Phase 1b/2 Safety and Efficacy of APR-246 w/Azacitidine for tx of TP53 Mutant Myeloid Neoplasms

Conditions:Cancer, Blood Cancer, Blood Cancer, Blood Cancer, Blood Cancer, Hematology, Hematology
Therapuetic Areas:Hematology, Oncology
Age Range:18 - Any
Start Date:May 5, 2017
End Date:May 2021

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A Phase 1b/2 Study to Evaluate the Safety and Efficacy of APR-246 in Combination With Azacitidine for the Treatment of TP53 Mutant Myeloid Neoplasms

The main purpose of this study is to determine the safe and recommended dose of APR-246 in
combination with azacitidine as well as to see if this combination of therapy improves
overall survival.

Participants will be treated for a total of 6 cycles. For participants responding or who have
stable disease following cycle 6, treatment may continue until one of the following criteria

- Inter-current illness that prevents further administration of treatment,

- Unacceptable adverse event(s),

- Participant decides to withdraw from the study, or

- General or specific changes in the participant's condition render the participant
unacceptable for further treatment in the judgment of the investigator.

- Evidence of disease progression by the International Working Group (IWG) 2006 criteria.

Participants who wish not to continue treatment at time of disease assessment at end of cycle
6 will complete their end of treatment visit upon completion of cycle 6.

Inclusion Criteria:

- Has signed the Informed Consent (ICF) and is able to comply with protocol

- Has adequate organ function according to study protocol guidelines.

- Age ≥18 years at the time of signing the informed consent form.

- Documented diagnosis of myelodysplastic syndrome (MDS), MDS/ myeloproliferative
neoplasm (MPN), chronic myelomonocytic leukemia (CMML) or oligoblastic AML (20-30%
myeloblasts) by World Health Organization (WHO) criteria.

- Documentation of a TP53 gene mutation by NGS based on central or local evaluation.

- For TP53 mutant patients with lower risk MDS (i.e., low or intermediate-1 risk by the
International Prognostic Scoring System (IPSS)) and isolated deletion of 5q (del(5q)),
failure of prior treatment with at least 4 full cycles of lenalidomide defined as no
response to treatment, loss of response at any time point, progressive disease, or
intolerance to therapy.

- An Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2 is

- If of childbearing potential, negative pre-treatment urine or serum pregnancy test.

- If of childbearing potential, willing to use an effective form of contraception such
as hormonal birth control, intrauterine device or double barrier method during
chemotherapy treatment and for at least six months thereafter.

Exclusion Criteria:

- Known history of HIV or active hepatitis B or active hepatitis C infection (testing
not mandatory).

- Has any of the following cardiac abnormalities (as determined by treating MD): a.
Symptomatic congestive heart failure; b. Myocardial infarction less than or equal to 6
months prior to enrollment; c. Unstable angina pectoris; d. Serious uncontrolled
cardiac arrhythmia; e. QTc ≥ 470 msec

- Concomitant malignancies or previous malignancies with less than a 1-year disease free
interval at the time of signing consent. Potential participants with adequately
resected basal or squamous cell carcinoma of the skin, or adequately resected
carcinoma in situ (e.g., cervix) may enroll irrespective of the time of diagnosis.

- Use of cytotoxic chemotherapeutic agents, or experimental agents (agents that are not
commercially available) for the treatment of MDS, MDS/MPN, CMML or AML within 14 days
of the first day of study drug treatment.

- No concurrent use of erythroid stimulating agents, G-CSF, GM-CSF is allowed during
study except in cases of febrile neutropenia where G-CSF can be used for short term.
Growth factors must be stopped 14 days prior to study.

- Women who are pregnant or breastfeeding.
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(212) 746-1067
Principal Investigator: Gail Roboz, MD
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