NCT06641414

Brief Summary

A global multicenter, randomized, double-blind, placebo-controlled, pivotal phase III study. To evaluate overall survival (OS) of Lisaftoclax (APG-2575) combined with azacitidine (AZA) vs. placebo combined with azacitidine in newly diagnosed patients with HR-MDS.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
490

participants targeted

Target at P50-P75 for phase_3

Timeline
43mo left

Started Jan 2025

Longer than P75 for phase_3

Geographic Reach
2 countries

2 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress27%
Jan 2025Dec 2029

First Submitted

Initial submission to the registry

October 12, 2024

Completed
3 days until next milestone

First Posted

Study publicly available on registry

October 15, 2024

Completed
3 months until next milestone

Study Start

First participant enrolled

January 22, 2025

Completed
4.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2029

Expected
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2029

Last Updated

November 20, 2025

Status Verified

November 1, 2025

Enrollment Period

4.4 years

First QC Date

October 12, 2024

Last Update Submit

November 18, 2025

Conditions

Higher-risk Myelodysplastic Syndrome

Keywords

Higher-risk Myelodysplastic SyndromeLisaftoclax

Outcome Measures

Primary Outcomes (1)

  • Overall Survival（OS）

    The primary endpoint was overall survival (OS), defined as the time from the date of randomization to the date of death of any cause.

    Up to 5 years

Secondary Outcomes (1)

  • Safety evaluation based on the adverse event concurrence

    Up to 5 years

Study Arms (2)

Lisaftoclax (APG-2575) combined with Azacitidine

EXPERIMENTAL
Drug: Lisaftoclax (APG-2575)Drug: Azacitidine Injection

Placebo combined with Azacitidine

ACTIVE COMPARATOR
Drug: Azacitidine InjectionOther: Placebo

Interventions

Lisaftoclax (APG-2575)DRUG

QD, oral administration.

Lisaftoclax (APG-2575) combined with Azacitidine
Azacitidine InjectionDRUG

QD, hypodermic or intravenous injection.

Lisaftoclax (APG-2575) combined with AzacitidinePlacebo combined with Azacitidine
PlaceboOTHER

QD, oral administration.

Placebo combined with Azacitidine

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Newly diagnosed higher-risk MDS.
  • ECOG score of ≤2.
  • Expected survival ≥ 3 months.
  • Adequate organ function.
  • Female subjects of potential childbearing potential have a negative urine or serum pregnancy test before dosing. Subjects of childbearing potential as well as their partners voluntarily use contraception deemed effective by the investigator during the treatment period and for at least six months after the last dose of study drug.
  • Able to understand and voluntarily sign a written informed consent form, which must be signed prior to the performance of any trial-specified study procedures.
  • Subjects are able to complete study procedures and follow-up examinations.

You may not qualify if:

  • Concomitant other malignancies or prior malignancies with disease-free intervals of less than 1 year at the time of signing the informed consent.
  • Have undergone hematopoietic stem cell transplantation.
  • Uncontrolled active infection
  • Use of moderately potent inducers and moderately potent inhibitors of CYP3A4 within 14 days prior to the first dose of study drug.
  • MDS or other conditions that cannot be administered enterally.
  • Any condition that the subject is deemed to be inappropriate to participate in this study after evaluation by the investigator.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

MD Anderson Cancer Center

Houston, Texas, 77054, United States

RECRUITING

Peking University People's Hospital

Beijing, Beijing Municipality, 100033, China

RECRUITING

MeSH Terms

Interventions

LisaftoclaxAzacitidine

Intervention Hierarchy (Ancestors)

Aza CompoundsOrganic ChemicalsCytidinePyrimidine NucleosidesPyrimidinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsNucleosidesNucleic Acids, Nucleotides, and NucleosidesRibonucleosides

Study Officials

  • Xiaojun Huang, M.D., Ph.D.

    Peking University People's Hospital

    STUDY CHAIR

  • Guillermo Garcia-Manero, M.D.

    M.D. Anderson Cancer Center

    STUDY CHAIR

Central Study Contacts

Yifan Zhai, M.D., Ph.D.

CONTACT

+86-20-28068501yzhai@ascentage.com

Qian Niu, M.D.

CONTACT

Qian.Niu@ascentage.com

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 12, 2024

First Posted

October 15, 2024

Study Start

January 22, 2025

Primary Completion (Estimated)

June 1, 2029

Study Completion (Estimated)

December 1, 2029

Last Updated

November 20, 2025

Record last verified: 2025-11

Locations

CN(1)US(1)