Trial Outcomes & Findings for Dose Escalation Study in Female Subjects With Breast Cancer Receiving Aromatase Inhibitor or Tamoxifen (NCT NCT04080297)

NCT ID: NCT04080297

Last Updated: 2020-02-28

Results Overview

Number of participants with indicated AE receiving Q-122

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

21 participants

Primary outcome timeframe

4 weeks

Results posted on

2020-02-28

Participant Flow

Participant milestones

Participant milestones
Measure
100 mg Q-122
Dosage was 100 mg Q-122 administered orally as two 50 mg capsules once daily for 28 days.
200 mg Q-122
Dosage was 200 mg Q-122 administered orally as four 50 mg capsules once daily for 28 days.
Overall Study
STARTED
10
11
Overall Study
COMPLETED
8
8
Overall Study
NOT COMPLETED
2
3

Reasons for withdrawal

Reasons for withdrawal
Measure
100 mg Q-122
Dosage was 100 mg Q-122 administered orally as two 50 mg capsules once daily for 28 days.
200 mg Q-122
Dosage was 200 mg Q-122 administered orally as four 50 mg capsules once daily for 28 days.
Overall Study
Adverse Event
1
3
Overall Study
concurrent illness
1
0

Baseline Characteristics

Dose Escalation Study in Female Subjects With Breast Cancer Receiving Aromatase Inhibitor or Tamoxifen

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
100 mg Q-122
n=10 Participants
Dosage was 100 mg Q-122 administered orally as two 50 mg capsules once daily for 28 days.
200 mg Q-122
n=11 Participants
Dosage was 200 mg Q-122 administered orally as four 50 mg capsules once daily for 28 days.
Total
n=21 Participants
Total of all reporting groups
Age, Continuous
56.5 years
n=5 Participants
57 years
n=7 Participants
56.8 years
n=5 Participants
Sex: Female, Male
Female
10 Participants
n=5 Participants
11 Participants
n=7 Participants
21 Participants
n=5 Participants
Sex: Female, Male
Male
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 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
Race (NIH/OMB)
Black or African American
10 Participants
n=5 Participants
9 Participants
n=7 Participants
19 Participants
n=5 Participants
Race (NIH/OMB)
White
0 Participants
n=5 Participants
2 Participants
n=7 Participants
2 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants

PRIMARY outcome

Timeframe: 4 weeks

Population: Analyses were performed using the results from the safety analysis population.

Number of participants with indicated AE receiving Q-122

Outcome measures

Outcome measures
Measure
100 mg Q-122
n=10 Participants
Dosage was 100 mg Q-122 administered orally as two 50 mg capsules once daily for 28 days.
200 mg Q-122
n=11 Participants
Dosage was 200 mg Q-122 administered orally as four 50 mg capsules once daily for 28 days.
Adverse Event (AE) Reporting of Q-122
7 Participants
7 Participants

PRIMARY outcome

Timeframe: 4 weeks

Population: Analyses were performed using the results from the safety analysis population.

Number of participants with indicated SAE receiving Q-122

Outcome measures

Outcome measures
Measure
100 mg Q-122
n=10 Participants
Dosage was 100 mg Q-122 administered orally as two 50 mg capsules once daily for 28 days.
200 mg Q-122
n=11 Participants
Dosage was 200 mg Q-122 administered orally as four 50 mg capsules once daily for 28 days.
Serious Adverse Event (SAE) Reporting of Q-122
0 Participants
1 Participants

PRIMARY outcome

Timeframe: Baseline to 4 weeks

Population: Analyses were performed using the intent-to-treat (ITT) population.

Mean change in frequency of moderate to severe vasomotor symptoms. Daily patient (paper) diaries will be used as the primary efficacy collection tool. Change from baseline represents the mean change from the daily average frequency calculated at baseline to the daily average frequency calculated for the last week the subject was on drug. The hot flash severity categories are defined clinically as follow: mild, sensation of heat without perspiration; moderate, sensation of heat with perspiration, but subject is able to continue with activity; and severe. sensation of heat with sweating, sufficiently severe to result in discontinuation of activity.

Outcome measures

Outcome measures
Measure
100 mg Q-122
n=10 Participants
Dosage was 100 mg Q-122 administered orally as two 50 mg capsules once daily for 28 days.
200 mg Q-122
n=11 Participants
Dosage was 200 mg Q-122 administered orally as four 50 mg capsules once daily for 28 days.
Change in Frequency of Moderate to Severe Vasomotor Symptoms.
Baseline
9.86 Hot flashes/day
Standard Deviation 3.18
8.58 Hot flashes/day
Standard Deviation 1.63
Change in Frequency of Moderate to Severe Vasomotor Symptoms.
Change from baseline
-5.81 Hot flashes/day
Standard Deviation 3.71
-5.60 Hot flashes/day
Standard Deviation 3.13

PRIMARY outcome

Timeframe: Baseline to 4 weeks

Population: Analyses were performed using the intent-to-treat (ITT) population.

Percent reduction in frequency of moderate to severe vasomotor symptoms. Daily patient (paper) diaries will be used as the primary efficacy collection tool. The hot flash severity categories are defined clinically as follows: mild, sensation of heat without perspiration; moderate, sensation of heat with perspiration, but subject is able to continue with activity; and severe, sensation of heat with sweating, sufficiently severe to result in discontinuation of activity.

Outcome measures

Outcome measures
Measure
100 mg Q-122
n=9 Participants
Dosage was 100 mg Q-122 administered orally as two 50 mg capsules once daily for 28 days.
200 mg Q-122
n=11 Participants
Dosage was 200 mg Q-122 administered orally as four 50 mg capsules once daily for 28 days.
Percent Change in Frequency of Moderate to Severe Vasomotor Symptoms.
-61.46 Percent change
Standard Deviation 38.85
-67.55 Percent change
Standard Deviation 39.27

SECONDARY outcome

Timeframe: Baseline to 4 weeks

Population: Analyses were performed using the intent-to-treat (ITT) population. The ITT population was defined as all subjects who received at least one dose of study medication and have an evaluable primary or secondary efficacy measurement after baseline.

For moderate-to-severe (mod/sev) hot flashes (HF), a score will be calculated by multiplying the number of moderate-to-severe HFs by their severity to determine the HF (mod/sev) index score, using the following formula: HFSSmod/sev = (number of moderate hot flashes/day × 2) + (number of severe hot flashes/day x 3). The Average Daily HFSSmod/sev for each week will be calculated by dividing the total of daily HFSSmod/sev by the number of days observations will be recorded in that week. The change in score is of clinical significance, with a lower score representing less moderate to severe hot flashes and a higher score representing a greater number of moderate to severe hot flashes.

Outcome measures

Outcome measures
Measure
100 mg Q-122
n=10 Participants
Dosage was 100 mg Q-122 administered orally as two 50 mg capsules once daily for 28 days.
200 mg Q-122
n=11 Participants
Dosage was 200 mg Q-122 administered orally as four 50 mg capsules once daily for 28 days.
Change in Hot Flash Severity Score
Baseline
24.91 Hot flash severity score (HFSS)/day
Standard Deviation 10.76
20.64 Hot flash severity score (HFSS)/day
Standard Deviation 4.06
Change in Hot Flash Severity Score
Treatment - Week 1
17.67 Hot flash severity score (HFSS)/day
Standard Deviation 13.87
10.09 Hot flash severity score (HFSS)/day
Standard Deviation 6.74
Change in Hot Flash Severity Score
Treatment - Week 2
12.41 Hot flash severity score (HFSS)/day
Standard Deviation 12.58
9.51 Hot flash severity score (HFSS)/day
Standard Deviation 7.41
Change in Hot Flash Severity Score
Treatment - Week 3
10.11 Hot flash severity score (HFSS)/day
Standard Deviation 12.81
9.35 Hot flash severity score (HFSS)/day
Standard Deviation 8.73
Change in Hot Flash Severity Score
Treatment - Week 4
11.14 Hot flash severity score (HFSS)/day
Standard Deviation 14.23
7.89 Hot flash severity score (HFSS)/day
Standard Deviation 10.06

SECONDARY outcome

Timeframe: Baseline to 4 weeks

Population: Analyses were performed using the intent-to-treat (ITT) population.

For moderate-to-severe (mod/sev) hot flashes (HF), a score will be calculated by multiplying the number of moderate-to-severe HFs by their severity to determine the HF (mod/sev) index score, using the following formula: HFSSmod/sev = (number of moderate hot flashes/day × 2) + (number of severe hot flashes/day x 3). The Average Daily HFSSmod/sev for each week will be calculated by dividing the total of daily HFSSmod/sev by the number of days observations will be recorded in that week.

Outcome measures

Outcome measures
Measure
100 mg Q-122
n=9 Participants
Dosage was 100 mg Q-122 administered orally as two 50 mg capsules once daily for 28 days.
200 mg Q-122
n=11 Participants
Dosage was 200 mg Q-122 administered orally as four 50 mg capsules once daily for 28 days.
Percent Change in Hot Flash Severity Score
-61.97 Percent change
Standard Deviation 36.03
-67.55 Percent change
Standard Deviation 40.81

SECONDARY outcome

Timeframe: Baseline and 4 weeks

Population: Analyses were performed using the intent-to-treat (ITT) population. The ITT population was defined as all subjects who received at least one dose of study medication and had an evaluable primary or secondary efficacy measurement after baseline.

Greene Climacteric Scale: A comprehensive assessment divided into psychological, physical and vasomotor areas. The scale includes 21 symptoms, subject will score the severity of each symptom with the following score system: 0 = not at all; 1 = a little; 2 = quite a bit; and 3 = extremely. The results represent the total combined score, which can range from 0 to 63. A lower score represents a better outcome.

Outcome measures

Outcome measures
Measure
100 mg Q-122
n=10 Participants
Dosage was 100 mg Q-122 administered orally as two 50 mg capsules once daily for 28 days.
200 mg Q-122
n=11 Participants
Dosage was 200 mg Q-122 administered orally as four 50 mg capsules once daily for 28 days.
Symptoms Associated With Postmenopausal Status
Total Score - Baseline
18.1 score on a scale
Standard Deviation 6.15
19.1 score on a scale
Standard Deviation 12.38
Symptoms Associated With Postmenopausal Status
Total Score - 4 weeks
6.5 score on a scale
Standard Deviation 5.95
4.5 score on a scale
Standard Deviation 2.88

Adverse Events

100 mg Q-122

Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths

200 mg Q-122

Serious events: 1 serious events
Other events: 7 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
100 mg Q-122
n=10 participants at risk
Dosage was 100 mg Q-122 administered orally as two 50 mg capsules once daily for 28 days.
200 mg Q-122
n=11 participants at risk
Dosage was 200 mg Q-122 administered orally as four 50 mg capsules once daily for 28 days.
Reproductive system and breast disorders
Breast pain
0.00%
0/10
9.1%
1/11 • Number of events 1

Other adverse events

Other adverse events
Measure
100 mg Q-122
n=10 participants at risk
Dosage was 100 mg Q-122 administered orally as two 50 mg capsules once daily for 28 days.
200 mg Q-122
n=11 participants at risk
Dosage was 200 mg Q-122 administered orally as four 50 mg capsules once daily for 28 days.
Investigations
Aspartate aminotransferase increase
10.0%
1/10 • Number of events 1
0.00%
0/11
Investigations
Blood thyroid stimulating hormone increased
0.00%
0/10
9.1%
1/11 • Number of events 1
Investigations
Blood triglycerides increased
0.00%
0/10
9.1%
1/11 • Number of events 1
Investigations
Blood uric acid increased
10.0%
1/10 • Number of events 1
9.1%
1/11 • Number of events 1
Investigations
Weight increased
0.00%
0/10
9.1%
1/11 • Number of events 1
Nervous system disorders
Coordination abnormal
0.00%
0/10
9.1%
1/11 • Number of events 1
Nervous system disorders
Dizziness
10.0%
1/10 • Number of events 1
0.00%
0/11
Nervous system disorders
Headache
0.00%
0/10
18.2%
2/11 • Number of events 3
Nervous system disorders
Insomnia
0.00%
0/10
9.1%
1/11 • Number of events 1
Gastrointestinal disorders
Gastroenteritis viral
10.0%
1/10 • Number of events 1
0.00%
0/11
Gastrointestinal disorders
Nausea
0.00%
0/10
9.1%
1/11 • Number of events 1
Gastrointestinal disorders
Oral herpes
0.00%
0/10
9.1%
1/11 • Number of events 1
Skin and subcutaneous tissue disorders
Contusion
10.0%
1/10 • Number of events 2
0.00%
0/11
Skin and subcutaneous tissue disorders
Pruritus
0.00%
0/10
9.1%
1/11 • Number of events 1
Metabolism and nutrition disorders
Hyperglycaemia
10.0%
1/10 • Number of events 1
0.00%
0/11
Metabolism and nutrition disorders
Hypertriglyceridaemia
10.0%
1/10 • Number of events 1
0.00%
0/11
Psychiatric disorders
Anxiety
0.00%
0/10
9.1%
1/11 • Number of events 1
Psychiatric disorders
Euphoric mood
0.00%
0/10
9.1%
1/11 • Number of events 1
General disorders
Flank pain
0.00%
0/10
9.1%
1/11 • Number of events 1
Infections and infestations
Urinary tract infection
0.00%
0/10
9.1%
1/11 • Number of events 1
Renal and urinary disorders
Haematuria
10.0%
1/10 • Number of events 1
0.00%
0/11
Reproductive system and breast disorders
Breast pain
0.00%
0/10
9.1%
1/11 • Number of events 1
Reproductive system and breast disorders
Breast mass
0.00%
0/10
9.1%
1/11 • Number of events 1
Reproductive system and breast disorders
Vulvovaginal burning sensation
10.0%
1/10 • Number of events 1
0.00%
0/11

Additional Information

Rob Crombie

Chief Exceutive Officer

Phone: +61 3 9657 0731

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place