A Multi-Site Break Through Cancer Trial: Targeting Measurable Residual Disease in Patients With Acute Myeloid Leukemia: A Phase 1/2 Study of Tagraxofusp, Azacitidine, and Venetoclax

Status: Recruiting
Location: See all (2) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY

The purpose of this research study is to test the safety and efficacy of a new drug combination with three agents, azacitidine, venetoclax and tagraxofusp. Leftover (residual) leukemia disease that is not visible by eye can be increase the chance of disease recurrence. This research study is to determine if the combination therapy can safely help to control residual Acute Myeloid Leukemia (AML) and to prevent disease recurrence. The names of the study drugs involved in this study are: * Tagraxofusp (a type of CD123-directed cytotoxin) * Azacitidine (a type of standard of care cytidine nucleoside analog) * Venetoclax (a type of standard of care BCL-2 inhibitor)

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• Age ≥ 18 years.

• History of known diagnosis of Acute Myeloid Leukemia (including de novo, secondary or AML arising from MDS).

• Subjects must be in CR, CRi, or CRh with \<5% morphologic blasts in bone marrow

• Any evidence of CD123+ by central assessment.

• Participants must have measurable disease, defined as ≥ 0.1% by multiparametric flow cytometric assay as assessed by central laboratory

• ECOG performance status ≤2 (see Appendix A).

• Subjects must have adequate organ and marrow function as defined below:

‣ total bilirubin ≤ 1.5 x institutional upper limit of normal unless due to Gilbert or non-hepatic in origin

⁃ AST(SGOT) and ALT(SGPT) ≤ 3.0 × institutional upper limit of normal

⁃ Creatinine clearance ≥ 45 ml/min GFR by MDRD

• Albumin ≥ 3.2 g/dL

• Left ventricular ejection fraction ≥ institutional lower limit of normal by MUGA or echocardiogram within 30 days of first protocol treatment. This can be locally assessed.

• Pregnancy potential: Female subjects of childbearing potential must have negative results for pregnancy test. Females with reproductive potential are advised to use effective contraception during study treatment and for at least 6 months after last dose. Similarly, males with female partners of reproductive potential are advised to use effective contraception during treatment and for at least 3 months after the last dose. Men must agree to abstain from donating sperm.

• Subject is able and willing to adhere to the study visit schedule and other protocol requirements

Locations
United States
Massachusetts
Brigham and Women's Hospital
RECRUITING
Boston
Dana-Farber Cancer Institute
RECRUITING
Boston
Contact Information
Primary
Jacqueline Garcia, MD
jacqueline_garcia@dfci.harvard.edu
617-632-6349
Backup
Jacqueline Garcia, MD
jacqueline_garcia@dfci.harvard.edu
Time Frame
Start Date: 2026-02
Estimated Completion Date: 2028-12-31
Participants
Target number of participants: 31
Treatments
Experimental: Arm 1: Phase 1 Dose Escalation
Up to 12 participants will be enrolled in a standard 3+3 design to determine the maximum tolerated dose/RP2D for Tagraxofusp in combination with azacitidine and venetoclax starting at Dose Level 1 and escalating to Dose Level 2 according to dose-limiting toxicity rules.~* Baseline visit~* Cycles 1 through 2 (28 day cycles):~ * Days 1 through 7: Predetermined dose of Azacitidine 1x daily.~ * Days 4 through 6: Predetermined dose of Tagraxofusp 1x daily.~ * Days 1 and 14: Predetermined dose of Venetoclax 1x daily~ * Day 28: bone marrow aspiration and biopsy~* Cycles 3 through 24 (28 day cycles):~ * Days 1 through 7: Predetermined dose of Azacitidine 1x daily.~ * Days 4 through 6: Predetermined dose of Tagraxofusp 1x daily.~ * Days 1 and 14: Predetermined dose of Venetoclax 1x daily~* Bone marrow aspirations/biopsies on Day 1 of Cycles 4, 6, 9, 12, and then every 3 cycles.~* End of treatment visit with bone marrow aspiration and biopsy~* Follow up for up to 2 years
Experimental: Arm 2: Phase 2 Dose Expansion
19 Participants will receive Tagraxofusp at the RP2D in combination with Azacitidine and Venetoclax and will complete:~* Baseline visit~* Cycles 1 through 2 (28 day cycles):~ * Days 1 through 7: Predetermined dose of Azacitidine 1x daily.~ * Days 4 through 6: Predetermined dose of Tagraxofusp 1x daily.~ * Days 1 and 14: Predetermined dose of Venetoclax 1x daily~ * Day 28: bone marrow aspiration and biopsy~* Cycles 3 through 24 (28 day cycles):~ * Days 1 through 7: Predetermined dose of Azacitidine 1x daily.~ * Days 4 through 6: Predetermined dose of Tagraxofusp 1x daily.~ * Days 1 and 14: Predetermined dose of Venetoclax 1x daily~* Bone marrow aspirations/biopsies on Day 1 of Cycles 4, 6, 9, 12, and then every 3 cycles.~* End of treatment visit with bone marrow aspiration and biopsy~* Follow up for up to 2 years
Related Therapeutic Areas
Sponsors
Leads: Jacqueline Garcia, MD
Collaborators: Stemline Therapeutics, Inc., Break Through Cancer Foundation

This content was sourced from clinicaltrials.gov

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