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Prostate CancerPhase 1/2NCT01171898

Safety, Pharmacokinetic and Proof-of-Concept Study of ARN-509 (Apalutamide) in Castration-Resistant Prostate Cancer (CRPC)

A Phase 1/2 study testing ARN-509 (Phase 1) and ARN-509 (Phase 2) in people with Prostate Cancer, run by Aragon Pharmaceuticals, Inc. at 15 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The purpose of this study is to assess the safety and activity of ARN-509 in men with advanced castration resistant prostate cancer. Patients will first be enrolled into Phase 1 of the study to identify a tolerable dose for the Phase 2 portion of the study. Read more about this study

Who is it for?
Ages 18 years and older, men only
Conditions the study is for
Prostate Cancer
What is being tested?
ARN-509 (Phase 1) and ARN-509 (Phase 2)
Could I receive a placebo?
No, everyone receives an active treatment
How many people
127 participants
Where
15 sitesUnited States
Study dates
26 Jul 2010to 22 Jul 2025
Phase
Phase 1/2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • Men only
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Histologically or cytologically proven prostate cancer with high risk for development of metastases, defined as either a PSA value >=8 ng/mL within the last 3 months or PSA Doubling Time <=10 months
  • Ongoing androgen depletion therapy with a Gonadotropin Releasing Hormone (GnRH) analogue or inhibitor, or orchiectomy (i.e., surgical or medical castration)
  • Castrate levels of serum testosterone of less than or equal to 50 ng/dL
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0-1
  • A life expectancy of at least 3 months
  • Histologically or cytologically proven prostate cancer with progressive disease based on either PSA or radiographic progression
Show all 16Show fewer
  • Ongoing androgen depletion therapy with a Gonadotropin Releasing Hormone (GnRH) analogue or inhibitor, or orchiectomy (i.e., surgical or medical castration)
  • Castrate levels of serum testosterone of less than or equal to 50 ng/dL
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0-1
  • A life expectancy of at least 3 months
  • Histologically or cytologically proven prostate cancer with progressive disease based on either PSA or radiographic progression
  • Ongoing androgen depletion therapy with a Gonadotropin Releasing Hormone (GnRH) analogue or inhibitor, or orchiectomy (i.e., surgical or medical castration)
  • Castrate levels of serum testosterone of less than or equal to 50 ng/dL
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0-1
  • A life expectancy of at least 3 months
  • Patients must have received a minimum of 6 months of abiraterone treatment prior to disease progression

Who cannot join

  • Distant metastases, including CNS and vertebral or meningeal involvement
  • Prior treatment with MDV3100
  • Prior treatment with abiraterone
  • Prior treatment with ketoconazole
  • Concurrent treatment with medications known to have seizure potential
  • Concurrent treatment with corticosteroids. If they are already on steroids, patients will be allowed to enroll on the study but will need to taper off as soon as possible.
Show all 28Show fewer
  • QTc > 450 msec
  • History of seizure or condition that may predispose to seizure
  • Evidence of severe or uncontrolled systemic disease or HIV infection
  • METASTATIC CRPC, TREATMENT-NAIVE
  • History of, or current metastases in the brain or untreated spinal cord compression
  • Prior treatment with MDV3100
  • Prior treatment with abiraterone
  • Prior treatment with ketoconazole
  • Concurrent treatment with medications known to have seizure potential
  • Concurrent treatment with corticosteroids. If they are already on steroids, patients will be allowed to enroll on the study but will need to taper off as soon as possible.
  • QTc > 450 msec
  • History of seizure or condition that may predispose to seizure
  • Evidence of severe or uncontrolled systemic disease or HIV infection
  • METASTATIC CRPC, CHEMOTHERAPY-NAIVE, POST-ABIRATERONE
  • History of, or current metastases in the brain or untreated spinal cord compression
  • Prior treatment with MDV3100
  • Prior treatment with ketoconazole
  • Concurrent treatment with medications known to have seizure potential
  • Concurrent treatment with corticosteroids. If they are already on steroids, patients will be allowed to enroll on the study but will need to taper off as soon as possible.
  • QTc > 450 msec
  • History of seizure or condition that may predispose to seizure
  • Evidence of severe or uncontrolled systemic disease or HIV infection

Trial locations and contacts

15 sites in 1 country.

15 sites in 1 country

United States15 sites

  • Study site

    Ann Arbor, Michigan

  • Study site

    Atlanta, Georgia

  • Study site

    Baltimore, Maryland

  • Study site

    Boston, Massachusetts

  • Study site

    Dallas, Texas

  • Study site

    Lancaster, Pennsylvania

  • Study site

    Madison, Wisconsin

  • Study site

    Myrtle Beach, South Carolina

  • Study site

    New York, New York

  • Study site

    Omaha, Nebraska

  • Study site

    Portland, Oregon

  • Study site

    Raleigh, North Carolina

  • Study site

    San Diego, California

  • Study site

    San Francisco, California

  • Study site

    Seattle, Washington

Other trials in United States

About this study

Official title: An Open-Label, Phase 1/2, Safety, Pharmacokinetic and Proof-of-Concept Study of ARN-509 in Patients With Progressive Advanced Castration-Resistant Prostate Cancer

The purpose of this study is to assess the safety and activity of ARN-509 in men with advanced castration resistant prostate cancer. Patients will first be enrolled into Phase 1 of the study to identify a tolerable dose for the Phase 2 portion of the study. In the Phase 2, 3 different cohorts of patients will be enrolled to evaluate the safety and activity of ARN-509.

Conditions
Prostate Cancer
Being tested
ARN-509 (Phase 1), ARN-509 (Phase 2)
Keywords
Non-Metastatic, Rising PSA, Castration-Resistant, Treatment-Naive, Post-abiraterone
Medical subject headings
Prostatic Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ARN-509 (Phase 1)Drug

    ARN-509 will be administered at a starting dose of 30 milligram per day (mg/day), with escalations to 60 mg, 90 mg, 120 mg, 180 mg, 240 mg, 300 mg, 390 mg, and 480 mg daily.

  • ARN-509 (Phase 2)Drug

    ARN-509 will be administered at Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D), determined in Phase 1.

Study groups in NCT01171898
Study groupRoleWhat they receive
Dose Escalation Cohort (Phase 1)ExperimentalARN-509 will be administered at a starting dose of 30 milligram per day (mg/day), with escalations to 60 mg, 90 mg, 120 mg, 180 mg, 240 mg, 300 mg, 390 mg, and 480 mg daily. Once Recommended Phase 2 Dose (RP2D) has been selected, Phase 1 participants being treated at the lower dose levels will be allowed to escalate to the RP2D level at the discretion of the primary investigator.
Receives: Drug: ARN-509 (Phase 1)
Non-metastatic CRPC (Phase 2)ExperimentalParticipants with non-metastatic, treatment-naive Castration-Resistant Prostate Cancer (CRPC) with rapidly rising Prostate Specific Antigen (PSA) will be enrolled. ARN-509 will be administered at Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D), determined in Phase 1.
Receives: Drug: ARN-509 (Phase 2)
Treatment-naive metastatic CRPC (Phase 2)ExperimentalParticipants with treatment-naive metastatic CRPC will be enrolled. ARN-509 will be administered at MTD and/or RP2D, determined in Phase 1.
Receives: Drug: ARN-509 (Phase 2)
Post-abiraterone metastatic CRPC (Phase 2)ExperimentalParticipants with metastatic CRPC that are chemotherapy-naive, but have been previously treated with abiraterone will be enrolled. ARN-509 will be administered at MTD and/or RP2D, determined in Phase 1.
Receives: Drug: ARN-509 (Phase 2)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Phase 1 and 2: Percentage of Participants With Greater Than or Equal to (>=) 50 Percent (%) Reduction in Prostate-Specific Antigen (PSA) at Week 12

    Percentage of participants with >=50% decrease in PSA compared to baseline were assessed at Week 12. PSA progression was defined by the protocol-specific Prostate Cancer Working Group 2 (PCWG2) criteria: PSA increase greater than or equal to [>=] 25 percent [%] and >=2 nanogram per milliliter [ng/mL] above the nadir confirmed >=3 weeks later; or >=25% and >=2 ng/mL above baseline PSA after 12 weeks.

    Measured: Week 12

Secondary outcomes (4)

  1. Phase 1 and 2: Median Time to PSA Progression

    Time to PSA progression was measured as the time interval from the date of the first dose to the date of PSA progression as defined by the PCWG2 criteria. PCWG2 criteria: For participants who achieved >=50% decrease from the baseline PSA, assessment of time to disease progression was when the PSA increased 25% and at a minimum of 2 ng/mL above the nadir at 3 or more weeks later. For participants without a PSA decrease, the time for progression was calculated at the time when the PSA progression was >=25% and >=2 ng/mL after 12 weeks.

    Measured: Up to approximately 7 years

  2. Phase 2: Median Metastasis-Free Survival (MFS)

    MFS was defined as the time from the start of treatment until new metastatic lesions were observed on Computed Tomography/ Magnetic Resonance Imaging (CT/MRI) scans or radionuclide bone scans (according to PCWG2 criteria: appearance of >=2 new lesions, and, for the first reassessment only, a confirmatory scan performed 6 or more weeks later that showed at least 2 or more additional new lesions) or death, whichever occurred first.

    Measured: Up to approximately 7 years

  3. Phase 1 and 2: Progression-free Survival (PFS)

    PFS was defined as the time from randomization to the radiographic disease progression or death, whichever occurred first. Radiographic progression defined by at least one of the following: a) Soft tissue progression by modified RECIST confirmed on repeat imaging >= 6 weeks later; b) Progression by bone scans: 1) first bone scan with >= 2 new lesions compared to baseline observed <12 weeks from start date and confirmed on a second bone scan >=6 weeks later that showed >=2 additional lesions (a total of >=4 new lesions compared to baseline); or 2) first bone scan with >=2 new lesions compared to baseline observed >=12 weeks from start date and the new lesions verified on the next bone scan >=6 weeks later (a total of >=2 new lesions compared to baseline).

    Measured: Up to approximately 7 years

  4. Phase 1 and 2: Objective Response Rate

    Objective Response Rate was defined as the percentage of participants with best overall response (OR) of confirmed complete response (CR) or partial response (PR) according to Response Evaluation Criteria in Solid Tumors, Version 1.0 (RECIST). Where, CR defined as disappearance of all target lesions. Any pathological lymph nodes must had reduction in short axis to less than 10 millimeter (mm). PR defined as at least 30 percent (%) decrease in sum of the diameters of the target lesions. Confirmed responses were those that persisted on repeat imaging study for at least 4 weeks after initial documentation of response.

    Measured: Up to approximately 7 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted29 Jul 2010 (estimated)
  2. Study start26 Jul 2010
  3. Primary completion20 Aug 2012
  4. Study completion22 Jul 2025

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
127 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01171898
Sponsor's protocol ID
CR103304
Other IDs
ARN-509-001
Status last verified
Jun 2026
First submitted
27 Jul 2010
Last updated
5 Jun 2026
Data monitoring committee
No

Publications

  • Rathkopf DE, Smith MR, Ryan CJ, Berry WR, Shore ND, Liu G, Higano CS, Alumkal JJ, Hauke R, Tutrone RF, Saleh M, Chow Maneval E, Thomas S, Ricci DS, Yu MK, de Boer CJ, Trinh A, Kheoh T, Bandekar R, Scher HI, Antonarakis ES. Androgen receptor mutations in patients with castration-resistant prostate cancer treated with apalutamide. Ann Oncol. 2017 Sep 1;28(9):2264-2271. doi: 10.1093/annonc/mdx283.

    PubMed 28633425

  • Smith MR, Antonarakis ES, Ryan CJ, Berry WR, Shore ND, Liu G, Alumkal JJ, Higano CS, Chow Maneval E, Bandekar R, de Boer CJ, Yu MK, Rathkopf DE. Phase 2 Study of the Safety and Antitumor Activity of Apalutamide (ARN-509), a Potent Androgen Receptor Antagonist, in the High-risk Nonmetastatic Castration-resistant Prostate Cancer Cohort. Eur Urol. 2016 Dec;70(6):963-970. doi: 10.1016/j.eururo.2016.04.023. Epub 2016 May 6.

    PubMed 27160947

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing ARN-509 (Phase 1) and ARN-509 (Phase 2) in people with Prostate Cancer, run by Aragon Pharmaceuticals, Inc. at 15 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 16 inclusion and 28 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 5 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 15 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 27 Jul 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Phase 1 and 2: Percentage of Participants With Greater Than or Equal to (>=) 50 Percent (%) Reduction in Prostate-Specific Antigen (PSA) at Week 12 (time frame: Week 12). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01171898, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jun 2026.

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