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OncoMatch/Clinical Trials/NCT04275518

A Phase Ib Study of APG-115 Single Agent or in Combination With Azacitidine or Cytarabine in Patients With AML and MDS.

Is NCT04275518 recruiting? Yes, currently enrolling (Jun 2026). This Phase 1 trial studies multiple treatments including APG-115 and Azacitidine for acute myeloid leukemia (aml).

Phase 1RecruitingAscentage Pharma Group Inc.NCT04275518Data as of Jun 2026Location: China

Treatment: APG-115 · Azacitidine · CytarabineAcute myeloid leukemia is a malignant disorder characterized by the rapid, uncontrolled proliferation of malignant clonal hematopoietic stem cells that accumulate as immature, undifferentiated cells (blasts) in the bone marrow and circulation. APG-115 is a potent and orally active small-molecule MDM2 inhibitor, it binds to MDM2 protein and shows potent cell growth inhibitory activity in vitro with low nanomolar potencies in a subset of human cancer cell lines. APG-115 has demonstrated its strong antitumor activities with either daily or less frequent dosing-schedules in the acute leukemia xenograft models. This is a phase 1b, open-label, three-stages study that will initially evaluate the safety and PK/PD profile of APG-115 as a single agent, followed by a combination of APG-115 + azacytidine or cytarabine in R/R AML or MDS subjects. Patients will continue treatment for maximally 6 cycles or until progression of disease or unacceptable toxicity is observed or administrative discontinuation whichever occurs first. Patients who continue to be benefit after 6 cycles' treatment will receive additional cycles of treatment until progression of disease, unacceptable toxicity is observed or administrative discontinuation. (As long as it is proven safe).

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Extracted eligibility criteria

Treatments studied

Chemotherapy

AzacitidineCytarabine

Other

APG-115

Cancer type

Acute Myeloid Leukemia

Myelodysplastic Syndrome

Performance status

ECOG 0–1(Restricted strenuous activity)

Prior therapy

Cannot have received: leukemia biotherapy

Patients must not have had leukemia biotherapy 4 weeks prior to starting investigational drug

Cannot have received: small molecular targeted drug therapy

less than 5 half-lives small molecular targeted drug therapy

Cannot have received: any anti-cancer therapy

28 days any anti-cancer therapy (whichever is longer)

Cannot have received: allogeneic hematopoietic stem cell transplant

Participants who have received allogeneic HSCT

Cannot have received: autologous hematopoietic stem cell transplant

Exception: within 12 months

autologous HSCT within 12 months

Cannot have received: MDM2-p53 inhibitor

Any prior systemic MDM2-p53 inhibitor treatment

Lab requirements

Blood counts

white blood cell count < 50 × 10^9/L (hydroxyurea permitted to meet this criterion)

Kidney function

Liver function

Adequate organ function; Subject has a white blood cell count< 50 × 10^9/L. Note: Hydroxyurea is permitted to meet this criterion.

Structured fields extracted by AI. May contain errors — verify against the official protocol.

Frequently asked questions

Is NCT04275518 currently recruiting?

Yes, this trial is currently recruiting patients.

Are there prior therapy exclusions?

Yes. Prior leukemia biotherapy, small molecular targeted drug therapy, any anti-cancer therapy disqualifies patients from enrollment.

Could you qualify for this trial?

Enter your biomarker results to see how this trial's eligibility criteria match your specific cancer profile.

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