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BY002 IIT Study in R/R Acute Leukemia

NCT07270770 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 18

Last updated 2026-03-18

No results posted yet for this study

Summary

This is a single-center, open-label, investigator-initiated phase 1 study designed to evaluate the safety, pharmacokinetics (PK), and preliminary efficacy of the menin inhibitor BY002 in patients with relapsed or refractory acute leukemia. Eligible subjects include adult patients (≥18 years) with AML, ALL, or MPAL, excluding APL, who carry KMT2A rearrangement or NPM1 mutation and have no better treatment options.

The study will be conducted in a dose-escalation design (3+3) , followed by expansion at the recommended dose. BY002 is administered orally in 28-day cycles until disease progression, unacceptable toxicity, HSCT, withdrawal, or death.

The primary objectives are to determine the incidence of dose-limiting toxicities (DLTs) and serious adverse events (SAEs), and to define the maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D). Secondary objectives include characterization of PK parameters, evaluation of safety (AEs, laboratory tests, vital signs, ECG), and assessment of efficacy endpoints such as complete remission (CR), composite remission (CRc), overall response rate (ORR), duration of response (DOR), event-free survival (EFS), relapse-free survival (RFS), overall survival (OS), and cumulative incidence of relapse (CIR). Exploratory objectives include analysis of pharmacodynamic biomarkers (e.g., HOXA9, MEIS1, CD11b) and correlation of baseline genetic mutations (e.g., NPM1, KMT2A, FLT3, TP53, NUP98) with clinical outcomes.

Conditions

  • Acute Leukemia
  • KMT2A Rearrangements or NPM1 Mutations Acute Leukemia

Interventions

DRUG

BY002

BY002 capsule (oral) * Starting dose: 50 mg BID * Dose escalation: 100 mg BID → 150 mg BID → 200 mg BID (3+3 design) * Treatment cycle: 28 days, repeated until disease progression, unacceptable toxicity, HSCT, withdrawal, or death

Sponsors & Collaborators

  • The First Affiliated Hospital of Soochow University

    lead OTHER

Principal Investigators

  • Suning Chen, MD, PhD · The First Affiliated Hospital of Soochow University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
16 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-03-15
Primary Completion
2027-03-31
Completion
2027-07-31

Countries

  • China

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07270770 on ClinicalTrials.gov