Trial Outcomes & Findings for Safety, Tolerability, and Pharmacokinetic Study of EVP-6124 in Patients With Schizophrenia (NCT NCT01556763)

NCT ID: NCT01556763

Last Updated: 2012-06-21

Results Overview

Safety and tolerability was measured by number of reported adverse events (serious and non-serious) and repeated clinical evaluation of physical examinations, vital signs, 12-lead electrocardiogram (ECG), 24-hour continuous cardiac monitoring, and laboratory tests (hematology/blood chemistry/urinalysis).

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

21 participants

Primary outcome timeframe

Screening (Day -5 for continuous cardiac monitoring) to Day 22

Results posted on

2012-06-21

Participant Flow

Participant milestones

Participant milestones
Measure
Placebo
Matching placebo was administered as one capsule per day for 21 days.
EVP-6124 (1.0 mg/Day)
EVP-6124 was administered as one 1.0 mg capsule per day for 21 days.
EVP-6124 (0.3 mg/Day)
EVP-6124 was administered as one 0.3 mg capsule per day for 21 days.
Overall Study
STARTED
4
9
8
Overall Study
COMPLETED
4
8
8
Overall Study
NOT COMPLETED
0
1
0

Reasons for withdrawal

Reasons for withdrawal
Measure
Placebo
Matching placebo was administered as one capsule per day for 21 days.
EVP-6124 (1.0 mg/Day)
EVP-6124 was administered as one 1.0 mg capsule per day for 21 days.
EVP-6124 (0.3 mg/Day)
EVP-6124 was administered as one 0.3 mg capsule per day for 21 days.
Overall Study
Withdrawal by Subject
0
1
0

Baseline Characteristics

Safety, Tolerability, and Pharmacokinetic Study of EVP-6124 in Patients With Schizophrenia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
EVP-6124 (1.0 mg/Day)
n=9 Participants
EVP-6124 was administered as one 1.0 mg capsule per day for 21 days.
Placebo
n=4 Participants
Matching placebo was administered as one capsule per day for 21 days.
EVP-6124 (0.3 mg/Day)
n=8 Participants
EVP-6124 was administered as one 0.3 mg capsule per day for 21 days.
Total
n=21 Participants
Total of all reporting groups
Age, Categorical
<=18 years
0 Participants
n=7 Participants
0 Participants
n=5 Participants
0 Participants
n=5 Participants
0 Participants
n=4 Participants
Age, Categorical
Between 18 and 65 years
9 Participants
n=7 Participants
4 Participants
n=5 Participants
8 Participants
n=5 Participants
21 Participants
n=4 Participants
Age, Categorical
>=65 years
0 Participants
n=7 Participants
0 Participants
n=5 Participants
0 Participants
n=5 Participants
0 Participants
n=4 Participants
Age Continuous
43.1 years
STANDARD_DEVIATION 11.0 • n=7 Participants
40.0 years
STANDARD_DEVIATION 11.6 • n=5 Participants
51.4 years
STANDARD_DEVIATION 6.9 • n=5 Participants
45.7 years
STANDARD_DEVIATION 10.4 • n=4 Participants
Sex: Female, Male
Female
3 Participants
n=7 Participants
0 Participants
n=5 Participants
3 Participants
n=5 Participants
6 Participants
n=4 Participants
Sex: Female, Male
Male
6 Participants
n=7 Participants
4 Participants
n=5 Participants
5 Participants
n=5 Participants
15 Participants
n=4 Participants
Region of Enrollment
United States
9 participants
n=7 Participants
4 participants
n=5 Participants
8 participants
n=5 Participants
21 participants
n=4 Participants

PRIMARY outcome

Timeframe: Screening (Day -5 for continuous cardiac monitoring) to Day 22

Population: All randomized patients who ingested at least one dose of study drug or placebo.

Safety and tolerability was measured by number of reported adverse events (serious and non-serious) and repeated clinical evaluation of physical examinations, vital signs, 12-lead electrocardiogram (ECG), 24-hour continuous cardiac monitoring, and laboratory tests (hematology/blood chemistry/urinalysis).

Outcome measures

Outcome measures
Measure
Placebo
n=4 Participants
Matching placebo was administered as one capsule per day for 21 days.
EVP-6124 (1.0 mg/Day)
n=9 Participants
EVP-6124 was administered as one 1.0 mg capsule per day for 21 days.
EVP-6124 (0.3 mg/Day)
n=8 Participants
EVP-6124 was administered as one 0.3 mg capsule per day for 21 days.
Number of Participants With Serious and Non-serious Adverse Events Spontaneously Reported by Subject and/or Observed by Investigator.
No Adverse Events Reported
4 participants
0
4 participants
0
3 participants
0
Number of Participants With Serious and Non-serious Adverse Events Spontaneously Reported by Subject and/or Observed by Investigator.
Serious Adverse Events
0 participants
0
0 participants
0
1 participants
0
Number of Participants With Serious and Non-serious Adverse Events Spontaneously Reported by Subject and/or Observed by Investigator.
Non-Serious Adverse Events
0 participants
0
5 participants
0
5 participants
0

PRIMARY outcome

Timeframe: Days 1 and 21

Population: All patients receiving aripiprazole.

Blood samples for pharmacokinetic (PK) analyses were taken before dosing with EVP-6124 on Days 1 and 21.

Outcome measures

Outcome measures
Measure
Placebo
n=7 Participants
Matching placebo was administered as one capsule per day for 21 days.
EVP-6124 (1.0 mg/Day)
n=3 Participants
EVP-6124 was administered as one 1.0 mg capsule per day for 21 days.
EVP-6124 (0.3 mg/Day)
EVP-6124 was administered as one 0.3 mg capsule per day for 21 days.
EVP-6124 Maximum Plasma Concentration (Cmax), Patients on Aripiprazole
Day 1
581.0 pg/mL
Standard Deviation 149.8
210.0 pg/mL
Standard Deviation 39.3
EVP-6124 Maximum Plasma Concentration (Cmax), Patients on Aripiprazole
Day 21
2058.6 pg/mL
Standard Deviation 393.2
968.3 pg/mL
Standard Deviation 90.1

PRIMARY outcome

Timeframe: Days 1 and 21

Population: All patients receiving aripiprazole.

Blood samples for PK analyses were taken before dosing with EVP-6124 on Days 1 and 21.

Outcome measures

Outcome measures
Measure
Placebo
n=7 Participants
Matching placebo was administered as one capsule per day for 21 days.
EVP-6124 (1.0 mg/Day)
n=3 Participants
EVP-6124 was administered as one 1.0 mg capsule per day for 21 days.
EVP-6124 (0.3 mg/Day)
EVP-6124 was administered as one 0.3 mg capsule per day for 21 days.
EVP-6124 Time to Maximum Concentration (Tmax), Patients on Aripiprazole
Day 21
6.0 hr
Interval 2.0 to 8.0
8.0 hr
Interval 1.0 to 8.0
EVP-6124 Time to Maximum Concentration (Tmax), Patients on Aripiprazole
Day 1
8.0 hr
Interval 2.0 to 8.0
8.0 hr
Interval 6.0 to 8.0

PRIMARY outcome

Timeframe: Days 1 and 21

Population: All patients receiving aripiprazole.

Blood samples for PK analyses were taken before dosing with EVP-6124 on Days 1 and 21.

Outcome measures

Outcome measures
Measure
Placebo
n=7 Participants
Matching placebo was administered as one capsule per day for 21 days.
EVP-6124 (1.0 mg/Day)
n=3 Participants
EVP-6124 was administered as one 1.0 mg capsule per day for 21 days.
EVP-6124 (0.3 mg/Day)
EVP-6124 was administered as one 0.3 mg capsule per day for 21 days.
EVP-6124 Area Under the Curve (AUC[0-24 h]), Patients on Aripiprazole
Day 1
10,966 pg*hr/mL
Standard Deviation 2831
3838 pg*hr/mL
Standard Deviation 1044
EVP-6124 Area Under the Curve (AUC[0-24 h]), Patients on Aripiprazole
Day 21
42,042 pg*hr/mL
Standard Deviation 9623
20,560 pg*hr/mL
Standard Deviation 2699

PRIMARY outcome

Timeframe: Days 1 and 21

Population: Patients receiving aripiprazole for whom blood samples were available for analysis.

Blood samples for PK analyses were taken before dosing with EVP-6124 on Days 1 and 21.

Outcome measures

Outcome measures
Measure
Placebo
n=1 Participants
Matching placebo was administered as one capsule per day for 21 days.
EVP-6124 (1.0 mg/Day)
n=1 Participants
EVP-6124 was administered as one 1.0 mg capsule per day for 21 days.
EVP-6124 (0.3 mg/Day)
EVP-6124 was administered as one 0.3 mg capsule per day for 21 days.
EVP-6124 Half-life (T[1/2]), Patients on Aripiprazole
Day 1
39.0 hr
Standard Deviation NA
Data for only one patient were available.
43.1 hr
Standard Deviation NA
Data for only one patient were available.
EVP-6124 Half-life (T[1/2]), Patients on Aripiprazole
Day 21
NA hr
Standard Deviation NA
The patient withdrew consent after receiving study drug for 3 days.
116.6 hr
Standard Deviation NA
Data for only one patient were available.

PRIMARY outcome

Timeframe: Days 1 and 21

Population: All patients receiving paliperidone/risperidone.

Blood samples for PK analyses were taken before dosing with EVP-6124 on Days 1 and 21.

Outcome measures

Outcome measures
Measure
Placebo
n=1 Participants
Matching placebo was administered as one capsule per day for 21 days.
EVP-6124 (1.0 mg/Day)
n=5 Participants
EVP-6124 was administered as one 1.0 mg capsule per day for 21 days.
EVP-6124 (0.3 mg/Day)
EVP-6124 was administered as one 0.3 mg capsule per day for 21 days.
EVP-6124 Maximum Plasma Concentration (Cmax), Patients on Paliperidone/Risperidone
Day 1
315.0 pg/mL
Standard Deviation NA
Data for only one patient were available.
165.0 pg/mL
Standard Deviation 40.2
EVP-6124 Maximum Plasma Concentration (Cmax), Patients on Paliperidone/Risperidone
Day 21
1510.0 pg/mL
Standard Deviation NA
Data for only one patient were available.
545.4 pg/mL
Standard Deviation 130.2

PRIMARY outcome

Timeframe: Days 1 and 21

Population: All patients receiving paliperidone/risperidone.

Blood samples for PK analyses were taken before dosing with EVP-6124 on Days 1 and 21.

Outcome measures

Outcome measures
Measure
Placebo
n=1 Participants
Matching placebo was administered as one capsule per day for 21 days.
EVP-6124 (1.0 mg/Day)
n=5 Participants
EVP-6124 was administered as one 1.0 mg capsule per day for 21 days.
EVP-6124 (0.3 mg/Day)
EVP-6124 was administered as one 0.3 mg capsule per day for 21 days.
EVP-6124 Time to Maximum Concentration (Tmax), Patients on Paliperidone/Risperidone
Day 1
6.0 hr
Data for only one patient were available.
8.0 hr
Interval 6.0 to 8.0
EVP-6124 Time to Maximum Concentration (Tmax), Patients on Paliperidone/Risperidone
Day 21
6.0 hr
Data for only one patient were available.
8.0 hr
Interval 8.0 to 8.0

PRIMARY outcome

Timeframe: Days 1 and 21

Population: All patients receiving paliperidone/risperidone.

Blood samples for PK analyses were taken before dosing with EVP-6124 on Days 1 and 21.

Outcome measures

Outcome measures
Measure
Placebo
n=1 Participants
Matching placebo was administered as one capsule per day for 21 days.
EVP-6124 (1.0 mg/Day)
n=5 Participants
EVP-6124 was administered as one 1.0 mg capsule per day for 21 days.
EVP-6124 (0.3 mg/Day)
EVP-6124 was administered as one 0.3 mg capsule per day for 21 days.
EVP-6124 Area Under the Curve (AUC[0-24 h]), Patients on Paliperidone/Risperidone
Day 1
6359 pg*hr/ml
Standard Deviation NA
Data for only one patient were available.
3110 pg*hr/ml
Standard Deviation 852
EVP-6124 Area Under the Curve (AUC[0-24 h]), Patients on Paliperidone/Risperidone
Day 21
33,042 pg*hr/ml
Standard Deviation NA
Data for only one patient were available.
11,888 pg*hr/ml
Standard Deviation 3095

PRIMARY outcome

Timeframe: Days 1 and 21

Population: Patients receiving paliperidone/risperidone for whom blood samples were available for analysis.

Blood samples for PK analyses were taken before dosing with EVP-6124 on Days 1 and 21.

Outcome measures

Outcome measures
Measure
Placebo
n=1 Participants
Matching placebo was administered as one capsule per day for 21 days.
EVP-6124 (1.0 mg/Day)
n=2 Participants
EVP-6124 was administered as one 1.0 mg capsule per day for 21 days.
EVP-6124 (0.3 mg/Day)
EVP-6124 was administered as one 0.3 mg capsule per day for 21 days.
EVP-6124 Half-life (T[1/2]), Patients on Paliperidone/Risperidone
Day 1
92.0 hr
Standard Deviation NA
Data for only one patient were available.
45.8 hr
Standard Deviation 17.7
EVP-6124 Half-life (T[1/2]), Patients on Paliperidone/Risperidone
Day 21
NA hr
Standard Deviation NA
Sample not analyzed.
78.1 hr
Standard Deviation 8.3

SECONDARY outcome

Timeframe: Days -1 to 20

Population: Subjects providing valid and measurable N100 responses.

N100 auditory evoked potential response (amplitude measured in microvolts) using the sensory gating paradigm. Measured by electroencephalography (EEG) as the amplitude ratio of test stimulus to conditioning stimulus. Plotted on a unitless scale of 0 to 2. Normalization is suggested by a lower value.

Outcome measures

Outcome measures
Measure
Placebo
n=2 Participants
Matching placebo was administered as one capsule per day for 21 days.
EVP-6124 (1.0 mg/Day)
n=5 Participants
EVP-6124 was administered as one 1.0 mg capsule per day for 21 days.
EVP-6124 (0.3 mg/Day)
n=5 Participants
EVP-6124 was administered as one 0.3 mg capsule per day for 21 days.
N100 Gating Ratio
1.648 ratio
Standard Error 0.29
0.801 ratio
Standard Error 0.19
0.951 ratio
Standard Error 0.20

SECONDARY outcome

Timeframe: Days -1 to 20

Population: Subjects providing valid and measurable P50 responses.

P50 auditory evoked potential response (amplitude measured in microvolts) using sensory gating paradigm. Measured by EEG as amplitude difference (conditioning stimulus minus test stimulus). Plotted on a scale of -0.2 to 0.8 microvolts. Normalization is suggested by a higher value.

Outcome measures

Outcome measures
Measure
Placebo
n=2 Participants
Matching placebo was administered as one capsule per day for 21 days.
EVP-6124 (1.0 mg/Day)
n=5 Participants
EVP-6124 was administered as one 1.0 mg capsule per day for 21 days.
EVP-6124 (0.3 mg/Day)
n=5 Participants
EVP-6124 was administered as one 0.3 mg capsule per day for 21 days.
P50 Amplitude Difference
-0.17 microvolts
Standard Error 0.38
0.67 microvolts
Standard Error 0.21
-0.06 microvolts
Standard Error 0.25

SECONDARY outcome

Timeframe: Days -1 to 20

Population: Subjects providing valid and measurable MMN responses.

Mismatch negativity (MMN) auditory evoked potential response (amplitude in microvolts) using orienting paradigm. Measured by EEG and calculated as the voltage difference over 100-200 msec following stimulus onset (rare stimulus minus frequent stimulus). Plotted on a scale of -1.2 to 0.2 microvolts. Normalization is suggested by a more negative value.

Outcome measures

Outcome measures
Measure
Placebo
n=4 Participants
Matching placebo was administered as one capsule per day for 21 days.
EVP-6124 (1.0 mg/Day)
n=8 Participants
EVP-6124 was administered as one 1.0 mg capsule per day for 21 days.
EVP-6124 (0.3 mg/Day)
n=7 Participants
EVP-6124 was administered as one 0.3 mg capsule per day for 21 days.
MMN Summed Amplitude
0.14 microvolts
Standard Error 0.33
-1.15 microvolts
Standard Error 0.24
-0.61 microvolts
Standard Error 0.25

SECONDARY outcome

Timeframe: Days -1 to 20

Population: Subjects providing valid and measurable P300 responses.

P300 auditory evoked potential response (amplitude in microvolts) using orienting paradigm. Measured by EEG and calculated as the peak amplitude over 250-500 msec following stimulus onset (rare stimulus minus frequent stimulus). Plotted on a scale of -0.4 to 1.2 microvolts. Normalization is suggested by a more positive value.

Outcome measures

Outcome measures
Measure
Placebo
n=4 Participants
Matching placebo was administered as one capsule per day for 21 days.
EVP-6124 (1.0 mg/Day)
n=8 Participants
EVP-6124 was administered as one 1.0 mg capsule per day for 21 days.
EVP-6124 (0.3 mg/Day)
n=7 Participants
EVP-6124 was administered as one 0.3 mg capsule per day for 21 days.
P300 Peak Amplitude
-0.3 microvolts
Standard Error 0.31
1.08 microvolts
Standard Error 0.22
0.78 microvolts
Standard Error 0.24

Adverse Events

Placebo

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

EVP-6124 (1.0 mg/Day)

Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths

EVP-6124 (0.3 mg/Day)

Serious events: 1 serious events
Other events: 5 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Placebo
n=4 participants at risk
Matching placebo was administered as one capsule per day for 21 days.
EVP-6124 (1.0 mg/Day)
n=9 participants at risk
EVP-6124 was administered as one 1.0 mg capsule per day for 21 days.
EVP-6124 (0.3 mg/Day)
n=8 participants at risk
EVP-6124 was administered as one 0.3 mg capsule per day for 21 days.
Psychiatric disorders
Psychiatric symptom
0.00%
0/4 • Screening to Day 22
0.00%
0/9 • Screening to Day 22
12.5%
1/8 • Number of events 1 • Screening to Day 22

Other adverse events

Other adverse events
Measure
Placebo
n=4 participants at risk
Matching placebo was administered as one capsule per day for 21 days.
EVP-6124 (1.0 mg/Day)
n=9 participants at risk
EVP-6124 was administered as one 1.0 mg capsule per day for 21 days.
EVP-6124 (0.3 mg/Day)
n=8 participants at risk
EVP-6124 was administered as one 0.3 mg capsule per day for 21 days.
Infections and infestations
Tinea cruris
0.00%
0/4 • Screening to Day 22
0.00%
0/9 • Screening to Day 22
12.5%
1/8 • Number of events 1 • Screening to Day 22
Investigations
Neutrophil count increased
0.00%
0/4 • Screening to Day 22
11.1%
1/9 • Number of events 1 • Screening to Day 22
0.00%
0/8 • Screening to Day 22
Investigations
White blood cell count increased
0.00%
0/4 • Screening to Day 22
11.1%
1/9 • Number of events 1 • Screening to Day 22
0.00%
0/8 • Screening to Day 22
Psychiatric disorders
Psychiatric symptom
0.00%
0/4 • Screening to Day 22
0.00%
0/9 • Screening to Day 22
12.5%
1/8 • Number of events 1 • Screening to Day 22
Skin and subcutaneous tissue disorders
Skin erosion
0.00%
0/4 • Screening to Day 22
0.00%
0/9 • Screening to Day 22
12.5%
1/8 • Number of events 1 • Screening to Day 22
Skin and subcutaneous tissue disorders
Skin irritation
0.00%
0/4 • Screening to Day 22
55.6%
5/9 • Number of events 5 • Screening to Day 22
62.5%
5/8 • Number of events 5 • Screening to Day 22

Additional Information

Maria Gawryl, Ph.D., Vice President, Regulatory Affairs & Drug Development

EnVivo Pharmaceuticals, Inc.

Phone: 617-225-4264

Results disclosure agreements

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

Restriction type: OTHER