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enzalutamide

Phase 3

Prostate Cancer | Small molecule | Oncology |Pfizer, Inc.|Last Updated: Aug 12, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLEDDMCBiomarker
Total Trials6
Total Enrollment3,316

FDA Designations

No designations recorded

Clinical trial landscape

enzalutamide · 23 trials · 19 indications

Phase 3 6Phase 2 14Phase 1 3
NCT02677896A Study of Enzalutamide Plus Androgen Deprivation Therapy (ADT) Versus Placebo Plus ADT in Patients With Metastatic Hormone Sensitive Prostate Cancer (mHSPC)Metastatic Hormone Sensitive Prostate Cancer
COMPLETED1,150 Analytics
NCT02288247A Study to Assess the Benefit of Treatment Beyond Progression With Enzalutamide in Men Who Are Starting Treatment With Docetaxel After Worsening of Their Prostate Cancer When Taking Enzalutamide AloneMetastatic Castration Resistant Prostate Cancer
COMPLETED688 Analytics
NCT02319837Safety and Efficacy Study of Enzalutamide Plus Leuprolide in Patients With Nonmetastatic Prostate Cancer (EMBARK)Hormone Sensitive Prostate Cancer
ACTIVE NOT_RECRUITING1,068 Analytics
NCT02003924Safety and Efficacy Study of Enzalutamide in Patients With Nonmetastatic Castration-Resistant Prostate CancerNonmetastatic Castration-Resistant Prostate Cancer
COMPLETED1,401 Analytics
NCT01212991A Safety and Efficacy Study of Oral MDV3100 in Chemotherapy-Naive Patients With Progressive Metastatic Prostate CancerProstate Cancer
COMPLETED1,717 Analytics
NCT00974311Safety and Efficacy Study of MDV3100 in Patients With Castration-Resistant Prostate Cancer Who Have Been Previously Treated With Docetaxel-based ChemotherapyCastration-Resistant Prostate Cancer
COMPLETED1,199 Analytics
PHASE3COMPLETED
A Study of Enzalutamide Plus Androgen Deprivation Therapy (ADT) Versus Placebo Plus ADT in Patients With Metastatic Hormone Sensitive Prostate Cancer (mHSPC)
Metastatic Hormone Sensitive Prostate CancerUnlock trial analytics
PHASE3COMPLETED
A Study to Assess the Benefit of Treatment Beyond Progression With Enzalutamide in Men Who Are Starting Treatment With Docetaxel After Worsening of Their Prostate Cancer When Taking Enzalutamide Alone
Metastatic Castration Resistant Prostate CancerUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
Safety and Efficacy Study of Enzalutamide Plus Leuprolide in Patients With Nonmetastatic Prostate Cancer (EMBARK)
Hormone Sensitive Prostate CancerUnlock trial analytics
PHASE3COMPLETED
Safety and Efficacy Study of Enzalutamide in Patients With Nonmetastatic Castration-Resistant Prostate Cancer
Nonmetastatic Castration-Resistant Prostate CancerUnlock trial analytics
PHASE3COMPLETED
A Safety and Efficacy Study of Oral MDV3100 in Chemotherapy-Naive Patients With Progressive Metastatic Prostate Cancer
Prostate CancerUnlock trial analytics
PHASE3COMPLETED
Safety and Efficacy Study of MDV3100 in Patients With Castration-Resistant Prostate Cancer Who Have Been Previously Treated With Docetaxel-based Chemotherapy
Castration-Resistant Prostate CancerUnlock trial analytics

Study Endpoints

Primary Endpoints

Radiographic Progression-Free Survival (rPFS) Based on Independent Central Review (ICR) of Bone Scan According to Prostate Cancer Clinical Trials Working Group 2 (PCWG2) Criteria
From the date of randomization to the first objective evidence of rPD at any time or death (maximum duration was 26.6 months)

rPFS was calculated as the time from the date of randomization to the first objective evidence of radiographic progression disease (rPD) at any time or death up to 24 weeks after study drug discontinuation without documented radiographic progression, whichever occurred first. rPD was defined as progressive disease by Response Evaluation Criteria in Solid Tumors version 1.1 for soft tissue disease or by appearance of 2 or more new lesions on bone scan compared to baseline or week 13 according to PCWG2 criteria, as assessed by ICR or death. In participants with no rPFS event, rPFS was censored on the date of last evaluable radiographic assessment prior to the data analysis cutoff date. In participants with no baseline radiographic assessment, participants with no postbaseline radiographic assessments and participants with all postbaseline radiographic assessments documented as "not evaluable (NE)," rPFS was censored on the date of randomization.

rPFS Based on ICR of Bone Scan According to Protocol Assessment Criteria
From the date of randomization to the first objective evidence of rPD at any time or death (maximum duration was 26.6 months)

rPFS was calculated as the time from the date of randomization to the first objective evidence of radiographic progression disease (rPD) at any time or death up to 24 weeks after study drug discontinuation without documented radiographic progression, whichever occurred first. rPD was defined as progressive disease by Response Evaluation Criteria in Solid Tumors version 1.1 for soft tissue disease or by appearance of 2 or more new lesions on bone scan compared to baseline for week 13 or the best response on treatment for week 25 or later assessments, as assessed by ICR or death. In participants with no rPFS event, rPFS was censored on the date of last evaluable radiographic assessment prior to the data analysis cutoff date. In participants with no baseline radiographic assessment, participants with no postbaseline radiographic assessments and participants with all postbaseline radiographic assessments documented as "not evaluable (NE)," rPFS was censored on the date of randomization.

Progression Free Survival (PFS)
From date of randomization to the earliest of either documented disease progression (median duration: 35 weeks)

PFS: time from randomization (Period 2 Week 1) to earliest progression event. Progression is defined as radiographic progression, unequivocal clinical progression, or death on study. Radiographic progression is defined for bone disease by appearance of ≥ 2 new lesions on whole-body radionuclide bone scan per Prostate Cancer Clinical Trials Working Group 2 (PCWG2) criteria (i.e., unconfirmed progressive disease) that needs to be confirmed in the next assessment (i.e., progressive disease in the next assessment) or for soft tissue disease by Response Evaluation Criteria in Solid Tumors (RECIST) 1.1. Unequivocal clinical progression is defined as new onset cancer pain requiring chronic administration of opiate analgesia or deterioration from prostate cancer of Eastern Cooperative Oncology Group (ECOG) performance status score to ≥ 3, or initiation of subsequent lines of cytotoxic chemotherapy or radiation therapy or surgical intervention due to complications of tumor progression.

Metastasis-free Survival (MFS) Compared Between Enzalutamide Plus Leuprolide and Placebo Plus Leuprolide
From randomization until radiographic progression or death without radiographic progression, whichever occurred first (up to Month 98 when at least 197 MFS events occurred among the 3 treatment groups)

MFS was defined as the duration of time in months between randomization and the earliest objective evidence of radiographic progression by central imaging or death without radiographic progression, whichever occurred first. Radiographic progression for soft tissue disease was defined by Response Evaluation Criteria in Solid Tumors, version 1.1 (RECIST 1.1). Radiographic progression for bone disease was defined as the appearance of 1 or more metastatic lesions on bone scan (a bone scan assesses 5 regions of the skeleton, including skull, thorax, spine, pelvis, and extremities). Confirmation with a second imaging modality (plain film, computed tomography \[CT\], or magnetic resonance imaging \[MRI\]) was to be required when bone lesions were found in a single region on the bone scan. Appearance of metastatic lesions in 2 or more of the 5 regions on a bone scan was not to require confirmation with a second imaging modality.

Metastasis Free Survival (MFS)
From randomization until radiographic progression at any time, or death within 112 days of treatment discontinuation, whichever occurred first (until the data cut-off date of 28 June 2017, maximum duration of treatment: 42.8 months)

MFS:time from randomization to first date of radiographic progression (RP) (by Blinded independent central radiology review \[BICR\]) at any time or death within 112 days of treatment discontinuation without evidence of RP.RP for bone disease:appearance of 1 or more metastatic lesions on bone scan.RP for soft tissue disease:per Response Evaluation Criteria in Solid Tumors,\[RECIST 1.1\])-at least a 20 percent (%) increase in the sum of diameters of target lesions,taking as reference the smallest sum on study (includes the baseline sum if smallest on study).Participants who did not have MFS event at the time of analysis data cut-off (28 June 2017) were censored at date of last assessment showing no objective evidence of RP prior to skeletal-related event or two or more consecutive missed tumor assessments. Participants who were randomized but later confirmed to have metastatic disease before randomization were censored on date of randomization. Analysis was based on Kaplan-Meier estimates.

Overall Survival
During study period (up to 3 years)

Overall survival was defined as the time from randomization to death due to any cause. For patients who were alive at the time of the analysis data cutoff, overall survival was censored at the last date the patient was known to be alive or analysis data cutoff date, whichever was first. This included patients who were known to have died after the data analysis cutoff date. Patients with no post-baseline survival information were censored on the date of randomization.

Radiographic Progression-free Survival (rPFS)
During study period (up to 20 months)

Radiographic progression-free survival was defined as the time from randomization to the first objective evidence of radiographic disease progression assessed by independent central radiology review or death due to any cause within 168 days after treatment discontinuation, whichever was first. Radiographic disease progression was evaluated by CT scan or MRI and radionuclide bone scans at regularly scheduled visits. Radiographic disease progression in bone required a confirmatory scan. Radiographic disease progression in soft tissue did not require a confirmatory scan for purposes of analysis. Radiographic disease progression was evaluated by independent central radiology review using RECIST 1.1 for soft tissue disease and PCWG2 guidelines for bone disease. Patients who did not reach the endpoint were censored at their last assessment.

Time to Prostate Cancer Progression
From the date of randomization until the date of the cancer progression (pathological or therapeutic) (up to study completion date, 28 Aug 2020; approximately 50 months)

Time to cancer prostate progression (pathological or therapeutic): time (in months) from date of randomization until the date of cancer progression (pathological or therapeutic). Pathological progression: increase in primary or secondary Gleason pattern by greater than or equal (\>=) 1 or higher proportion of cancer positive cores (\>=15 percent \[%\] increase). Therapeutic progression: earliest occurrence of primary therapy for prostate cancer (prostatectomy/radiation/focal therapy/systemic therapy). Medians and 95% CIs were calculated with the Kaplan-Meier (KM) method. Participants with no cancer progression at the time of study completion, discontinuation or death were censored at the last assessment date. Participants switching therapy during the study were censored at the time of the initial therapy switch, and participants discontinuing therapy were censored at the time of study discontinuation.

Overall Survival (OS)
From date of randomization up to data cut-off date 02 Oct 2017 (approximately 22 months); median follow-up time was 14.65 months for enzalutamide and 13.83 for placebo.

OS was defined as the time from the date of randomization until the documented date of death from any cause. Participants who were still alive at the time of the data cut-off date was censored on the last date known to be alive or at the data cutoff date, whichever occurs first. Results based on Kaplan-Meier estimates.

Safety profile of enzalutamide assessed by adverse events (AEs), serious AEs (SAEs), vital signs, electrocardiograms (ECGs) and laboratory measurements
Up to 30 days after the last dose of enzalutamide or prior to the initiation of another anticancer therapy, whichever comes first (up to 18 months)
Clinical Benefit Rate (CBR)
Tumor assessments were performed every 8 weeks through week 49, and then every 12 weeks thereafter until disease progression or death; the median duration of treatment was 70 days, and the maximum was 660 days.

Clinical benefit rate was defined as the percentage of evaluable participants with best objective response of confirmed complete response or partial response per Response Evaluation Criteria in Solid Tumors (RECIST) 1.1, or prolonged stable disease (≥ 24 weeks). Complete response (CR) was defined as the disappearance of all target and non-target lesions and no new lesions, and lymph nodes all \< 10 mm in short axis. Partial response (PR) was defined as disappearance of target lesions or a ≥ 30% decrease in the size of target lesions, with persistence of non-target lesions and no new lesions. Stable disease (SD) was defined as \< 30% decrease and \< 20% increase in the size of target lesions, persistence of non-target lesions, and no new lesions. PR and CR required confirmation with equivalent or improved assessment no less than 4 weeks after the date that PR or CR was first observed. SD required confirmation with equivalent or improved assessment no less than 8 weeks after enrollment.

Number of Participants With Adverse Events (AEs)
From the first dose of study drug administration up 30 days following the last dose of study drug date, with a median duration of treatment of 10.1 months.

A treatment-emergent adverse event (TEAE) was defined as an adverse event occurring or worsening between the start of study treatment date and the latest date of 30 days after the last dose date or the 30-day follow-up visit date, and not later than the data cut-off date or the date of death. AEs, including abnormal clinical laboratory values, were graded using the National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE) guidelines (V4.03).

Progression Free Survival (PFS) Based on Independent Central Review (ICR) Assessment
From randomization until the data cut-off date of 19 October 2014, median duration of treatment was 11.6 months in the enzalutamide arm and 5.8 months in the bicalutamide arm.

PFS is the time from randomization to the date of the first progression event detected. A progression event was defined as objective evidence of radiographic disease progression based on the assessments by the ICR, skeletal-related event, initiation of new antineoplastic therapy or death by any cause, whichever occurred first. Radiographic disease progression was defined as either a progression in soft tissue on computed tomography (CT)/magnetic resonance imaging (MRI) scan according to Response Evaluation Criteria in Solid Tumors (RECIST) 1.1, and/or a progression in bone lesions on bone scan (≥ 2 new bone lesions) confirmed by the next bone scan. A skeletal-related event was any radiation therapy or surgery to bone, pathologic bone fracture, spinal cord compression or change in antineoplastic therapy to treat bone pain. The initiation of new antineoplastic therapy included any new therapy for the treatment of disease progression after the study drug administration started.

Number of participants with adverse events
Until End of Study (Up to 96 Months)
Percentage of Participants With at Least (>=) 1 Responding Bone Lesion Assessed by Total Sodium Fluoride (NaF) Standardized Uptake Value [SUVtotal] at Third 18F-NaF PET/CT Imaging (NaF-3) Schedule
At NaF-3 schedule: at time of PSA, or radiographic(bone or soft tissue), or clinically relevant progression, or at 2years without progression after treatment initiation, whichever occurred first(From first dose of study drug up to maximum of 34.1 months)

Bone lesion responded: if its change from baseline in SUVtotal is below limit of agreement (LOA, no specific value, based upon test/retest analysis using software). SUVtotal: total NaF uptake,indicated tumor burden across all bone lesions/in individual lesions reflecting bone-metastatic prostate cancer.NaF-3 performed on any of these: 1) prostate-specific antigen (PSA) progression(increase of \>=25% and absolute increase of \>=2.0 ng/mL above nadir); 2) bone progressive disease(PD)(appearance of \>=2 new lesions after screening assessed by technetium Tc 99m medronate \[99mTc-MDP\] bone scintigraphy); 3) soft tissue PD; 4) clinically relevant progression by investigator; 5) at 2 years without progression after treatment initiation. PD, RECIST1.1:\>=20% increase in sum of diameters of target lesions,(reference smallest sum on study, included baseline sum if that is smallest on study),relative increase of 20%,sum of diameters indicated absolute increase of \>=5mm, appearance of \>=1 new lesions.

Progression Free Survival (PFS): Intent-to-Treat (ITT) Population By Interactive Web Recognition System (IWRS)
From randomization until PD, last tumor assessment without PD before new antitumor treatment initiation or death due to any cause, whichever occurred first (up to 3 years)

PFS was defined as the time in months from randomization to the first documentation of progression of disease (PD) or death on study due to any cause, whichever occurred first. PD according to response evaluation criteria in solid tumors version 1.1 (RECIST 1.1) was defined as greater than or equal to (\>=) 20 percent (%) increase in the sum of diameters of the target lesions taking as a reference the smallest sum recorded since the start of treatment or unequivocal progression in non-target lesions or the appearance of 1 or more new lesions. The analysis of PFS was based on investigator assessment of disease progression. Participants who were not known to have had a PFS event at the analysis date were censored at last tumor assessment date prior to data cutoff or date of new treatment initiation, whichever occurred first.

Progression Free Survival (PFS): Diagnostic Positive (DX+) Population By Interactive Web Recognition System (IWRS)
From randomization until PD, last tumor assessment without PD before new antitumor treatment initiation or death due to any cause, whichever occurred first (up to 3 years)

PFS was defined as the time in months from randomization to the first documentation of PD or death on study due to any cause, whichever occurred first. PD according to RECIST 1.1, was defined as \>= 20% increase in the sum of diameters of the target lesions taking as a reference the smallest sum recorded since the start of treatment or unequivocal progression in non-target lesions or the appearance of 1 or more new lesions. The analysis of PFS was based on investigator assessment of disease progression. Participants who were not known to have had a PFS event at the analysis date were censored at last tumor assessment date prior to data cutoff or date of new treatment initiation, whichever occurred first.

Percentage of Participants With Clinical Benefit at Week 16: Evaluable Population
Week 16

Percentage of participants with a clinical benefit at Week 16 defined as percentage of participants with a best response of complete response (CR), partial response(PR), stable disease(SD) for \>=16 weeks on radiologic imaging based on Investigator assessment using Response Evaluation Criteria in Solid Tumors version 1.1(RECIST 1.1). An estimate of the percentage and its exact 2-sided 85% confidence interval(CI) were calculated using the Blaker method. As per RECIST 1.1, CR defined as disappearance of all target, non-target lesions and normalization of tumor marker level and all lymph nodes decreased to non-pathological in size \<10mm short axis. PR: At least 30% decrease in sum of longest diameter (LD) of target lesions taking as reference baseline sum of LD, without progression of non-target lesions, no appearance of new lesions. SD: Neither sufficient reduction to qualify as PR nor sufficient increase to qualify as PD, using the smallest sum diameters during the study as a reference.

Percentage of Participants With Clinical Benefit at Week 16: Intent-to-Treat (ITT) Population
Week 16

Percentage of participants with a clinical benefit at Week 16 defined as percentage of participants with a best response of CR, PR, or SD for \>= 16 weeks on radiologic imaging based on Investigator assessment using RECIST 1.1. An estimate of the percentage and its exact 2-sided 85% CI were calculated using the Blaker method. As per RECIST 1.1, CR defined as disappearance of all target, non-target lesions and normalization of tumor marker level and all lymph nodes decreased to non-pathological in size \<10 mm short axis. PR: At least 30% decrease in sum of LD of target lesions taking as reference baseline sum of LD, without progression of non-target lesions, no appearance of new lesions. SD: Neither sufficient reduction to qualify as PR nor sufficient increase to qualify as PD, using the smallest sum diameters during the study as a reference.

Pathologic Complete Response Rate
Day 180

Pathologic complete response rate was defined as percentage of participants with pathologic complete response. Pathologic complete response rate following triplet therapy (enzalutamide in combination with leuprolide and dutasteride) and enzalutamide alone when administered as neoadjuvant therapy for 180 days prior to prostatectomy in participants with localized prostate cancer. Pathologic complete response was defined as the absence of morphologically identifiable carcinoma in the prostatectomy specimen, as assessed by the local and central pathologist.

Change From Baseline in Bone Marrow Testosterone
Baseline, Week 9

Bone marrow biopsies were performed at the Day 1 visit prior to initiation of enzalutamide administration. Repeat bone marrow biopsies were performed at the Week 9 visit. If a repeat bone marrow was not performed at the Week 9 visit or if a patient discontinued the study before the Week 9 visit, a bone marrow biopsy was obtained at the Safety Follow-up visit. Assessment of intratumoral testosterone was assessed by liquid chromatography mass spectrometry.

Change From Baseline in Bone Marrow Dihydrotestosterone
Baseline, Week 9

Bone marrow biopsies were performed at the Day 1 visit prior to initiation of enzalutamide administration. Repeat bone marrow biopsies were performed at the Week 9 visit. If a repeat bone marrow was not performed at the Week 9 visit or if a patient discontinued the study before the Week 9 visit, a bone marrow biopsy was obtained at the Safety Follow-up visit. Assessment of intratumoral dihydrotestosterone was assessed by liquid chromatography mass spectrometry.

Change From Baseline in Bone Marrow Testosterone at Week 9 by Prostate-Specific Antigen (PSA) Response Status
Baseline, Week 9

Bone marrow biopsies were performed at the Day 1 visit prior to initiation of enzalutamide administration. Repeat bone marrow biopsies were performed at the Week 9 visit. If a repeat bone marrow was not performed at the Week 9 visit or if a patient discontinued the study before the Week 9 visit, a bone marrow biopsy was obtained at the Safety Follow-up visit. Assessment of intratumoral testosterone was assessed by liquid chromatography mass spectrometry. Serum samples for measurement of PSA levels were obtained at baseline prior to initiation of enzalutamide administration and at the Week 9 visit. The change from baseline in bone marrow testosterone levels at Week 9 was correlated with PSA response status at Week 9.

Change From Baseline in Bone Marrow Dihydrotestosterone at Week 9 by Prostate-Specific Antigen (PSA) Response Status
Baseline, Week 9

Bone marrow biopsies were performed at the Day 1 visit prior to initiation of enzalutamide administration. Repeat bone marrow biopsies were performed at the Week 9 visit. If a repeat bone marrow was not performed at the Week 9 visit or if a patient discontinued the study before the Week 9 visit, a bone marrow biopsy was obtained at the Safety Follow-up visit. Assessment of intratumoral dihydrotestosterone was assessed by liquid chromatography mass spectrometry. Serum samples for measurement of PSA levels were obtained at baseline prior to initiation of enzalutamide administration and at the Week 9 visit. The change from baseline in bone marrow dihydrotestosterone levels at Week 9 was correlated with PSA response status at Week 9.

Pharmacokinetics (PK) of Digoxin in combination with rosuvastatin in plasma: maximum concentration (Cmax)
Up to Day 71

Cmax will be recorded from the pharmacokinetic (PK) plasma samples collected.

Pharmacokinetics (PK) of Digoxin in combination with rosuvastatin in plasma: area under the concentration-time curve from the time of dosing to the last measurable concentration (AUClast)
Up to Day 71

AUClast will be recorded from the pharmacokinetic (PK) plasma samples collected.

Pharmacokinetics (PK) of Digoxin in combination with rosuvastatin in plasma: area under the concentration time curve from the time of dosing extrapolated to time infinity (AUCinf)
Up to Day 71

AUCinf will be recorded from the pharmacokinetic (PK) plasma samples collected.

Rate of dose limiting toxicity (DLT)
28 Days

Rate of dose limiting toxicity (DLT) in the first 28 days of study therapy by dose level when escalating doses of crizotinib are combined with enzalutamide and when appropriate a GnRH agonist.

Dose-Escalation Phase: Percentage of Participants With Dose-limiting Toxicity (DLTs)
Baseline up to Day 35

DLTs were defined as any of following events related to the study drug: Any adverse event (AE) consistent with a seizure of any grade; Grade greater than equal to (\>=) 3 fatigue, diarrhea, nausea, or vomiting that did not improve to Grade 1 within 14 days of initiating standard of care therapy; any Grade \>=3 hematologic toxicity with the following modifications: 1) Grade \>=3 platelet count associated with bleeding, 2) Grade \>=3 absolute neutrophil count that persists for 7 or more days or that was associated with fevers (febrile neutropenia); Grade \>=3 any other non-hematological toxicity that was determined to be related to study drug.

Percentage of Participants With Adverse Events of Grade 3 or Higher Severity by National Cancer Institute Common Toxicity Criteria For Adverse Events (NCI CTCAE) (Version 4.03)
Day 1 up to 30 days after the last dose of study drug or before initiation of a new systemic antitumor treatment, whichever occurred first (up to 3.5 years)

An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. AE was assessed according to severity grading based on NCI CTCAE Version 4.03 and defined as Grade 3 AEs = severe or medically significant events but not immediately life-threatening, hospitalization or prolongation of hospitalization indicated, disabling, limiting self-care activities of daily living; Grade 4 AEs = life-threatening, urgent intervention indicated; Grade 5 AEs = death related to adverse event.

Percentage of Participants With Treatment-Emergent Serious Adverse Events (SAEs)
Day 1 up to 30 days after the last dose of study drug or before initiation of a new systemic antitumor treatment, whichever occurred first (up to 3.5 years)

An AE was any untoward medical occurrence in a participant who received study treatment without regard to possibility of causal relationship. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dying), persistent or significant disability or incapacity, congenital anomaly. Treatment-emergent are events between first dose of study drug and up to 30 days after last dose of study drug that were absent before treatment or that worsened relative to pretreatment state. AEs included both serious and non-serious adverse events.

Percentage of Participants Who Discontinued the Study Drug Due to Adverse Events or Serious Adverse Events
Day 1 up to 30 days after the last dose of study drug or before initiation of a new systemic antitumor treatment, whichever occurred first (up to 3.5 years)

An AE was any untoward medical occurrence in a participant who received study treatment without regard to possibility of causal relationship. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dying), persistent or significant disability or incapacity, congenital anomaly.

Percentage of Participants Who Require Dose Reductions Due to Adverse Events
Day 1 up to 30 days after the last dose of study drug or before initiation of a new systemic antitumor treatment, whichever occurred first (up to 3.5 years)

An AE was any untoward medical occurrence in a participant who received study treatment without regard to possibility of causal relationship. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dying), persistent or significant disability or incapacity, congenital anomaly.

Percentage of Participants With Potentially Clinically Significant Change From Baseline in Vital Signs
Day 1 up to 30 days after the last dose of study drug or before initiation of a new systemic antitumor treatment, whichever occurred first (up to 3.5 years)

Absolute systolic blood pressure (SBP) greater than (\>) 180 millimeter of mercury (mm Hg) and an increase of \>40 mm Hg from baseline; absolute SBP less than (\<) 90 mm Hg and an decrease of \>30 mm Hg from baseline. Absolute diastolic blood pressure (DBP) \>105 mm Hg and an increase of \>30 mm Hg from baseline; absolute DBP \<50 mm Hg and an increase of \>20 mm Hg from baseline. Absolute heart rate \>120 beats per minute (bpm) and an increase of \>30 bpm from baseline; absolute heart rate \<50 bpm and decrease of \>20 bpm from baseline. Participants with any of these abnormalities were reported for this outcome in each arm.

Dose-Escalation Phase: Maximum Plasma Concentration (Cmax) of Enzalutamide and Its Metabolites After Single Dose
pre-dose, 0.5, 1, 2, 4, 6, 24, 48, 72 and 96 hours post dose on Day 1

Carboxylic Acid (M1) and N-desmethyl (M2) are two metabolites of Enzalutamide.

Dose-Escalation Phase: Time to Reach Maximum Plasma Concentration (Tmax) of Enzalutamide and Its Metabolites After Single Dosing
pre-dose, 0.5, 1, 2, 4, 6, 24, 48, 72 and 96 hours post dose on Day 1

Carboxylic Acid (M1) and N-desmethyl (M2) are two metabolites of Enzalutamide.

Dose-Escalation Phase: Area Under the Plasma Concentration-time Curve From Time Zero to 24 Hours (AUC24h) of Enzalutamide and Its Metabolites After Single Dose
pre-dose, 0.5, 1, 2, 4, 6 and 24 hours post dose on Day 1

Carboxylic Acid (M1) and N-desmethyl (M2) are two metabolites of Enzalutamide.

Dose-Escalation Phase: Area Under the Plasma Concentration-time Curve From Time Zero to 72 Hours (AUC72h) of Enzalutamide After Single Dosing
pre-dose, 0.5, 1, 2, 4, 6, 24, 48 and 72 hours post dose on Day 1
Dose-Escalation Phase: Area Under the Plasma Concentration-Time Profile From Time Zero Extrapolated to Infinite Time (AUCinf) of Enzalutamide After Single Dosing
pre-dose, 0.5, 1, 2, 4, 6, 24, 48, 72 and 96 hours post dose on Day 1

AUCinf = Area under the plasma concentration versus time curve (AUC) from time zero (pre-dose) to extrapolated infinite time (0 - inf).

Dose-Escalation Phase: Terminal Elimination Half-Life (t1/2) of Enzalutamide After Single Dosing
pre-dose, 0.5, 1, 2, 4, 6, 24, 48, 72 and 96 hours post dose on Day 1

Terminal elimination half-life is the time measured for the plasma concentration of Enzalutamide to decrease by one-half of its initial concentration.

Dose Escalation Phase: Apparent Oral Clearance (CL/F) of Enzalutamide After Single Dosing
pre-dose, 0.5, 1, 2, 4, 6, 24, 48, 72 and 96 hours post dose on Day 1

Apparent oral clearance of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological processes. It was obtained after oral dose (apparent oral clearance) is influenced by the fraction of the dose absorbed.

Dose Escalation Phase: Apparent Volume of Distribution (Vz/F) of Enzalutamide After Single Dosing
pre-dose, 0.5, 1, 2, 4, 6, 24, 48, 72 and 96 hours post dose on Day 1

Volume of distribution is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. Apparent volume of distribution after oral dose (Vz/F) is influenced by the fraction absorbed.

Dose-Escalation Phase: Maximum Plasma Concentration (Cmax) of Enzalutamide and Its Metabolites After Multiple Dosing
pre-dose, 0.5, 1, 2, 4, 6, and 24 hours post-dose on Day 50

Carboxylic Acid (M1) and N-desmethyl (M2) are two metabolites of Enzalutamide.

Dose-Escalation Phase: Time to Reach Maximum Plasma Concentration (Tmax) of Enzalutamide and Its Metabolites After Multiple Dosing
pre-dose, 0.5, 1, 2, 4, 6, and 24 hours post-dose on Day 50

Carboxylic Acid (M1) and N-desmethyl (M2) are two metabolites of Enzalutamide.

Dose-Escalation Phase: Area Under the Curve From Time Zero to End of Dosing Interval (AUCtau) of Enzalutamide and Its Metabolites After Multiple Dosing
pre-dose, 0.5, 1, 2, 4, 6, and 24 hours post-dose on Day 50

Area under the plasma concentration versus time-curve from time zero to end of dosing interval (AUCtau), where dosing interval was 24 hours. Carboxylic Acid (M1) and N-desmethyl (M2) are two metabolites of Enzalutamide.

Dose-Escalation Phase: Apparent Oral Clearance (CL/F) of Enzalutamide After Multiple Dosing
pre-dose, 0.5, 1, 2, 4, 6, and 24 hours post-dose on Day 50

Apparent oral clearance of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological processes. It was obtained after oral dose (apparent oral clearance) is influenced by the fraction of the dose absorbed.

Dose-Escalation Phase: Peak-to-Trough Ratio of Enzalutamide and Its Metabolites After Multiple Dosing
pre-dose, 0.5, 1, 2, 4, 6, and 24 hours post-dose on Day 50

Peak-to-trough ratio was calculated by dividing Cmax with Cmin of Enzalutamide and its Metabolites. Carboxylic Acid (M1) and N-desmethyl (M2) are two metabolites of Enzalutamide. Cmax was maximum plasma concentration during the dosing interval and Cmin was minimum observed plasma concentration during the dosing interval.

Dose-Escalation Phase: Accumulation Ratio of AUC24 of Enzalutamide and Its Metabolites After Multiple Dosing
pre-dose, 0.5, 1, 2, 4, 6, and 24 hours post-dose on Day 1 and 50

Accumulation Ratio was defined as the ratio of AUC24 of Day 50 to AUC24 of Day 1, where AUC24 was area under the plasma concentration-time curve from time zero to 24 hours post-dose. Carboxylic Acid (M1) and N-desmethyl (M2) are two metabolites of Enzalutamide.

Secondary Endpoints

Overall Survival (OS)
From randomization to death due to any cause (maximum duration was 58.6 months)
Time to Prostate Specific Antigen (PSA) Progression
From the date of randomization to the first observation of PSA progression (maximum duration was 26.6 months)
Time to Start of New Antineoplastic Therapy
From randomization to the date of the first dose administration of the first antineoplastic therapy (maximum duration was 58.6 months)
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Enzalutamide + Androgen Deprivation Therapy (ADT)EXPERIMENTALParticipants received 160 mg enzalutamide orally once daily during double-blind treatment period until radiographic progression was documented or until the participants started an investigational agent or new therapy for treatment of prostate cancer or until any other discontinuation criterion was met. Eligible participants who received enzalutamide during double-blind treatment period and provided informed consent to take part in open-label period continued to receive 160 mg enzalutamide orally once daily in open-label period until disease progression, unacceptable toxicity or any other discontinuation criteria were met. ADT (either bilateral orchiectomy or Luteinizing hormone-releasing hormone (LHRH) agonist/antagonist) was maintained during study treatment as per standard of care and provided by the site's pharmacy stock.
Placebo + Androgen Deprivation Therapy (ADT)PLACEBO_COMPARATORParticipants received matching placebo orally once daily during double-blind treatment period until radiographic progression was documented or until the participants started an investigational agent or new therapy for treatment of prostate cancer or until any other discontinuation criterion was met. ADT (either bilateral orchiectomy or LHRH agonist/antagonist) was maintained during study treatment as per standard of care and provided by the site's pharmacy stock.
Placebo followed by EnzalutamideEXPERIMENTALEligible participants who received enzalutamide matching placebo during double-blind treatment period and provided informed consent to take part in open-label period switched to receive 160 mg enzalutamide orally once daily in open-label period until disease progression, unacceptable toxicity or any other discontinuation criteria were met. ADT (either bilateral orchiectomy or LHRH agonist/antagonist) was maintained during study treatment as per standard of care and provided by the site's pharmacy stock.
EnzalutamideEXPERIMENTALParticipants (Pts) received OL enzalutamide 160 mg QD from Day 1 in Period 1 (P1) until they were either randomized to Period 2 (P2), were ineligible, experienced intolerable toxicity, withdrew, or died. Pts with confirmed disease progression in P1, meeting eligibility, were randomized to receive placebo/enzalutamide 160 mg QD with docetaxel 75 mg/m\^2 in 1hour infusion every 3 weeks \& prednisolone 5mg twice daily in P2. Docetaxel \& prednisolone were administered for upto 10 cycles (1 cycle=3 weeks) or more per investigator discretion, while placebo/enzalutamide continued until progression, toxicity, withdrawal, or death. Extension(EXT) phase was available for Pts in P1\& P2 who were not meeting primary endpoint. Enzalutamide continued until disease progression, intolerable toxicity, withdrawal, or death. Pts not entering EXT discontinued and received local care. Those benefiting from enzalutamide in EXT at study closure continued in study 9785-CL-0123 or via commercial enzalutamide.
PlaceboPLACEBO_COMPARATORParticipants received an OL treatment with enzalutamide 160 mg capsules, orally once daily from day 1 in period 1 until randomization to period 2 treatment, confirmation of ineligibility for period 2 treatment, intolerable toxicity, withdrawal, or death, whichever occurred first. Participants with confirmed disease progression on enzalutamide in period 1 and who continued to meet all eligibility criteria received placebo matched to enzalutamide, orally once daily in combination with docetaxel 75 mg/m\^2 in a one-hour infusion every 3 weeks and prednisolone 5 mg orally twice daily, DB in period 2. Docetaxel and prednisolone were administered up to 10 cycles (1 cycle = 3 weeks) or additional cycles as assessed by the investigator and enzalutamide was administered until disease progression, intolerable toxicity, withdrawal or death, whichever occurred first. Participants discontinued the study and received local standard of care.
Enzalutamide plus leuprolideEXPERIMENTALEnzalutamide (160 mg) administered as four 40-mg capsules by mouth once daily in combination with leuprolide administered as as a single intramuscular or subcutaneous injection once every 12 weeks
Enzalutamide monotherapyEXPERIMENTALEnzalutamide (160 mg) administered as four 40-mg capsules by mouth once daily
Leuprolide plus placeboACTIVE_COMPARATOREnzalutamide placebo (placebo) capsules (identical in appearance to enzalutamide) administered as 4 capsules by mouth once daily in combination with leuprolide administered as a single intramuscular or subcutaneous injection once every 12 weeks. The randomized blinded portion of the study has concluded following primary endpoint analyses. In the Open Label Period the placebo is no longer applicable in this study arm, and patients continue to receive leuprolide alone.
Active Surveillance (AS)OTHERParticipants did not receive any study treatment in this arm but were on continued active surveillance (AS) for 1 year of treatment period. Following the 1-year treatment period, all participants were followed for 1 additional year. Post the 1-year follow up, participants then started the continued follow-up period, in which, participants were followed up every 3 months for these 2 years, after which follow-up was either every 6 months up to 36 months or until the end of study (total duration of follow up in the study was approximately up to 35.9 months).
Enzalutamide 160 mgEXPERIMENTALParticipants received enzalutamide 160 milligrams (mg) capsules, orally QD during double blind treatment period until disease progression, unacceptable toxicity, or any other discontinuation criterion was met. Eligible participants received enzalutamide 160 mg capsules, orally QD during open label period until disease progression, unacceptable toxicity, or any other discontinuation criterion was met. Median treatment duration was 64 days.
Enzalutamide + TrastuzumabEXPERIMENTALParticipants received 160 mg enzalutamide orally once daily and 6 mg/kg trastuzumab administered by intravenous infusion or subcutaneous injection every 21 days. Participants continued on treatment until disease progression, unacceptable toxicity or any other discontinuation criteria were met.
Enzalutamide + Abiraterone + PrednisoneEXPERIMENTALParticipants received enzalutamide combined with abiraterone acetate once daily plus prednisone twice daily.
BicalutamideACTIVE_COMPARATORParticipants received bicalutamide 50 mg orally once daily until confirmed radiographic disease progression, skeletal-related event or the initiation of a new antineoplastic therapy.
enzalutamide plus abiraterone acetate and prednisoneEXPERIMENTALSubjects enrolling from study 9785-CL-0011 or MDV3100-10 (PLATO) study may receive abiraterone acetate once daily and prednisone twice daily, in addition to enzalutamide once daily
Enzalutamide plus leuprolide acetateEXPERIMENTALSubjects enrolling from study MDV3100-13 (EMBARK) study may receive leuprolide acetate once every 12 weeks in addition to enzalutamide once daily
Enzalutamide & exemestaneEXPERIMENTALEnzalutamide 160 mg/day administered as four 40mg soft gelatin capsules by mouth once daily with or without food and exemestane 50mg (two 25mg tablets overencapsulated as a single capsule during the blinded portion of the study and two 25mg tablets after unblinding) once daily after food.
Placebo & exemestaneACTIVE_COMPARATORPlacebo and exemestane 25mg (overencapsulated to match 50mg dose during the blinded portion of the study and one 25mg tablet without placebo after unblinding) once daily after food.
Enzalutamide aloneEXPERIMENTALEnzalutamide 160 mg, orally, once daily
Enzalutamide & Leuprolide & DutasterideEXPERIMENTALEnzalutamide 160 mg, orally, once daily and leuprolide 22.5 mg, intramuscular injection, every 3 months, and dutasteride, 0.5 mg, orally, once daily
digoxin plus rosuvastatin and enzalutamideEXPERIMENTALParticipants will receive a single oral dose cocktail containing 0.25 mg digoxin and 10 mg rosuvastatin on Day 1 and 64. A single oral dose of placebo to match enzalutamide will be given on Day 1 and 160 mg enzalutamide once daily on Days 8 through 71.
crizotinib and enzalutamideEXPERIMENTALA traditional 3+3 dose escalation scheme will be used to identify the recommended phase 2 dose (RP2D) of crizotinib when used in combination with standard fixed dose enzalutamide. * Crizotinib- given orally daily-28 day cycle * Enzalutamide- given orally daily-28 day cycle
enzalutamide (80-mg with increase to 160 mg)EXPERIMENTALenzalutamide be provided as two or four 40-mg capsules by mouth daily
enzalutamide and anastrozoleEXPERIMENTALenzalutamide (160 mg) administered as four 40-mg capsules by mouth once daily in combination with anastrozole (1 mg) administered as one 1-mg tablet by mouth once daily.
enzalutamide and exemestane 25 mgEXPERIMENTALenzalutamide (160 mg) administered as four 40-mg capsules by mouth once daily in combination with exemestane administered as one 25-mg tablet daily
enzalutamide and exemestane 50 mgEXPERIMENTALenzalutamide (160 mg) administered as four 40-mg capsules by mouth once daily in combination with exemestane administered as two 25-mg tablets daily
enzalutamide and fulvestrantEXPERIMENTALenzalutamide (160 mg) administered as four 40-mg capsules by mouth once daily in combination with fulvestrant (500 mg) administered as two 250-mg intramuscular injections every 28 days

Interventions

NameTypeDescription
EnzalutamideDRUGOral
PlaceboDRUGOral
DocetaxelDRUGintravenous infusion
PrednisoloneDRUGOral
Placebo (No longer applicable in Open Label study period)DRUGSugar pill to mimic enzalutamide
Leuprolide Open LabelDRUG -
Active SurveillanceOTHER -
TrastuzumabDRUGIntravenous infusion (IV) or subcutaneous injection if it is standard of care within a country
abiraterone acetateDRUGParticipants received 1000 mg of abiraterone acetate orally once daily (4 tablets, 250 mg each).
prednisoneDRUGParticipants received 5 mg of prednisone orally twice daily (2 tablets, 5 mg each).
bicalutamideDRUGtablets
Leuprolide acetateDRUGSubjects enrolling from study MDV3100-13 (EMBARK) study may receive leuprolide acetate once every 12 weeks in addition to enzalutamide once daily
exemestaneDRUG25mg (overencapsulated to match 50mg dose during the blinded portion of the study and one 25mg tablet after unblinding) by mouth once daily after food.
Placebo (for enzalutamide)DRUGSugar pill manufactured to mimic enzalutamide administered as four soft gelatin capsules by mouth once daily with or without food.
LeuprolideDRUG -
DutasterideDRUG -
enzalutamide PlaceboDRUGoral
digoxinDRUGoral
rosuvastatinDRUGoral
CrizotinibDRUGCrizotinib is an ATP-competitive small-molecule inhibitor of the ALK, c-Met/HGFR, RON, and ROS receptor tyrosine kinases.
anastrozoleDRUG1 mg/day
fulvestrantDRUG500 mg every 28 days
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Eligibility Criteria

Age Range18 Years to N/A
SexMALE
Healthy VolunteersNo
Study Sites203

Inclusion Criteria: * Subject is considered an adult according to local regulation at the time of signing informed consent. * Subject is diagnosed with histologically or cytologically confirmed adenocarcinoma of the prostate without neuroendocrine differentiation, signet cell or small cell histolog...

Countries:United StatesArgentinaAustraliaBelgiumCanadaChileDenmarkFinlandFranceGermanyIsraelItalyJapanNetherlandsNew ZealandPolandRomaniaRussiaSlovakiaSouth KoreaSpainSwedenTaiwanUnited KingdomAustriaCzechiaGreeceNorwaySwitzerlandTurkey (Türkiye)BrazilChinaHong KongMalaysiaSerbiaSingaporeThailandUkraineLithuaniaSouth AfricaPuerto RicoGeorgiaMoldovaIreland
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Competitive Landscape -Prostate Cancer 254 trials

Recent Changes (Last 90 Days)

MEDIUMAug 12, 2026NCT02960022Status: RECRUITING → ACTIVE_NOT_RECRUITING
MEDIUMAug 12, 2026NCT02960022Status: RECRUITING → ACTIVE_NOT_RECRUITING
LOWJul 31, 2026NCT02960022lastUpdatePostDate: changed
LOWJul 31, 2026NCT02960022lastUpdatePostDate: changed
LOWJul 9, 2026NCT02319837lastUpdatePostDate: changed
LOWJul 9, 2026NCT02319837lastUpdatePostDate: changed

Frequently asked questions about enzalutamide

What is Enzalutamide used for?

Enzalutamide is a small molecule oncology drug being studied for advanced hepatocellular carcinoma, nonmetastatic castration-resistant prostate cancer, breast cancer, prostate carcinoma metastatic to the bone, metastatic castration-resistant prostate cancer, and HER2 amplified cancers. It is currently in Phase 2 clinical development.

Who makes Enzalutamide?

Enzalutamide is being developed by Pfizer, Inc., which trades under the ticker PFE. The drug is a small molecule in the oncology therapeutic area and is currently in Phase 2 clinical trials.

What phase is Enzalutamide in?

Enzalutamide is in Phase 2 clinical development. It is an investigational drug and has not been approved by the FDA. There are six clinical trials listed for the drug, with one active and five completed.

What clinical trials is Enzalutamide in?

Enzalutamide has been studied in several clinical trials, including NCT01650194, which tested it in combination with abiraterone acetate in bone metastatic castration-resistant prostate cancer, and NCT02384382, a PET/CT bone imaging study in castration-resistant prostate cancer. NCT02960022 is an active study for subjects who previously participated in an enzalutamide clinical study.

Is Enzalutamide the same as MDV3100?

Yes, Enzalutamide is also known as MDV3100. The clinical trial NCT01650194, which studied the safety and tolerability of enzalutamide in combination with abiraterone acetate, refers to the drug as enzalutamide (MDV3100).

How does Enzalutamide work?

Enzalutamide is a small molecule that targets the androgen receptor pathway. It is being studied in prostate cancer and other oncology indications, where it interferes with androgen receptor signaling to potentially slow tumor growth.