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Clinical Trial 20375

Cancer Type: Malignant Hematology
Study Type: Treatment
NCT#: NCT04214860

Phase: Phase I
Principal Investigator: Sallman, David

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Study Title

Phase I Study of APR-246 in Combination with Venetoclax and Azacitidine in TP53-Mutant Myeloid Malignancies


This clinical trial is a Phase I, open-label, dose-finding and cohort expansion study to determine the safety and preliminary efficacy of APR-246 in combination with venetoclax and azacitidine in patients with myeloid malignancies.


To evaluate the safety and tolerability of administration of APR-246 in combination with venetoclax and azacitidine in patients with TP53-mutant myeloid malignancies.



Therapy (NOS)


APR-246 (); GDC-0199 (Venetoclax); Venetoclax (); azacitidine (5-azacitidine)

Inclusion Criteria

Inclusion Criteria:

  • Signed informed consent and ability to comply with protocol requirements.
  • Documented diagnosis of AML according to World Health Organization WHO) classification. For patients with newly diagnosed AML bone marrow and/or blood evidence of AML with >20% blasts is required. For patients with newly diagnosed AML, prior therapy with HMA and/or any chemotherapeutic agent for MDS is excluded
  • Adequate organ function as defined per protocol.
  • Age 18 years or older
  • At least one TP53 mutation, based on central TP53 testing results. If local mutation and/or p53 IHC results become available before central TP53 mutation results, enrollment may be made based on local results with Medical Monitor approval.
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2
  • Projected life expectancy > 12 weeks.
  • Negative serum or urine pregnancy test
  • Females of childbearing potential and males with female partners of childbearing potential must be willing to use an effective form of contraception

  • Exclusion Criteria

    Exclusion Criteria:

  • Prior treatment for TP53-mutant AML (*dependent upon treatment arm assigned).
  • Known history of MPN (i.e., polycythemia vera, essential thrombocythemia and primary myelofibrosis are excluded).
  • Known history of HIV or active hepatitis B or active hepatitis C infection.
  • Any of the following cardiac abnormalities: (a) Myocardial infarction within six months prior to registration; (b) New York Heart Association Class III or IV heart failure or known left ventricular ejection fraction (LVEF) > Concomitant malignancies for which patients are receiving active therapy
  • Known active CNS involvement from AML.
  • Malabsorption syndrome
  • Pregnancy or lactation.
  • Active uncontrolled systemic infection (viral, bacterial or fungal).

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