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Evaluating AK117 with Azacitidine for higher-risk Myelodysplastic Syndromes

A Randomized, Double-blind, Placebo-controlled, Multicenter Phase 2 Study of AK117/Placebo in Combination With Azacitidine in Patients With Newly Diagnosed Higher-risk Myelodysplastic Syndromes

Phase 2 Interventional Akeso · NCT06196203

This study is testing if a new treatment combining AK117 with azacitidine can help people recently diagnosed with higher-risk myelodysplastic syndromes feel better compared to standard treatment.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment90 (estimated)
Ages18 Years and up
SexAll
SponsorAkeso Industry-sponsored
Locations15 sites (Los Angeles, California and 14 other locations)
Trial IDNCT06196203 on ClinicalTrials.gov

What this trial studies

This Phase 2 clinical trial is a randomized, double-blind, placebo-controlled study designed to assess the efficacy and safety of AK117 in combination with azacitidine in patients who have been newly diagnosed with higher-risk myelodysplastic syndromes (HR-MDS). Participants will be randomly assigned to receive either the treatment or a placebo, and their responses will be monitored over time. The study aims to determine if the combination therapy can improve patient outcomes compared to standard treatment. It involves multiple centers to ensure a diverse patient population and robust data collection.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 and older who have been newly diagnosed with higher-risk myelodysplastic syndromes.

Not a fit: Patients with lower-risk myelodysplastic syndromes or those who do not meet the inclusion criteria will likely not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with higher-risk myelodysplastic syndromes, potentially improving survival and quality of life.

How similar studies have performed: Other studies have shown promise in using combination therapies for myelodysplastic syndromes, but the specific combination of AK117 and azacitidine is being evaluated for the first time in this context.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Age ≥ 18 years old at the time of enrolment.
* Eastern Cooperative Oncology Group (ECOG) performance status score of 0 to 2.
* Expected life expectancy ≥ 3 months.
* Newly diagnosed HR-MDS, according to the 2016 World Health Organization (WHO) classification with the presence of \< 20% blasts in bone marrow or peripheral blood; Overall IPSS-R score ≥ 3.5.
* Ability to undergo the study-required bone marrow sample collection procedures.
* Suitable venous access for the study-required blood sampling (i.e., including PK and immunogenicity).
* Female patients of childbearing age must have negative serum pregnancy test results before randomization or per region-specific guidance documented in the informed consent and a negative urine pregnancy test on the day of first dose prior to dosing.
* Female patients of childbearing potential having sex with an unsterilized male partner must agree to use a highly effective method of contraception from the beginning of screening until 180 days after the last dose of the study treatment.
* Unsterilized male patients having sex with a female partner of childbearing potential must agree to use an effective method of contraception from the beginning of screening until 180 days after the last dose of study treatment.

Exclusion Criteria:

* MDS evolving from a pre-existing myeloproliferative neoplasm (MPN), myelodysplastic/myeloproliferative neoplasms (MDS/MPN).
* Prior treatment with Cluster of Differentiation (CD) 47 or Signal-regulatory protein alpha (SIRPα)-targeting agents.
* Concurrently participating in another interventional clinical study, unless it is an observational (non-interventional) clinical study or during the follow-up period of an interventional study.
* Patients who previously diagnosed with another malignancy and have any evidence of residual disease.
* Known allergy to any component of any study drug; known history of severe hypersensitivity to other monoclonal antibodies.
* Patients with any psychiatric or social factor which the investigator deems may interfere with the patient's ability to comply with the requirements of the study.
* Patients with current hypertension with systolic blood pressure ≥ 160 mmHg or diastolic blood pressure ≥ 100 mmHg after oral antihypertensive therapy.
* Patients with known cardiopulmonary disease defined as unstable angina, clinically significant arrhythmia, congestive heart failure (New York Heart Association Class III or IV), decompensated cirrhosis, nephrotic syndrome, uncontrolled metabolic disorders.
* Patients who are breastfeeding or plans to breastfeed during the study.
* Other conditions where the investigator considers the patient inappropriate for enrollment.

Where this trial is running

Los Angeles, California and 14 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 Higher-risk Myelodysplastic Syndromes
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.