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Evaluating APG-115 alone or with Azacitidine for blood cancers

A Phase Ib/II Study of APG-115 Alone or in Combination With Azacitidine in Patients With Relapse/Refractory AML, CMML or MDS

Phase1; Phase2 Interventional Ascentage Pharma Group Inc. · NCT04358393

This study is testing a new treatment called APG-115, both on its own and with another drug, to see how safe and effective it is for people with certain types of blood cancers.

Quick facts

PhasePhase1; Phase2
Study typeInterventional
Enrollment69 (estimated)
Ages18 Years and up
SexAll
SponsorAscentage Pharma Group Inc. Industry-sponsored
Drugs / interventionschemotherapy, immunotherapy, radiation
Locations8 sites (Gilbert, Arizona and 7 other locations)
Trial IDNCT04358393 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the safety and tolerability of APG-115, a novel treatment, in patients with relapsed or refractory acute myeloid leukemia (AML), chronic myelomonocytic leukemia (CMML), or high-risk myelodysplastic syndrome (MDS). The study is divided into two parts: the first part assesses APG-115 as a single agent, while the second part evaluates its effectiveness when combined with 5-azacitidine. The trial employs a dose escalation design to determine the optimal dosing regimen for both treatments over 28-day cycles.

Who should consider this trial

Good fit: Ideal candidates include patients with relapsed or refractory AML, CMML, or high-risk MDS who have no available standard therapies.

Not a fit: Patients with stable disease or those who have not exhausted standard treatment options may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with difficult-to-treat blood cancers.

How similar studies have performed: While this approach is novel, similar studies targeting blood cancers have shown promising results with combination therapies.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Patients with a diagnosis of histologically confirmed relapsed or refractory AML, CMML, or high-risk MDS (overall revised international prognostic scoring system (IPSS-R) score \> 3, including intermediate, high, or very high risk) by World Health Organization (WHO) classification for which no available standard therapies are indicated or anticipated to result in a durable response.
2. Adequate organ function as defined below:

   1. Liver function (total bilirubin \< or = 1.5 x upper limit of normal (ULN), aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) \<3 x ULN
   2. Kidney function (defined as a calculated creatinine clearance ≥ 60 mL/min; determined via urine collection for 24-hour creatinine clearance or by the Cockcroft Gault formula)
   3. Known cardiac ejection fraction of \> or = 45% within the past 3 months
3. Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 2
4. A negative serum pregnancy test is required within 1 week for all women of childbearing potential prior to enrolling on this trial.
5. Patient must have the ability to understand the requirements of the study and signed informed consent. A signed informed consent by the patient or legally authorized representative is required prior to their enrollment on the protocol.
6. Subject must have a projected life expectancy of at least 12 weeks.
7. Subject has a white blood cell count \< 25 × 10˄9/L. Note: Hydroxyurea is permitted to meet this criteria.

Exclusion:

1. Pregnant women are excluded.
2. Uncontrolled intercurrent illness including, but not limited to active uncontrolled infection, symptomatic congestive heart failure (NYHA Class III or IV), unstable angina pectoris, clinically significant cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
3. Have had leukemia therapy for 14 days prior to starting investigational drug. However, patients with rapidly proliferative disease may receive hydroxyurea as needed until 24 hours prior to starting therapy on this protocol and during the first cycle of study.
4. Have acute promyelocytic leukemia.
5. Active infection requiring systemic antibiotic/antifungal medication, known clinically active hepatitis B or C, or HIV infection.
6. Have received allogeneic hematopoietic stem cell transplant (HSCT) within 12 months prior to the first dose, or who have active/ongoing graft-versus host disease (GVHD), or require continued treatment with systemic immunosuppressive agents (calcineurin inhibitors within 4 weeks prior to the first dose), or received autologous hematopoietic stem cell transplantation within 6 months prior to the first dose.
7. Documented hypersensitivity to any of the components of the therapy program
8. Active, uncontrolled central nervous system (CNS) leukemia will not be eligible.
9. Men and women of childbearing potential who do not practice contraception. Women of childbearing potential and men must agree to use at least 1 form of barrier birth control (such as condom) prior to study entry and for the duration of study participation.
10. Any prior systemic MDM2-p53 inhibitor treatment
11. Any other condition or circumstance that would, in the opinion of the investigator, make the patient unsuitable for participation in the study.
12. History of other malignancies within 2 years prior to study entry, with the exception of:

    1. Adequately treated in situ carcinoma of the cervix uteri or carcinoma in situ of breast
    2. Basal cell carcinoma of the skin or localized squamous cell carcinoma of the skin
    3. Previous malignancy confined and surgically resected (or treated with other modalities) with curative intention: requires discussion with sponsor
13. Failure to have recovered (Grade \> 1) from prior treatment (including chemotherapy, targeted therapy, immunotherapy, experimental agents, radiation, or surgery)
14. Significant screening electrocardiogram (ECG) abnormalities including left bundle branch block, 2nd degree atrioventricular (AV) block type II, 3rd degree block, or corrected QT interval (QTc) ≥470 msec

Where this trial is running

Gilbert, Arizona and 7 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions AMLAcute Myeloid LeukemiaChronic Myelomonocytic LeukemiaCMMLMyelodysplastic SyndromesHigh-risk Myelodysplastic SyndromeMDSTP53
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.