Trial Outcomes & Findings for Phase II, Dose Finding Study of GTx-758 (NCT NCT01393119)
NCT ID: NCT01393119
Last Updated: 2024-04-22
Results Overview
Percentage of Patients Note: Due to the study being terminated early, per FDA, the secondary assessment of maintenance was unable to be assessed. Hence, all summaries provided are for subjects in the two loading dose groups of 1000 mg BID and 1500mg BID only. PLEASE NOTE: Study was terminated early, per FDA, the secondary assessment of maintenance was unable to be assessed. Hence, the efficacy summary provided are for subjects in the two loading dose groups of 1000 mg BID and 1500mg BID only, poling across maintenance doses within each loading dose. Safety/ITT - 27 and 28 patients, respectively mITT - 18 and 19 patients, respectively Note: mITT includes patients that meet the requirements for the efficacy analyses This format was agreed to by the PRS review team, per email communication on guidnace for presenting the data.
TERMINATED
PHASE2
77 participants
Day 1-28
2024-04-22
Participant Flow
77 patients were screened with 19 discontinuing the study due to screen failure. 77-19=58 randomized subjects. Please note, 3 of these 58 subjects were randomized but not treated, as noted below, hence n=55 subjects are provided data for results summaries. Note: Due to study being terminated early, per FDA, secondary assessment of maintenance was not assessed. Hence, all summaries are for the two loading dose groups of 1000 mg BID and 1500mg BID only.
Screen Failure subjects were not included in the outputs from the previous company who owned the product.
Participant milestones
| Measure |
Loading Dose 1000mg GTx-758 BID and Maintenance Dose of 1000mg GTx-758
Loading Dose 1000mg GTx-758 BID and Maintenance Dose of 1000mg GTx-758
|
Loading Dose 1000mg GTx-758 BID and Maintenance Dose of 2000mg GTx-758
Loading Dose 1000mg GTx-758 BID and Maintenance Dose of 2000mg GTx-758
|
Loading Dose 1500mg GTx-758 BID and Maintenance Dose of 1000mg GTx-758
Loading Dose 1000mg GTx-758 BID and Maintenance Dose of 2000mg GTx-758
|
Loading Dose 1500mg GTx-758 BID and Maintenance Dose of 2000mg GTx-758
Loading Dose 1500mg GTx-758 BID and Maintenance Dose of 2000mg GTx-758
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
14
|
14
|
14
|
16
|
|
Overall Study
COMPLETED
|
0
|
0
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
14
|
14
|
14
|
16
|
Reasons for withdrawal
| Measure |
Loading Dose 1000mg GTx-758 BID and Maintenance Dose of 1000mg GTx-758
Loading Dose 1000mg GTx-758 BID and Maintenance Dose of 1000mg GTx-758
|
Loading Dose 1000mg GTx-758 BID and Maintenance Dose of 2000mg GTx-758
Loading Dose 1000mg GTx-758 BID and Maintenance Dose of 2000mg GTx-758
|
Loading Dose 1500mg GTx-758 BID and Maintenance Dose of 1000mg GTx-758
Loading Dose 1000mg GTx-758 BID and Maintenance Dose of 2000mg GTx-758
|
Loading Dose 1500mg GTx-758 BID and Maintenance Dose of 2000mg GTx-758
Loading Dose 1500mg GTx-758 BID and Maintenance Dose of 2000mg GTx-758
|
|---|---|---|---|---|
|
Overall Study
Adverse Event
|
1
|
1
|
0
|
1
|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
1
|
0
|
|
Overall Study
Lack of Efficacy
|
1
|
0
|
2
|
1
|
|
Overall Study
Protocol Violation
|
0
|
0
|
1
|
0
|
|
Overall Study
Death
|
0
|
0
|
0
|
1
|
|
Overall Study
Randomized but Not treated
|
1
|
0
|
1
|
1
|
|
Overall Study
Sponsor Termination of Study
|
11
|
12
|
9
|
12
|
Baseline Characteristics
Phase II, Dose Finding Study of GTx-758
Baseline characteristics by cohort
| Measure |
Loading Dose 1000mg GTx-758 BID and Maintenance Dose of 1000mg GTx-758
n=13 Participants
Loading Dose 1000mg GTx-758 BID and Maintenance Dose of 1000mg GTx-758
|
Loading Dose 1000mg GTx-758 BID and Maintenance Dose of 2000mg GTx-758
n=14 Participants
Loading Dose 1000mg GTx-758 BID and Maintenance Dose of 2000mg GTx-758
|
Loading Dose 1500mg GTx-758 BID and Maintenance Dose of 1000mg GTx-758
n=13 Participants
Loading Dose 1500mg GTx-758 BID and Maintenance Dose of 1000mg GTx-758
|
Loading Dose 1500mg GTx-758 BID and Maintenance Dose of 2000mg GTx-758
n=15 Participants
Loading Dose 1500mg GTx-758 BID and Maintenance Dose of 2000mg GTx-758
|
Total
n=55 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Continuous
Age, Continuous
|
64.8 years
STANDARD_DEVIATION 4.9 • n=5 Participants
|
67.1 years
STANDARD_DEVIATION 8.4 • n=7 Participants
|
68.0 years
STANDARD_DEVIATION 5.4 • n=5 Participants
|
68.9 years
STANDARD_DEVIATION 8.2 • n=4 Participants
|
67.25 years
STANDARD_DEVIATION 6.9 • n=21 Participants
|
|
Sex/Gender, Customized
Males
|
13 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
15 Participants
n=4 Participants
|
55 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
13 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
13 Participants
n=4 Participants
|
53 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
13 Participants
n=21 Participants
|
|
Race (NIH/OMB)
White
|
11 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
10 Participants
n=4 Participants
|
40 Participants
n=21 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
PRIMARY outcome
Timeframe: Day 1-28Population: Modified Intent to Treat Population
Percentage of Patients Note: Due to the study being terminated early, per FDA, the secondary assessment of maintenance was unable to be assessed. Hence, all summaries provided are for subjects in the two loading dose groups of 1000 mg BID and 1500mg BID only. PLEASE NOTE: Study was terminated early, per FDA, the secondary assessment of maintenance was unable to be assessed. Hence, the efficacy summary provided are for subjects in the two loading dose groups of 1000 mg BID and 1500mg BID only, poling across maintenance doses within each loading dose. Safety/ITT - 27 and 28 patients, respectively mITT - 18 and 19 patients, respectively Note: mITT includes patients that meet the requirements for the efficacy analyses This format was agreed to by the PRS review team, per email communication on guidnace for presenting the data.
Outcome measures
| Measure |
1000mg GTx-758 BID
n=18 Participants
Loading dose 1000mg GTx-758 BID + maintenance dose of 1000mg/2000mg GTx-758 daily Combined
|
1500 mg GTx-758 BID
n=19 Participants
Loading dose of 1500 mg GTx-758 BID + maintenance dose of 1000mg/2000mg GTx-758 daily Combined
|
|---|---|---|
|
Percentage of Subjects That Reach Castration by Day 28
|
15 Participants
|
16 Participants
|
Adverse Events
1000mg GTx-758 BID
1000 mg GTx-758 BID
1500 mg GTx-758 BID
1500mg GTx-758 BID
Serious adverse events
| Measure |
1000mg GTx-758 BID
n=13 participants at risk
Loading dose 1000mg GTx-758 BID + maintenance dose of 1000mg GTx-758 daily
|
1000 mg GTx-758 BID
n=14 participants at risk
Loading dose of 1000mg GTx-758 BID + maintenance dose of 2000mg GTx-758 daily
|
1500 mg GTx-758 BID
n=13 participants at risk
Loading dose of 1500 mg GTx-758 BID + maintenance dose of 1000mg GTx-758 daily
|
1500mg GTx-758 BID
n=15 participants at risk
Loading dose of 1500mg of GTx-758 BID + maintenance dose of 2000mg GTx-758 daily
|
|---|---|---|---|---|
|
Nervous system disorders
Stroke
|
0.00%
0/13
|
0.00%
0/14
|
0.00%
0/13
|
6.7%
1/15 • Number of events 1
|
|
Cardiac disorders
Congestive Heart Failure
|
0.00%
0/13
|
0.00%
0/14
|
0.00%
0/13
|
6.7%
1/15 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary Embolism
|
0.00%
0/13
|
7.1%
1/14 • Number of events 1
|
0.00%
0/13
|
13.3%
2/15 • Number of events 2
|
|
Vascular disorders
DVT
|
7.7%
1/13 • Number of events 1
|
0.00%
0/14
|
0.00%
0/13
|
0.00%
0/15
|
|
General disorders
Non-Cardiac Chest pain
|
7.7%
1/13 • Number of events 1
|
0.00%
0/14
|
0.00%
0/13
|
0.00%
0/15
|
Other adverse events
| Measure |
1000mg GTx-758 BID
n=13 participants at risk
Loading dose 1000mg GTx-758 BID + maintenance dose of 1000mg GTx-758 daily
|
1000 mg GTx-758 BID
n=14 participants at risk
Loading dose of 1000mg GTx-758 BID + maintenance dose of 2000mg GTx-758 daily
|
1500 mg GTx-758 BID
n=13 participants at risk
Loading dose of 1500 mg GTx-758 BID + maintenance dose of 1000mg GTx-758 daily
|
1500mg GTx-758 BID
n=15 participants at risk
Loading dose of 1500mg of GTx-758 BID + maintenance dose of 2000mg GTx-758 daily
|
|---|---|---|---|---|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/13
|
0.00%
0/14
|
7.7%
1/13
|
13.3%
2/15
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/13
|
21.4%
3/14
|
7.7%
1/13
|
20.0%
3/15
|
|
General disorders
Fatigue
|
30.8%
4/13
|
7.1%
1/14
|
7.7%
1/13
|
26.7%
4/15
|
|
General disorders
Influenza Like Illness
|
15.4%
2/13
|
0.00%
0/14
|
7.7%
1/13
|
0.00%
0/15
|
|
General disorders
Odema Peripheral
|
0.00%
0/13
|
14.3%
2/14
|
0.00%
0/13
|
0.00%
0/15
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
7.7%
1/13
|
0.00%
0/14
|
7.7%
1/13
|
13.3%
2/15
|
|
Infections and infestations
Urinary Tract Infection
|
15.4%
2/13
|
7.1%
1/14
|
0.00%
0/13
|
0.00%
0/15
|
|
Investigations
Electrocardiogram QT Prolonged
|
0.00%
0/13
|
14.3%
2/14
|
0.00%
0/13
|
0.00%
0/15
|
|
Musculoskeletal and connective tissue disorders
Pain in Extremity
|
15.4%
2/13
|
7.1%
1/14
|
0.00%
0/13
|
13.3%
2/15
|
|
Renal and urinary disorders
Pollakiuria
|
7.7%
1/13
|
0.00%
0/14
|
0.00%
0/13
|
13.3%
2/15
|
|
Reproductive system and breast disorders
Breast Pain
|
38.5%
5/13
|
7.1%
1/14
|
15.4%
2/13
|
20.0%
3/15
|
|
Reproductive system and breast disorders
Gynaecomastia
|
0.00%
0/13
|
14.3%
2/14
|
15.4%
2/13
|
6.7%
1/15
|
|
Respiratory, thoracic and mediastinal disorders
Nasal Congestion
|
0.00%
0/13
|
14.3%
2/14
|
7.7%
1/13
|
0.00%
0/15
|
|
Vascular disorders
DVT
|
15.4%
2/13
|
0.00%
0/14
|
0.00%
0/13
|
0.00%
0/15
|
|
Vascular disorders
Hot Flush
|
7.7%
1/13
|
14.3%
2/14
|
15.4%
2/13
|
0.00%
0/15
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place