Trial Outcomes & Findings for HCQ on PAR-4 Levels in Patients With Resectable Solid Tumors (NCT NCT02232243)
NCT ID: NCT02232243
Last Updated: 2021-06-28
Results Overview
Number of patients with 2-fold change in Par-4 levels from baseline to day 14
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
10 participants
Primary outcome timeframe
Baseline and day 14
Results posted on
2021-06-28
Participant Flow
This study was initiated July 2015 at the University of Kentucky and enrolled 10 adult patients with early stage solid malignancies.
10 patients enrolled in this dose escalation study. In Period 1, three patients were assigned to receive 200mg HCQ twice daily. Based on toxicity and PAR-4 levels, in Period 2 the next four patients received 400mg HCQ twice daily. Following additional analysis, in Period 3 the final three patients received 200mg HCQ twice daily.
Participant milestones
| Measure |
Initial: Hydroxychloroquine 400mg HCQ
These patients received 200mg hydroxychloroquine (HCQ) twice daily (400mg/day total) for 14 days prior to surgery.
|
Secondary: Hydroxychloroquine 800mg HCQ
These patients received 400mg hydroxychloroquine (HCQ) twice daily (800mg/day total) for 14 days prior to surgery.
|
Tertiary: Hydroxychloroquine 400mg HCQ
Based on data from primary and secondary groups, these patients received 200mg hydroxychloroquine (HCQ) twice daily (400mg/day total) for 14 days prior to surgery.
|
|---|---|---|---|
|
First Dose (200mg HCQ Twice Daily)
STARTED
|
3
|
0
|
0
|
|
First Dose (200mg HCQ Twice Daily)
COMPLETED
|
3
|
0
|
0
|
|
First Dose (200mg HCQ Twice Daily)
NOT COMPLETED
|
0
|
0
|
0
|
|
Second Dose (400mg HCQ Twice Daily)
STARTED
|
0
|
4
|
0
|
|
Second Dose (400mg HCQ Twice Daily)
COMPLETED
|
0
|
3
|
0
|
|
Second Dose (400mg HCQ Twice Daily)
NOT COMPLETED
|
0
|
1
|
0
|
|
Third Dose(200mg HCQ Twice Daily)
STARTED
|
0
|
0
|
3
|
|
Third Dose(200mg HCQ Twice Daily)
COMPLETED
|
0
|
0
|
3
|
|
Third Dose(200mg HCQ Twice Daily)
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
| Measure |
Initial: Hydroxychloroquine 400mg HCQ
These patients received 200mg hydroxychloroquine (HCQ) twice daily (400mg/day total) for 14 days prior to surgery.
|
Secondary: Hydroxychloroquine 800mg HCQ
These patients received 400mg hydroxychloroquine (HCQ) twice daily (800mg/day total) for 14 days prior to surgery.
|
Tertiary: Hydroxychloroquine 400mg HCQ
Based on data from primary and secondary groups, these patients received 200mg hydroxychloroquine (HCQ) twice daily (400mg/day total) for 14 days prior to surgery.
|
|---|---|---|---|
|
Second Dose (400mg HCQ Twice Daily)
Protocol Violation
|
0
|
1
|
0
|
Baseline Characteristics
One patient was unanalyzable
Baseline characteristics by cohort
| Measure |
Initial: Hydroxychloroquine 400mg HCQ
n=3 Participants
Hydroxychloroquine 14 days
Hydroxychloroquine: Hydroxychloroquine (200mg twice per day; 400mg total) was given to subjects for up to 14 days prior to surgery.
|
Secondary: Hydroxychloroquine 800mg HCQ
n=4 Participants
Hydroxychloroquine 14 days
Hydroxychloroquine: Hydroxychloroquine (400mg twice per day; 800mg total) was given to subjects for up to 14 days prior to surgery.
|
Tertiary: Hydroxychloroquine 400mg HCQ
n=3 Participants
Hydroxychloroquine 14 days
Hydroxychloroquine: Hydroxychloroquine (200mg twice per day; 400mg total) was given to subjects for up to 14 days prior to surgery.
|
Total
n=10 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants • One patient was unanalyzable
|
0 Participants
n=7 Participants • One patient was unanalyzable
|
0 Participants
n=5 Participants • One patient was unanalyzable
|
0 Participants
n=4 Participants • One patient was unanalyzable
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=5 Participants • One patient was unanalyzable
|
3 Participants
n=7 Participants • One patient was unanalyzable
|
3 Participants
n=5 Participants • One patient was unanalyzable
|
8 Participants
n=4 Participants • One patient was unanalyzable
|
|
Age, Categorical
>=65 years
|
1 Participants
n=5 Participants • One patient was unanalyzable
|
1 Participants
n=7 Participants • One patient was unanalyzable
|
0 Participants
n=5 Participants • One patient was unanalyzable
|
2 Participants
n=4 Participants • One patient was unanalyzable
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants • One patient was unanalyzable
|
0 Participants
n=7 Participants • One patient was unanalyzable
|
1 Participants
n=5 Participants • One patient was unanalyzable
|
1 Participants
n=4 Participants • One patient was unanalyzable
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants • One patient was unanalyzable
|
4 Participants
n=7 Participants • One patient was unanalyzable
|
2 Participants
n=5 Participants • One patient was unanalyzable
|
9 Participants
n=4 Participants • One patient was unanalyzable
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants • One patient was unanalyzable
|
0 Participants
n=7 Participants • One patient was unanalyzable
|
0 Participants
n=5 Participants • One patient was unanalyzable
|
0 Participants
n=4 Participants • One patient was unanalyzable
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=5 Participants • One patient was unanalyzable
|
4 Participants
n=7 Participants • One patient was unanalyzable
|
3 Participants
n=5 Participants • One patient was unanalyzable
|
10 Participants
n=4 Participants • One patient was unanalyzable
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants • One patient was unanalyzable
|
0 Participants
n=7 Participants • One patient was unanalyzable
|
0 Participants
n=5 Participants • One patient was unanalyzable
|
0 Participants
n=4 Participants • One patient was unanalyzable
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants • one patient unanalyzable
|
0 Participants
n=7 Participants • one patient unanalyzable
|
0 Participants
n=5 Participants • one patient unanalyzable
|
0 Participants
n=4 Participants • one patient unanalyzable
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants • one patient unanalyzable
|
0 Participants
n=7 Participants • one patient unanalyzable
|
0 Participants
n=5 Participants • one patient unanalyzable
|
0 Participants
n=4 Participants • one patient unanalyzable
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants • one patient unanalyzable
|
0 Participants
n=7 Participants • one patient unanalyzable
|
0 Participants
n=5 Participants • one patient unanalyzable
|
0 Participants
n=4 Participants • one patient unanalyzable
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants • one patient unanalyzable
|
0 Participants
n=7 Participants • one patient unanalyzable
|
0 Participants
n=5 Participants • one patient unanalyzable
|
0 Participants
n=4 Participants • one patient unanalyzable
|
|
Race (NIH/OMB)
White
|
3 Participants
n=5 Participants • one patient unanalyzable
|
4 Participants
n=7 Participants • one patient unanalyzable
|
3 Participants
n=5 Participants • one patient unanalyzable
|
10 Participants
n=4 Participants • one patient unanalyzable
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants • one patient unanalyzable
|
0 Participants
n=7 Participants • one patient unanalyzable
|
0 Participants
n=5 Participants • one patient unanalyzable
|
0 Participants
n=4 Participants • one patient unanalyzable
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants • one patient unanalyzable
|
0 Participants
n=7 Participants • one patient unanalyzable
|
0 Participants
n=5 Participants • one patient unanalyzable
|
0 Participants
n=4 Participants • one patient unanalyzable
|
|
Region of Enrollment
United States
|
3 participants
n=5 Participants
|
4 participants
n=7 Participants
|
3 participants
n=5 Participants
|
10 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Baseline and day 14Number of patients with 2-fold change in Par-4 levels from baseline to day 14
Outcome measures
| Measure |
Initial: Hydroxychloroquine 400mg HCQ
n=3 Participants
These initial 3 patients received 200mg hydroxychloroquine (HCQ) twice daily (400mg/day total) for 14 days prior to surgery.
Hydroxychloroquine, 200mg twice dailiy: Hydroxychloroquine, 200mg twice daily (400mg/day total) was given to subjects for up to 14 days prior to surgery.
|
Secondary: Hydroxychloroquine 800mg HCQ
n=3 Participants
Based on data analysis from the initial patients, these patients received 400mg hydroxychloroquine (HCQ) twice daily (800mg/day total) for 14 days prior to surgery.
Hydroxychloroquine, 400mg twice daily: Hydroxychloroquine, 400mg twice daily (800mg/day total) was given to subjects for up to 14 days prior to surgery.
|
Tertiary: Hydroxychloroquine 400mg HCQ
n=3 Participants
Based on data from the primary and secondary groups, patients received 200mg hydroxychloroquine (HCQ) twice daily (400mg/day total) for 14 days prior to surgery.
Hydroxychloroquine, 200mg twice dailiy: Hydroxychloroquine, 200mg twice daily (400mg/day total) was given to subjects for up to 14 days prior to surgery.
|
|---|---|---|---|
|
Patients With Elevated Par-4 Levels
|
3 Participants
|
2 Participants
|
1 Participants
|
PRIMARY outcome
Timeframe: Day 14Population: All patients
Dose (mg twice daily) wherein 70% of patients exhibit a 2-fold increase in Par-4 levels with a dose-limiting toxicity of no more than 30%.
Outcome measures
| Measure |
Initial: Hydroxychloroquine 400mg HCQ
n=9 Participants
These initial 3 patients received 200mg hydroxychloroquine (HCQ) twice daily (400mg/day total) for 14 days prior to surgery.
Hydroxychloroquine, 200mg twice dailiy: Hydroxychloroquine, 200mg twice daily (400mg/day total) was given to subjects for up to 14 days prior to surgery.
|
Secondary: Hydroxychloroquine 800mg HCQ
Based on data analysis from the initial patients, these patients received 400mg hydroxychloroquine (HCQ) twice daily (800mg/day total) for 14 days prior to surgery.
Hydroxychloroquine, 400mg twice daily: Hydroxychloroquine, 400mg twice daily (800mg/day total) was given to subjects for up to 14 days prior to surgery.
|
Tertiary: Hydroxychloroquine 400mg HCQ
Based on data from the primary and secondary groups, patients received 200mg hydroxychloroquine (HCQ) twice daily (400mg/day total) for 14 days prior to surgery.
Hydroxychloroquine, 200mg twice dailiy: Hydroxychloroquine, 200mg twice daily (400mg/day total) was given to subjects for up to 14 days prior to surgery.
|
|---|---|---|---|
|
Optimal Biologic Dose of Hydroxychloroquine
|
200 mg twice daily
|
—
|
—
|
Adverse Events
Initial: Hydroxychloroquine 400mg HCQ
Serious events: 0 serious events
Other events: 1 other events
Deaths: 1 deaths
Secondary: Hydroxychloroquine 800mg HCQ
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Tertiary: Hydroxychloroquine 400mg HCQ
Serious events: 0 serious events
Other events: 1 other events
Deaths: 1 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Initial: Hydroxychloroquine 400mg HCQ
n=3 participants at risk
Hydroxychloroquine 14 days
Hydroxychloroquine: Hydroxychloroquine (200mg twice daily; 400mg total) was given to subjects for up to 14 days prior to surgery.
|
Secondary: Hydroxychloroquine 800mg HCQ
n=4 participants at risk
Hydroxychloroquine 14 days
Hydroxychloroquine: Hydroxychloroquine (400mg twice daily; 800mg total) was given to subjects for up to 14 days prior to surgery.
|
Tertiary: Hydroxychloroquine 400mg HCQ
n=3 participants at risk
Hydroxychloroquine 14 days
Hydroxychloroquine: Hydroxychloroquine (200mg twice daily, 400mg total) was given to subjects for up to 14 days prior to surgery.
|
|---|---|---|---|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/3 • 14 days
Patients were queried about adverse events during study visits.
|
25.0%
1/4 • Number of events 1 • 14 days
Patients were queried about adverse events during study visits.
|
33.3%
1/3 • Number of events 2 • 14 days
Patients were queried about adverse events during study visits.
|
|
Gastrointestinal disorders
Diarrhea
|
33.3%
1/3 • Number of events 1 • 14 days
Patients were queried about adverse events during study visits.
|
0.00%
0/4 • 14 days
Patients were queried about adverse events during study visits.
|
0.00%
0/3 • 14 days
Patients were queried about adverse events during study visits.
|
|
General disorders
Abdominal Pain
|
33.3%
1/3 • Number of events 1 • 14 days
Patients were queried about adverse events during study visits.
|
0.00%
0/4 • 14 days
Patients were queried about adverse events during study visits.
|
0.00%
0/3 • 14 days
Patients were queried about adverse events during study visits.
|
|
Eye disorders
Floaters
|
0.00%
0/3 • 14 days
Patients were queried about adverse events during study visits.
|
25.0%
1/4 • Number of events 1 • 14 days
Patients were queried about adverse events during study visits.
|
0.00%
0/3 • 14 days
Patients were queried about adverse events during study visits.
|
|
General disorders
Anorexia
|
0.00%
0/3 • 14 days
Patients were queried about adverse events during study visits.
|
25.0%
1/4 • Number of events 1 • 14 days
Patients were queried about adverse events during study visits.
|
0.00%
0/3 • 14 days
Patients were queried about adverse events during study visits.
|
|
Eye disorders
Blurred Vision
|
0.00%
0/3 • 14 days
Patients were queried about adverse events during study visits.
|
25.0%
1/4 • Number of events 1 • 14 days
Patients were queried about adverse events during study visits.
|
0.00%
0/3 • 14 days
Patients were queried about adverse events during study visits.
|
|
Renal and urinary disorders
Urinary Tract Infection
|
0.00%
0/3 • 14 days
Patients were queried about adverse events during study visits.
|
0.00%
0/4 • 14 days
Patients were queried about adverse events during study visits.
|
33.3%
1/3 • Number of events 1 • 14 days
Patients were queried about adverse events during study visits.
|
|
Cardiac disorders
Chest Pain
|
0.00%
0/3 • 14 days
Patients were queried about adverse events during study visits.
|
25.0%
1/4 • Number of events 1 • 14 days
Patients were queried about adverse events during study visits.
|
0.00%
0/3 • 14 days
Patients were queried about adverse events during study visits.
|
|
Vascular disorders
Thromboembolism
|
0.00%
0/3 • 14 days
Patients were queried about adverse events during study visits.
|
25.0%
1/4 • Number of events 1 • 14 days
Patients were queried about adverse events during study visits.
|
0.00%
0/3 • 14 days
Patients were queried about adverse events during study visits.
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/3 • 14 days
Patients were queried about adverse events during study visits.
|
25.0%
1/4 • Number of events 1 • 14 days
Patients were queried about adverse events during study visits.
|
0.00%
0/3 • 14 days
Patients were queried about adverse events during study visits.
|
|
Nervous system disorders
Headache
|
0.00%
0/3 • 14 days
Patients were queried about adverse events during study visits.
|
25.0%
1/4 • Number of events 1 • 14 days
Patients were queried about adverse events during study visits.
|
0.00%
0/3 • 14 days
Patients were queried about adverse events during study visits.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place