Actively Recruiting

Phase 1
Age: 16Years +
All Genders
NCT07270770

BY002 IIT Study in R/R Acute Leukemia

Led by The First Affiliated Hospital of Soochow University · Updated on 2026-03-18

18

Participants Needed

1

Research Sites

71 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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

Official Title

BY002 IIT Study in R/R Acute Leukemia

Who Can Participate

Age: 16Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Age 16 years or older
  • Confirmed diagnosis of AML, ALL, or MPAL according to WHO 2022 criteria
  • Relapsed or refractory disease after at least one prior therapy
  • Presence of KMT2A rearrangement or NPM1 mutation (preferred but not required)
  • ECOG performance status between 0 and 2
  • Adequate organ function including ANC ≥1.0 × 10⁹/L (unless due to leukemia), platelets ≥50 × 10⁹/L (unless due to leukemia), ALT/AST ≤2.5 × upper limit of normal, bilirubin ≤1.5 × upper limit of normal, creatinine clearance ≥50 mL/min
  • Negative pregnancy test for women of childbearing potential
  • Willingness to use effective contraception during the study and for 90 days after last dose
  • Signed informed consent
Not Eligible

You will not qualify if you...

  • Active central nervous system leukemia (prior treated and controlled CNS involvement allowed)
  • Significant liver disease including viral hepatitis or cirrhosis (HBsAg positive requires negative HBV DNA, HCV antibody positive requires negative HCV RNA)
  • Known HIV infection
  • Pregnant or breastfeeding women
  • Significant cardiac disease including congenital long QT syndrome, QTcF >450 msec, recent myocardial infarction, unstable angina, coronary artery bypass within 6 months, or congestive heart failure NYHA class II or higher
  • History of another malignancy within 5 years except certain treated skin or in-situ cancers
  • Autologous HSCT or CAR-T therapy within 60 days or unresolved toxicities from these treatments
  • Allogeneic HSCT within 100 days, active graft-versus-host disease, or ongoing immunosuppressive therapy
  • Anti-leukemia therapy within 2 weeks before study entry (except hydroxyurea)
  • Prior investigational drug use within specified washout periods
  • Unresolved toxicities greater than grade 1 from prior therapy (except alopecia)
  • Uncontrolled active infections requiring hospitalization or intravenous antibiotics within 14 days unless resolved
  • Active tuberculosis
  • Conditions impairing oral intake or absorption
  • Known severe allergy to menin inhibitors or components of BY002
  • Poor compliance or inability to complete the study as judged by investigator
  • Any serious disease or condition that increases risk, interferes with treatment, confounds results, or predicts survival less than 6 months

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

1
2
3
+1

Trial Site Locations

Total: 1 location

1

The First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, China, 215000

Actively Recruiting

Loading map...

Research Team

J

Jing Lu Doctor, MD, PhD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

Not the Right Trial for You?

Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.

Already have an account? Log in here

BY002 IIT Study in R/R Acute Leukemia | DecenTrialz