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A Study of Tagraxofusp in Combination With Venetoclax and Azacitidine in Adults With Untreated CD123+ Acute Myeloid Leukemia Who Cannot Undergo Intensive Chemotherapy

Phase 2
Recruiting
Conditions
Acute Myeloid Leukemia
Interventions
Registration Number
NCT06456463
Lead Sponsor
Stemline Therapeutics, Inc.
Brief Summary

This study will be divided into 2 parts (Part 1 and Part 2). Part 1 will evaluate 2 doses of tagraxofusp (9 and 12 micrograms/kilogram/day \[μg/kg/day\]), used in combination with venetoclax and azacitidine, to determine the dose for Part 2. This determined dose, in combination with venetoclax and azacitidine, will then be further evaluated in Part 2 in 2 cohorts (TP53 mutated and TP53 wild type). Both parts will be conducted in participants with previously untreated CD123+ AML who are ineligible for intensive chemotherapy.

Detailed Description

Not available

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
76
Inclusion Criteria
  • Previously untreated with histological confirmation of AML by World Health Organization 2022 criteria and are ineligible for treatment with a standard cytarabine and anthracycline induction regimen due to age, or comorbidity.

  • Participant has any level of CD123 expression on blasts confirmed centrally by flow cytometry.

  • Must be considered ineligible for intensive chemotherapy, defined by the following:

    • ≥75 years of age; or

    • ≥18 to 74 years of age with at least 1 of the following comorbidities:

      • Eastern Cooperative Oncology Group (ECOG) performance status of 2 or 3.
      • Diffusing capacity of the lung for carbon monoxide of ≤65% or forced expiratory volume in 1 second ≤65%.
      • Baseline creatinine clearance ≥30 to <45 milliliters/minute calculated by the Cockcroft Gault formula or measured by 24-hour urine collection.
      • Hepatic disorder with total bilirubin >1.5 x upper limit of normal.
      • Any other comorbidity that the investigator judges to be incompatible with intensive chemotherapy must be reviewed and approved by the Sponsor.
  • ECOG performance status:

    • 0 to 2 for participants ≥75 years of age, or
    • 0 to 3 for participants ≥18 to 74 years of age.

Key

Exclusion Criteria
  • Participant has received prior therapy for AML.
  • Willing and able to receive standard induction therapy.
  • Treatment for an antecedent hematologic disease with a hypomethylating agent, venetoclax, tagraxofusp, purine analogue, cytarabine, intensive chemotherapy, chimeric antigen receptor-T therapy, or other experimental therapies.
  • AML with central nervous system involvement.

Note: Other inclusion/exclusion criteria may apply.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
Part 1 - Tagraxofusp (9 μg/kg/day)TagraxofuspParticipants will receive tagraxofusp in combination with venetoclax and azacitidine.
Part 1 - Tagraxofusp (9 μg/kg/day)VenetoclaxParticipants will receive tagraxofusp in combination with venetoclax and azacitidine.
Part 1 - Tagraxofusp (9 μg/kg/day)AzacitidineParticipants will receive tagraxofusp in combination with venetoclax and azacitidine.
Part 1 - Tagraxofusp (12 μg/kg/day)TagraxofuspParticipants will receive tagraxofusp in combination with venetoclax and azacitidine.
Part 1 - Tagraxofusp (12 μg/kg/day)VenetoclaxParticipants will receive tagraxofusp in combination with venetoclax and azacitidine.
Part 1 - Tagraxofusp (12 μg/kg/day)AzacitidineParticipants will receive tagraxofusp in combination with venetoclax and azacitidine.
Part 2 - Tagraxofusp (Selected Dose) and TP53 Wild TypeTagraxofuspParticipants (TP53 wild type) will receive tagraxofusp in combination with venetoclax and azacitidine.
Part 2 - Tagraxofusp (Selected Dose) and TP53 Wild TypeVenetoclaxParticipants (TP53 wild type) will receive tagraxofusp in combination with venetoclax and azacitidine.
Part 2 - Tagraxofusp (Selected Dose) and TP53 Wild TypeAzacitidineParticipants (TP53 wild type) will receive tagraxofusp in combination with venetoclax and azacitidine.
Part 2 - Tagraxofusp (Selected Dose) and TP53 MutatedTagraxofuspParticipants (TP53 mutated) will receive tagraxofusp in combination with venetoclax and azacitidine.
Part 2 - Tagraxofusp (Selected Dose) and TP53 MutatedVenetoclaxParticipants (TP53 mutated) will receive tagraxofusp in combination with venetoclax and azacitidine.
Part 2 - Tagraxofusp (Selected Dose) and TP53 MutatedAzacitidineParticipants (TP53 mutated) will receive tagraxofusp in combination with venetoclax and azacitidine.
Primary Outcome Measures
NameTimeMethod
Part 1: Determination of Part 2 Selected Dose of Tagraxofusp When Administered in Combination with Venetoclax and AzacitidineCycles 1-4 (up to 112 days; 28 days/cycle)
Part 2: Number of Participants Achieving a Best Overall Response (BOR) of Complete Remission (CR)Cycles 1-4 (up to 112 days; 28 days/cycle)
Secondary Outcome Measures
NameTimeMethod
Parts 1 and 2: Number of Participants Achieving a BOR of CRCycles 1-6 (up to 168 days; 28 days/cycle)
Parts 1 and 2: Time to First CRCycles 1-6 (up to 168 days; 28 days/cycle)

The time to first CR will be defined as the time from randomization (Cycle 1, Day 1) to the date of first documented CR.

Parts 1 and 2: Duration of ResponseCycles 1-6 (up to 168 days; 28 days/cycle)
Parts 1 and 2: Number of Participants Achieving a BOR of CR, CR with Incomplete Hematologic Recovery (CRi), or CR with Partial Hematologic Recovery (CRh)Cycles 1-4 (up to 112 days; 28 days/cycle)
Parts 1 and 2: Number of Participants Who Bridged to Stem Cell Transplant (SCT) Through Study TreatmentUp to approximately 6 years
Parts 1 and 2: Plasma Concentration of Free Tagraxofusp, Venetoclax, and AzacitidinePredose, up to 8 hours post dose (Days 4, 5, 6, 7, 14; Cycles 1-6; 28 days/cycle)
Parts 1 and 2: Time to First Composite CRCycles 1-4 (up to 112 days; 28 days/cycle)

The time to first composite CR will be defined as the time from randomization (Cycle 1, Day 1) to the date of first documented CR, CRi, or CRh.

Parts 1 and 2: Number of Participants Achieving a BOR of CR or CRiCycles 1-6 (up to 168 days; 28 days/cycle)
Parts 1 and 2: Time to first CR/CRiCycles 1-6 (up to 168 days; 28 days/cycle)

The time to first CR/CRi will be defined as the time from randomization (Cycle 1, Day 1) to the date of first documented CR or CRi.

Parts 1 and 2: Event-free Survival (EFS)Up to approximately 6 years

EFS will be defined as the time from the date of randomization (Cycle 1, Day 1) until the date of treatment failure, hematologic relapse after CR/CRi/CRh, or death from any cause, whichever occurs first.

Parts 1 and 2: CR with Minimal Residual Disease (MRD) NegativeCycles 1-6 (up to 168 days; 28 days/cycle)

Defined as the number of participants with a presence of marrow MRD of less than 0.01% at the time of CR.

Parts 1 and 2: Exposure-response of Free Tagraxofusp When Administered in Combination with Venetoclax and AzacitidineUp to approximately 6 years

The exposure-response relationship will be assessed utilizing the CR rate/composite CR rate and the number of participants experiencing adverse events of interest. This model-based analysis will be conducted to compare the exposure and response of free tagraxofusp, venetoclax, and azacitidine with venetoclax and azacitidine. Results will be reported as percent probability, wherein changes in the percent probability would indicate corresponding changes in the response rates with changes in exposure.

Parts 1 and 2: Number of Participants With Serum Anti-drug Antibodies for Tagraxofusp, Venetoclax, and AzacitidineDay 4 of each cycle (each cycle is 28 days) up to the end of study (approximately 6 years)

Trial Locations

Locations (30)

University of California, Los Angeles

🇺🇸

Los Angeles, California, United States

University of Miami Sylvester Comprehensive Cancer Center

🇺🇸

Miami, Florida, United States

AdventHealth Cancer Institute

🇺🇸

Orlando, Florida, United States

Dana Farber Cancer Institute (DFCI)

🇺🇸

Boston, Massachusetts, United States

Massachusetts General Hospital

🇺🇸

Boston, Massachusetts, United States

Beth Israel Deaconess Medical Center

🇺🇸

Boston, Massachusetts, United States

Henry Ford Health

🇺🇸

Detroit, Michigan, United States

Washington University - Siteman Cancer Center

🇺🇸

Saint Louis, Missouri, United States

John Theurer Cancer Center - Hackensack Meridian Health

🇺🇸

Hackensack, New Jersey, United States

Rutgers Cancer Institute

🇺🇸

New Brunswick, New Jersey, United States

Roswell Park Comprehensive Cancer Center

🇺🇸

Buffalo, New York, United States

North Shore University Hospital

🇺🇸

Manhasset, New York, United States

NYU Langone Health

🇺🇸

New York, New York, United States

Columbia University Irving Medical Center

🇺🇸

New York, New York, United States

Novant Health Presbyterian Medical Center

🇺🇸

Charlotte, North Carolina, United States

Novant Health Derrick L Davis Cancer Center

🇺🇸

Winston-Salem, North Carolina, United States

Cleveland Clinic Foundation

🇺🇸

Cleveland, Ohio, United States

Sarah Cannon, the Cancer Institute of HCA Healthcare

🇺🇸

Nashville, Tennessee, United States

Tennessee Oncology

🇺🇸

Nashville, Tennessee, United States

Baylor Scott & White Health

🇺🇸

Dallas, Texas, United States

University of Texas MD Anderson Cancer Center

🇺🇸

Houston, Texas, United States

Huntsman Cancer Institute

🇺🇸

Salt Lake City, Utah, United States

Concord Repatriation General Hospital

🇦🇺

Concord, New South Wales, Australia

Townsville Hospital

🇦🇺

Townsville, Queensland, Australia

Royal Adelaide Hospital

🇦🇺

Adelaide, South Australia, Australia

Box Hill Hospital

🇦🇺

Box Hill, Victoria, Australia

Monash Medical Centre

🇦🇺

Clayton, Victoria, Australia

St. Vincents Hospital

🇦🇺

Fitzroy, Victoria, Australia

Austin Hospital

🇦🇺

Heidelberg, Victoria, Australia

Royal Perth Hospital

🇦🇺

Perth, Western Australia, Australia

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