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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03918447




Registration number
NCT03918447
Ethics application status
Date submitted
12/04/2019
Date registered
17/04/2019

Titles & IDs
Public title
A Trial of Bardoxolone Methyl in Patients With ADPKD - FALCON
Scientific title
A Phase 3 Trial of the Efficacy and Safety of Bardoxolone Methyl in Patients With Autosomal Dominant Polycystic Kidney Disease
Secondary ID [1] 0 0
402-C-1808
Universal Trial Number (UTN)
Trial acronym
FALCON
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Autosomal Dominant Polycystic Kidney 0 0
ADPKD 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Renal and Urogenital 0 0 0 0
Other renal and urogenital disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Bardoxolone methyl oral capsule
Treatment: Drugs - Placebo oral capsule

Experimental: Maximum bardoxolone methyl dose of 20 mg - Patients 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.

Experimental: Maximum bardoxolone methyl dose 30 mg - Patients 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.

Placebo comparator: Placebo - Patients 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.


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

Treatment: Drugs: Placebo oral capsule
Capsule containing an inert placebo
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Off-treatment Period: Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 108
Timepoint [1] 0 0
Baseline, Week 108
Primary outcome [2] 0 0
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs
Timepoint [2] 0 0
From first dose of the study drug up to end of follow-up (up to Week 112)
Secondary outcome [1] 0 0
Treatment Period: Change From Baseline in eGFR at Week 100
Timepoint [1] 0 0
Baseline, Week 100

Eligibility
Key inclusion criteria
* 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.
Minimum age
12 Years
Maximum age
70 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* 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;
* 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Other
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
29/05/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
8/08/2023
Sample size
Target
Accrual to date
Final
667
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
Renal Research - Gosford
Recruitment hospital [2] 0 0
John Hunter Hospital - New Lambton
Recruitment hospital [3] 0 0
Westmead Hospital - Sydney
Recruitment hospital [4] 0 0
Sunshine Coast University Hospital - Birtinya
Recruitment hospital [5] 0 0
Royal Brisbane and Women's Hospital - Herston
Recruitment hospital [6] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [7] 0 0
Monash Health - Clayton
Recruitment hospital [8] 0 0
Melbourne Health - Parkville
Recruitment hospital [9] 0 0
Melbourne Renal Research Group - Reservoir
Recruitment hospital [10] 0 0
Fiona Stanley Hospital - Murdoch
Recruitment postcode(s) [1] 0 0
2250 - Gosford
Recruitment postcode(s) [2] 0 0
2305 - New Lambton
Recruitment postcode(s) [3] 0 0
2145 - Sydney
Recruitment postcode(s) [4] 0 0
4575 - Birtinya
Recruitment postcode(s) [5] 0 0
4029 - Herston
Recruitment postcode(s) [6] 0 0
5000 - Adelaide
Recruitment postcode(s) [7] 0 0
3168 - Clayton
Recruitment postcode(s) [8] 0 0
3050 - Parkville
Recruitment postcode(s) [9] 0 0
3073 - Reservoir
Recruitment postcode(s) [10] 0 0
6150 - Murdoch
Recruitment outside Australia
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United States of America
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Alabama
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Arizona
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Louisiana
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Massachusetts
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Roma
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Hokkaido
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Kanagawa
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Miyagi
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Niigata
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Nottingham
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Swansea

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Reata, a wholly owned subsidiary of Biogen
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://vivli.org/


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03918447