Triple Negative Breast CancerPhase 2NCT02368691

Efficacy and Safety of GTx-024 in Patients With Androgen Receptor-Positive Triple Negative Breast Cancer (AR+ TNBC)

A Phase 2 study testing GTx-024 in people with Triple Negative Breast Cancer, run by GTx at 7 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Trial summary

The purpose of this study is to determine if GTx-024 is effective and safe in the treatment of patients with advanced, androgen receptor positive triple negative breast cancer (AR+ TNBC). Read more about this study

Who is it for?: Ages 18 years and older, women only
Conditions the study is for: Triple Negative Breast Cancer
What is being tested?: GTx-024
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 32 participants
Where: 7 sitesUnited States
Study dates: Jun 2015to 22 Sep 2017
Phase: Phase 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
Women only
No healthy volunteers
Adult, Older adult

Who may be able to join

Able and willing to give voluntary, written and signed, informed consent
Women ≥ 18 years of age
Women with TNBC who have received at least one but no more than two prior chemotherapy regimens for TNBC
Confirmation of AR+ (defined as ≥ 10% nuclear AR staining by immunohistochemistry [IHC]) TNBC in either the primary or metastatic lesion, assessed during the screening period by a local laboratory or by medical history
TNBC confirmed by medical history as: human epidermal growth factor receptor 2 [HER2]-negative (confirmed by IHC 0, 1+ regardless of fluorescence in situ hybridization [FISH] ratio; IHC 2+ with FISH ratio lower than 2.0 or HER2 gene copy less than 6.0; FISH ratio of 0, indicating gene deletion, when positive and negative in situ hybridization [ISH] controls are present); estrogen receptor (ER) negative (confirmed as ER expression less than or equal to 1% positive tumor nuclei); progesterone receptor negative (confirmed as progesterone receptor expression less than or equal to 1% positive tumor nuclei)
Availability of paraffin embedded or formalin fixed tumor tissue; OR, a minimum of 10 and up to 20 slides of archived tumor tissue for central laboratory confirmation of AR status and molecular subtyping. Metastatic tumor tissue is preferred when possible

Show all 22Show fewer

Subjects must have either measurable disease or bone-only non-measurable disease, evaluable according to RECIST 1.1
Subjects with bone metastases should be treated with intravenous bisphosphonates or subcutaneous denosumab (or investigator preferred standard of care) prior to and during the trial, unless there is a contraindication or subject intolerance to these therapies
Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1 at the time of screening and enrollment
Negative pregnancy test in women of childbearing potential (premenopausal or less than 12 months of amenorrhea post-menopause, and who have not undergone surgical sterilization), no more than 7 days before the first dose of study treatment
For women of childbearing potential who are sexually active, agreement to use a highly effective, non-hormonal form of contraception during and for at least 6 months after completion of study treatment; OR, a fertile male partner willing and able to use effective non-hormonal of contraception (barrier method of contraception in conjunction with spermicidal jelly, or surgical sterilization) during and for at least 6 months after completion of study treatment
Adequate organ function as shown by:
Absolute neutrophil count ≥ 1,000 cells/mm3
Platelet count ≥ 100,000 cells/mm3
Hemoglobin ≥ 9 g/dL
Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 Upper Limit of the Normal range (ULN) (or ≤ 5 if hepatic metastases are present)
Total serum bilirubin ≤ 2.0 × ULN (unless the subject has documented Gilbert Syndrome)
Alkaline phosphatase levels ≤ 2.5 × ULN (≤ 5 × ULN in subjects with liver metastasis)
Serum creatinine < 2.0 mg/dL or 177 μmol/L
International normalized ratio (INR), activated partial thromboplastin time (aPTT), or partial thromboplastin time (PTT) < 1.5 × ULN (unless on anticoagulant treatment at screening)
Able to swallow capsules
Any toxicity from prior chemotherapy has resolved or Grade 1 (NCI-CTCAE, Version 4.0)

Who cannot join

Life expectancy < 4 months;
Subjects with radiographic evidence of central nervous system (CNS) metastases as assessed by computerized tomography (CT) or magnetic resonance imaging (MRI) that are not well controlled (symptomatic or requiring control with continuous corticosteroid therapy [e.g., dexamethasone]). Note: Subjects with CNS metastases are permitted to participate in the study if the CNS metastases are medically well controlled prior to screening (as assessed by the Investigator) after receiving local therapy (irradiation, surgery, etc.)
Radiotherapy within 14 days prior to first dose of study treatment
Have, in the judgment of the Investigator, a clinically significant concurrent illness or psychological, familial, sociological, geographical, or other concomitant condition that would not permit adequate follow-up and compliance with the study protocol
Positive hepatitis B virus (HBV) and/or hepatitis C virus (HCV) infection at screening
Positive human immunodeficiency virus (HIV) infection at screening

Show all 22Show fewer

Prior treatment with any anti-androgens, including but not limited to, enzalutamide and bicalutamide
Major surgery within 28 days of the first dose of study treatment
Be currently taking or have previously taken testosterone, methyltestosterone, oxandrolone (Oxandrin®), oxymetholone, danazol, fluoxymesterone (Halotestin®), testosterone-like agents (such as dehydroepiandrosterone, androstenedione, and other androgenic compounds, including herbals), or anti-androgens
Treatment with any of the following hormone replacement therapies, unless discontinued at least 14 days prior to the first dose of study treatment:
Estrogens
Megesterol acetate
Treatment with any investigational agent within 28 days before the first dose of study treatment
Another active cancer (excluding adequately treated basal cell carcinoma or cervical intraepithelial neoplasia [CIN]/cervical carcinoma in situ or melanoma in situ). Prior history of other cancer is allowed as long as there is no active disease within the prior 5 years
Subject has a concomitant medical condition that precludes adequate study treatment compliance or assessment, or increases subject risk, in the opinion of the Investigator, such as but not limited to:
Myocardial infarction or arterial thromboembolic events within 6 months prior to baseline or severe or unstable angina, New York Heart Association (NYHA) Class III or IV disease, or a QTcB (corrected according to Bazett's formula) interval > 470 msec; serious uncontrolled cardiac arrhythmia grade II or higher according to NYHA; uncontrolled hypertension (systolic > 150 and/or diastolic > 100 mm Hg)
Acute and chronic active infectious disorders and non-malignant medical illnesses that are uncontrolled or whose control may be jeopardized by the complications of this study therapy
Impairment of gastrointestinal function or gastrointestinal disease that may significantly alter the absorption of study drugs (e.g., ulcerative disease, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome)
Current treatment with intravenous bisphosphonate or denosumab with elevated serum calcium corrected for albumin or ionized calcium levels outside institutional normal limits at screening
History of non-compliance to medical regimens
Subjects unwilling to or unable to comply with the protocol procedures as assessed by the Investigator
Concurrent participation in another therapeutic clinical trial

Trial locations and contacts

7 sites in 1 country.

City, postcode or hospital

7 sites in 1 country

United States7 sites

St. Vincent Frontier Cancer Center
Billings, Montana, 59102
Holy Cross Hospital
Fort Lauderdale, Florida, 33308
US Oncology / Texas Oncology, P.A.
Houston, Texas, 77024
Lakeland Regional Health Care/Cancer Center
Lakeland, Florida, 33805
The West Clinic, PC
Memphis, Tennessee, 38120
University of Miami Sylvester Comprehensive Cancer Center
Miami, Florida, 33136
H. Lee Moffitt Cancer Center and Research Institute
Tampa, Florida, 33612

Other trials in United States

About this study

Official title: A Phase 2 Open Label, Multi-Center, Multinational Study Investigating The Efficacy and Safety Of GTx-024 On Advanced, Androgen Receptor-Positive Triple Negative Breast Cancer (AR+ TNBC)

The purpose of this study is to determine if GTx-024 is effective and safe in the treatment of patients with advanced, androgen receptor positive triple negative breast cancer (AR+ TNBC).

Conditions: Triple Negative Breast Cancer
Being tested: GTx-024
Medical subject headings: Triple Negative Breast Neoplasms

What participants receive

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

GTx-024Drug
Also called Enobosarm, Ostarine, S-22
GTx-024 softgel capsules will be administered once-daily to a total dose of 18 mg

Study groups in NCT02368691
Study group	Role	What they receive
GTx-024	Experimental	GTx-024 capsules, 18 mg PO once-daily for up to 12 months Receives: Drug: GTx-024

What is being measured

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

Clinical Benefit Rate, in Centrally Confirmed Androgen Receptor Positive (AR+) Subjects
To estimate the clinical benefit rate (defined as complete response, partial response, or stable disease) according to Response Evaluation Criteria in Solid Tumors v 1.1 (RECIST) as assessed by CT or MRI in subjects with centrally confirmed AR+ status. Clinical Benefit Rate=CR+PR+SD. Complete Response (CR), disappearance of all target lesions; Partial Response (PR), >=30% decrease in the sum of the longest diameter of target lesions; Stable Disease, <30% decrease in sum of the longest diameter of target lesions.
Measured: Sixteen (16) weeks

Secondary outcomes (6)

Clinical Benefit Rate, in Full Analysis Set
To estimate the clinical benefit rate in all subjects who receive at least one dose of study medication (full analysis set), regardless of central confirmation of AR status.
Measured: Sixteen (16) weeks
Best Overall Response
To assess best overall response as measured by RECIST 1.1 from the start of study treatment until the end of treatment taking into account any requirement for confirmation. Best overall response is reported as the best response (CR, PR or SD) by CT or MRI up through 11 months of treatment, median duration of treatment 1.9 months.
Measured: From treatment initiation to end of treatment (up through 11 months of treatment, median duration of treatment 1.9 months).
Progression Free Survival
To assess progression free survival (PFS) defined as the time elapsed between initiation of treatment and tumor progression as measured by RECIST 1.1 or death.
Measured: From treatment initiation to tumor progression or death. PFS was assessed up to 11 months of treatment; median duration of treatment, 1.9 months
Time-to-progression
To assess time to progression defined as time elapsed between treatment initiation and tumor progression as measured by RECIST 1.1 or death due to disease progression.
Measured: From treatment initiation to tumor progression or death. Time to progression was assessed up to 11 months of treatment; median duration of treatment, 1.9 months
Duration of Response
To assess the duration of response defined as the time from documentation of tumor response to disease progression or death
Measured: From time of documented tumor response to tumor progression or death. Duration of response was assessed through 11 months; median treatment duration, 1.9 months
Objective Response Rate
To estimate the objective response rate (defined as complete response or partial response) according to RECIST 1.1.
Measured: Sixteen (16) weeks

Timeline

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

Jun 201522 Sep 2017

First posted23 Feb 2015 (estimated)
Study startJun 2015
Primary completion22 Sep 2017
Study completion22 Sep 2017

Why it stopped: Lack of Efficacy

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Triple Negative Breast CancerStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

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 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 32 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02368691
Sponsor's protocol ID: G200901
Status last verified: Oct 2020
First submitted: 10 Feb 2015
Last updated: 18 Nov 2020
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Study at a glance

From the registry record, last updated by the sponsor on 18 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2 Open Label, Multi-Center, Multinational Study Investigating The Efficacy and Safety Of GTx-024 On Advanced, Androgen Receptor-Positive Triple Negative Breast Cancer (AR+ TNBC)
ClinicalTrials.gov ID: NCT02368691
Acronym / sponsor ID: G200901
Conditions: Triple Negative Breast Cancer
Phase: Phase 2
Study type: Interventional
Recruitment status: TerminatedReason stopped: Lack of Efficacy
Enrolment: 32 participants (actual)
Lead sponsor: GTxIndustry (pharma, biotech, devices)
Study start: Jun 2015 (actual)
Primary completion: 22 Sep 2017 (actual)
Study completion: 22 Sep 2017 (actual)
First posted: 23 Feb 2015 (estimated)
Results: Posted 30 Oct 2020

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

The purpose of this study is to determine if GTx-024 is effective and safe in the treatment of patients with advanced, androgen receptor positive triple negative breast cancer (AR+ TNBC).

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
GTx-024	Experimental	Drug: GTx-024GTx-024 capsules, 18 mg PO once-daily for up to 12 months

Who may qualify

Ages: 18 years and older
Sex: Female only
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (22 listed)

Able and willing to give voluntary, written and signed, informed consent
Women ≥ 18 years of age
Women with TNBC who have received at least one but no more than two prior chemotherapy regimens for TNBC
Confirmation of AR+ (defined as ≥ 10% nuclear AR staining by immunohistochemistry [IHC]) TNBC in either the primary or metastatic lesion, assessed during the screening period by a local laboratory or by medical history
TNBC confirmed by medical history as: human epidermal growth factor receptor 2 [HER2]-negative (confirmed by IHC 0, 1+ regardless of fluorescence in situ hybridization [FISH] ratio; IHC 2+ with FISH ratio lower than 2.0 or HER2 gene copy less than 6.0; FISH ratio of 0, indicating gene deletion, when positive and negative in situ hybridization [ISH] controls are present); estrogen receptor (ER) negative (confirmed as ER expression less than or equal to 1% positive tumor nuclei); progesterone receptor negative (confirmed as progesterone receptor expression less than or equal to 1% positive tumor nuclei)

Key exclusion criteria (22 listed)

Life expectancy < 4 months;
Subjects with radiographic evidence of central nervous system (CNS) metastases as assessed by computerized tomography (CT) or magnetic resonance imaging (MRI) that are not well controlled (symptomatic or requiring control with continuous corticosteroid therapy [e.g., dexamethasone]). Note: Subjects with CNS metastases are permitted to participate in the study if the CNS metastases are medically well controlled prior to screening (as assessed by the Investigator) after receiving local therapy (irradiation, surgery, etc.)
Radiotherapy within 14 days prior to first dose of study treatment
Have, in the judgment of the Investigator, a clinically significant concurrent illness or psychological, familial, sociological, geographical, or other concomitant condition that would not permit adequate follow-up and compliance with the study protocol
Positive hepatitis B virus (HBV) and/or hepatitis C virus (HCV) infection at screening

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
GTx-024Also: Enobosarm, Ostarine, S-22GTx-024 softgel capsules will be administered once-daily to a total dose of 18 mg	Drug	GTx-024

Primary outcome

Clinical Benefit Rate, in Centrally Confirmed Androgen Receptor Positive (AR+) Subjects
Time frame: Sixteen (16) weeks
To estimate the clinical benefit rate (defined as complete response, partial response, or stable disease) according to Response Evaluation Criteria in Solid Tumors v 1.1 (RECIST) as assessed by CT or MRI in subjects with centrally confirmed AR+ status. Clinical Benefit Rate=CR+PR+SD. Complete Response (CR), disappearance of all target lesions; Partial Response (PR), >=30% decrease in the sum of the longest diameter of target lesions; Stable Disease, <30% decrease in sum of the longest diameter of target lesions.

6 secondary outcomes

Clinical Benefit Rate, in Full Analysis Set
Time frame: Sixteen (16) weeks
Best Overall Response
Time frame: From treatment initiation to end of treatment (up through 11 months of treatment, median duration of treatment 1.9 months).
Progression Free Survival
Time frame: From treatment initiation to tumor progression or death. PFS was assessed up to 11 months of treatment; median duration of treatment, 1.9 months
Time-to-progression
Time frame: From treatment initiation to tumor progression or death. Time to progression was assessed up to 11 months of treatment; median duration of treatment, 1.9 months
Duration of Response
Time frame: From time of documented tumor response to tumor progression or death. Duration of response was assessed through 11 months; median treatment duration, 1.9 months
Objective Response Rate
Time frame: Sixteen (16) weeks

Results and safety

Results posted to the registry on 30 Oct 2020, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
GTx-024	32	0

Primary outcome: Clinical Benefit Rate, in Centrally Confirmed Androgen Receptor Positive (AR+) Subjects

Time frame: Sixteen (16) weeks. Count of participants. Unit: Participants

Measure	GTx-024 n = 18
Value	1

Population analysed: Subjects who were centrally confirmed as AR+

6 secondary outcomes are reported in the full results.

Adverse events

Time frame: one year. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
GTx-024	5 of 32 (15.6%)	28 of 32 (87.5%)	13 of 32

Limitations and caveats (from the sponsor): Early termination due to lack of efficacy and limited data reported.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 18 Nov 2020.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Triple Negative Breast Cancer

RecruitingPhase 3
A Study of Camrelizumab Plus Chemotherapy in Combination With or Without Famitinib as Neoadjuvant Therapy in Participants With Triple Negative Breast Cancer (BCTOP-T-N01)
TNBC - Triple-Negative Breast Cancer
RecruitingPhase 2
BrAin Metastasis in tripLe Negateive Breast Cancer: IvoneScimab and Trop2 ADC
Triple-Negative Breast Cancer (TNBC), Brain Metastases
Active, not recruitingPhase 1/2
Phase I/II Study of the Anti-Programmed Death Ligand-1 Durvalumab Antibody (MEDI4736) in Combination With Olaparib and/or Cediranib for Advanced Solid Tumors and Advanced or Recurrent Ovarian, Triple Negative Breast, Lung, Prostate and Colorectal Can...
Colorectal Neoplasms, Breast Neoplasms
RecruitingPhase 2
Study of Patritumab Deruxtecan Plus Pembrolizumab With Other Anticancer Agents in Participants With High-Risk Early-Stage Triple-Negative or Hormone Receptor-Low Positive/HER-2 Negative Breast Cancer (MK-1022-010, HERTHENA-Breast-03)
Breast Neoplasms, Breast Cancer
RecruitingObservational study
Collection of CSF Samples From Participants With Metastatic Triple Negative Breast Cancer (TNBC) and HER2+ Breast Cancer With no Prior History Nor Active Radiographically Detectable Brain Metastases
Breast Cancer, Breast Carcinoma

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Hope S Rugo, MD principal investigator

University of California, San Francisco

7 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing GTx-024 in people with Triple Negative Breast Cancer, run by GTx at 7 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 22 inclusion and 22 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: stopped early, as of the sponsor's last update on 18 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 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 phase mean?

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 30 Oct 2020). 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?

Clinical Benefit Rate, in Centrally Confirmed Androgen Receptor Positive (AR+) Subjects (time frame: Sixteen (16) weeks). 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 NCT02368691.

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 18 Nov 2020.