Trial Outcomes & Findings for Biomarker and Safety Study of Clozapine in Patients With Benign Ethnic Neutropenia (BEN) (NCT NCT02404155)
NCT ID: NCT02404155
Last Updated: 2023-01-31
Results Overview
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
274 participants
Primary outcome timeframe
24 week period baseline and endpoint
Results posted on
2023-01-31
Participant Flow
Participant milestones
| Measure |
Clozapine
Clozapine
|
|---|---|
|
Overall Study
STARTED
|
274
|
|
Overall Study
COMPLETED
|
227
|
|
Overall Study
NOT COMPLETED
|
47
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Biomarker and Safety Study of Clozapine in Patients With Benign Ethnic Neutropenia (BEN)
Baseline characteristics by cohort
| Measure |
Clozapine
n=274 Participants
Clozapine
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
274 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
40.7 years
STANDARD_DEVIATION 11.5 • n=5 Participants
|
|
Sex: Female, Male
Female
|
106 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
168 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
137 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
130 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
255 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
11 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
150 participants
n=5 Participants
|
|
Region of Enrollment
Nigeria
|
124 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 24 week period baseline and endpointPopulation: Genetic information was only able to be collected and analyzed on 249 of t he 274 overall participants.
Outcome measures
| Measure |
Clozapine
n=249 Participants
Clozapine
|
|---|---|
|
Change in White Blood Cell (WBC) (mm3) and Absolute Neutrophil Counts (ANC) (mm3) in Persons According to Presence of the DARC Null Allele.
Baseline ANC for CC genotype
|
2755.1 cells/mm3
Standard Deviation 1271.5
|
|
Change in White Blood Cell (WBC) (mm3) and Absolute Neutrophil Counts (ANC) (mm3) in Persons According to Presence of the DARC Null Allele.
Endpoint ANC for CC genotype
|
3079.9 cells/mm3
Standard Deviation 1563.0
|
|
Change in White Blood Cell (WBC) (mm3) and Absolute Neutrophil Counts (ANC) (mm3) in Persons According to Presence of the DARC Null Allele.
Baseline ANC for CT genotype
|
4360.6 cells/mm3
Standard Deviation 1779.0
|
|
Change in White Blood Cell (WBC) (mm3) and Absolute Neutrophil Counts (ANC) (mm3) in Persons According to Presence of the DARC Null Allele.
Endpoint ANC for CT genotype
|
4450.3 cells/mm3
Standard Deviation 2398.0
|
|
Change in White Blood Cell (WBC) (mm3) and Absolute Neutrophil Counts (ANC) (mm3) in Persons According to Presence of the DARC Null Allele.
Baseline WBC for CC genotype
|
5478.9 cells/mm3
Standard Deviation 1688.1
|
|
Change in White Blood Cell (WBC) (mm3) and Absolute Neutrophil Counts (ANC) (mm3) in Persons According to Presence of the DARC Null Allele.
Endpoint WBC for CC genotype
|
5766.1 cells/mm3
Standard Deviation 1946.4
|
|
Change in White Blood Cell (WBC) (mm3) and Absolute Neutrophil Counts (ANC) (mm3) in Persons According to Presence of the DARC Null Allele.
Baseline WBC for CT genotype
|
7802.0 cells/mm3
Standard Deviation 2142.0
|
|
Change in White Blood Cell (WBC) (mm3) and Absolute Neutrophil Counts (ANC) (mm3) in Persons According to Presence of the DARC Null Allele.
Endpoint WBC for CT genotype
|
7702.7 cells/mm3
Standard Deviation 2607.5
|
PRIMARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Clozapine
n=274 Participants
Clozapine
|
|---|---|
|
Number of Episodes of Agranulocytosis (Count).
|
1 Episodes
|
Adverse Events
Clozapine
Serious events: 19 serious events
Other events: 264 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Clozapine
n=274 participants at risk
Clozapine
|
|---|---|
|
Gastrointestinal disorders
Prolapsed Rectum
|
0.36%
1/274 • For each participants 6 months of enrollment
|
|
Musculoskeletal and connective tissue disorders
Stroke
|
0.73%
2/274 • For each participants 6 months of enrollment
|
|
Nervous system disorders
Seizure
|
0.73%
2/274 • For each participants 6 months of enrollment
|
|
Cardiac disorders
Pulmonary Emobolism
|
0.36%
1/274 • For each participants 6 months of enrollment
|
|
Cardiac disorders
Myocarditis
|
0.36%
1/274 • For each participants 6 months of enrollment
|
|
General disorders
Hospitalization for Clinical Worsening
|
1.1%
3/274 • For each participants 6 months of enrollment
|
|
Gastrointestinal disorders
Diverticulitis
|
0.36%
1/274 • For each participants 6 months of enrollment
|
|
Blood and lymphatic system disorders
Severe Neutropenia
|
0.36%
1/274 • For each participants 6 months of enrollment
|
|
General disorders
Dehydration
|
1.1%
3/274 • For each participants 6 months of enrollment
|
|
Infections and infestations
Urinary Tract Infection
|
0.36%
1/274 • For each participants 6 months of enrollment
|
|
General disorders
Atypical Neuroleptic Malignant Syndrome/ Drug Fever
|
0.36%
1/274 • For each participants 6 months of enrollment
|
|
Infections and infestations
Infection/Cellulitis
|
0.73%
2/274 • For each participants 6 months of enrollment
|
|
General disorders
Pneumonia
|
0.36%
1/274 • For each participants 6 months of enrollment
|
|
Gastrointestinal disorders
Severe Constipation
|
0.36%
1/274 • For each participants 6 months of enrollment
|
|
Gastrointestinal disorders
Diffuse Ileus
|
0.36%
1/274 • For each participants 6 months of enrollment
|
|
General disorders
Pleural Effusion
|
0.36%
1/274 • For each participants 6 months of enrollment
|
|
General disorders
Unresponsiveness
|
0.36%
1/274 • For each participants 6 months of enrollment
|
Other adverse events
| Measure |
Clozapine
n=274 participants at risk
Clozapine
|
|---|---|
|
Gastrointestinal disorders
Abdominal Pain
|
33.2%
91/274 • For each participants 6 months of enrollment
|
|
General disorders
Anorexia
|
18.6%
51/274 • For each participants 6 months of enrollment
|
|
General disorders
Bruising
|
4.4%
12/274 • For each participants 6 months of enrollment
|
|
Gastrointestinal disorders
Constipation
|
32.1%
88/274 • For each participants 6 months of enrollment
|
|
Gastrointestinal disorders
Diarrhea
|
23.4%
64/274 • For each participants 6 months of enrollment
|
|
General disorders
Dizziness
|
40.9%
112/274 • For each participants 6 months of enrollment
|
|
General disorders
Dry Mouth
|
23.7%
65/274 • For each participants 6 months of enrollment
|
|
Gastrointestinal disorders
Enuresis
|
25.5%
70/274 • For each participants 6 months of enrollment
|
|
General disorders
Fever
|
12.8%
35/274 • For each participants 6 months of enrollment
|
|
General disorders
Headache
|
31.4%
86/274 • For each participants 6 months of enrollment
|
|
General disorders
Insomnia
|
19.3%
53/274 • For each participants 6 months of enrollment
|
|
General disorders
Malaise
|
52.6%
144/274 • For each participants 6 months of enrollment
|
|
General disorders
Mucosal
|
8.0%
22/274 • For each participants 6 months of enrollment
|
|
Gastrointestinal disorders
Nausea
|
28.1%
77/274 • For each participants 6 months of enrollment
|
|
General disorders
Rash
|
13.1%
36/274 • For each participants 6 months of enrollment
|
|
Nervous system disorders
Restlessness
|
28.8%
79/274 • For each participants 6 months of enrollment
|
|
Endocrine disorders
Salivation
|
68.2%
187/274 • For each participants 6 months of enrollment
|
|
General disorders
Sedation
|
52.9%
145/274 • For each participants 6 months of enrollment
|
|
Musculoskeletal and connective tissue disorders
Stiffness
|
24.1%
66/274 • For each participants 6 months of enrollment
|
|
General disorders
Sore Throat
|
17.9%
49/274 • For each participants 6 months of enrollment
|
|
Nervous system disorders
Tremors
|
26.6%
73/274 • For each participants 6 months of enrollment
|
|
General disorders
Urticaria
|
21.5%
59/274 • For each participants 6 months of enrollment
|
|
Gastrointestinal disorders
Vomiting
|
22.3%
61/274 • For each participants 6 months of enrollment
|
|
Gastrointestinal disorders
Weight loss
|
10.6%
29/274 • For each participants 6 months of enrollment
|
|
General disorders
Tinnitus
|
13.5%
37/274 • For each participants 6 months of enrollment
|
|
General disorders
Abnormal Ejaculation/Erectile Dysfunction
|
0.73%
2/274 • For each participants 6 months of enrollment
|
|
Cardiac disorders
Chest Pain
|
2.2%
6/274 • For each participants 6 months of enrollment
|
|
Metabolism and nutrition disorders
Diabetes/Hyperglycemia
|
1.1%
3/274 • For each participants 6 months of enrollment
|
|
General disorders
Disorientation/Confusion
|
0.73%
2/274 • For each participants 6 months of enrollment
|
|
General disorders
Edema
|
0.73%
2/274 • For each participants 6 months of enrollment
|
|
Cardiac disorders
Myocarditis
|
0.36%
1/274 • For each participants 6 months of enrollment
|
|
General disorders
Myoclonus
|
1.1%
3/274 • For each participants 6 months of enrollment
|
Additional Information
Dr. Deanna L. Kelly, Pharm.D., BCPP
Maryland Psychiatric Research Center
Phone: 410-402-6860
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place