Trial Outcomes & Findings for Efficacy and Safety of GTx-024 in Patients With Estrogen Receptor (ER)+/Androgen Receptor (AR)+ Breast Cancer (NCT NCT02463032)

Last Updated: 2020-12-09

Results Overview

To estimate the clinical benefit rate (defined as complete response, partial response, or stable disease) according to RECIST 1.1, in subjects with estrogen receptor positive/androgen receptor positive (ER+/AR+) BC who have centrally confirmed AR+ status. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions assessed by CT or MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR; Clinical Benefit Rate (CBR)= CR + PR + SD.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

136 participants

Primary outcome timeframe

24 weeks

Results posted on

2020-12-09

Participant Flow

Participant milestones

Participant milestones
Measure	GTx-024 9 mg Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 9 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.	GTx-024 18 mg Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 18 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.
Overall Study STARTED	72	64
Overall Study COMPLETED	1	0
Overall Study NOT COMPLETED	71	64

Reasons for withdrawal

Reasons for withdrawal
Measure	GTx-024 9 mg Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 9 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.	GTx-024 18 mg Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 18 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.
Overall Study Lack of Efficacy	61	50
Overall Study Death	8	8
Overall Study Adverse Event	2	6

Baseline Characteristics

Efficacy and Safety of GTx-024 in Patients With Estrogen Receptor (ER)+/Androgen Receptor (AR)+ Breast Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	GTx-024 9 mg n=72 Participants Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 9 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.	GTx-024 18 mg n=64 Participants Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 18 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.	Total n=136 Participants Total of all reporting groups
Age, Continuous	61 years n=5 Participants	62 years n=7 Participants	61 years n=5 Participants
Sex: Female, Male Female	72 Participants n=5 Participants	64 Participants n=7 Participants	136 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	70 Participants n=5 Participants	59 Participants n=7 Participants	129 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants	5 Participants n=7 Participants	7 Participants n=5 Participants
Androgen Receptor (AR) status positive	50 Participants n=5 Participants	52 Participants n=7 Participants	102 Participants n=5 Participants

PRIMARY outcome

Timeframe: 24 weeks

Outcome measures

Outcome measures
Measure	GTx-024 9 mg n=50 Participants Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 9 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.	GTx-024 18 mg n=52 Participants Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 18 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.
Clinical Benefit Rate, in Centrally Confirmed Androgen Receptor (AR)+ Subjects	16 participants	15 participants

SECONDARY outcome

Timeframe: 24 weeks

To estimate the clinical benefit response rate in all subjects randomized who receive at least one dose of study medication (the FAS) regardless of AR status as determined by the central laboratory. o estimate the clinical benefit rate (defined as complete response, partial response, or stable disease) according to RECIST 1.1, in subjects with estrogen receptor positive/androgen receptor positive (ER+/AR+) BC who have centrally confirmed AR+ status. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions assessed by CT or MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR; Clinical Benefit Rate (CBR)= CR + PR + SD.

Outcome measures

Outcome measures
Measure	GTx-024 9 mg n=72 Participants Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 9 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.	GTx-024 18 mg n=64 Participants Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 18 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.
Clinical Benefit Rate, in Full Analysis Set	18 participants	17 participants

SECONDARY outcome

Timeframe: 24 weeks

Outcome measures

Outcome measures
Measure	GTx-024 9 mg n=50 Participants Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 9 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.	GTx-024 18 mg n=52 Participants Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 18 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.
Objective Response (CR + PR) in AR+ Patients PR	2 participants	2 participants
Objective Response (CR + PR) in AR+ Patients CR	2 participants	0 participants

SECONDARY outcome

Timeframe: From treatment initiation to end of treatment

To estimate the best overall response of GTx-024 9 mg and 18 mg

Outcome measures

Outcome measures
Measure	GTx-024 9 mg n=50 Participants Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 9 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.	GTx-024 18 mg n=52 Participants Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 18 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.
Best Overall Response in AR+ Patients PR	2 participants	2 participants
Best Overall Response in AR+ Patients CR	2 participants	0 participants

SECONDARY outcome

Timeframe: From randomization to tumor progression or death

To estimate the progression free survival of subjects receiving Gtx-024 9 mg and 18 mg. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions. Subjects were assessed up through 24 months.

Outcome measures

Outcome measures
Measure	GTx-024 9 mg n=72 Participants Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 9 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.	GTx-024 18 mg n=64 Participants Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 18 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.
Progression Free Survival in All Subjects	5.3 months Due to the study ending prematurely and the limited data available, the confidence interval was not reported. This data is NOT available as it was never provided to GTx- the median was the only reported value. All good cause efforts to locate the data have been exhausted, data are missing and hence not available to be reported.	2.9 months Due to the study ending prematurely and the limited data available, the confidence interval was not reported. This data is NOT available as it was never provided to GTx- the median was the only reported value. Due to the study ending prematurely and the limited data available, the confidence interval was never provided to GTx- the median was the only reported value. All good cause efforts to locate the data have been exhausted, data are missing and hence not available to be reported.

SECONDARY outcome

Timeframe: From randomization to tumor progression or death

To estimate the time to progression in subjects receiving Gtx-024 9 mg and 18 mg in all subjects. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions. Time to progression was assessed up through 24 months.

Outcome measures

Outcome measures
Measure	GTx-024 9 mg n=72 Participants Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 9 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.	GTx-024 18 mg n=64 Participants Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 18 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.
Time to Progression in All Subjects	5.3 months Due to the study ending prematurely and the limited data available, the confidence interval was not reported. This data is NOT available as it was never provided to GTx- the median was the only reported value. All good cause efforts to locate the data have been exhausted, data are missing and hence not available to be reported.	2.9 months Due to the study ending prematurely and the limited data available, the confidence interval was not reported. This data is NOT available as it was never provided to GTx- the median was the only reported value. All good cause efforts to locate the data have been exhausted, data are missing and hence not available to be reported.

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 24 months

To describe the safety profile of GTx-024 9 mg and 18 mg in all subjects randomized and treated. Reported adverse events were described by system organ class (SOC) as opposed to individual events

Outcome measures

Outcome measures
Measure	GTx-024 9 mg n=72 Participants Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 9 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.	GTx-024 18 mg n=64 Participants Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 18 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.
Number Subjects Experiencing Adverse Events	71 Participants	56 Participants

Adverse Events

GTx-024 9 mg

Serious events: 6 serious events

Other events: 71 other events

Deaths: 8 deaths

GTx-024 18 mg

Serious events: 10 serious events

Other events: 56 other events

Deaths: 8 deaths

Serious adverse events

Serious adverse events
Measure	GTx-024 9 mg n=72 participants at risk Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 9 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.	GTx-024 18 mg n=64 participants at risk Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 18 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.
Endocrine disorders diabetes	1.4% 1/72 • 24 months	0.00% 0/64 • 24 months
Investigations hypercalcemia	1.4% 1/72 • 24 months	6.2% 4/64 • 24 months
Investigations increased serum creatinine	0.00% 0/72 • 24 months	1.6% 1/64 • 24 months
Renal and urinary disorders acute kidney failure	1.4% 1/72 • 24 months	1.6% 1/64 • 24 months
Cardiac disorders cardiac failure	0.00% 0/72 • 24 months	3.1% 2/64 • 24 months
Blood and lymphatic system disorders anemia and marrow failure	0.00% 0/72 • 24 months	1.6% 1/64 • 24 months
Infections and infestations sepsis	2.8% 2/72 • 24 months	3.1% 2/64 • 24 months
Respiratory, thoracic and mediastinal disorders pneumothorax	1.4% 1/72 • 24 months	0.00% 0/64 • 24 months
Investigations increased AST	1.4% 1/72 • 24 months	0.00% 0/64 • 24 months
Gastrointestinal disorders nausea	1.4% 1/72 • 24 months	0.00% 0/64 • 24 months
Musculoskeletal and connective tissue disorders pain	1.4% 1/72 • 24 months	1.6% 1/64 • 24 months
Infections and infestations cellulitis	1.4% 1/72 • 24 months	0.00% 0/64 • 24 months
Infections and infestations pneumonia	0.00% 0/72 • 24 months	1.6% 1/64 • 24 months
Gastrointestinal disorders gastritis	0.00% 0/72 • 24 months	1.6% 1/64 • 24 months
Cardiac disorders hypertension	1.4% 1/72 • 24 months	1.6% 1/64 • 24 months
Cardiac disorders myocardial infarction	0.00% 0/72 • 24 months	1.6% 1/64 • 24 months
Infections and infestations h pylori infection	0.00% 0/72 • 24 months	1.6% 1/64 • 24 months
Investigations tumor flare	0.00% 0/72 • 24 months	3.1% 2/64 • 24 months
Investigations pyrexia	0.00% 0/72 • 24 months	1.6% 1/64 • 24 months

Other adverse events

Other adverse events
Measure	GTx-024 9 mg n=72 participants at risk Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 9 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.	GTx-024 18 mg n=64 participants at risk Drug: GTx-024 GTx-024 softgel capsules will be administered once daily to a total dose of 18 mg GTx-024: To determine whether either or both doses result in an acceptable clinical benefit rate.
Gastrointestinal disorders GI disorder	34.7% 25/72 • 24 months	28.1% 18/64 • 24 months
General disorders general disorder	34.7% 25/72 • 24 months	18.8% 12/64 • 24 months
Investigations investigations	23.6% 17/72 • 24 months	23.4% 15/64 • 24 months
Musculoskeletal and connective tissue disorders musculoskeletal	15.3% 11/72 • 24 months	18.8% 12/64 • 24 months
Metabolism and nutrition disorders metabolism	11.1% 8/72 • 24 months	17.2% 11/64 • 24 months
Nervous system disorders nervous system	13.9% 10/72 • 24 months	10.9% 7/64 • 24 months
Skin and subcutaneous tissue disorders skin	8.3% 6/72 • 24 months	12.5% 8/64 • 24 months

Additional Information

Mary Breitmeyer

Oncternal

Phone: 858-434-1113

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place