Acute LeukemiaPhase 1NCT07270770
BY002 IIT Study in R/R Acute Leukemia
A Phase 1 study testing BY002 in people with Acute Leukemia and KMT2A Rearrangements or NPM1 Mutations Acute Leukemia, run by The First Affiliated Hospital of Soochow University at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Acute Leukemia, KMT2A Rearrangements or NPM1 Mutations Acute Leukemia
- What is being tested?
- BY002
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteChina
- Study dates
- 15 Mar 2026to 31 Jul 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age ≥16 years.
- Confirmed diagnosis of AML, ALL, or MPAL per WHO 2022 criteria.
- Relapsed or refractory disease after ≥1 prior therapy.
- Presence of KMT2A rearrangement or NPM1 mutation (preferred, but not exclusive).
- ECOG performance status 0-2.
- Adequate organ function:
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- ANC ≥1.0 × 10⁹/L (unless cytopenia due to leukemia)
- Platelets ≥50 × 10⁹/L (unless due to leukemia)
- ALT/AST ≤2.5 × ULN, bilirubin ≤1.5 × ULN
- Creatinine clearance ≥50 mL/min
- Negative pregnancy test for women of childbearing potential.
- Willing to use effective contraception during study and 90 days after last dose.
- Signed informed consent.
Who cannot join
- Active central nervous system (CNS) leukemia. (Prior CNS involvement allowed if treated and controlled; CNS prophylaxis permitted.)
- History of significant liver disease, including viral hepatitis or cirrhosis:
- HBsAg positive must have negative HBV DNA.
- HCV antibody positive must have negative HCV RNA.
- Known HIV infection.
- Pregnant or breastfeeding women.
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- Significant cardiac disease:
- Congenital long QT syndrome or QTcF >450 msec.
- Acute myocardial infarction, unstable angina, or coronary artery bypass within 6 months.
- Congestive heart failure ≥ NYHA class II.
- History of another malignancy within 5 years, except adequately treated basal cell carcinoma of the skin, in-situ breast cancer, or in-situ cervical cancer.
- Autologous HSCT or CAR-T therapy within 60 days, or unresolved toxicities from ASCT/CAR-T.
- Allogeneic HSCT within 100 days, or active GVHD, or requiring ongoing immunosuppressive therapy.
- Anti-leukemia therapy within 2 weeks before study entry (hydroxyurea permitted).
- Prior investigational drug use: <2 weeks or <5 half-lives for small molecules; <4 weeks or <5 half-lives for biologics (whichever is shorter).
- Unresolved toxicities > grade 1 from prior anti-leukemia therapy (except alopecia).
- Uncontrolled active infection:
- Mild infections manageable with oral/topical treatment are allowed.
- Serious infections requiring hospitalization/IV antibiotics within 14 days excluded, unless resolved.
- Febrile neutropenia without infection evidence may be eligible if afebrile >72 h without antipyretics.
- Active tuberculosis excluded.
- Conditions impairing oral intake or absorption (e.g., swallowing difficulty, short bowel syndrome, gastroparesis).
- Known severe allergy to Menin inhibitors or any component of BY002.
- Investigator judges poor compliance or inability to complete study.
- Any other serious disease, abnormality, or condition that may increase risk, interfere with study drug, confound results, or expected survival ≤6 months.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, 215000
Lu Jing, MD, +86-13771836270, lujing@suda.edu.cn
About this study
Official title: A Single-center, Open-label, Investigator-Initiated Trial to Evaluate the Safety, Pharmacokinetics, and Efficacy of Menin Inhibitor BY002 in Patients With Relapsed or Refractory Acute Leukemia
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.
Read the detailed description
Relapsed/refractory (R/R) acute leukemia remains a severe disease with poor prognosis, and current treatment options are limited. Patients carrying genetic alterations such as KMT2A rearrangements or NPM1 mutations are particularly challenging, as these mutations are associated with high relapse rates and treatment resistance. Existing therapies have shown limited benefit in this population, highlighting an urgent unmet need for novel targeted approaches.
BY002 is a selective menin inhibitor developed in China. Preclinical studies have demonstrated that menin inhibition can downregulate leukemogenic genes such as HOXA9 and MEIS1, induce differentiation, and restore sensitivity to treatment. Importantly, BY002 shows potential to overcome resistance driven by existing mutations including KMT2A rearrangements and NPM1 mutations, thereby offering a new therapeutic strategy for these high-risk patients.
This study is a single-center, open-label, investigator-initiated phase 1 trial designed to evaluate the safety, pharmacokinetics (PK), and preliminary efficacy of BY002 in adult patients with relapsed or refractory acute leukemia. The trial adopts a 3+3 dose-escalation design with accelerated titration at the starting dose, followed by expansion at the recommended dose.
Patients will receive oral BY002 in 28-day cycles until disease progression, unacceptable toxicity, hematopoietic stem cell transplantation, withdrawal, or death. Safety assessments will include the incidence of dose-limiting toxicities (DLTs), serious adverse events (SAEs), and overall adverse events, as well as laboratory tests, vital signs, and ECG monitoring. PK will be assessed by plasma concentrations of BY002 and its metabolite M1, together with parameters such as Cmax, Tmax, t1/2, AUC, CL/F, and V/F.
Secondary endpoints will evaluate efficacy according to ELN 2022 criteria, including 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 analyses will assess changes in pharmacodynamic biomarkers (e.g., HOXA9, MEIS1, CD11b) before and after treatment, and investigate the correlation between baseline genetic alterations (such as KMT2A rearrangement, NPM1 mutation, FLT3 mutation/fusion, TP53 mutation, or NUP98 fusion) and clinical outcomes.
This trial will generate first-in-human data on BY002 in relapsed/refractory acute leukemia and provide essential evidence to guide subsequent phase 2/3 clinical development.
- Being tested
- BY002
- Keywords
- KMT2A rearrangements or NPM1 mutations, relapsed or refractory acute leukemia, Menin Inhibitor
- Medical subject headings
- Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BY002Drug
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 group | Role | What they receive |
|---|---|---|
| BY002 treatment | Experimental | 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 Receives: Drug: BY002 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of dose-limiting toxicities (DLT)
A DLT is defined as any ≥Grade 3 non-hematologic or ≥Grade 4 hematologic toxicity considered related to BY002, occurring during the DLT evaluation period. The number and proportion of participants with DLTs will be summarized.
Measured: 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).
Incidence of serious adverse events (SAEs)
SAEs will be defined and classified according to ICH E2A and CTCAE v5.0. The number and proportion of participants experiencing ≥1 SAE will be summarized.
Measured: From Cycle 1 Day 1 (C1D1) until 30 days after the last dose.
Secondary outcomes (3)
Incidence, type, and severity of all adverse events (AEs)
AEs will be coded per MedDRA and graded by NCI CTCAE v5.0. Number, type, severity, and causality of AEs will be summarized.
Measured: From first dose until 30 days after the last dose.
Plasma concentrations of BY002 and its primary metabolite (M1)
PK parameters including Cmax, Tmax, AUC, t1/2, and CL/F will be calculated using non-compartmental analysis following single-dose and multiple-dose administration.
Measured: Cycle 0 Day 1 (single-dose PK) and throughout treatment cycles, up to 30 days after the last dose.
Proportion of participants achieving complete remission (CR/CRi) per ELN 2022 criteria
Response will be evaluated according to ELN 2022 criteria, including CR, CRi, PR, and MRD negativity rates.
Measured: From first dose until disease progression, relapse, or death, assessed up to approximately 24 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Dec 2025
- Study start15 Mar 2026
- Primary completion31 Mar 2027 (estimated)
- Study completion31 Jul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The First Affiliated Hospital of Soochow University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Chen Suning, The First Affiliated Hospital of Soochow University
- Investigators
- Suning Chen, MD, PhD, Principal investigator, The First Affiliated Hospital of Soochow University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07270770
- Sponsor's protocol ID
- 2nd_Menin inhibitor
- Status last verified
- Mar 2026
- First submitted
- 2 Oct 2025
- Last updated
- 18 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 18 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-center, Open-label, Investigator-Initiated Trial to Evaluate the Safety, Pharmacokinetics, and Efficacy of Menin Inhibitor BY002 in Patients With Relapsed or Refractory Acute Leukemia
- ClinicalTrials.gov ID
- NCT07270770
- Acronym / sponsor ID
- 2nd_Menin inhibitor
- Keywords
- KMT2A rearrangements or NPM1 mutations, relapsed or refractory acute leukemia, Menin Inhibitor
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Soochow UniversityUniversity, hospital or other
- Study start
- 15 Mar 2026 (actual)
- Primary completion
- 31 Mar 2027 (estimated)
- Study completion
- 31 Jul 2027 (estimated)
- First posted
- 8 Dec 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Relapsed/refractory (R/R) acute leukemia remains a severe disease with poor prognosis, and current treatment options are limited. Patients carrying genetic alterations such as KMT2A rearrangements or NPM1 mutations are particularly challenging, as these mutations are associated with high relapse rates and treatment resistance. Existing therapies have shown limited benefit in this population, highlighting an urgent unmet need for novel targeted approaches.
BY002 is a selective menin inhibitor developed in China. Preclinical studies have demonstrated that menin inhibition can downregulate leukemogenic genes such as HOXA9 and MEIS1, induce differentiation, and restore sensitivity to treatment. Importantly, BY002 shows potential to overcome resistance driven by existing mutations including KMT2A rearrangements and NPM1 mutations, thereby offering a new therapeutic strategy for these high-risk patients.
This study is a single-center, open-label, investigator-initiated phase 1 trial designed to evaluate the safety, pharmacokinetics (PK), and preliminary efficacy of BY002 in adult patients with relapsed or refractory acute leukemia. The trial adopts a 3+3 dose-escalation design with accelerated titration at the starting dose, followed by expansion at the recommended dose.
Patients will receive oral BY002 in 28-day cycles until disease progression, unacceptable toxicity, hematopoietic stem cell transplantation, withdrawal, or death. Safety assessments will include the incidence of dose-limiting toxicities (DLTs), serious adverse events (SAEs), and overall adverse events, as well as laboratory tests, vital signs, and ECG monitoring. PK will be assessed by plasma concentrations of BY002 and its metabolite M1, together with parameters such as Cmax, Tmax, t1/2, AUC, CL/F, and V/F.
Secondary endpoints will evaluate efficacy according to ELN 2022 criteria, including 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 analyses will assess changes in pharmacodynamic biomarkers (e.g., HOXA9, MEIS1, CD11b) before and after treatment, and investigate the correlation between baseline genetic alterations (such as KMT2A rearrangement, NPM1 mutation, FLT3 mutation/fusion, TP53 mutation, or NUP98 fusion) and clinical outcomes.
This trial will generate first-in-human data on BY002 in relapsed/refractory acute leukemia and provide essential evidence to guide subsequent phase 2/3 clinical development.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BY002 treatment | Experimental | Drug: BY002BY002 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 |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (13 listed)
- Age ≥16 years.
- Confirmed diagnosis of AML, ALL, or MPAL per WHO 2022 criteria.
- Relapsed or refractory disease after ≥1 prior therapy.
- Presence of KMT2A rearrangement or NPM1 mutation (preferred, but not exclusive).
- ECOG performance status 0-2.
Key exclusion criteria (25 listed)
- Active central nervous system (CNS) leukemia. (Prior CNS involvement allowed if treated and controlled; CNS prophylaxis permitted.)
- History of significant liver disease, including viral hepatitis or cirrhosis:
- HBsAg positive must have negative HBV DNA.
- HCV antibody positive must have negative HCV RNA.
- Known HIV infection.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BY002BY002 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 | Drug | BY002 treatment |
Primary outcome
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).
A DLT is defined as any ≥Grade 3 non-hematologic or ≥Grade 4 hematologic toxicity considered related to BY002, occurring during the DLT evaluation period. The number and proportion of participants with DLTs will be summarized.
Incidence of serious adverse events (SAEs)
Time frame: From Cycle 1 Day 1 (C1D1) until 30 days after the last dose.
SAEs will be defined and classified according to ICH E2A and CTCAE v5.0. The number and proportion of participants experiencing ≥1 SAE will be summarized.
3 secondary outcomes
Incidence, type, and severity of all adverse events (AEs)
Time frame: From first dose until 30 days after the last dose.
Plasma concentrations of BY002 and its primary metabolite (M1)
Time frame: Cycle 0 Day 1 (single-dose PK) and throughout treatment cycles, up to 30 days after the last dose.
Proportion of participants achieving complete remission (CR/CRi) per ELN 2022 criteria
Time frame: From first dose until disease progression, relapse, or death, assessed up to approximately 24 months.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Mar 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Suning Chen, MD, PhD principal investigator
The First Affiliated Hospital of Soochow University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing BY002 in people with Acute Leukemia and KMT2A Rearrangements or NPM1 Mutations Acute Leukemia, run by The First Affiliated Hospital of Soochow University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 25 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 18 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jing Lu Doctor, MD, PhD (86+0512-67781137, gloriajlu@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the 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).). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Mar 2026.