BY002 IIT Study in R/R Acute Leukemia

Sponsor
The First Affiliated Hospital of Soochow University
Study ID
NCT07270770
Phase
PHASE1
Status
Recruiting
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Conditions

  • Acute Leukemia
  • KMT2A Rearrangements or NPM1 Mutations Acute Leukemia

Eligibility Criteria

Sex
ALL
Age
16 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • BY002 — DRUG
    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

Study Details

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.

Key Dates

First listed
Dec 8, 2025
Start date
Mar 15, 2026
Status verified
Mar 2026
Primary completion
Mar 31, 2027
Completion
Jul 31, 2027

Study Design

Enrollment
18 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: BY002 treatment
    BY002 capsule (oral) * 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

Primary Outcome Measure

Incidence of dose-limiting toxicities (DLT) [ Time Frame: From Cycle 1 Day 1 (C1D1) through the end of the DLT observation period (Day 1-28; may extend to Day 42 if toxicity evaluation is delayed). ]

Central Contacts

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