,requires hospitalization/prolongation of existing hospitalisation,substantial disruption of ability to conduct normal life functions,congenital anomaly or is an important medical event. AEs \& SAEs that occurred during treatment and within 30 days after last dose were considered TE.

Outcome measures

Outcome measures
Measure	Bardoxolone Methyl n=335 Participants During the treatment period, the participants received bardoxolone methyl capsules, QD at a starting dose of 5 mg, followed by dose-escalation to 10 mg at Week 2, and to 20 mg at Week 4. If the eligibility UACR was \>300 mg/g, the dose was increased to 30 mg starting from Week 6 until Week 100. Participants continued to be assessed during the off-treatment period up to Week 112.	Placebo n=331 Participants During the treatment period, participants received bardoxolone methyl matching-placebo capsules, orally, QD up to Week 100, with sham titration to maintain the blinding. Participants did not receive a bardoxolone methyl matching placebo capsule during the off-treatment period between Weeks 100 and 112.
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs TEAEs	314 Participants	296 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs Serious TEAEs	38 Participants	26 Participants

SECONDARY outcome

Timeframe: Baseline, Week 100

Population: ITT included all enrolled participants categorized by their randomized treatment group (whether or not they received study drug). 'Overall number of participants analyzed' indicates the number of participants with an eGFR assessment at Week 100.

eGFR is a measure of kidney function assessed through blood/serum. eGFR was measured in mL/min/1.73 m\^2. Higher eGFRs represent better/improved kidney function. Lower eGFRs represent poorer/decreased kidney function. A negative change from baseline in eGFR indicates worsened kidney function.

Outcome measures

Outcome measures
Measure	Bardoxolone Methyl n=168 Participants During the treatment period, the participants received bardoxolone methyl capsules, QD at a starting dose of 5 mg, followed by dose-escalation to 10 mg at Week 2, and to 20 mg at Week 4. If the eligibility UACR was \>300 mg/g, the dose was increased to 30 mg starting from Week 6 until Week 100. Participants continued to be assessed during the off-treatment period up to Week 112.	Placebo n=167 Participants During the treatment period, participants received bardoxolone methyl matching-placebo capsules, orally, QD up to Week 100, with sham titration to maintain the blinding. Participants did not receive a bardoxolone methyl matching placebo capsule during the off-treatment period between Weeks 100 and 112.
Treatment Period: Change From Baseline in eGFR at Week 100	1.31 mL/min/1.73 m^2 Standard Error 0.550	-6.64 mL/min/1.73 m^2 Standard Error 0.549

Adverse Events

Bardoxolone Methyl

Serious events: 38 serious events

Other events: 299 other events

Deaths: 1 deaths

Placebo

Serious events: 26 serious events

Other events: 255 other events

Deaths: 0 deaths

Serious adverse events

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