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

Phase 2

Recruiting

• Conditions: Refractory Acute Myeloid Leukemia; Relapsed Acute Myeloid Leukemia; Minimal Residual Disease; Myelodysplastic Syndromes
• Interventions: Drug: PD-1 inhibitor, Venetoclax, Decitabine, Azacytidine

• Registration Number: NCT06536959
• Lead Sponsor: Beijing 302 Hospital

Brief Summary

The efficiency and safety of PD-1 inhibitor in combination with venetoclax and hypomethylation agent in relapsed/refractory acute myeloid leukemia or high-risk myelodysplastic syndrome remain uncertain. In this study, the investigators aimed to assess safety and response to a new PD-1 inhibitor-based triple-drug combination regimen (venetoclax + hypomethylation agent + PD-1 inhibitor) in relapsed/refractory acute myeloid leukemia and high-risk myelodysplastic syndrome patients, or who had positive minimal residual disease.

Detailed Description

Not available

Study Timeline

• Status Verified: 2024-07
• Study Start: 2024-07-18
• Study First Submitted: 2024-07-31
• Study First Submitted QC: 2024-07-31
• Last Update Submitted: 2024-07-31
• Study First Posted: 2024-08-05
• Last Update Posted: 2024-08-05
• Primary Completion: 2026-07-31
• Study Completion: 2027-07-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 67

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
VA-PD1i	PD-1 inhibitor, Venetoclax, Decitabine, Azacytidine	Patients are treated with PD-1 inhibitor combined with venetoclax and decitabine/azacytidine.

Primary Outcome Measures

Name	Time	Method
Complete minimal residual disease (MRD) Response Rate	At the end of Cycle 2 (each cycle is 28 days)	Percentage of subjects with MRD negative or MRD \< 0.01%
MRD Response Rate	At the end of Cycle 2 (each cycle is 28 days)	Percentage of subjects with MRD \< 0.1% detectable by multicolor flow cytometry
Complete remission rate	At the end of Cycle 2 (each cycle is 28 days)	percentage of subjects with complete remission (CR) and incomplete hematologic recovery (CRi)

Secondary Outcome Measures

Name	Time	Method
Relapse-Free Survival	24 months	Time interval from leukemia free state to the first recurrence or death
Duration of response	24 months	Time interval from morphologic/MRD response to loss of response or death
Adverse events	start of treatment to 2 weeks after end of treatment	Number of subjects with adverse events
Overall Survival	24 months	Time interval from start of treatment until death or last follow-up

Trial Locations

Locations (1)

Xiao-ning Gao; 🇨🇳 Beijing, Beijing, China