A Phase 1/2 interventional study of Exicorilant and Enzalutamide in Metastatic Castration-Resistant Prostate Cancer, sponsored by Corcept Therapeutics. Terminated at 9 sites in 2 countries. Open to male participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-05-15.
Sponsored by Corcept Therapeutics · Phase 1/2, Interventional, and Treatment
This study consists of a dose escalation study with an expansion phase to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD), and preliminary efficacy of exicorilant (CORT125281) in combination with enzalutamide in patients with metastatic castration-resistant prostate cancer (mCRPC) to identify a recommended dose (RD) for Phase 2 studies.
Exicorilant is a selective glucocorticoid receptor (GR) antagonist. In this study, exicorilant will be administered orally in combination with enzalutamide to patients with mCRPC to evaluate the safety, tolerability, PK, PD, and preliminary efficacy of the regimen. The study consists of 2 phases: a dose-determination phase and an expansion phase. The dose determination phase is designed to determine dose-limiting toxicities and the RD of exicorilant plus enzalutamide in patients with mCRPC. Once the recommended dosing regimen has been determined, the following expansion cohorts will be enrolled and treated with exicorilant plus enzalutamide at the RD level.
Abiraterone-Resistant Cohort: Patients who have progressed during treatment with abiraterone, and have received no other androgen receptor (AR)-blocking therapies.
AR Antagonist-Resistant Cohort: Patients who have progressed during treatment with enzalutamide or other second-generation AR inhibitors.
The effect of food on exicorilant PK will be assessed in a portion of the patients enrolled in the Expansion Phase. The 2 expansion cohorts will be enrolled in parallel.
In each phase of the study, routine assessments of safety and tolerability will be performed and samples will be collected to determine standard PK parameters for exicorilant, enzalutamide, and their major metabolites. PD, quality of life evaluations and preliminary evaluations of antitumor activity of exicorilant with enzalutamide will be performed throughout the study.
7,046 studies on the registry are indexed under Prostatic Neoplasms; 1,580 are open to participants now.
This study's enrollment of 39 is below the median of 60 across 5,348 interventional studies indexed under Prostatic Neoplasms.
Browse Prostatic Neoplasms studies →Corcept Therapeutics is the lead sponsor of 73 studies on the registry; 3 are open to participants now.
Of its 32 completed or terminated interventional studies of FDA-regulated products, 9 (28%) have results posted.
Counted across the registry records on this site, refreshed daily.
Major Inclusion Criteria:
Dose-Determination Phase Segment 2: Currently receiving enzalutamide with a rising PSA as follows:
Expansion Phase: Patients must have progressed while receiving an androgen-directed therapy as follows:
Major Exclusion Criteria:
Dose Determination Phase and Expansion Phases will exclude patients for the following:
Dose-Determination Phase (Segment 1 only):
Expansion Phase Abi-Resistant Cohort:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Cancer deposits in the brain from a cancer that started elsewhere in the body (CNS metastases include the spinal cord).
Studies differ: some leave out any brain metastases, some accept ones treated and stable for some weeks. Check what this study allows.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Histologically or cytologically confirmed
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Washout period
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Brain metastases
Cancer deposits in the brain from a cancer that started elsewhere in the body (CNS metastases include the spinal cord).
Studies differ: some leave out any brain metastases, some accept ones treated and stable for some weeks. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CYP3A (CYP3A4) inhibitors and inducers
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients will receive lead-in enzalutamide monotherapy once daily for 28 days. Patients will then receive combination treatment with twice-daily exicorilant 180 mg (total daily dose 360 mg) and enzalutamide once daily in 28-day dosing cycles.
Drug: Exicorilant · Drug: Enzalutamide
Patients will receive lead-in enzalutamide monotherapy once daily for 28 days. Patients will then receive combination treatment with twice-daily exicorilant 140 mg (total daily dose 280 mg) and enzalutamide once daily in 28-day dosing cycles.
Drug: Exicorilant · Drug: Enzalutamide
Patients will not receive lead-in enzalutamide monotherapy. Patients will receive combination treatment with twice-daily exicorilant 140 mg (total daily dose 280 mg) and enzalutamide once daily in 28-day dosing cycles.
Drug: Exicorilant · Drug: Enzalutamide
Patients will receive treatment with once-daily exicorilant starting at 240 mg and titrating to 280 mg and then to 320 mg at 2-week intervals, as tolerated, in combination with enzalutamide once daily in 28-day dosing cycles. Patients in this arm are reported at the highest titrated dose of exicorilant achieved: 240 mg exicorilant.
Drug: Exicorilant · Drug: Enzalutamide
Patients will receive treatment with once-daily exicorilant starting at 240 mg and titrating to 280 mg and then to 320 mg at 2-week intervals, as tolerated, in combination with enzalutamide once daily in 28-day dosing cycles. Patients in this arm are reported at the highest titrated dose of exicorilant achieved: 280 mg exicorilant.
Drug: Exicorilant · Drug: Enzalutamide
Patients will receive treatment with once-daily exicorilant starting at 240 mg and titrating to 280 mg and then to 320 mg at 2-week intervals, as tolerated, in combination with enzalutamide once daily in 28-day dosing cycles. Patients in this arm are reported at the highest titrated dose of exicorilant achieved: 320 mg exicorilant.
Drug: Exicorilant · Drug: Enzalutamide
Patients will receive combination treatment with once-daily exicorilant 240 mg, enzalutamide once daily, and placebo in 28-day dosing cycles.
Drug: Exicorilant · Drug: Enzalutamide · Drug: Placebo
Patients who have progressed during treatment with abiraterone and no other androgen receptor-blocking therapies will receive exicorilant and enzalutamide.
Drug: Exicorilant · Drug: Enzalutamide
Subcohort (first 10 patients enrolled into Cohort A). Patients enrolled into this subcohort will receive a single dose of exicorilant at Cycle 1 Day -7 and a single dose of exicorilant at Cycle 1 Day 1 30 minutes after a standard breakfast to assess the effect of food on pharmacokinetic (PK)parameters. Patients will then begin exicorilant in combination with enzalutamide on Cycle 1 Day 2 and continue in 28-day dosing cycles.
Drug: Exicorilant · Drug: Enzalutamide
Patients who progressed during treatment with enzalutamide or second-generation androgen receptor-blocking (ARant) therapies will receive a daily dose of exicorilant and enzalutamide.
Drug: Exicorilant · Drug: Enzalutamide
Exicorilant is supplied as capsules for oral dosing
Also known as: CORT125281
Enzalutamide will be taken orally
Also known as: Xtandi
Placebo capsules to match the appearance of the exicorilant capsules
Number of Patients With One or More Dose-Limiting Toxicity (DLT)
Assess the maximum tolerated dose (MTD) and/or biologically active doses of exicorilant in combination with enzalutamide to identify the recommended dose (RD) for Phase 2 studies based on the number of patients who experienced a DLT while receiving exicorilant in combination with enzalutamide. DLTs were defined as any of the protocol-specified toxicities that the Investigator considered possibly or probably related to study drug that occurred during the DLT-evaluation period. The MTD is defined as the highest dose at which the DLT rate was \<33%.
Time frame: From first dose of exicorilant through completion of Cycle 1 (up to 28 days) for Segment 1 and from first dose of exicorilant through completion of Cycle 3 (up to 84 days) for Segment 2
Number of Patients With One or More Treatment-Emergent Adverse Events
The safety of each treatment group will be assessed by evaluating the incidence of treatment-emergent adverse events.
Time frame: Up to 27 months for Segment 1 and up to 19 months for Segment 2
Area Under the Concentration Versus Time Curve (AUC) of Plasma Exicorilant: Segment 1
AUC from time zero to 12 hours postdose (AUC0-12) calculated using linear up and log down method.
Time frame: Predose and at time intervals up to 12 hours postdose on Cycle 1 Day 1 and Cycle 2 Day 1
Maximum Observed Concentration (Cmax) of Plasma Exicorilant: Segment 1
Maximum observed concentration over the dosing interval
Time frame: Predose and at time intervals up to 12 hours postdose on Cycle 1 Day 1 and Cycle 2 Day 1
AUC of Plasma Enzalutamide: Segment 1
AUC from time zero to 24 hours postdose (AUC0-24) calculated using linear up and log down method.
Time frame: Predose and at time intervals up to 24 hours postdose on Cycle 1 Day -1, Cycle 1 Day 1, and Cycle 2 Day 1
Cmax of Plasma Enzalutamide: Segment 1
Maximum observed concentration over the dosing interval
Time frame: Predose and at time intervals up to 24 hours postdose on Cycle 1 Day -1, Cycle 1 Day 1, and Cycle 2 Day 1
AUC of Plasma N-Desmethyl Enzalutamide: Segment 1
AUC0-24 calculated using linear up and log down method.
Time frame: Predose and at time intervals up to 24 hours postdose on Cycle 1 Day -1, Cycle 1 Day 1, and Cycle 2 Day 1
Cmax of Plasma N-Desmethyl Enzalutamide: Segment 1
Maximum observed concentration over the dosing interval
Time frame: Predose and at time intervals up to 24 hours postdose on Cycle 1 Day -1, Cycle 1 Day 1, and Cycle 2 Day 1
AUC of Plasma Enzalutamide Carboxylic Acid: Segment 1
AUC0-24 calculated using linear up and log down method.
Time frame: Predose and at time intervals up to 24 hours postdose on Cycle 1 Day -1, Cycle 1 Day 1, and Cycle 2 Day 1
Cmax of Plasma Enzalutamide Carboxylic Acid: Segment 1
Maximum observed concentration over the dosing interval
Time frame: Predose and at time intervals up to 24 hours postdose on Cycle 1 Day -1, Cycle 1 Day 1, and Cycle 2 Day 1
AUC of Plasma Exicorilant: Segment 2
AUC from time zero to 6 hours postdose (AUC0-6) calculated using linear up and log down method. Patients in Arm B did not receive dose escalations, but they were sampled for pharmacokinetic analysis on the same time frame as the Arm A patients: Cycle 1 Day 15, Week 4, and Week 6.
Time frame: Predose and at time intervals up to 6 hours on Cycle 1 Day 15 (before dose escalation), and at 2 weeks after each dose escalation (Week 4 for escalation from 240 mg to 280 mg exicorilant and Week 6 for escalation from 280 mg to 320 mg exicorilant).
Cmax of Plasma Exicorilant: Segment 2
Maximum observed concentration over the dosing interval. Patients in Arm B did not receive dose escalations, but they were sampled for pharmacokinetic analysis on the same time frame as the Arm A patients: Cycle 1 Day 15, Week 4, and Week 6.
Time frame: Predose and at time intervals up to 6 hours on Cycle 1 Day 15 (before dose escalation), and at 2 weeks after each dose escalation (Week 4 for escalation from 240 mg to 280 mg exicorilant and Week 6 for escalation from 280 mg to 320 mg exicorilant).
AUC of Plasma Enzalutamide: Segment 2
AUC from time zero to 6 hours postdose (AUC0-6) calculated using linear up and log down method. Patients in Arm B did not receive dose escalations, but they were sampled for pharmacokinetic analysis on the same time frame as the Arm A patients: Cycle 1 Day 15, Week 4, and Week 6.
Time frame: Predose and at time intervals up to 6 hours on Cycle 1 Day 15 (before dose escalation), and at 2 weeks after each dose escalation (Week 4 for escalation from 240 mg to 280 mg exicorilant and Week 6 for escalation from 280 mg to 320 mg exicorilant).
Cmax of Plasma Enzalutamide: Segment 2
Maximum observed concentration over the dosing interval. Patients in Arm B did not receive dose escalations, but they were sampled for pharmacokinetic analysis on the same time frame as the Arm A patients: Cycle 1 Day 15, Week 4, and Week 6.
Time frame: Predose and at time intervals up to 6 hours on Cycle 1 Day 15 (before dose escalation), and at 2 weeks after each dose escalation (Week 4 for escalation from 240 mg to 280 mg exicorilant and Week 6 for escalation from 280 mg to 320 mg exicorilant).
AUC of Plasma N-Desmethyl Enzalutamide: Segment 2
AUC from time zero to 6 hours postdose (AUC0-6) calculated using linear up and log down method. Patients in Arm B did not receive dose escalations, but they were sampled for pharmacokinetic analysis on the same time frame as the Arm A patients: Cycle 1 Day 15, Week 4, and Week 6.
Time frame: Predose and at time intervals up to 6 hours on Cycle 1 Day 15 (before dose escalation), and at 2 weeks after each dose escalation (Week 4 for escalation from 240 mg to 280 mg exicorilant and Week 6 for escalation from 280 mg to 320 mg exicorilant).
Cmax of Plasma N-Desmethyl Enzalutamide: Segment 2
Maximum observed concentration over the dosing interval. Patients in Arm B did not receive dose escalations, but they were sampled for pharmacokinetic analysis on the same time frame as the Arm A patients: Cycle 1 Day 15, Week 4, and Week 6.
Time frame: Predose and at time intervals up to 6 hours on Cycle 1 Day 15 (before dose escalation), and at 2 weeks after each dose escalation (Week 4 for escalation from 240 mg to 280 mg exicorilant and Week 6 for escalation from 280 mg to 320 mg exicorilant).
AUC of Plasma Enzalutamide Carboxylic Acid: Segment 2
AUC from time zero to 6 hours postdose (AUC0-6) calculated using linear up and log down method. Patients in Arm B did not receive dose escalations, but they were sampled for pharmacokinetic analysis on the same time frame as the Arm A patients: Cycle 1 Day 15, Week 4, and Week 6.
Time frame: Predose and at time intervals up to 6 hours on Cycle 1 Day 15 (before dose escalation), and at 2 weeks after each dose escalation (Week 4 for escalation from 240 mg to 280 mg exicorilant and Week 6 for escalation from 280 mg to 320 mg exicorilant).
Cmax of Plasma Enzalutamide Carboxylic Acid: Segment 2
Maximum observed concentration over the dosing interval. Patients in Arm B did not receive dose escalations, but they were sampled for pharmacokinetic analysis on the same time frame as the Arm A patients: Cycle 1 Day 15, Week 4, and Week 6.
Time frame: Predose and at time intervals up to 6 hours on Cycle 1 Day 15 (before dose escalation), and at 2 weeks after each dose escalation (Week 4 for escalation from 240 mg to 280 mg exicorilant and Week 6 for escalation from 280 mg to 320 mg exicorilant).
Objective Response Rate (ORR)
Confirmed ORR is defined as the proportion of patients with measurable disease at Baseline who achieve a complete regression (CR) or partial regression (PR) by Prostate Cancer Clinical Trials Working Group 3 (PCWG3) / Modified Response Evaluation Criteria in Solid Tumors v1.1 (mRECIST) criteria, after confirmation.
Time frame: Up to 22 months
Number of Patients With ≥50% Reduction in Prostate-Specific Antigen (PSA)
Time frame: Up to 39 months
Time to PSA Progression
Assess the time to PSA progression defined as the first occurrence of 50% or greater increase in PSA levels. Kaplan-Meier estimates of time to PSA progression were calculated as (earliest date of PSA progression or censoring - date of first study treatment + 1)/30.4375.
Time frame: Up to 39 months
Percentage of Patients Who Are Progression-Free by PSA Criteria at 4, 6, and 12 Months
Assess the percentage of patients who are progression-free by PSA criteria, or death. PSA progression was defined as the first occurrence of 50% or greater increase in PSA levels. Values are Kaplan-Meier estimates of the patients progression free at the time points specified.
Time frame: 4, 6, and 12 months
Time to First Symptomatic Skeletal Event (SSE)
Assess the time to first SSE defined as symptomatic fracture, radiation or surgery to bone, or spinal cord compression. Kaplan-Meier estimates of time to first SSE were calculated as (earliest date of SSE or censoring - date of first study treatment + 1)/30.4375.
Time frame: Up to 39 months
Time to Progression by Radiographic Criteria
Assess radiographic progression free survival (PFS) defined as the time interval from first dose of study drug (exicorilant and/or enzalutamide) to the date when the first site of disease progression is found on computerized tomography (CT), magnetic resonance imaging (MRI), or radionucleotide bone scan per PCWG3/mRECIST v1.1, or death whichever occurs first. The data values are Kaplan-Meier estimates.
Time frame: Up to 22 months
Percentage of Patients Who Are Progression-Free by Radiographic Criteria at 4, 6, and 12 Months
Assess the percentage of patients who are progression-free by radiographic criteria per PCWG3/RECIST v1.1, or death whichever occurs first. Values are Kaplan-Meier estimates of the patients progression free at the time points specified.
Time frame: 4, 6, and 12 months
Time to Progression by Clinical or Radiographic Criteria
Determine PFS by clinical or radiographic criteria, or death, whichever occurs first. Clinical progression was defined as treatment discontinuation due to disease progression by investigator assessment per PCWG3/mRECIST v1.1, or by PSA criteria. The data values are Kaplan-Meier estimates.
Time frame: Up to 22 months
Percentage of Patients Who Are Progression-Free by Clinical or Radiographic Criteria at 4, 6, and 12 Months
Assess the percentage of patients who are progression-free by clinical or radiographic measures at 4, 6, and 12 months. Values are Kaplan-Meier estimates of the patients progression free at the time points specified.
Time frame: 4, 6, and 12 months
Time to Progression by Clinical or Biochemical Criteria
Determine PFS by clinical criteria or biochemical criteria (defined as treatment discontinuation due to clinical progression by investigator assessment, or by PSA criteria) PCWG3/mRECIST v1.1, or death whichever occurs first. The data values are Kaplan-Meier estimates.
Time frame: Up to 33 months
Percentage of Patients Who Are Progression-Free by Clinical or Biochemical Criteria at 4, 6, and 12 Months
Assess the percentage of patients who are progression-free by clinical or biochemical criteria (defined as treatment discontinuation due to clinical progression by investigator assessment or by PSA criteria) PCWG3/mRECIST v1.1, or death whichever occurs first at 4, 6, and 12 months. Values are Kaplan-Meier estimates of the patients progression free at the time points specified.
Time frame: 4, 6, and 12 months
Duration of Response (DOR)
Determine the DOR as defined as the time from the first occurrence of a documented objective tumor response to the time of radiographic progression (per investigator using PCWG3/mRECIST v1.1 criteria) or death from any cause on study, whichever occurs first. DOR was calculated as (earliest date of progression, death, or censoring - date of first documented objective response +1)/30.4375. The data values are Kaplan-Meier estimates.
Time frame: Up to 11 months
Overall Survival (OS)
Determine OS assessed as the time from the first dose of study drug (exicorilant and/or enzalutamide) to the date of death from any cause. The data values are Kaplan-Meier estimates.
Time frame: Up to 52 months
By Sponsor decision, no patients were enrolled in the study after the Dose Determination Segment 2; thus, no patients were enrolled in the Dose Expansion arms.
| Milestone | Enrolled But Not Allocated | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant | Dose Expansion - Abi-Resistant Cohort (Open-label) | Dose Expansion - Abi-Resistant Cohort Food Effect (Open-label) | Dose Expansion - ARant-Resistant Cohort (Open-label) |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Started | 2 | 2 | 3 | 7 | 8 | 1 | 10 | 6 | 0 | 0 | 0 |
| Completed | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Not completed | 2 | 2 | 3 | 7 | 8 | 1 | 10 | 6 | 0 | 0 | 0 |
| Withdrew: Death | 1 | 1 | 1 | 5 | 1 | 0 | 5 | 4 | 0 | 0 | 0 |
| Withdrew: Study terminated by sponsor | 0 | 0 | 2 | 2 | 7 | 1 | 3 | 2 | 0 | 0 | 0 |
| Withdrew: Withdrawal by subject | 1 | 0 | 0 | 0 | 0 | 0 | 2 | 0 | 0 | 0 | 0 |
| Withdrew: Investigator decision | 0 | 1 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
Assess the maximum tolerated dose (MTD) and/or biologically active doses of exicorilant in combination with enzalutamide to identify the recommended dose (RD) for Phase 2 studies based on the number of patients who experienced a DLT while receiving exicorilant in combination with enzalutamide. DLTs were defined as any of the protocol-specified toxicities that the Investigator considered possibly or probably related to study drug that occurred during the DLT-evaluation period. The MTD is defined as the highest dose at which the DLT rate was \<33%.
| Participants | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant |
|---|---|---|---|---|---|---|---|
| Number of Patients With One or More Dose-Limiting Toxicity (DLT) | 2 | 1 | 0 | 4 | 1 | 0 | 1 |
The safety of each treatment group will be assessed by evaluating the incidence of treatment-emergent adverse events.
| Participants | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant |
|---|---|---|---|---|---|---|---|
| Number of Patients With One or More Treatment-Emergent Adverse Events | 2 | 3 | 7 | 8 | 1 | 10 | 6 |
AUC from time zero to 12 hours postdose (AUC0-12) calculated using linear up and log down method.
| ng-hr/ml | Dose Determination Segment 1 (Open-label): Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label): Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label):Cohort 3 - 280 mg Exicorilant |
|---|---|---|---|
| Cycle 1 Day 1 | 387 ± 11.3 | 275 ± 64.2 | 477 ± 82.4 |
| Cycle 2 Day 1 | 433 ± 14.8 | 431 ± 29.0 | 747 ± 82.2 |
Maximum observed concentration over the dosing interval
| ng/ml | Dose Determination Segment 1 (Open-label): Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label): Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label): Cohort 3 - 280 mg Exicorilant |
|---|---|---|---|
| Cycle 1 Day 1 | 84.2 ± 24.5 | 91.6 ± 34.5 | 186.1 ± 70.5 |
| Cycle 2 Day 1 | 108 ± 59.2 | 125 ± 15.4 | 174 ± 68.7 |
AUC from time zero to 24 hours postdose (AUC0-24) calculated using linear up and log down method.
| ng-hr/ml | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant |
|---|---|---|---|
| Cycle 1 Day -1 lead-in | 281499 ± 2.7 | 255623 ± 20.0 | — |
| Cycle 1 Day 1 | 307849 | 278573 ± 25.6 | 36979 ± 21.4 |
| Cycle 2 Day 1 | 334761 ± 3.8 | 410557 ± 16.3 | 386651 ± 15.3 |
Maximum observed concentration over the dosing interval
| ng/ml | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant |
|---|---|---|---|
| Cycle 1 Day -1 lead-in | 15112 ± 15.5 | 12759 ± 27.5 | — |
| Cycle 1 Day 1 | 15500 ± 0.9 | 14186 ± 13.0 | 4260 ± 36.5 |
| Cycle 2 Day 1 | 16497 ± 2.6 | 19669 ± 7.9 | 19338 ± 11.6 |
AUC0-24 calculated using linear up and log down method.
| ng-hr/ml | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant |
|---|---|---|---|
| Cycle 1 Day -1 lead-in | 143300 | 121320 ± 26.1 | — |
| Cycle 1 Day 1 | — | 146225 ± 21.0 | 717 |
| Cycle 2 Day 1 | 180628 ± 21.5 | 181855 ± 7.8 | 146580 ± 42.7 |
Maximum observed concentration over the dosing interval
| ng/ml | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant |
|---|---|---|---|
| Cycle 1 Day -1 lead-in | 7835 ± 14.3 | 5812 ± 12.6 | — |
| Cycle 1 Day 1 | 8570 ± 18.3 | 7074 ± 20.4 | 112 ± 1152.2 |
| Cycle 2 Day 1 | 7760 ± 21.2 | 7996 ± 6.8 | 6742 ± 46.1 |
AUC0-24 calculated using linear up and log down method.
| ng-hr/ml | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant |
|---|---|---|---|
| Cycle 1 Day -1 lead-in | 19108 | 34493 | — |
| Cycle 1 Day 1 | 125786 | 52471 ± 26.3 | 1287 |
| Cycle 2 Day 1 | 80970 ± 219.0 | 52579 ± 23.1 | 63388 ± 93.3 |
Maximum observed concentration over the dosing interval
| ng/ml | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant |
|---|---|---|---|
| Cycle 1 Day -1 lead-in | 2286 ± 121.4 | 1930 ± 39.5 | — |
| Cycle 1 Day 1 | 3181 ± 113.8 | 2554 ± 26.6 | 127 ± 180.0 |
| Cycle 2 Day 1 | 3719 ± 235.9 | 2862 ± 31.6 | 3088 ± 88.0 |
AUC from time zero to 6 hours postdose (AUC0-6) calculated using linear up and log down method. Patients in Arm B did not receive dose escalations, but they were sampled for pharmacokinetic analysis on the same time frame as the Arm A patients: Cycle 1 Day 15, Week 4, and Week 6.
| ng-hr/ml | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant (Patients in Arm B Did Not Dose Escalate) |
|---|---|---|---|---|
| Cycle 1 Day 15 before dose escalation | 1086 ± 43.4 | 511 | 1125 ± 56.1 | 1201 ± 77.0 |
| After dose escalation from 240 mg to 280 mg exicorilant. | 974 | — | 1424 ± 53.3 | 990 ± 33.2 |
| After dose escalation from 280 mg to 320 mg exicorilant. | — | — | 1589 ± 38.8 | 1012 ± 48.3 |
Maximum observed concentration over the dosing interval. Patients in Arm B did not receive dose escalations, but they were sampled for pharmacokinetic analysis on the same time frame as the Arm A patients: Cycle 1 Day 15, Week 4, and Week 6.
| ng/ml | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant (Patients in Arm B Did Not Dose Escalate) |
|---|---|---|---|---|
| Cycle 1 Day 15 before dose escalation | 402 ± 34.7 | 157 | 418 ± 61.7 | 495 ± 80.7 |
| After dose escalation from 240 mg to 280 mg exicorilant | 247 | — | 528 ± 58.6 | 398 ± 63.8 |
| After dose escalation from 280 mg to 320 mg exicorilant | — | — | 534 ± 56.8 | 374 ± 65.9 |
AUC from time zero to 6 hours postdose (AUC0-6) calculated using linear up and log down method. Patients in Arm B did not receive dose escalations, but they were sampled for pharmacokinetic analysis on the same time frame as the Arm A patients: Cycle 1 Day 15, Week 4, and Week 6.
| ng-hr/ml | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant (Patients in Arm B Did Not Dose Escalate) |
|---|---|---|---|---|
| Cycle 1 Day 15 before dose escalation | 93894 ± 27.5 | 90563 | 98157 ± 26.1 | 95735 ± 8.5 |
| After dose escalation from 240 mg to 280 mg exicorilant | 150022 | — | 102373 ± 20.3 | 96684 ± 17.6 |
| After dose escalation from 280 mg to 320 mg exicorilant | — | — | 111632 ± 12.4 | 95653 ± 11.8 |
Maximum observed concentration over the dosing interval. Patients in Arm B did not receive dose escalations, but they were sampled for pharmacokinetic analysis on the same time frame as the Arm A patients: Cycle 1 Day 15, Week 4, and Week 6.
| ng/ml | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant (Patients in Arm B Did Not Dose Escalate) |
|---|---|---|---|---|
| Cycle 1 Day 15 before dose escalation | 17609 ± 28.3 | 16800 | 18004 ± 25.6 | 18440 ± 15.2 |
| After dose escalation from 240 mg to 280 mg exicorilant | 27000 | — | 18899 ± 17.9 | 18408 ± 22.8 |
| After dose escalation from 280 mg to 320 mg exicorilant | — | — | 20235 ± 13.2 | 18157 ± 17.0 |
AUC from time zero to 6 hours postdose (AUC0-6) calculated using linear up and log down method. Patients in Arm B did not receive dose escalations, but they were sampled for pharmacokinetic analysis on the same time frame as the Arm A patients: Cycle 1 Day 15, Week 4, and Week 6.
| ng-hr/ml | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant (Patients in Arm B Did Not Dose Escalate) |
|---|---|---|---|---|
| Cycle 1 Day 15 before dose escalation | 67872 ± 7.0 | 34131 | 50752 ± 20.6 | 49744 ± 21.0 |
| After dose escalation from 240 mg to 280 mg exicorilant | 49995 | — | 51977 ± 22.0 | 50353 ± 28.2 |
| After dose escalation from 280 mg to 320 mg exicorilant | — | — | 48255 ± 11.2 | 48411 ± 20.6 |
Maximum observed concentration over the dosing interval. Patients in Arm B did not receive dose escalations, but they were sampled for pharmacokinetic analysis on the same time frame as the Arm A patients: Cycle 1 Day 15, Week 4, and Week 6.
| ng/ml | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant (Patients in Arm B Did Not Dose Escalate) |
|---|---|---|---|---|
| Cycle 1 Day 15 before dose escalation | 12448 ± 7.7 | 6520 | 9350 ± 22.8 | 10116 ± 24.3 |
| After dose escalation from 240 mg to 280 mg exicorilant | 8600 | — | 9682 ± 20.4 | 9873 ± 28.4 |
| After dose escalation from 280 mg to 320 mg exicorilant | — | — | 8870 ± 13.4 | 9185 ± 23.8 |
AUC from time zero to 6 hours postdose (AUC0-6) calculated using linear up and log down method. Patients in Arm B did not receive dose escalations, but they were sampled for pharmacokinetic analysis on the same time frame as the Arm A patients: Cycle 1 Day 15, Week 4, and Week 6.
| ng-hr/ml | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant (Patients in Arm B Did Not Dose Escalate) |
|---|---|---|---|---|
| Cycle 1 Day 15 before dose escalation | 25662 ± 55.7 | 14108 | 24863 ± 41.6 | 19310 ± 56.9 |
| After dose escalation from 240 mg to 280 mg exicorilant | 86613 | — | 25995 ± 42.6 | 23393 ± 21.1 |
| After dose escalation from 280 mg to 320 mg exicorilant | — | — | 31814 ± 20.8 | 18047 ± 43.6 |
Maximum observed concentration over the dosing interval. Patients in Arm B did not receive dose escalations, but they were sampled for pharmacokinetic analysis on the same time frame as the Arm A patients: Cycle 1 Day 15, Week 4, and Week 6.
| ng/ml | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant (Patients in Arm B Did Not Dose Escalate) |
|---|---|---|---|---|
| Cycle 1 Day 15 before dose escalation | 4693 ± 50.8 | 2480 | 4572 ± 40.8 | 3713 ± 58.3 |
| After dose escalation from 240 mg to 280 mg exicorilant | 15400 | — | 4821 ± 43.1 | 4745 ± 15.1 |
| After dose escalation from 280 mg to 320 mg exicorilant | — | — | 6032 ± 24.8 | 3524 ± 44.1 |
Confirmed ORR is defined as the proportion of patients with measurable disease at Baseline who achieve a complete regression (CR) or partial regression (PR) by Prostate Cancer Clinical Trials Working Group 3 (PCWG3) / Modified Response Evaluation Criteria in Solid Tumors v1.1 (mRECIST) criteria, after confirmation.
| Participants | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant |
|---|---|---|---|---|---|---|---|
| Objective Response Rate (ORR) | 1 | 0 | 1 | 0 | 0 | 0 | 0 |
| Participants | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant |
|---|---|---|---|---|---|---|---|
| Number of Patients With ≥50% Reduction in Prostate-Specific Antigen (PSA) | 2 | 0 | 3 | 0 | 0 | 1 | 0 |
Assess the time to PSA progression defined as the first occurrence of 50% or greater increase in PSA levels. Kaplan-Meier estimates of time to PSA progression were calculated as (earliest date of PSA progression or censoring - date of first study treatment + 1)/30.4375.
| months | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant |
|---|---|---|---|---|---|---|---|
| Time to PSA Progression | NA (NA to NA) | NA (0.99 to NA) | 38.90 (0.99 to NA) | 1.43 (0.49 to NA) | 1.41 | 1.41 (0.49 to NA) | 3.25 (0.95 to NA) |
Assess the percentage of patients who are progression-free by PSA criteria, or death. PSA progression was defined as the first occurrence of 50% or greater increase in PSA levels. Values are Kaplan-Meier estimates of the patients progression free at the time points specified.
| Percentage of patients | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant |
|---|---|---|---|---|---|---|---|
| 4 months | 0 (NA to NA) | NA (NA to NA) | 85.71 (33.41 to 97.86) | 0 (NA to NA) | 0 | 40.00 (12.27 to 67.02) | 50.00 (11.09 to 80.37) |
| 6 months | 0 (NA to NA) | NA (NA to NA) | 85.71 (33.41 to 97.86) | 0 (NA to NA) | 0 | 40.00 (12.27 to 67.02) | 33.33 (4.61 to 67.56) |
| 12 months | 0 (NA to NA) | NA (NA to NA) | 85.71 (33.41 to 97.86) | 0 (NA to NA) | 0 | 26.67 (4.76 to 56.34) | 16.67 (0.77 to 51.68) |
Assess the time to first SSE defined as symptomatic fracture, radiation or surgery to bone, or spinal cord compression. Kaplan-Meier estimates of time to first SSE were calculated as (earliest date of SSE or censoring - date of first study treatment + 1)/30.4375.
| months | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant |
|---|---|---|---|---|---|---|---|
| Time to First Symptomatic Skeletal Event (SSE) | 0 (NA to NA) | NA (2.83 to NA) | NA (25.79 to NA) | NA (NA to NA) | NA (NA to NA) | NA (NA to NA) | NA (NA to NA) |
Assess radiographic progression free survival (PFS) defined as the time interval from first dose of study drug (exicorilant and/or enzalutamide) to the date when the first site of disease progression is found on computerized tomography (CT), magnetic resonance imaging (MRI), or radionucleotide bone scan per PCWG3/mRECIST v1.1, or death whichever occurs first. The data values are Kaplan-Meier estimates.
| months | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant |
|---|---|---|---|---|---|---|---|
| Time to Progression by Radiographic Criteria | 18.46 (NA to NA) | 1.81 (0.82 to NA) | 6.21 (0.49 to NA) | 5.55 (1.02 to NA) | 0 | 5.08 (1.02 to NA) | NA (3.91 to NA) |
Assess the percentage of patients who are progression-free by radiographic criteria per PCWG3/RECIST v1.1, or death whichever occurs first. Values are Kaplan-Meier estimates of the patients progression free at the time points specified.
| Percentage of patients | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant |
|---|---|---|---|---|---|---|---|
| 4 months | 100.0 (100.0 to 100.0) | 0.00 (NA to NA) | 83.33 (27.31 to 97.47) | 62.50 (14.19 to 89.31) | 0 | 66.67 (28.17 to 87.83) | 83.33 (27.31 to 97.47) |
| 6 months | 100.0 (100.0 to 100.0) | 0.00 (NA to NA) | 62.50 (14.19 to 89.31) | 0.00 (NA to NA) | 0 | 33.33 (5.26 to 66.38) | 62.50 (14.19 to 89.31) |
| 12 months | 100.0 (100.0 to 100.0) | 0.00 (NA to NA) | 41.67 (5.60 to 76.65) | 0.00 (NA to NA) | 0 | 33.33 (5.26 to 66.38) | 62.50 (14.19 to 89.31) |
Determine PFS by clinical or radiographic criteria, or death, whichever occurs first. Clinical progression was defined as treatment discontinuation due to disease progression by investigator assessment per PCWG3/mRECIST v1.1, or by PSA criteria. The data values are Kaplan-Meier estimates.
| months | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant |
|---|---|---|---|---|---|---|---|
| Time to Progression by Clinical or Radiographic Criteria | 18.46 (NA to NA) | 1.81 (0.82 to NA) | 6.21 (0.49 to NA) | 1.84 (0.99 to NA) | 0 | 3.68 (1.02 to NA) | NA (3.91 to NA) |
Assess the percentage of patients who are progression-free by clinical or radiographic measures at 4, 6, and 12 months. Values are Kaplan-Meier estimates of the patients progression free at the time points specified.
| Percentage of patients | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant |
|---|---|---|---|---|---|---|---|
| 4 months | 100.0 (100.0 to 100.0) | 0.00 (NA to NA) | 83.33 (27.31 to 97.47) | 44.44 (6.62 to 78.49) | 0 | 50.00 (18.36 to 75.32) | 83.33 (27.31 to 97.47) |
| 6 months | 100.0 (100.0 to 100.0) | 0.00 (NA to NA) | 62.50 (14.19 to 89.31) | 0.00 (NA to NA) | 0 | 33.33 (6.33 to 64.58) | 62.50 (14.19 to 89.31) |
| 12 months | 100.0 (100.0 to 100.0) | 0.00 (NA to NA) | 41.67 (5.60 to 76.65) | 0.00 (NA to NA) | 0 | 33.33 (6.33 to 64.58) | 62.50 (14.19 to 89.31) |
Determine PFS by clinical criteria or biochemical criteria (defined as treatment discontinuation due to clinical progression by investigator assessment, or by PSA criteria) PCWG3/mRECIST v1.1, or death whichever occurs first. The data values are Kaplan-Meier estimates.
| months | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant |
|---|---|---|---|---|---|---|---|
| Time to Progression by Clinical or Biochemical Criteria | 32.72 (NA to NA) | 5.85 (NA to NA) | 11.96 (3.68 to NA) | 3.55 (0.99 to NA) | 0 | 6.77 (1.41 to 16.53) | NA (4.86 to NA) |
Assess the percentage of patients who are progression-free by clinical or biochemical criteria (defined as treatment discontinuation due to clinical progression by investigator assessment or by PSA criteria) PCWG3/mRECIST v1.1, or death whichever occurs first at 4, 6, and 12 months. Values are Kaplan-Meier estimates of the patients progression free at the time points specified.
| Percentage of patients | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant |
|---|---|---|---|---|---|---|---|
| 4 months | 100.0 (100.0 to 100.0) | 100.0 (100.0 to 100.0) | 80.00 (20.38 to 96.92) | 41.67 (1.12 to 84.31) | 0 | 70.00 (32.87 to 89.19) | 100.0 (100.0 to 100.0) |
| 6 months | 100.0 (100.0 to 100.0) | 0.00 (NA to NA) | 60.00 (12.57 to 88.18) | NA (NA to NA) | 0 | 58.33 (22.98 to 82.07) | 75.00 (12.79 to 96.05) |
| 12 months | 100.0 (100.0 to 100.0) | 0.00 (NA to NA) | 40.00 (5.20 to 75.28) | NA (NA to NA) | 0 | 35.00 (8.48 to 63.98) | 75.00 (12.79 to 96.05) |
Determine the DOR as defined as the time from the first occurrence of a documented objective tumor response to the time of radiographic progression (per investigator using PCWG3/mRECIST v1.1 criteria) or death from any cause on study, whichever occurs first. DOR was calculated as (earliest date of progression, death, or censoring - date of first documented objective response +1)/30.4375. The data values are Kaplan-Meier estimates.
| months | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant |
|---|---|---|---|---|---|---|---|
| Duration of Response (DOR) | — | — | 10.97 | — | — | — | — |
Determine OS assessed as the time from the first dose of study drug (exicorilant and/or enzalutamide) to the date of death from any cause. The data values are Kaplan-Meier estimates.
| months | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant |
|---|---|---|---|---|---|---|---|
| Overall Survival (OS) | 32.72 (NA to NA) | NA (5.85 to NA) | 13.73 (4.63 to NA) | NA (3.55 to NA) | NA (NA to NA) | 13.96 (4.47 to NA) | 17.23 (10.02 to NA) |
Collected over Treatment-emergent adverse events were reported from randomization up to 27 months for Segment 1 and up to 19 months for Segment 2. All-cause mortality was reported from enrollment up to 52 months.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Enrolled But Not Allocated | 1/2 (50%) | — | — |
| Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | 1/2 (50%) | 1/2 (50%) | 2/2 (100%) |
| Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | 1/3 (33.3%) | 1/3 (33.3%) | 3/3 (100%) |
| Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | 5/7 (71.4%) | 2/7 (28.6%) | 7/7 (100%) |
| Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | 1/8 (12.5%) | 2/8 (25%) | 8/8 (100%) |
| Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | 0/1 (0%) | 0/1 (0%) | 1/1 (100%) |
| Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | 5/10 (50%) | 2/10 (20%) | 10/10 (100%) |
| Dose Determination Segment 2: Arm B - 240 mg Exicorilant | 4/6 (66.7%) | 0/6 (0%) | 6/6 (100%) |
| Event | Enrolled But Not Allocated | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant |
|---|---|---|---|---|---|---|---|---|
| FatigueGeneral disorders | — | 1/2 | 0/3 | 0/7 | 0/8 | 0/1 | 0/10 | 0/6 |
| Musculoskeletal painMusculoskeletal and connective tissue disorders | — | 1/2 | 0/3 | 0/7 | 0/8 | 0/1 | 0/10 | 0/6 |
| Pancreatitis acuteGastrointestinal disorders | — | 0/2 | 1/3 | 0/7 | 0/8 | 0/1 | 0/10 | 0/6 |
| Spinal fractureInjury, poisoning and procedural complications | — | 0/2 | 0/3 | 1/7 | 0/8 | 0/1 | 0/10 | 0/6 |
| Pleural effusionRespiratory, thoracic and mediastinal disorders | — | 0/2 | 0/3 | 1/7 | 0/8 | 0/1 | 0/10 | 0/6 |
| Back painMusculoskeletal and connective tissue disorders | — | 0/2 | 0/3 | 0/7 | 1/8 | 0/1 | 0/10 | 0/6 |
| Bone painMusculoskeletal and connective tissue disorders | — | 0/2 | 0/3 | 0/7 | 1/8 | 0/1 | 0/10 | 0/6 |
| Confusional statePsychiatric disorders | — | 0/2 | 0/3 | 0/7 | 1/8 | 0/1 | 0/10 | 0/6 |
| SepsisInfections and infestations | — | 0/2 | 0/3 | 0/7 | 0/8 | 0/1 | 1/10 | 0/6 |
| Urinary retentionRenal and urinary disorders | — | 0/2 | 0/3 | 0/7 | 0/8 | 0/1 | 1/10 | 0/6 |
| Event | Enrolled But Not Allocated | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant |
|---|---|---|---|---|---|---|---|---|
| AnaemiaBlood and lymphatic system disorders | — | 0/2 | 0/3 | 0/7 | 2/8 | 1/1 | 3/10 | 2/6 |
| Abdominal painGastrointestinal disorders | — | 1/2 | 1/3 | 1/7 | 1/8 | 1/1 | 0/10 | 3/6 |
| FlatulenceGastrointestinal disorders | — | 0/2 | 0/3 | 0/7 | 0/8 | 1/1 | 0/10 | 0/6 |
| NauseaGastrointestinal disorders | — | 0/2 | 1/3 | 1/7 | 1/8 | 1/1 | 2/10 | 1/6 |
| VomitingGastrointestinal disorders | — | 0/2 | 0/3 | 0/7 | 0/8 | 1/1 | 0/10 | 0/6 |
| FatigueGeneral disorders | — | 2/2 | 0/3 | 4/7 | 5/8 | 0/1 | 6/10 | 5/6 |
| Lipase increasedInvestigations | — | 0/2 | 0/3 | 0/7 | 3/8 | 1/1 | 0/10 | 4/6 |
| Lymphocyte count decreasedInvestigations | — | 0/2 | 0/3 | 0/7 | 0/8 | 1/1 | 1/10 | 2/6 |
| Back painMusculoskeletal and connective tissue disorders | — | 2/2 | 1/3 | 4/7 | 4/8 | 1/1 | 5/10 | 5/6 |
| Musculoskeletal painMusculoskeletal and connective tissue disorders | — | 2/2 | 0/3 | 0/7 | 3/8 | 0/1 | 0/10 | 0/6 |
The Safety Population included all patients who received ≥1 dose of exicorilant.
| Age, Continuous(years) | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant | Total |
|---|---|---|---|---|---|---|---|---|
| Median | 67.5 (59 to 76) | 60.0 (53 to 71) | 70.0 (58 to 80) | 72.0 (60 to 82) | 75.0 (75 to 75) | 64.0 (53 to 76) | 66.0 (55 to 74) | 70.0 (53 to 82) |
| Sex: Female, Male(Participants) | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant | Total |
|---|---|---|---|---|---|---|---|---|
| Female | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Male | 2 | 3 | 7 | 8 | 1 | 10 | 6 | 37 |
| Ethnicity (NIH/OMB)(Participants) | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant | Total |
|---|---|---|---|---|---|---|---|---|
| Hispanic or Latino | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Not Hispanic or Latino | 1 | 3 | 7 | 7 | 1 | 8 | 5 | 32 |
| Unknown or Not Reported | 1 | 0 | 0 | 1 | 0 | 2 | 1 | 5 |
| Race (NIH/OMB)(Participants) | Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant | Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant | Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant | Dose Determination Segment 2: Arm B - 240 mg Exicorilant | Total |
|---|---|---|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 | 0 | 0 | 1 | 1 | 2 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Black or African American | 0 | 1 | 0 | 1 | 0 | 2 | 0 | 4 |
| White | 2 | 2 | 7 | 4 | 1 | 5 | 5 | 26 |
| More than one race | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 | 3 | 0 | 2 | 0 | 5 |
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