The Use of Trifluoperazine in Transfusion Dependent DBA

• Register: ClinicalTrials.gov
• Last refreshed on: 26 October 2021
• Main ID: NCT03966053
• Date of registration: 23/05/2019
• Prospective Registration: Yes
• Primary sponsor: Adrianna Vlachos
• Public title: The Use of Trifluoperazine in Transfusion Dependent DBA DBA
• Scientific title: Phase I/II, Open Label Study to Determine Safety of Trifluoperazine (TFP) in Adults With Red Blood Cell Transfusion-Dependent Diamond Blackfan Anemia
• Date of first enrolment: September 13, 2019
• Target sample size: 2
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT03966053
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Adrianna Vlachos, MD
• Affiliation: Northwell Health

Key inclusion & exclusion criteria

Inclusion Criteria:

- Men and women age: 18 years and <65 years of age.

- Weight: =45 kilograms.

- DBA diagnosed according to the DBA criteria (Vlachos, 2008)

- RBC transfusion-dependence (defined as 2 units packed RBCs per 28 days averaged over
84 days [12 weeks] prior to study entry)

- Calculated creatinine clearance > 30 mL/min

- Karnofsky performance status scale score = 70

- Female subjects of childbearing potential must have a negative serum pregnancy test
and use highly effective methods of birth control during the study

- Male subjects must agree to use a latex condom during any sexual contact with females
of childbearing potential while participating in the study

- Agreement to adhere to the study visit schedule, understand and comply with all
protocol requirements.

Exclusion Criteria:

- Liver: aspartate aminotransferase (AST) > 5 x the upper limit of normal (ULN), alanine
aminotransferase (ALT) >5 x ULN, or bilirubin > 5 x ULN

- Heart disease (New York Heart Association classification of = 3)

- History of angina

- Uncontrolled hypertension

- Subjects currently responsive to corticosteroids for treatment of DBA.

- Treatment with another investigational drug or device <56 days pre-study entry.

- Pregnant or lactating females

- Any history of severe allergic reaction requiring the use of epinephrine

- Known hypersensitivity to the study drug or other phenothiazines

- History or presence of extrapyramidal signs

- History of cancer



Age minimum: 18 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Diamond Blackfan Anemia

Pure Red Cell Aplasia

Intervention(s)

Drug: Trifluoperazine

Primary Outcome(s)

Number of participants with treatment-related adverse events as assessed by the Simpson-Angus Scale and CTCAE v4.0 [Time Frame: 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.]

Secondary ID(s)

17-0748

Source(s) of Monetary Support

Please refer to primary and secondary sponsors