Trial Outcomes & Findings for Neuro-pharmacological Properties of Repurposed Posaconazole in Glioblastoma: A Phase 0 Clinical Trial (NCT NCT04825275)
NCT ID: NCT04825275
Last Updated: 2025-12-17
Results Overview
Assessment of the concentration of drug in the dialysate fluid.
Recruitment status
TERMINATED
Study phase
EARLY_PHASE1
Target enrollment
7 participants
Primary outcome timeframe
Collected over a 24-hour period after surgery (biopsy or resection)
Results posted on
2025-12-17
Participant Flow
Seven participants gave informed consent to participate in the clinical trial. One participant withdrew consent prior to beginning dosing posaconazole.
Participants were assigned to the Control Arm if their tumor resection surgery was scheduled more than 7-10 days after informed consent was obtained.
Participant milestones
| Measure |
Posaconazole
Participants will be taking 300 mg of the study drug (three 100 mg tablets) by mouth twice a day the first day and then 300 mg once a day until the day of biopsy or surgery. On the day of biopsy or surgery, participants will take their medication the morning of their biopsy or surgery (before the operation). Participants will then take the last dose of the medication in the morning of the day after their biopsy or surgery. Participants will be given 12 days' worth of the study drug (pills) and verbally instructed how and when to take them.
|
Control
Participants will not undergo any intervention.
|
|---|---|---|
|
Overall Study
STARTED
|
3
|
4
|
|
Overall Study
COMPLETED
|
2
|
4
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
| Measure |
Posaconazole
Participants will be taking 300 mg of the study drug (three 100 mg tablets) by mouth twice a day the first day and then 300 mg once a day until the day of biopsy or surgery. On the day of biopsy or surgery, participants will take their medication the morning of their biopsy or surgery (before the operation). Participants will then take the last dose of the medication in the morning of the day after their biopsy or surgery. Participants will be given 12 days' worth of the study drug (pills) and verbally instructed how and when to take them.
|
Control
Participants will not undergo any intervention.
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
Baseline Characteristics
Since CSF was not collected prior to tumor resection surgery (or posaconazole dosing), the baseline neuro-pharmacokinetic profile of posaconazole is not applicable.
Baseline characteristics by cohort
| Measure |
Posaconazole
n=2 Participants
Participants will be taking 300 mg of the study drug (three 100 mg tablets) by mouth twice a day the first day and then 300 mg once a day until the day of biopsy or surgery. On the day of biopsy or surgery, participants will take their medication the morning of their biopsy or surgery (before the operation). Participants will then take the last dose of the medication in the morning of the day after their biopsy or surgery. Participants will be given 12 days' worth of the study drug (pills) and verbally instructed how and when to take them.
|
Control
n=4 Participants
Participants will not undergo any intervention.
|
Total
n=6 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=6 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=2 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=6 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=2 Participants
|
3 Participants
n=4 Participants
|
5 Participants
n=6 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=6 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=2 Participants
|
4 Participants
n=4 Participants
|
6 Participants
n=6 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=6 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=6 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=6 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=6 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=2 Participants
|
4 Participants
n=4 Participants
|
5 Participants
n=6 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=2 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=6 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=6 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=6 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
2 Participants
n=2 Participants
|
4 Participants
n=4 Participants
|
6 Participants
n=6 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=6 Participants
|
PRIMARY outcome
Timeframe: Collected over a 24-hour period after surgery (biopsy or resection)Population: The fourth control participant's samples were unavailable for analysis.
Assessment of the concentration of drug in the dialysate fluid.
Outcome measures
| Measure |
Posaconazole
n=2 Participants
Participants will be taking 300 mg of the study drug (three 100 mg tablets) by mouth twice a day the first day and then 300 mg once a day until the day of biopsy or surgery. On the day of biopsy or surgery, participants will take their medication the morning of their biopsy or surgery (before the operation). Participants will then take the last dose of the medication in the morning of the day after their biopsy or surgery. Participants will be given 12 days' worth of the study drug (pills) and verbally instructed how and when to take them.
|
Control
n=3 Participants
Participants will not undergo any intervention.
|
|---|---|---|
|
Posaconazole Concentration in Cerebrospinal Fluid Using Microdialysis Catheters
|
NA ng/mL
Standard Deviation NA
Results were less than the limit of detection.
|
NA ng/mL
Standard Deviation NA
Results were less than the limit of detection.
|
SECONDARY outcome
Timeframe: from Baseline to Visit 7 (14 days +/- 7 days post-op)Population: Control participants did not take the study drug.
Measured through the Grade and Frequency of adverse events, based on the CTCAE v5.0 criteria
Outcome measures
| Measure |
Posaconazole
n=2 Participants
Participants will be taking 300 mg of the study drug (three 100 mg tablets) by mouth twice a day the first day and then 300 mg once a day until the day of biopsy or surgery. On the day of biopsy or surgery, participants will take their medication the morning of their biopsy or surgery (before the operation). Participants will then take the last dose of the medication in the morning of the day after their biopsy or surgery. Participants will be given 12 days' worth of the study drug (pills) and verbally instructed how and when to take them.
|
Control
Participants will not undergo any intervention.
|
|---|---|---|
|
Number of Participants Able to Tolerate Preoperative Steady-state Dosing of Posaconazole
|
2 Participants Who Tolerated Dosing
|
—
|
SECONDARY outcome
Timeframe: Within 24 hours after biopsy or tumor resectionPopulation: The fourth control participant's samples were unavailable for analysis.
Measured using a hexokinase-2 ELISA with a range of 1.56 ng/mL - 100 ng/mL on tumor tissue.
Outcome measures
| Measure |
Posaconazole
n=2 Participants
Participants will be taking 300 mg of the study drug (three 100 mg tablets) by mouth twice a day the first day and then 300 mg once a day until the day of biopsy or surgery. On the day of biopsy or surgery, participants will take their medication the morning of their biopsy or surgery (before the operation). Participants will then take the last dose of the medication in the morning of the day after their biopsy or surgery. Participants will be given 12 days' worth of the study drug (pills) and verbally instructed how and when to take them.
|
Control
n=3 Participants
Participants will not undergo any intervention.
|
|---|---|---|
|
Posaconazole Effect on Hexokinase 2 Concentration Within Tumor Tissue
|
NA ng/mg
Standard Deviation NA
Results were all less than the limit of detection.
|
NA ng/mg
Standard Deviation NA
Results were all less than the limit of detection.
|
SECONDARY outcome
Timeframe: Within 24 hours after biopsy or tumor resectionMeasured using Ki-67 proliferation index with of a range of 0% proliferation (no proliferation) to 100% (high proliferation). Ki-67 is a marker found in actively dividing cells. A higher Ki-67 value may signify a more aggressive tumor. Ki-67 is routinely measured during pathology analysis of tumor samples.
Outcome measures
| Measure |
Posaconazole
n=2 Participants
Participants will be taking 300 mg of the study drug (three 100 mg tablets) by mouth twice a day the first day and then 300 mg once a day until the day of biopsy or surgery. On the day of biopsy or surgery, participants will take their medication the morning of their biopsy or surgery (before the operation). Participants will then take the last dose of the medication in the morning of the day after their biopsy or surgery. Participants will be given 12 days' worth of the study drug (pills) and verbally instructed how and when to take them.
|
Control
n=4 Participants
Participants will not undergo any intervention.
|
|---|---|---|
|
Posaconazole Effect on Tumor Proliferation in Tumor Tissue
|
25 % cells actively dividing
Standard Deviation 0.05
|
39 % cells actively dividing
Standard Deviation 0.134
|
SECONDARY outcome
Timeframe: Within 24 hours after biopsy or tumor resectionPopulation: The fourth control participant's samples were unavailable for analysis.
Measured using a BCL-2 ELISA with a range of 0.156 ng/mL to 10 ng/mL.
Outcome measures
| Measure |
Posaconazole
n=2 Participants
Participants will be taking 300 mg of the study drug (three 100 mg tablets) by mouth twice a day the first day and then 300 mg once a day until the day of biopsy or surgery. On the day of biopsy or surgery, participants will take their medication the morning of their biopsy or surgery (before the operation). Participants will then take the last dose of the medication in the morning of the day after their biopsy or surgery. Participants will be given 12 days' worth of the study drug (pills) and verbally instructed how and when to take them.
|
Control
n=3 Participants
Participants will not undergo any intervention.
|
|---|---|---|
|
Posaconazole Effect on Cell Death in Tumor Tissue
|
0.3078 ng/mL
Standard Deviation 0.2418
|
0.5363 ng/mL
Standard Deviation 0.2908
|
SECONDARY outcome
Timeframe: Within 24 hours after biopsy or tumor resectionPopulation: The fourth control participant's samples were unavailable for analysis.
Measured using a CD31 ELISA with a range of 31.2 pg/mL to 2000 pg/mL.
Outcome measures
| Measure |
Posaconazole
n=2 Participants
Participants will be taking 300 mg of the study drug (three 100 mg tablets) by mouth twice a day the first day and then 300 mg once a day until the day of biopsy or surgery. On the day of biopsy or surgery, participants will take their medication the morning of their biopsy or surgery (before the operation). Participants will then take the last dose of the medication in the morning of the day after their biopsy or surgery. Participants will be given 12 days' worth of the study drug (pills) and verbally instructed how and when to take them.
|
Control
n=3 Participants
Participants will not undergo any intervention.
|
|---|---|---|
|
Posaconazole Effect on Angiogenesis in Tumor Tissue
|
2130 pg/mL
Standard Deviation 1500
|
2240 pg/mL
Standard Deviation 499.8
|
SECONDARY outcome
Timeframe: Collected over a 24-hour period after surgery (biopsy or resection)Population: The entirety of the cerebrospinal fluid collected from the microdialysis catheters was consumed during the posaconazole concentration analysis. Since the catheters yielded a low volume of sample, none was available to test lactate concentration, thus, correlation of posaconazole profile to lactate concentration could not be analyzed for any of the participants.
The concentration vs. time profile of posaconazole will be correlated with the concentration vs. time profile of lactate in the cerebrospinal fluid.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Collected over a 24-hour period after surgery (biopsy or resection)Population: The entirety of the cerebrospinal fluid collected from the microdialysis catheters was consumed during the posaconazole concentration analysis. Since the catheters yielded a low volume of sample, none was available to test pyruvate concentration, thus, correlation of posaconazole profile to pyruvate concentration could not be analyzed for any of the participants.
The concentration vs. time profile of posaconazole will be correlated with the concentration vs. time profile of pyruvate in the cerebrospinal fluid.
Outcome measures
Outcome data not reported
Adverse Events
Posaconazole
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Control
Serious events: 2 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Posaconazole
n=2 participants at risk
Participants will be taking 300 mg of the study drug (three 100 mg tablets) by mouth twice a day the first day and then 300 mg once a day until the day of biopsy or surgery. On the day of biopsy or surgery, participants will take their medication the morning of their biopsy or surgery (before the operation). Participants will then take the last dose of the medication in the morning of the day after their biopsy or surgery. Participants will be given 12 days' worth of the study drug (pills) and verbally instructed how and when to take them.
|
Control
n=4 participants at risk
Participants will not undergo any intervention.
|
|---|---|---|
|
Injury, poisoning and procedural complications
Wound Complication
|
0.00%
0/2 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
25.0%
1/4 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
|
Vascular disorders
Hypertension
|
0.00%
0/2 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
25.0%
1/4 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
Other adverse events
| Measure |
Posaconazole
n=2 participants at risk
Participants will be taking 300 mg of the study drug (three 100 mg tablets) by mouth twice a day the first day and then 300 mg once a day until the day of biopsy or surgery. On the day of biopsy or surgery, participants will take their medication the morning of their biopsy or surgery (before the operation). Participants will then take the last dose of the medication in the morning of the day after their biopsy or surgery. Participants will be given 12 days' worth of the study drug (pills) and verbally instructed how and when to take them.
|
Control
n=4 participants at risk
Participants will not undergo any intervention.
|
|---|---|---|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
50.0%
1/2 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
25.0%
1/4 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
50.0%
1/2 • Number of events 2 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
75.0%
3/4 • Number of events 3 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
|
Investigations
Platelet Count Decreased
|
50.0%
1/2 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
25.0%
1/4 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
|
Psychiatric disorders
Agitation
|
0.00%
0/2 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
25.0%
1/4 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
|
Psychiatric disorders
Confusion
|
0.00%
0/2 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
25.0%
1/4 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
|
Psychiatric disorders
Delirium
|
0.00%
0/2 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
25.0%
1/4 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
|
Investigations
Alanine Aminotransferase Increased
|
50.0%
1/2 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
0.00%
0/4 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
|
Investigations
Creatinine Increased
|
50.0%
1/2 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
0.00%
0/4 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
50.0%
1/2 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
0.00%
0/4 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
|
Metabolism and nutrition disorders
Hyponatremia
|
50.0%
1/2 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
0.00%
0/4 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
|
Investigations
Lymphocyte Count Decreased
|
50.0%
1/2 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
0.00%
0/4 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
|
Investigations
Renal Disorder - BUN Increased
|
50.0%
1/2 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
0.00%
0/4 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
|
Psychiatric disorders
Insomnia
|
0.00%
0/2 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
25.0%
1/4 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
|
Nervous system disorders
Asymptomatic Stroke
|
50.0%
1/2 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
0.00%
0/4 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
|
General disorders
Fatigue
|
50.0%
1/2 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
0.00%
0/4 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
|
Renal and urinary disorders
Urinary Retention
|
0.00%
0/2 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
25.0%
1/4 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
|
Investigations
Low HCT
|
0.00%
0/2 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
25.0%
1/4 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
|
Investigations
Low RBC
|
0.00%
0/2 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
25.0%
1/4 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
|
Investigations
Increased RDW
|
0.00%
0/2 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
25.0%
1/4 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
|
Metabolism and nutrition disorders
Hyperphosphatemia
|
50.0%
1/2 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
25.0%
1/4 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
|
Blood and lymphatic system disorders
Anemia
|
0.00%
0/2 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
25.0%
1/4 • Number of events 1 • Adverse event data were collected from the Baseline visit through Visit 7 (14+/-7 days Post-Op).
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place