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Autosomal Dominant Polycystic KidneyPhase 3FALCONNCT03918447

A Trial of Bardoxolone Methyl in Patients With ADPKD - FALCON

A Phase 3 study testing Bardoxolone methyl oral capsule in people with Autosomal Dominant Polycystic Kidney and ADPKD, run by Biogen at 137 sites in 10 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This international, multi-center, randomized, double-blind, placebo-controlled Phase 3 trial will study the safety, tolerability, and efficacy of bardoxolone methyl in qualified patients with ADPKD. Approximately 850 patients will be enrolled. Read more about this study

Who is it for?
Ages 12 to 70 years
Conditions the study is for
Autosomal Dominant Polycystic Kidney, ADPKD
What is being tested?
Bardoxolone methyl oral capsule
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
667 participants
Where
137 sitesin 10 countries
Study dates
29 May 2019to 8 Aug 2023
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 to 70 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Male and female patients 12 ≤ age ≤ 70 upon study consent;
  • Diagnosis of ADPKD by modified Pei-Ravine criteria (for adults 18≤ age ≤70 years): 1) at least 3 cysts per kidney by sonography or at least 5 cysts by CT or MRI with family history of ADPKD or 2) at least 10 cysts per kidney by any radiologic method and exclusion of other cystic kidney diseases if without family history;
  • Screening eGFR (average of Screen A and Screen B eGFR values) ≥ 30 to≤ 90 mL/min/1.73 m2 (12 to 55 years) or ≥ 30 to ≤ 44 mL/min/1.73 m2 (56 to 70 years):
  • 1) Patients with either screening eGFR ≥ 60 to ≤ 90 mL/min/1.73 m2 or age 56 to 70 years, must have evidence of ADPKD progression (i.e., eGFR decline of ≥ 2.0 mL/min/1.73 m2 per year, based on historical eGFR data and medical monitor discretion); 2)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;
  • Systolic blood pressure ≤ 140 mmHg and diastolic blood pressure ≤ 90 mmHg at Screen A or B visit after a period of rest.

Who cannot join

  • History of administration of polycystic kidney disease-modifying agents (somatostatin analogues) within 2 months prior to the Screen A visit;
  • B-type natriuretic peptide (BNP) level > 200 pg/mL at Screen A visit;
  • Uncontrolled diabetes (HbA1c > 11.0%) at Screen A visit;
  • Serum albumin < 3 g/dL at Screen A visit;
  • History of intracranial aneurysms;
  • Kidney or any other solid organ transplant recipient or a planned transplant during the study;
Show all 17Show fewer
  • Acute dialysis or acute kidney injury within 12 weeks prior to Screen A visit or during Screening;
  • History of clinically significant left-sided heart disease and/or clinically significant cardiac disease;
  • Systolic BP < 90 mm Hg at Screen A visit after a period of rest;
  • BMI < 18.5 kg/m2 at the Screen A visit;
  • History of malignancy within 5 years prior to Screen A visit, with the exception of localized skin or cervical carcinomas;
  • Systemic immunosuppression for more than 2 weeks, cumulatively, within the 12 weeks prior to randomization or anticipated need for immunosuppression during the study;
  • Untreated or uncontrolled active bacterial, fungal, or viral infection;
  • Participation in other interventional clinical studies within 30 days prior to Day 1;
  • Unwilling to practice acceptable methods of birth control (both males who have partners of child-bearing 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;
  • Women who are pregnant or breastfeeding;
  • Concomitant use of tolvaptan is excluded. Patients previously treated with tolvaptan must have discontinued drug for at least 2 months prior to Screen A visit

Trial locations and contacts

137 sites in 10 countries.

137 sites in 10 countries

United States83 sites

  • TTUHSC

    Amarillo, Texas, 79106

  • Arlington Nephrology, PA

    Arlington, Texas, 76015

  • Western Nephrology

    Arvada, Colorado, 80002

  • Mountain Kidney & Hypertension Associates

    Asheville, North Carolina, 28801

  • Emory University

    Atlanta, Georgia, 30322

  • University of Colorado Anschutz Medical Center

    Aurora, Colorado, 80045

  • Research Management, Inc.

    Austin, Texas, 78751

  • Research Management, Inc.

    Austin, Texas, 78758

  • University of Maryland School of Medicine

    Baltimore, Maryland, 21201

  • The Johns Hopkins University

    Baltimore, Maryland, 21287

  • Renal Associates of Baton Rouge

    Baton Rouge, Louisiana, 70808

  • Northeast Clinical Research Center

    Bethlehem, Pennsylvania, 18017

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35294

  • Tufts Medical Center

    Boston, Massachusetts, 02111

  • Beth Israel Deaconess Medical Center

    Boston, Massachusetts, 02215

  • University of Vermont Medical Center

    Burlington, Vermont, 05401

  • Boise Kidney & Hypertension, PLLC

    Caldwell, Idaho, 83605

  • Metrolina Nephrology Associates

    Charlotte, North Carolina, 28204

  • University of Virginia

    Charlottesville, Virginia, 22908

  • Northwestern University

    Chicago, Illinois, 60611

  • University of Chicago

    Chicago, Illinois, 60617

  • Cincinnati VA Medical Center

    Cincinnati, Ohio, 45220

  • University of Cincinnati College of Medicine

    Cincinnati, Ohio, 45267

  • Cleveland Clinic

    Cleveland, Ohio, 44195

  • Columbia Nephrology Associates, PA

    Columbia, South Carolina, 29203

  • Remington-Davis Clinical Research

    Columbus, Ohio, 43215

  • Liberty Research Center

    Dallas, Texas, 75230

  • Renal Disease Research Institute

    Dallas, Texas, 75235

  • Kidney Associates of Colorado

    Denver, Colorado, 80210

  • Denver Nephrologist, PC

    Denver, Colorado, 80230

  • Rancho Research Institute

    Downey, California, 90242

  • Davita Clinical Research

    El Paso, Texas, 79925

  • Nephrology Associates of Northern Virginia, Inc.

    Fairfax, Virginia, 22033

  • Nephrology Associates, P.C.

    Flushing, New York, 11355

  • AKDHC

    Glendale, Arizona, 85306

  • Division of Kidney Diseases and Hypertension

    Great Neck, New York, 11021

  • Nephrology Associates PC

    Homewood, Alabama, 35209

  • DaVita Med Center

    Houston, Texas, 77004

  • Nephrology Consultants, LLC

    Huntsville, Alabama, 35805

  • Paragon Health PC d/b/a Nephrology Center PC

    Kalamazoo, Michigan, 49007

  • University of Kansas Medical Center

    Kansas City, Kansas, 66224

  • Clinical Research Consultants, LLC

    Kansas City, Missouri, 64111

  • California Institute Renal Research

    La Mesa, California, 91942

  • KSOSN

    Las Vegas, Nevada, 89129

  • Georgia Nephrology, LLC

    Lawrenceville, Georgia, 30046

  • University of California, Los Angeles

    Los Angeles, California, 90025

  • Keck USC/LAC

    Los Angeles, California, 90033

  • Loyola University Chicago

    Maywood, Illinois, 60153

  • Boise Kidney & Hypertension, PLLC

    Meridian, Idaho, 83642

  • Aventiv Research, Inc

    Mesa, Arizona, 85210

  • University of Miami

    Miami, Florida, 33136

  • Pro-Care Research Center, Corp.

    Miami Gardens, Florida, 33014

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

  • NYU Winthrop Hospital

    Mineola, New York, 11501

  • Nephrology Associates, P.C.

    Nashville, Tennessee, 37205

  • Yale University School of Medicine

    New Haven, Connecticut, 06520

  • Amicis Research Center

    Northridge, California, 91324

  • Discovery Medical Research Group

    Ocala, Florida, 34471

  • Innovation Medical Research Center, Inc

    Palmetto Bay, Florida, 33157

  • Stanford University

    Palo Alto, California, 94304

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19014

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

  • Michigan Kidney Consultants

    Pontiac, Michigan, 48341

  • Volunteer Medical Research

    Port Charlotte, Florida, 33952

  • North Carolina Nephrology, P.A. 2nd Floor

    Raleigh, North Carolina, 27609

  • Apex Research of Riverside

    Riverside, California, 92505

  • Mayo Clinic

    Rochester, Minnesota, 55905

  • Clinical Advancement Center

    San Antonio, Texas, 78215

  • University of California San Francisco

    San Francisco, California, 94143

  • Swedish Medical Center

    Seattle, Washington, 98052

  • Northwest Louisiana Nephrology

    Shreveport, Louisiana, 71101

  • KidneyCare and Tranplant Services of New England

    Springfield, Massachusetts, 01104

  • Renal and Transplant Associates of New England, PC

    Springfield, Massachusetts, 01107

  • Washington University in St. Louis

    St Louis, Missouri, 63110

  • University of South Florida

    Tampa, Florida, 33606

  • Cotton O'Neil Clinical Research Center

    Topeka, Kansas, 66606

  • University of Arizona

    Tucson, Arizona, 85719

  • Milwaukee Nephrologists, SC

    Wauwatosa, Wisconsin, 53226

  • Western Nephrology and Mineral Bone Disease, PC

    Westminster, Colorado, 80031

  • Ascension Via Christi Research

    Wichita, Kansas, 67214

  • Kansas Nephrology Research Institute, LLC

    Wichita, Kansas, 67214

  • Brookview Hills Research Associates, LLC

    Winston-Salem, North Carolina, 27103

  • Florida Premier Research Institute, LLC

    Winter Park, Florida, 32789

Other trials in United States

Spain11 sites

  • Hospital Universitario de Badajoz

    Badajoz, 6070

  • Hospital Del Mar

    Barcelona, 8003

  • Fundacio Puigvert

    Barcelona, 8025

  • Hospital Universitario Reina Sofía

    Córdoba, 14004

  • Hospital Universitario Virgen de las Nieves

    Granada, 18012

  • Hospital Lucus Augusti

    Lugo, 27003

  • Fundación Jiménez Díaz

    Madrid, 28040

  • Hospital Universitario La Paz

    Madrid, 28046

  • Hospital de Getafe

    Madrid, 28905

  • Hospital Universitario Marques de Valdecilla

    Santander, 39008

  • Hospital Universitario Dr Peset

    Valencia, 46017

Other trials in Spain

Australia10 sites

  • Royal Adelaide Hospital

    Adelaide, South Australia, 5000

  • Sunshine Coast University Hospital

    Birtinya, Queensland, 4575

  • Monash Health

    Clayton, Victoria, 3168

  • Renal Research

    Gosford, New South Wales, 2250

  • Royal Brisbane and Women's Hospital

    Herston, Queensland, 4029

  • Fiona Stanley Hospital

    Murdoch, Western Australia, 6150

  • John Hunter Hospital

    New Lambton, New South Wales, 2305

  • Melbourne Health

    Parkville, Victoria, 3050

  • Melbourne Renal Research Group

    Reservoir, Victoria, 3073

  • Westmead Hospital

    Sydney, New South Wales, 2145

Other trials in Australia

Japan10 sites

  • Hokkaido University Hospital

    Hokkaido, 060-8648

  • Toranomon Hospital Kajigaya

    Kanagawa, 213-8587

  • Japan Community Healthcare Organization Sendai Hospital

    Miyagi, 981-8501

  • Niigata University Medical & Dental Hospital

    Niigata, 951-8520

  • Local Incorporated Administrative Agency Osaka City Hospital Organization Osaka City General Hospital

    Osaka, 534-0021

  • Osaka City University Hospital

    Osaka, 545-8586

  • Osaka University Hospital

    Osaka, 565-0871

  • Toranomon Hospital

    Tokyo, 105-8470

  • Juntendo University Hospital

    Tokyo, 113-8431

  • Tokyo Women's Medical University Hospital

    Tokyo, 162-8666

Other trials in Japan

France5 sites

  • University Hospital La Cavale Blanche

    Brest, 29200

  • Chu Grenoble Alpes

    Grenoble, 38043

  • Hospital Henri-Mondor AP-HP

    Le Kremlin-Bicêtre, 94270

  • CHU de Nantes

    Nantes, 44093

  • Hopital Necker, Universite Paris Descartes

    Paris, 75015

Other trials in France

Belgium4 sites

  • Nephrology, Cliniques U St-Luc

    Brussels, 1200

  • Universitair Ziekenhuis Brussel (VUB)

    Brussels, B-1090

  • University Hospitals Leuven, Dept. of Nephrology, Dialysis and Renal Transplantation

    Leuven, 3000

  • Chu Liege

    Liège, 4000

Other trials in Belgium

Italy4 sites

  • Renal Division, ASST Santi Paolo e Carlo

    Milan, 20153

  • Università di Modena e Reggio Emilia

    Modena, 41124

  • ICS Maugeri SpA SB

    Pavia, 27100

  • Fondazione Policlinico Gemelli

    Roma, 168

Other trials in Italy

United Kingdom4 sites

  • North Bristol NHS Trust

    Bristol, BS10 5NB

  • Manchester University NHS Foundation Trust

    Manchester, M13 9WL

  • Nottingham University Hospitals

    Nottingham, NG5 1PB

  • Morriston Hospital

    Swansea, SA66NL

Other trials in United Kingdom

Czechia3 sites

  • FN Brno

    Brno, 62500

  • Nephrology Dept., General Teaching Hospital

    Prague, 12808

  • IKEM

    Prague, 14021

Other trials in Czechia

Germany3 sites

  • Universitätsklinikum Essen

    Essen, 45147

  • Medizinische Hochschule Hannover

    Hanover, 30625

  • Klinikum rechts der Isar der TU München

    München, 81675

Other trials in Germany

About this study

Official title: A Phase 3 Trial of the Efficacy and Safety of Bardoxolone Methyl in Patients With Autosomal Dominant Polycystic Kidney Disease

This international, multi-center, randomized, double-blind, placebo-controlled Phase 3 trial will study the safety, tolerability, and efficacy of bardoxolone methyl in qualified patients with ADPKD. Approximately 850 patients will be enrolled.

Read the detailed description

This international, multi-center, randomized, double-blind, placebo-controlled Phase 3 trial will study the safety, tolerability, and efficacy of bardoxolone methyl in qualified patients with ADPKD.

Patients will be randomized 1:1 to either bardoxolone methyl or placebo. Patients receiving bardoxolone methyl will start with once-daily dosing at 5 mg and will dose-escalate to 10 mg at Week 2, to 20 mg at Week 4, and then to 30 mg at Week 6 (only if baseline ACR >300 mg/g) unless contraindicated clinically and approved by the medical monitor. Dose de-escalation is permitted during the study if indicated clinically, and subsequent dose re-escalation is also permitted to meet the dosing objective of the highest tolerated dose.

All patients in the study will follow the same visit and assessment schedule. Patients will continue to receive study drug or placebo through Week 100 and will not receive study drug or placebo during a 12-week off-treatment period between Weeks 100 and 112.

Being tested
Bardoxolone methyl oral capsule, Placebo oral capsule
Keywords
Bardoxolone Methyl, RTA 402, Autosomal Dominant Polycystic Kidney Disease, ADPKD
Medical subject headings
Polycystic Kidney, Autosomal Dominant

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Bardoxolone methyl oral capsuleDrug

    Also called RTA 402

    Bardoxolone methyl capsules dose escalated from 5 mg to a maximum of 20 or 30 mg, depending on baseline proteinuria status.

  • Placebo oral capsuleDrug

    Capsule containing an inert placebo

Study groups in NCT03918447
Study groupRoleWhat they receive
Maximum bardoxolone methyl dose of 20 mgExperimentalPatients randomized to receive bardoxolone methyl with a baseline ACR less than or equal to 300 mg/g will be titrated to a maximum dose of 20 mg. Patients will begin once-daily dosing with bardoxolone methyl capsules at 5 mg and will dose escalate to 10 mg at Week 2 and 20 mg at Week 4. Patients will continue to receive study drug through Week 100, and will not receive study drug during a 12-week off-treatment period between Weeks 100 and 112.
Receives: Drug: Bardoxolone methyl oral capsule
Maximum bardoxolone methyl dose 30 mgExperimentalPatients randomized to receive bardoxolone methyl with a baseline ACR greater than 300 mg/g will be titrated to a maximum dose of 30 mg. Patients will begin once-daily dosing with bardoxolone methyl capsules at 5 mg and will dose escalate to 10 mg at Week 2, 20 mg at Week 4 and 30 mg at Week 6. Patients will continue to receive study drug through Week 100, and will not receive study drug during a 12-week off-treatment period between Weeks 100 and 112.
Receives: Drug: Bardoxolone methyl oral capsule
PlaceboPlacebo comparatorPatients randomized to placebo will remain on placebo capsules throughout the study, undergoing sham titration. Patients will continue to receive placebo capsules through Week 100, and will not receive capsules during a 12-week off-treatment period between Weeks 100 and 112.
Receives: Drug: Placebo oral capsule

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Off-treatment Period: Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 108

    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.

    Measured: Baseline, Week 108

  2. Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs

    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.

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

Secondary outcomes (1)

  1. Treatment Period: Change From Baseline in eGFR 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.

    Measured: Baseline, Week 100

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Apr 2019
  2. Study start29 May 2019
  3. Primary completion8 Aug 2023
  4. Study completion8 Aug 2023

Why it stopped: Discontinuation of all bardoxolone chronic kidney disease programs

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Sequential
Masking
Triple blind
Who is masked
Participant, Care provider, Investigator
Enrolment
667 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03918447
Sponsor's protocol ID
402-C-1808
Acronym
FALCON
Status last verified
May 2025
First submitted
12 Apr 2019
Last updated
3 Jun 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

Publications

  • St Pierre K, Cashmore BA, Bolignano D, Zoccali C, Ruospo M, Craig JC, Strippoli GF, Mallett AJ, Green SC, Tunnicliffe DJ. Interventions for preventing the progression of autosomal dominant polycystic kidney disease. Cochrane Database Syst Rev. 2024 Oct 2;10(10):CD010294. doi: 10.1002/14651858.CD010294.pub3.

    PubMed 39356039

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Bardoxolone methyl oral capsule in people with Autosomal Dominant Polycystic Kidney and ADPKD, run by Biogen at 137 sites in 10 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 3 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 137 sites in United States, Spain, Australia, Japan, France and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 11 Jun 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Off-treatment Period: Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 108 (time frame: Baseline, Week 108). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03918447, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jun 2025.

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FALCON Clinical Trial NCT03918447 | AzTrials