Combining PD-1 inhibitor with venetoclax for treating tough cases of leukemia and myelodysplastic syndromes

A Study of VA Combined With PD-1 Inhibitor in the Treatment of Relapsed and Refractory AML and High-risk MDS

Phase 2 Interventional Beijing 302 Hospital · NCT06536959

This study is testing a new treatment that combines three drugs to see if it can help people with tough cases of leukemia and myelodysplastic syndromes who haven't responded to other treatments.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment67 (estimated)
Ages18 Years to 70 Years
SexAll
SponsorBeijing 302 Hospital Academic / other
Drugs / interventionschemotherapy, immunotherapy
Locations1 site (Beijing, Beijing Municipality)
Trial IDNCT06536959 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and effectiveness of a new treatment regimen that combines a PD-1 inhibitor with venetoclax and a hypomethylation agent for patients with relapsed or refractory acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS). The study aims to determine how well this triple-drug combination works in patients who have not responded to previous treatments or have minimal residual disease. Participants will undergo a series of assessments to monitor their response to the treatment and any potential side effects.

Who should consider this trial

Good fit: Ideal candidates include patients diagnosed with relapsed or refractory AML or high-risk MDS who require chemotherapy and have not responded to prior treatments.

Not a fit: Patients who are not diagnosed with relapsed or refractory AML or high-risk MDS, or those who are currently pregnant or breastfeeding, 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 forms of leukemia and myelodysplastic syndromes.

How similar studies have performed: While the combination of PD-1 inhibitors with other treatments is being explored, this specific approach in relapsed/refractory AML and high-risk MDS is relatively novel and has not been extensively tested.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

* Patients diagnosed with relapsed and refractory acute myeloid leukemia (AML) and patients diagnosed with myelodysplastic syndrome (MDS) who require chemotherapy treatment.
* Patients who did not respond or had disease recurrence after 1 course of induction chemotherapy or had positive immune residues after induction chemotherapy or positive molecular residues (if any) after induction chemotherapy.
* Voluntarily participate in clinical research and sign an informed consent form and be willing to follow and be able to complete all experimental procedures.
* The toxic and side effects caused by the last treatment should be recovered.
* Eastern Cooperative Oncology Group score of 0 to 3 points.
* The organ function is intact.

  * Alanine aminotransferase (ALT) and Aspartate aminotransferase (AST) ≤2×ULN (Upper Limit of Normal).
  * Creatinine≤2×ULN.
  * Bilirubin≤2×ULN.
* Karnofsky≥70.
* The expected survival period is at least 12 weeks.
* Non-pregnant, non-breastfeeding women.

Exclusion Criteria:

* Suffering from other untreated or unrelieved malignant tumors within 2 years.
* Major surgery, radiotherapy, chemotherapy, biological therapy, immunotherapy, and experimental therapy were performed within 2 weeks of the first medication.
* Suffering from any other known serious and/or uncontrolled disease (eg, uncontrolled diabetes; cardiovascular disease, including congestive heart failure New York Heart Association \[NYHA\] Class III or IV, 6 months patients with myocardial infarction and poorly controlled blood pressure); chronic renal failure; or active uncontrolled infection); the investigators considered unsuitable for this clinical trial.
* Patients who are unwilling or unable to comply with the protocol.
* Currently being treated with other systemic anti-tumor or anti-tumor research drugs.
* Women who are pregnant or breastfeeding.

Where this trial is running

Beijing, Beijing Municipality

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.
Conditions Relapsed Acute Myeloid LeukemiaRefractory Acute Myeloid LeukemiaMyelodysplastic SyndromesMinimal Residual DiseaseVenetoclaxHypomethylation agenPD-1 inhibitor
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.