NCT03437941

Brief Summary

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.

Trial Health

60
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
39

participants targeted

Target at P50-P75 for phase_1

Timeline
Completed

Started Feb 2018

Longer than P75 for phase_1

Geographic Reach
2 countries

9 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 16, 2018

Completed
1 month until next milestone

First Posted

Study publicly available on registry

February 19, 2018

Completed
1 day until next milestone

Study Start

First participant enrolled

February 20, 2018

Completed
3.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 13, 2021

Completed
1.2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

January 12, 2023

Completed
3.3 years until next milestone

Results Posted

Study results publicly available

May 15, 2026

Completed
Last Updated

May 15, 2026

Status Verified

April 1, 2026

Enrollment Period

3.6 years

First QC Date

January 16, 2018

Results QC Date

September 11, 2024

Last Update Submit

April 23, 2026

Conditions

Keywords

Prostate CancerCastration-Resistant Prostate CancerGlucocorticoid receptorAntagonistmCRPCenzalutamideandrogen receptor

Outcome Measures

Primary Outcomes (1)

  • 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%.

    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

Secondary Outcomes (30)

  • Number of Patients With One or More Treatment-Emergent Adverse Events

    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

    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

    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

    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

    Predose and at time intervals up to 24 hours postdose on Cycle 1 Day -1, Cycle 1 Day 1, and Cycle 2 Day 1

  • +25 more secondary outcomes

Study Arms (10)

Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg Exicorilant

EXPERIMENTAL

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: ExicorilantDrug: Enzalutamide

Dose Determination Segment 1 (Open-label) Cohort 2 - 280 mg Exicorilant

EXPERIMENTAL

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: ExicorilantDrug: Enzalutamide

Dose Determination Segment 1 (Open-label) Cohort 3 - 280 mg Exicorilant

EXPERIMENTAL

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: ExicorilantDrug: Enzalutamide

Dose Determination Segment 2: Arm A - Maximum Dose 240 mg Exicorilant

EXPERIMENTAL

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: ExicorilantDrug: Enzalutamide

Dose Determination Segment 2: Arm A - Maximum Dose 280 mg Exicorilant

EXPERIMENTAL

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: ExicorilantDrug: Enzalutamide

Dose Determination Segment 2: Arm A - Maximum Dose 320 mg Exicorilant

EXPERIMENTAL

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: ExicorilantDrug: Enzalutamide

Dose Determination Segment 2: Arm B - 240 mg Exicorilant

EXPERIMENTAL

Patients will receive combination treatment with once-daily exicorilant 240 mg, enzalutamide once daily, and placebo in 28-day dosing cycles.

Drug: ExicorilantDrug: EnzalutamideDrug: Placebo

Dose Expansion - Abi-Resistant Cohort (Open-label)

EXPERIMENTAL

Patients who have progressed during treatment with abiraterone and no other androgen receptor-blocking therapies will receive exicorilant and enzalutamide.

Drug: ExicorilantDrug: Enzalutamide

Dose Expansion - Abi-Resistant Cohort Food Effect (Open-label)

EXPERIMENTAL

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: ExicorilantDrug: Enzalutamide

Dose Expansion - ARant-Resistant Cohort (Open-label)

EXPERIMENTAL

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: ExicorilantDrug: Enzalutamide

Interventions

Placebo capsules to match the appearance of the exicorilant capsules

Dose Determination Segment 2: Arm B - 240 mg Exicorilant

Exicorilant is supplied as capsules for oral dosing

Also known as: CORT125281
Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg ExicorilantDose Determination Segment 1 (Open-label) Cohort 2 - 280 mg ExicorilantDose Determination Segment 1 (Open-label) Cohort 3 - 280 mg ExicorilantDose Determination Segment 2: Arm A - Maximum Dose 240 mg ExicorilantDose Determination Segment 2: Arm A - Maximum Dose 280 mg ExicorilantDose Determination Segment 2: Arm A - Maximum Dose 320 mg ExicorilantDose Determination Segment 2: Arm B - 240 mg ExicorilantDose Expansion - ARant-Resistant Cohort (Open-label)Dose Expansion - Abi-Resistant Cohort (Open-label)Dose Expansion - Abi-Resistant Cohort Food Effect (Open-label)

Enzalutamide will be taken orally

Also known as: Xtandi
Dose Determination Segment 1 (Open-label) Cohort 1 - 360 mg ExicorilantDose Determination Segment 1 (Open-label) Cohort 2 - 280 mg ExicorilantDose Determination Segment 1 (Open-label) Cohort 3 - 280 mg ExicorilantDose Determination Segment 2: Arm A - Maximum Dose 240 mg ExicorilantDose Determination Segment 2: Arm A - Maximum Dose 280 mg ExicorilantDose Determination Segment 2: Arm A - Maximum Dose 320 mg ExicorilantDose Determination Segment 2: Arm B - 240 mg ExicorilantDose Expansion - ARant-Resistant Cohort (Open-label)Dose Expansion - Abi-Resistant Cohort (Open-label)Dose Expansion - Abi-Resistant Cohort Food Effect (Open-label)

Eligibility Criteria

Age18 Years+
Sexmale
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Able to understand the purpose and risks of the study; willing and able to adhere to scheduled visits, treatment plans, laboratory tests, and other study evaluations and procedures, and provide written informed consent
  • Males ≥18 years of age at the time of signing consent
  • Histologically confirmed adenocarcinoma of the prostate with metastatic disease
  • Dose-Determination Phase Segment 1 and Expansion Phase: Progressive disease as defined by prostate-specific antigen (PSA) or imaging after most recent prior therapy. PSA ≥1 ng/mL, if a confirmed rise in PSA is the only indication of progression. Progression by PSA requires rising PSA over a previous reference value by at least 2 measurements obtained ≥1 week apart. PSA measurements can be collected during or after the most recent prior therapy.
  • Dose-Determination Phase Segment 2: Currently receiving enzalutamide with a rising PSA as follows:
  • Rising PSA: 25% increase over nadir and an absolute value of \>1 ng/mL by at least 2 measurements obtained ≥1 week apart. PSA measurements can be collected during or after the most recent prior therapy.
  • Patients must have received enzalutamide for a minimum of 12 weeks and have been on stable doses of enzalutamide ≥80 mg once daily for at least 4 weeks prior to Cycle 1 Day 1. Patients will continue enzalutamide without interruption during the Screening Period (no wash-out period). This will be the enzalutamide starting dose for combination with exicorilant beginning on Cycle 1 Day 1.
  • M0 disease is allowed
  • Expansion Phase: Patients must have progressed while receiving an androgen-directed therapy as follows:
  • Abiraterone-Resistant Cohort: Patients must have progressed during treatment with abiraterone
  • Androgen Receptor Antagonist-Resistant Cohort: Patients must have progressed during treatment with enzalutamide or second-generation AR-blocking therapies. Patients progressing on enzalutamide immediately prior to enrolling in this study must be on stable doses of enzalutamide. These patients will continue enzalutamide without interruption during the Screening Period (no wash-out period required).
  • Baseline tumor assessment performed within 28 days prior to the first dose of study treatment (exicorilant and/or on-study enzalutamide, whichever is earliest).
  • Prior surgical or chemical castration with serum testosterone \<1.7 nmol/L (50 ng/dL). If the method of castration is use of a luteinizing hormone-releasing hormone (LHRH) analogue, there must be a plan to maintain effective LHRH analogue treatment for the duration of the trial.
  • Consent to have all protocol-required pharmacodynamic biomarker samples, including the pretreatment and on treatment paired tumor biopsies (mandatory for a subset of patients)
  • Consent to provide mandatory pharmacogenomic blood sample (Dose-Determination Segment 1 only)
  • +4 more criteria

You may not qualify if:

  • Received chemotherapy, non-palliative radiotherapy, immunotherapy, or any investigational cancer therapies within 21 days prior to the first dose of exicorilant, or treatment with such therapies is planned during protocol treatment. Concomitant anticancer therapy is not permitted during the enzalutamide Lead-In Period during Dose-Determination Phase Segment 1.
  • More than 2 prior cytotoxic chemotherapy regimens for the treatment of mCRPC
  • Dose Determination Phase and Expansion Phases will exclude patients for the following:
  • Dose-Determination Phase (Segment 1 only):
  • Progressed during treatment with enzalutamide prior to Cycle 1 Day -28 (only applies to patients receiving enzalutamide Lead-In) or
  • Received prior second-generation anti-androgen and require urgent disease response or stabilization
  • Expansion Phase Abi-Resistant Cohort:
  • Received prior treatment with enzalutamide, or
  • Received prior second-generation anti-androgen and require urgent disease response or stabilization
  • Expansion Phase ARant-Resistant Cohort: Require urgent disease response or stabilization
  • Ongoing or anticipated therapy with hormone therapy (other than LHRH analogue), including any dose of megestrol acetate (Megace), finasteride (Proscar), dutasteride (Avodart) or received abiraterone within 28 days prior to the first dose of exicorilant
  • Contraindication or precaution for enzalutamide
  • Parenchymal brain metastases
  • Any clinically significant uncontrolled condition that may increase the risk to the study patient or that the Investigator considers places the patient at unacceptable risk
  • Received herbal products or alternative therapies that may decrease PSA levels or that may have hormonal anti-prostate cancer activity within 21 days of study treatment initiation or plans to initiate treatment with these products/alternative therapies during the entire duration of the study
  • +2 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (9)

Scottsdale

Scottsdale, Arizona, 85258, United States

Location

Detroit

Detroit, Michigan, 48201, United States

Location

Basking Ridge

Basking Ridge, New Jersey, 07920, United States

Location

New York

New York, New York, 10065, United States

Location

Portland

Portland, Oregon, 97239, United States

Location

Madison

Madison, Wisconsin, 53792, United States

Location

London

London, England, W1T7HA, United Kingdom

Location

Southampton

Southampton, England, SO16 6YD, United Kingdom

Location

Sutton

Sutton, Surrey, SM2 5PT, United Kingdom

Location

Related Publications (1)

  • Serritella AV, Shevrin D, Heath EI, Wade JL, Martinez E, Anderson A, Schonhoft J, Chu YL, Karrison T, Stadler WM, Szmulewitz RZ. Phase I/II Trial of Enzalutamide and Mifepristone, a Glucocorticoid Receptor Antagonist, for Metastatic Castration-Resistant Prostate Cancer. Clin Cancer Res. 2022 Apr 14;28(8):1549-1559. doi: 10.1158/1078-0432.CCR-21-4049.

MeSH Terms

Conditions

Prostatic NeoplasmsBulbo-Spinal Atrophy, X-Linked

Interventions

enzalutamide

Condition Hierarchy (Ancestors)

Genital Neoplasms, MaleUrogenital NeoplasmsNeoplasms by SiteNeoplasmsGenital Diseases, MaleGenital DiseasesUrogenital DiseasesProstatic DiseasesMale Urogenital DiseasesMuscular Atrophy, SpinalSpinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesMotor Neuron DiseaseNeuromuscular DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

Title
Medical Director
Organization
Corcept Therapeutics

Study Officials

  • William Guyer, PharmD

    Corcept Therapeutics

    STUDY DIRECTOR
  • Grace Mann, PhD

    Corcept Therapeutics

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 16, 2018

First Posted

February 19, 2018

Study Start

February 20, 2018

Primary Completion

October 13, 2021

Study Completion

January 12, 2023

Last Updated

May 15, 2026

Results First Posted

May 15, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will not share

Locations