Trial Outcomes & Findings for The Use of Trifluoperazine in Transfusion Dependent DBA (NCT NCT03966053)

Last Updated: 2022-12-14

Results Overview

Each subject will undergo weekly safety assessment using the Simpson-Angus Extrapyramidal Side Effects Scale to determine the safety of TFP in this new population of patients. The subjects will also undergo weekly bloodwork to evaluate for any liver or kidney abnormalities as well as a complete blood count and reticulocyte count. All dosed subjects will be followed for an additional 1 week after discontinuing study drug (post-study safety follow-up). There will be no more than 6 subjects enrolled at any particular time. Treatment will be discontinued for any subject if their Hb is \> 12 gm/dL , and not associated with RBC transfusions.

Recruitment status

TERMINATED

Study phase

PHASE1/PHASE2

Target enrollment

2 participants

Primary outcome timeframe

The subjects will be evaluated weekly for 4 weeks after the start of the 21-day course, 3 weeks while on the study drug and one week after completion.

Results posted on

2022-12-14

Participant Flow

Participant milestones

Participant milestones
Measure	Cohort A Cohort A: Three subjects will receive Trifluoperazine (TFP) 1 mg PO daily. * If there is no non-neurologic toxicity Grade 3 at the end of the 21 days, Cohort B will start. * If 1/3 subjects in Cohort A demonstrates toxicity Grade 3, an additional 3 subjects will be enrolled in Cohort A. * If 2 or more of the 6 subjects in Cohort A demonstrate toxicity Grade 3, the trial will be stopped; no MTD will be declared. * If less than 2 of the 6 subjects in Cohort A demonstrate toxicity Grade 3 within 21 days of starting therapy, Cohort B will start. Trifluoperazine: Trifluoperazine (TFP)1mg, 2mg, 5mg, or 10mg will be given once daily by mouth for 21 days
Overall Study STARTED	2
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Cohort A Cohort A: Three subjects will receive Trifluoperazine (TFP) 1 mg PO daily. * If there is no non-neurologic toxicity Grade 3 at the end of the 21 days, Cohort B will start. * If 1/3 subjects in Cohort A demonstrates toxicity Grade 3, an additional 3 subjects will be enrolled in Cohort A. * If 2 or more of the 6 subjects in Cohort A demonstrate toxicity Grade 3, the trial will be stopped; no MTD will be declared. * If less than 2 of the 6 subjects in Cohort A demonstrate toxicity Grade 3 within 21 days of starting therapy, Cohort B will start. Trifluoperazine: Trifluoperazine (TFP)1mg, 2mg, 5mg, or 10mg will be given once daily by mouth for 21 days
Overall Study Withdrawal by subject prior to starting study drug.	1

Baseline Characteristics

The Use of Trifluoperazine in Transfusion Dependent DBA

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cohort A n=2 Participants Cohort A: Three subjects will receive Trifluoperazine (TFP) 1 mg PO daily. * If there is no non-neurologic toxicity Grade 3 at the end of the 21 days, Cohort B will start. * If 1/3 subjects in Cohort A demonstrates toxicity Grade 3, an additional 3 subjects will be enrolled in Cohort A. * If 2 or more of the 6 subjects in Cohort A demonstrate toxicity Grade 3, the trial will be stopped; no MTD will be declared. * If less than 2 of the 6 subjects in Cohort A demonstrate toxicity Grade 3 within 21 days of starting therapy, Cohort B will start. Trifluoperazine: Trifluoperazine (TFP)1mg, 2mg, 5mg, or 10mg will be given once daily by mouth for 21 days
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 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	0 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: The subjects will be evaluated weekly for 4 weeks after the start of the 21-day course, 3 weeks while on the study drug and one week after completion.

Population: One subject received study drug. The other subject did not receive study drug.

Outcome measures

Outcome measures
Measure	Cohort A n=1 Participants Cohort A: Three subjects will receive Trifluoperazine (TFP) 1 mg PO daily. * If there is no non-neurologic toxicity Grade 3 at the end of the 21 days, Cohort B will start. * If 1/3 subjects in Cohort A demonstrates toxicity Grade 3, an additional 3 subjects will be enrolled in Cohort A. * If 2 or more of the 6 subjects in Cohort A demonstrate toxicity Grade 3, the trial will be stopped; no MTD will be declared. * If less than 2 of the 6 subjects in Cohort A demonstrate toxicity Grade 3 within 21 days of starting therapy, Cohort B will start. Trifluoperazine: Trifluoperazine (TFP)1mg, 2mg, 5mg, or 10mg will be given once daily by mouth for 21 days
Number of Participants With Treatment-related Adverse Events as Assessed by the Simpson-Angus Scale and CTCAE v4.0	0 Participants

Adverse Events

Cohort A

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Adrianna Vlachos, MD

Feinstein Institutes for Medical Research

Phone: 5165623757

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place