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Febuxostat

Phase 3

Cardiovascular Disease | Small molecule | Cardiovascular |Takeda Pharmaceutical Company Limited|Last Updated: Jun 14, 2018

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindACTIVE_CONTROLLEDDMC
Total Trials1
Total Enrollment6,198

FDA Designations

No designations recorded

Clinical trial landscape

Febuxostat · 13 trials · 8 indications

Phase 3 5Phase 2 6Phase 1 2
NCT01101035Cardiovascular Safety of Febuxostat and Allopurinol in Participants With Gout and Cardiovascular Comorbidities (CARES)Cardiovascular Disease
COMPLETED6,198 Analytics
NCT00430248Efficacy and Safety of Oral Febuxostat in Participants With GoutGout
COMPLETED2,269 Analytics
NCT00175019Allopurinol Versus Febuxostat in Subjects Completing the Phase 3 Trials C02-009 or C02-010Gout
COMPLETED1,086 Analytics
NCT00174915Phase 3, Febuxostat, Allopurinol and Placebo-Controlled Study in Gout Subjects.Gout
COMPLETED1,072 Analytics
NCT00102440Febuxostat Versus Allopurinol Control Trial in Subjects With GoutGout
COMPLETED760 Analytics
PHASE3COMPLETED
Cardiovascular Safety of Febuxostat and Allopurinol in Participants With Gout and Cardiovascular Comorbidities (CARES)
Cardiovascular DiseaseUnlock trial analytics
PHASE3COMPLETED
Efficacy and Safety of Oral Febuxostat in Participants With Gout
GoutUnlock trial analytics
PHASE3COMPLETED
Allopurinol Versus Febuxostat in Subjects Completing the Phase 3 Trials C02-009 or C02-010
GoutUnlock trial analytics
PHASE3COMPLETED
Phase 3, Febuxostat, Allopurinol and Placebo-Controlled Study in Gout Subjects.
GoutUnlock trial analytics
PHASE3COMPLETED
Febuxostat Versus Allopurinol Control Trial in Subjects With Gout
GoutUnlock trial analytics

Study Endpoints

Primary Endpoints

Percentage of Participants With Primary Major Adverse Cardiovascular Events (MACE) Composite (75% Interim Analysis)
Up to last dose of study drug (approximately 83 months)

Major adverse cardiovascular events (MACE) were defined as a composite of cardiovascular (CV) death, non-fatal myocardial infarction (MI), nonfatal stroke and unstable angina with urgent coronary revascularization; these events were adjudicated by an independent cardiovascular endpoints committee.

Percentage of Participants With Primary MACE Composite (Final Analysis)
Up to last dose of study drug (approximately 83 months)

Major adverse cardiovascular events (MACE) were defined as a composite of cardiovascular (CV) death, non-fatal myocardial infarction (MI), nonfatal stroke and unstable angina with urgent coronary revascularization; these events were adjudicated by an independent cardiovascular endpoints committee.

Percentage of Subjects Whose Serum Urate Level is <6.0 Milligrams Per Deciliter (mg/dL) at the Final Visit.
Last Visit on treatment (up to 6 months)

The percentage of subjects whose serum urate level was \<6.0 mg/dL at the Final Visit was summarized. The Final Visit was the last visit at which a serum urate value was collected.

Percentage of Subjects Whose Serum Urate Level Decreases to < 6.0 mg/dL at Month 1.
Month 1

Serum urate values were obtained at the Month 1 visit. The percentage of subjects whose serum urate was \<6.0 mg/dL at the Month 1 visit was summarized.

Percentage of Subjects Whose Serum Urate Level Decreases to < 6.0 mg/dL at Month 12.
Month 12

Serum urate values were obtained at the Month 12 visit. The percentage of subjects whose serum urate was \<6.0 mg/dL at the Month 12 visit was summarized.

Percentage of Subjects Whose Serum Urate Level Decreases to < 6.0 mg/dL at Month 24.
Month 24

Serum urate values were obtained at the Month 24 visit. The percentage of subjects whose serum urate was \<6.0 mg/dL at the Month 24 visit was summarized.

Percentage of Subjects Whose Serum Urate Level Decreases to < 6.0 mg/dL at Month 36.
Month 36

Serum urate values were obtained at the Month 36 visit. The percentage of subjects whose serum urate was \<6.0 mg/dL at the Month 36 visit was summarized.

Percentage of Subjects Whose Serum Urate Level Decreases to < 6.0 mg/dL at Last Visit on Treatment.
Last Visit on treatment (up to 40 months).

The percentage of subjects whose serum urate was \<6.0 mg/dL at the last visit on treatment was summarized. The last visit on treatment was the last visit at which a serum urate value was collected prior to any changes in drug and/or dose from the initial treatment assignment.

Percentage of Subjects Whose Last Three Serum Urate Levels Are <6.0 Milligram Per Deciliter (mg/dL).
Last 3 visits (any last 3 visits up to week 28)

Each subject's serum urate at the last 3 visits determined the subject's response for the primary efficacy variable. A subject who prematurely discontinued without least 3 postbaseline serum urate levels was considered a nonresponder; if at least 3 serum urate were obtained postbaseline, those 3 visits were used. The last 3 visits used may have differed for each subject.

Percentage of Subjects With the Last 3 Serum Urate Levels <6.0 Milligrams Per Deciliter (mg/dL)
Last 3 Visits (up to 52 weeks)

Each subject's serum urate at the last 3 visits determined the subject's response for the primary efficacy variable. A subject who prematurely discontinued without least 3 postbaseline serum urate levels was considered a nonresponder; if at least 3 serum urate were obtained postbaseline, those 3 visits were used. The last 3 visits used may have differed for each subject.

Change From Baseline in 24-hour Mean Systolic Blood Pressure (SBP) Measured by Ambulatory Blood Pressure Monitoring at Week 6
Baseline and Week 6

The change in 24-hour mean SBP measured at final visit or Week 6 relative to baseline. Ambulatory blood pressure monitoring measures blood pressure at regular intervals throughout the day and night. The 24-hour mean is the average of all measurements recorded for 24 hours after dosing.

Change From Baseline to Month 12 in Serum Creatinine
Baseline and Month 12

Renal function was assessed by measuring the change from Baseline in serum creatinine. Analyses were conducted by the Central Laboratory.

Mean Change From Baseline to Month 24 in the Modified Sharp/Van Der Heijde Erosion Score of the Single Affected Joint
Baseline and Month 24

The single affected joint was defined as the joint with the history of the first acute gout flare. Radiographs (X-rays) of this single joint in the hands or feet were evaluated using the modified Sharp/van der Heijde method. Each erosion was assessed using a 4-point scale where 0=no erosions (best) to 3=large erosion passing the mid-line (worst). Individual erosion scores were summed to a maximum erosion score of 5 for joints in the hands and 10 for joints in the feet. Higher scores indicated more joint damage. A negative change from Baseline indicated improvement.

Percent Change From Baseline to Month 6 in 24-hour Urine Uric Acid (uUA) Excretion
Baseline and Month 6

The change from Baseline to Month 6 in 24-hour urine uric acid is expressed as a percentage of the Baseline uUA value.

Percentage of Subjects Whose Serum Urate Level Decreases to or is Maintained at <6.0 mg/dL at Month 6 Visit.
Month 6

Serum urate values were obtained at the Month 6 visit. The percentage of subjects whose serum urate was \<6.0 mg/dL at the Month 6 visit was summarized.

Percentage of Subjects Whose Serum Urate Level Decreases to or is Maintained at <6.0 mg/dL at Month 12 Visit.
Month 12

Serum urate values were obtained at the Month 12 visit. The percentage of subjects whose serum urate was \<6.0 mg/dL at the Month 12 visit was summarized.

Percentage of Subjects Whose Serum Urate Level Decreases to or is Maintained at <6.0 mg/dL at Month 18 Visit.
Month 18

Serum urate values were obtained at the Month 18 visit. The percentage of subjects whose serum urate was \<6.0 mg/dL at the Month 18 visit was summarized.

Percentage of Subjects Whose Serum Urate Level Decreases to or is Maintained at <6.0 mg/dL at Month 24 Visit.
Month 24

Serum urate values were obtained at the Month 24 visit. The percentage of subjects whose serum urate was \<6.0 mg/dL at the Month 24 visit was summarized.

Percentage of Subjects Whose Serum Urate Level Decreases to or is Maintained at <6.0 mg/dL at Month 36 Visit.
Month 36

Serum urate values were obtained at the Month 36 visit. The percentage of subjects whose serum urate was \<6.0 mg/dL at the Month 36 visit was summarized.

Percentage of Subjects Whose Serum Urate Level Decreases to or is Maintained at <6.0 mg/dL at Month 48 Visit.
Month 48

Serum urate values were obtained at the Month 48 visit. The percentage of subjects whose serum urate was \<6.0 mg/dL at the Month 48 visit was summarized.

Percentage of Subjects Whose Serum Urate Level Decreases to or is Maintained at <6.0 mg/dL at Month 60 Visit.
Month 60

Serum urate values were obtained at the Month 60 visit. The percentage of subjects whose serum urate was \<6.0 mg/dL at the Month 60 visit was summarized.

Percentage of Subjects Whose Serum Urate Level Decreases to or is Maintained at <6.0 mg/dL at Final Visit.
Last Visit on treatment (up to 66 months).

The percentage of subjects whose serum urate was \<6.0 mg/dL at the final visit was summarized. The final visit was the last visit at which a serum urate value was collected.

Percentage of Subjects Whose Serum Urate Level Decreased to <6.0 Milligram Per Deciliter (mg/dL) at the Day 28 Visit.
Day 28.

Serum urate values were obtained at the Day 28 visit. The percentage of subjects whose serum urate decreased to \<6.0 mg/dL at the Day 28 visit was summarized.

Cmax: Maximum Observed Plasma Concentration for Febuxostat
Days 1 at multiple timepoints (up to 48 hours) post-dose
AUC(0-tau): Area Under the Plasma Concentration-time Curve During the Dosing Interval for Febuxostat
Days 1 pre-dose and at multiple timepoints (up to 48 hours) post-dose
AUC(0-inf): Area Under the Plasma Concentration-time Curve From Time 0 to Infinity for Febuxostat
Days 1 pre-dose and at multiple timepoints (up to 48 hours) post-dose
Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs)
Day 1 up to 30 days after last dose of drug (Day 31 for each of the 4 periods)
Number of Participants With Clinically Significant Change From Baseline in Vital Signs
Day 1 up to 30 days after last dose of drug (Day 31 for each of the 4 periods)
Number of Participants With Clinically Significant Change From Baseline in Physical Examination Findings
Day 1 up to 30 days after last dose of drug (Day 31 for each of the 4 periods)
Number of Participants With Clinically Significant Change From Baseline in Clinical Laboratory Evaluation
Day 1 up to 30 days after last dose of drug (Day 31 for each of the 4 periods)
Number of Participants With Clinically Significant Change From Baseline in 12-lead Electrocardiogram (ECG)
Day 1 up to 30 days after last dose of drug (Day 31 for each of the 4 periods)
Cmax: Maximum Observed Plasma Concentration for Febuxostat XR 80 mg in Fed (Regimen A) and Fasted (Regimen C) States
Day 1 pre-dose and at multiple timepoints (up to 48 hours) post dose

Maximum observed plasma concentration (Cmax) is the peak plasma concentration of a drug after administration, obtained directly from the plasma concentration-time curve. Participant blood samples were collected pre-dose and following a single oral dose.

Cmax: Maximum Observed Plasma Concentration for Febuxostat XR 40 mg (Regimen B) and Febuxostat XR 80 mg (Regimen C) in Fasted States
Day 1 pre-dose and at multiple time points (up to 48 hours) post dose

Maximum observed plasma concentration (Cmax) is the peak plasma concentration of a drug after administration, obtained directly from the plasma concentration-time curve. Participant blood samples were collected pre-dose and following a single oral dose.

AUCt: Area Under the Plasma Concentration-Time Curve (AUC) From Time 0 to Time of the Last Quantifiable Concentration for Febuxostat XR 80 mg in Fed (Regimen A) and Fasted (Regimen C) States
Day 1 pre-dose and at multiple timepoints (up to 48 hours) post dose

AUCt is a measure of total plasma exposure to the drug from Time 0 to Time of the Last Quantifiable Concentration. Participant blood samples were collected pre-dose and following a single oral dose.

AUCt: Area Under the Plasma Concentration-Time Curve (AUC) From Time 0 to Time of the Last Quantifiable Concentration for Febuxostat XR 40 mg (Regimen B) and Febuxostat XR 80 mg (Regimen C) in Fasted States
Day 1 pre-dose and at multiple timepoints (up to 48 hours) post dose

AUCt is a measure of total plasma exposure to the drug from Time 0 to Time of the Last Quantifiable Concentration (AUCt) Participant blood samples were collected pre-dose and following a single oral dose.

AUCinf: Area Under the Plasma Concentration-Time Curve From Time 0 to Infinity for Febuxostat XR 80 mg in Fed (Regimen A) and Fasted (Regimen C) States
Day 1 pre-dose and at multiple timepoints (up to 48 hours) post dose

AUCinf is a measure of total plasma exposure to the drug from time zero extrapolated to infinity. Participant blood samples were collected pre-dose and following a single oral dose.

AUCinf: Area Under the Plasma Concentration-Time Curve From Time 0 to Infinity for Febuxostat XR 40 mg (Regimen B) and Febuxostate XR 80 mg (Regimen C) in Fasted States
Day 1 pre-dose and at multiple timepoints (up to 48 hours) post dose

AUCinf is a measure of total plasma exposure to the drug from time zero extrapolated to infinity. Participant blood samples were collected pre-dose and following a single oral dose.

Secondary Endpoints

Percentage of Participants With Antiplatelet Trialists' Collaborative (APTC) Event
Up to last dose of study drug (approximately 83 months)
Percentage of Participants With Cardiovascular (CV) Death
Up to last dose of study drug (approximately 83 months)
Percentage of Participants With Non-fatal Myocardial Infarction (MI)
Up to last dose of study drug (approximately 83 months)
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
FebuxostatEXPERIMENTALFebuxostat 40 mg (or 80 mg beginning on week 4 if serum uric acid level was ≥6.0 mg/dL), tablets, orally, once daily for up to approximately 82 months.
AllopurinolACTIVE_COMPARATORAllopurinol 300 mg to 600 mg (increased in 100 mg increments each month until serum uric acid was \<6.0 mg/dL), tablets, orally, once daily for up to approximately 83 months to participants with mildly impaired renal function or normal renal function (estimated creatinine clearance \[eCLcr\] ≥60 mL/min) or allopurinol 200 mg to 400 mg (increased in 100 mg increments each month until serum uric acid was \<6.0 mg/dL), tablets, orally, once daily for up to approximately 83 months to participants with moderately impaired renal function (eCLcr ≥30 but \<60 mL/min).
Febuxostat 40 mg QDEXPERIMENTAL -
Febuxostat 80 mg QDEXPERIMENTAL -
Allopurinol 200 mg or 300 mg QDACTIVE_COMPARATOR(dependent on renal function)
Febuxostat 120 mg QDEXPERIMENTAL -
Allopurinol QDACTIVE_COMPARATOR -
Febuxostat 240 mg QDEXPERIMENTAL -
Placebo QDPLACEBO_COMPARATOR -
Allopurinol 300 mg QDACTIVE_COMPARATOR -
PlaceboPLACEBO_COMPARATORPlacebo-matching capsules, orally, twice daily for up to 12 months.
Febuxostat 30 mg BIDEXPERIMENTALFebuxostat 30 mg, capsules, orally, twice daily (BID) for up to 12 months.
Febuxostat 40/80 mg QDEXPERIMENTALParticipants initially received febuxostat 40 mg, capsules, once daily (QD) and one placebo-matching capsule QD and remained on this dose for up to 12 months if their serum urate (sUA) was \<6.0 mg/dL at the Day 14 visit. Participants whose sUA was ≥6.0 mg/dL at the Day 14 visit received febuxostat 80 mg, capsule, QD, and one placebo-matching capsule QD at the Month 1 visit, and for the remainder of the study.
Febuxostat 40 mg or 80 mgEXPERIMENTALFebuxostat 40 mg or 80 mg (based on serum urate levels at Day 14), capsules, orally, once daily for up to 24 Months.
1EXPERIMENTAL -
2EXPERIMENTAL -
3EXPERIMENTAL -
Sequence 1: ABDCEXPERIMENTALExperimental: Sequence 1: ABDC Febuxostat 80 mg extended-release (XR) capsule, orally, once, after a 10-hour fast, and 20 mL Maalox Advance Regular Strength liquid containing Aluminum Hydroxide 200 mg, Magnesium Hydroxide 200 mg, and Simethicone 20 mg/5 mL (hereafter referred as Maalox) or equivalent, orally, once, after a 10-hour fast, on Day 1 of Period 1 (A), followed by a 7-day washout period, followed by Maalox or equivalent, orally, once, after a 9-hour fast, and febuxostat 80 mg XR capsule, orally, once, after a 10-hour fast (1 hour after Maalox or equivalent), on Day 1 of Period 2 (B), followed by a 7-day washout period, followed by febuxostat 80 mg XR capsule, orally, once, after a 10-hour fast, on Day 1 of Period 3 (D), followed by a 7-day washout period, followed by febuxostat 80 mg XR capsule, orally, once, after a 10-hour fast, and Maalox or equivalent, orally, once, after an 11-hour fast (1 hour after febuxostat), on Day 1 of Period 4 (C).
Sequence 2: DACBEXPERIMENTALExperimental: Sequence 2: DACB Febuxostat 80 mg XR capsule, orally, once, after a 10-hour fast, on Day 1 of Period 1, followed by a 7-day washout period, followed by febuxostat 80 mg extended-release (XR) capsule, orally, once, after a 10-hour fast, and Maalox or equivalent, orally, once, after a 10-hour fast, on Day 1 of Period 2, followed by a 7-day washout period, followed by febuxostat 80 mg XR capsule, orally, once, after a 10-hour fast, and Maalox or equivalent, orally, once, after an 11-hour fast (1 hour after febuxostat), on Day 1 of Period 3, followed by a 7-day washout period, followed by Maalox or equivalent, orally, once, after a 9-hour fast, and febuxostat 80 mg XR capsule, orally, once, after a 10-hour fast (1 hour after Maalox or equivalent), on Day 1 of Period 4.
Sequence 3: CDBAEXPERIMENTALFebuxostat 80 mg XR capsule, orally, once, after a 10-hour fast, and Maalox or equivalent, orally, once, after an 11-hour fast (1 hour after febuxostat), on Day 1 of Period 1, followed by a 7-day washout period, followed by febuxostat 80 mg XR capsule, orally, once, after a 10-hour fast, on Day 1 of Period 2, followed by a 7-day washout period, followed by Maalox or equivalent, orally, once, after a 9-hour fast, and febuxostat 80 mg XR capsule, orally, once, after a 10-hour fast (1 hour after Maalox or equivalent), on Day 1 of Period 3, followed by a 7-day washout period, followed by Febuxostat 80 mg extended-release (XR) capsule, orally, once, after a 10-hour fast, and Maalox or equivalent, orally, once, after a 10-hour fast, on Day 1 of Period 4.
Sequence 4: BCADEXPERIMENTALMaalox or equivalent, orally, once, after a 9-hour fast, and febuxostat 80 mg XR capsule, orally, once, after a 10-hour fast (1 hour after Maalox), on Day 1 of Period 1, followed by a 7-day washout period, followed by febuxostat 80 mg XR capsule, orally, once, after a 10-hour fast, and Maalox or equivalent, orally, once, after an 11-hour fast (1 hour after febuxostat), on Day 1 of Period 2, followed by a 7-day washout period, followed by febuxostat 80 mg extended-release (XR) capsule, orally, once, after a 10-hour fast, and Maalox or equivalent, orally, once, after a 10-hour fast, on Day 1 of Period 3, followed by a 7-day washout period, followed by febuxostat 80 mg XR capsule, orally, once, after a 10-hour fast, on Day 1 of Period 4.
Treatment Sequence ABCEXPERIMENTALFebuxostat extended release (XR) 80 mg, capsules, orally, once on Day 1 of Period 1 after a high-fat meal, followed by a 7-day washout period, followed by febuxostat XR 40 mg, capsules, orally, once on Day 1 of Period 2 after a 10-hour fast, followed by a 7-day washout period, followed by febuxostat XR 80, capsules, orally, once on Day 1 of Period 3 after a 10-hour fast.
Treatment Sequence BCAEXPERIMENTALFebuxostat XR 40 mg, capsules, orally, once on Day 1 of Period 1 after a 10-hour fast, followed by a 7-day washout period, followed by febuxostat XR 80 mg, capsules, orally, once on Day 1 of Period 2 after a 10-hour fast, followed by a 7-day washout period, followed by Febuxostat XR 80 mg, capsules, orally, once on Day 1 of Period 3 after a high-fat meal.
Treatment Sequence CABEXPERIMENTALFebuxostat XR 80 mg, capsules, orally, once on Day 1 of Period 1 after a 10-hour fast, followed by a 7-day washout period, followed by febuxostat XR 80 mg, capsules, orally, once on Day 1 of Period 2 after a high-fat meal, followed by a 7-day washout period, followed by febuxostat XR 40 mg, capsules, orally, once on Day 1 of Period 3 after a 10-hour fast.

Interventions

NameTypeDescription
FebuxostatDRUGFebuxostat tablets
AllopurinolDRUGAllopurinol tablets
PlaceboDRUGPlacebo, orally, once daily for up to 28 weeks.
Placebo for FebuxostatDRUGFebuxostat placebo-matching capsules
Febuxostat XRDRUGFebuxostat extended-release (XR) capsules
Maalox Advance Regular Strength liquidDRUGMaalox Advance Regular Strength liquid containing Aluminum Hydroxide 200 mg, Magnesium Hydroxide 200 mg, and Simethicone 20 mg/5 mL or equivalent
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Eligibility Criteria

Age Range50 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites203

Inclusion Criteria: 1. The participant or the participant's legally acceptable representative signs and dates a written, informed consent form/Health Insurance Portability and Accountability Act (HIPAA) Authorization prior to the initiation of any study procedures. 2. The participant is male ≥50 ye...

Countries:United StatesMexico
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Frequently asked questions about Febuxostat

What is Febuxostat used for?

Febuxostat is a small molecule being studied for use in cardiovascular disease, gout, hyperuricosuria, joint damage, and renal impairment. It is also being evaluated in healthy volunteers. The drug is developed by Takeda Pharmaceutical Company Limited and is currently in Phase 3 clinical development.

What does Febuxostat target?

Febuxostat is a xanthine oxidase inhibitor that works by reducing the production of uric acid in the body. It is being studied for its effects on conditions related to high uric acid levels, such as gout and hyperuricosuria, as well as for potential benefits in cardiovascular disease and joint damage.

Who makes Febuxostat?

Febuxostat is developed by Takeda Pharmaceutical Company Limited, which is publicly traded under the ticker symbol TAK. The drug is a small molecule in Phase 3 clinical development for cardiovascular disease and other indications.

What phase is Febuxostat in?

Febuxostat is in Phase 3 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The drug has completed seven clinical trials, with no active trials currently ongoing.

What clinical trials is Febuxostat in?

Febuxostat has completed seven clinical trials, including NCT01077284 for hyperuricosuria and kidney stones, NCT01078389 for joint damage in gout, NCT01496469 for hypertension, and NCT02382640 for healthy volunteers. All trials are completed, with no active trials ongoing.

Is Febuxostat the same as Uloric?

Febuxostat is also known by the brand name Uloric. It is a xanthine oxidase inhibitor used to treat gout and hyperuricemia. The drug is being studied for additional indications such as cardiovascular disease and joint damage.