Trial Outcomes & Findings for Efficacy and Safety of GTx-024 in Patients With Androgen Receptor-Positive Triple Negative Breast Cancer (AR+ TNBC) (NCT NCT02368691)

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

32 participants

Primary outcome timeframe

Sixteen (16) weeks

Results posted on

2020-11-18

Participant Flow

Up to 55 patients were planned for enrollment

No patients were excluded from the study before drug assignment

Participant milestones

Participant milestones
Measure	GTx-024 GTx-024 capsules, 18 mg PO once-daily for up to 12 months GTx-024: GTx-024 softgel capsules will be administered once-daily to a total dose of 18 mg
Overall Study STARTED	32
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	32

Reasons for withdrawal

Reasons for withdrawal
Measure	GTx-024 GTx-024 capsules, 18 mg PO once-daily for up to 12 months GTx-024: GTx-024 softgel capsules will be administered once-daily to a total dose of 18 mg
Overall Study Death	13
Overall Study Lack of Efficacy	15
Overall Study Physician Decision	4

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	GTx-024 n=32 Participants GTx-024 capsules, 18 mg PO once-daily for up to 12 months GTx-024: GTx-024 softgel capsules will be administered once-daily to a total dose of 18 mg
Age, Continuous	65 years n=99 Participants
Sex: Female, Male Female	32 Participants n=99 Participants
Sex: Female, Male Male	0 Participants n=99 Participants
Race/Ethnicity, Customized Caucasian	27 Participants n=99 Participants
Region of Enrollment United States	32 Participants n=99 Participants
AR status-positive	18 participants n=99 Participants

PRIMARY outcome

Timeframe: Sixteen (16) weeks

Population: Subjects who were centrally confirmed as AR+

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.

Outcome measures

Outcome measures
Measure	GTx-024 n=18 Participants GTx-024 capsules, 18 mg PO once-daily for up to 12 months GTx-024: GTx-024 softgel capsules will be administered once-daily to a total dose of 18 mg
Clinical Benefit Rate, in Centrally Confirmed Androgen Receptor Positive (AR+) Subjects	1 Participants

SECONDARY outcome

Timeframe: Sixteen (16) weeks

Population: All subjects, regardless of AR status

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.

Outcome measures

Outcome measures
Measure	GTx-024 n=32 Participants GTx-024 capsules, 18 mg PO once-daily for up to 12 months GTx-024: GTx-024 softgel capsules will be administered once-daily to a total dose of 18 mg
Clinical Benefit Rate, in Full Analysis Set	2 participants

SECONDARY outcome

Timeframe: From treatment initiation to end of treatment (up through 11 months of treatment, median duration of treatment 1.9 months).

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.

Outcome measures

Outcome measures
Measure	GTx-024 n=32 Participants GTx-024 capsules, 18 mg PO once-daily for up to 12 months GTx-024: GTx-024 softgel capsules will be administered once-daily to a total dose of 18 mg
Best Overall Response	6 participants

SECONDARY outcome

Timeframe: From treatment initiation to tumor progression or death. PFS was assessed up to 11 months of treatment; median duration of treatment, 1.9 months

Population: All patients regardless of AR status

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.

Outcome measures

Outcome measures
Measure	GTx-024 n=32 Participants GTx-024 capsules, 18 mg PO once-daily for up to 12 months GTx-024: GTx-024 softgel capsules will be administered once-daily to a total dose of 18 mg
Progression Free Survival	1.9 months Interval 1.7 to 2.3

SECONDARY outcome

Timeframe: 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

Population: All subjects regardless of AR status

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.

Outcome measures

Outcome measures
Measure	GTx-024 n=32 Participants GTx-024 capsules, 18 mg PO once-daily for up to 12 months GTx-024: GTx-024 softgel capsules will be administered once-daily to a total dose of 18 mg
Time-to-progression	1.9 months Interval 1.7 to 2.0

SECONDARY outcome

Timeframe: 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

Population: All subjects regardless of AR status

To assess the duration of response defined as the time from documentation of tumor response to disease progression or death

Outcome measures

Outcome measures
Measure	GTx-024 n=32 Participants GTx-024 capsules, 18 mg PO once-daily for up to 12 months GTx-024: GTx-024 softgel capsules will be administered once-daily to a total dose of 18 mg
Duration of Response	1.9 months Interval 0.1 to 11.0

SECONDARY outcome

Timeframe: Sixteen (16) weeks

To estimate the objective response rate (defined as complete response or partial response) according to RECIST 1.1.

Outcome measures

Outcome measures
Measure	GTx-024 n=32 Participants GTx-024 capsules, 18 mg PO once-daily for up to 12 months GTx-024: GTx-024 softgel capsules will be administered once-daily to a total dose of 18 mg
Objective Response Rate	0 participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to twelve (12) months

To describe the safety profile in subjects with TNBC and centrally confirmed AR+ as well as in all subjects enrolled and treated.

Outcome measures

Outcome measures
Measure	GTx-024 n=32 Participants GTx-024 capsules, 18 mg PO once-daily for up to 12 months GTx-024: GTx-024 softgel capsules will be administered once-daily to a total dose of 18 mg
Number of Participants With Adverse Events	28 participants

Adverse Events

GTx-024

Serious events: 5 serious events

Other events: 28 other events

Deaths: 13 deaths

Serious adverse events

Serious adverse events
Measure	GTx-024 n=32 participants at risk GTx-024 capsules, 18 mg PO once-daily for up to 12 months GTx-024: GTx-024 softgel capsules will be administered once-daily to a total dose of 18 mg
Cardiac disorders CHF	3.1% 1/32 • Number of events 1 • one year
Cardiac disorders pleural effusion	3.1% 1/32 • Number of events 1 • one year
Vascular disorders pulmonary embolism	3.1% 1/32 • Number of events 1 • one year
Neoplasms benign, malignant and unspecified (incl cysts and polyps) tumor flare	3.1% 1/32 • Number of events 1 • one year
Renal and urinary disorders UTI	3.1% 1/32 • Number of events 1 • one year
Metabolism and nutrition disorders dehydration	3.1% 1/32 • Number of events 1 • one year
Gastrointestinal disorders constipation	3.1% 1/32 • Number of events 1 • one year
Musculoskeletal and connective tissue disorders rib fracture	3.1% 1/32 • Number of events 1 • one year

Other adverse events

Other adverse events
Measure	GTx-024 n=32 participants at risk GTx-024 capsules, 18 mg PO once-daily for up to 12 months GTx-024: GTx-024 softgel capsules will be administered once-daily to a total dose of 18 mg
Investigations investigations	40.6% 13/32 • Number of events 13 • one year
Gastrointestinal disorders GI	25.0% 8/32 • Number of events 8 • one year
Metabolism and nutrition disorders metabolism	25.0% 8/32 • Number of events 8 • one year
Blood and lymphatic system disorders blood disorder	12.5% 4/32 • Number of events 4 • one year
Nervous system disorders nervous system	9.4% 3/32 • Number of events 3 • one year
Skin and subcutaneous tissue disorders rash	9.4% 3/32 • Number of events 3 • one year

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