A Study of BL-M11D1 in Combination With Cytarabine + Daunorubicin or Venetoclax + Azacitidine in Patients With Acute Myeloid Leukemia

A Phase II/III Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetic Characteristics and Preliminary Efficacy of BL-M11D1 for Injection in Combination With Cytarabine + Daunorubicin or Venetoclax + Azacitidine in Patients With Acute Myeloid Leukemia

This study is an open, multicenter, dose-escalation and expansion, non-randomized phase II/III clinical trial to evaluate the safety, tolerability, pharmacokinetic characteristics, and preliminary efficacy of BL-M11D1 in combination with cytarabine + daunorubicin or venetoclax + azacitidine in patients with acute myeloid leukemia.

Study Overview

• Status: Not yet recruiting
• Conditions: Acute Myeloid Leukemia
• Intervention / Treatment: Drug: BL-M11D1; Drug: Cytarabine; Drug: Daunorubicin; Drug: Venetoclax; Drug: Azacitidine

Detailed Description

The study cohorts include: Cohort A: Untreated newly diagnosed acute myeloid leukemia patients treated with BL-M11D1 in combination with cytarabine + daunorubicin. Cohort B: Untreated newly diagnosed acute myeloid leukemia patients treated with BL-M11D1 in combination with venetoclax + azacitidine.

• Study Type: Interventional
• Enrollment (Estimated): 216
• Phase: Phase 2; Phase 3

Contacts and Locations

• Study Contact: Name: Sa Xiao, PHD; Phone Number: 15013238943; Email: xiaosa@baili-pharm.com

Study Locations

• China
  • Tianjin Municipality
    • Tianjin, Tianjin Municipality, China
      • Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences
      • Principal Investigator: Jianxiang Wang
      • Contact: Jianxiang Wang
      • Principal Investigator: Hui Wei

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Voluntarily sign the informed consent form and comply with the protocol requirements;
2. No gender restrictions;
3. Age: ≥18 years;
4. Expected survival time ≥3 months;
5. Newly diagnosed AML according to the World Health Organization (WHO) 2016 classification and confirmed by morphology;
6. ECOG performance status score ≤2;
7. Peripheral blood white blood cell count ≤25×10⁹/L before the first dose;
8. Organ function levels must meet the requirements;
9. For premenopausal women with childbearing potential, a pregnancy test must be performed within 7 days before starting treatment, and the serum/urine pregnancy test must be negative. Patients must not be breastfeeding; all enrolled patients (regardless of male or female) should adopt adequate barrier contraception throughout the entire treatment cycle and for 6 months after the end of treatment.

Exclusion Criteria:

1. Acute promyelocytic leukemia, acute transformation of chronic myeloid leukemia;
2. Previous treatment for AML;
3. Participation in other interventional or observational studies;
4. History of severe cardiovascular or cerebrovascular diseases within 6 months prior to screening;
5. Prolonged QT interval, complete left bundle branch block, third-degree atrioventricular block, frequent and uncontrollable arrhythmia;
6. Active autoimmune diseases and inflammatory diseases;
7. Diagnosis of other malignancies within 5 years prior to the first dose;
8. Poorly controlled hypertension;
9. Grade ≥3 lung disease as defined by CTCAE v5.0, history of interstitial lung disease requiring systemic steroid therapy, etc.;
10. Patients with central nervous system involvement;
11. Previous organ transplantation;
12. History of allergy to recombinant humanized antibodies or human-mouse chimeric antibodies, or allergy to any excipient component of BL-M11D1;
13. Positive human immunodeficiency virus antibody, active tuberculosis, active hepatitis B virus infection, or active hepatitis C virus infection;
14. Evidence of other clinically significant, poorly controlled infections requiring systemic treatment;
15. Clinically symptomatic or recurrent pleural, peritoneal, pelvic, or pericardial effusion requiring drainage;
16. Pregnant or lactating women;
17. Within 4 weeks prior to the first dose of the study drug, subjects must not have received any live vaccines or are not expected to receive live vaccines during the study participation;
18. Other conditions deemed by the investigator as unsuitable for participation in this clinical trial.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: BL-M11D1 in Combination with Cytarabine + Daunorubicin or Venetoclax + Azacitidine; Participants receive BL-M11D1 in Combination with Cytarabine + Daunorubicin or Venetoclax + Azacitidine for the first cycle (4 weeks). Participants with clinical benefit could receive additional treatment for more cycles. The administration will be terminated because of disease progression or intolerable toxicity occurring or other reasons.

Drug: BL-M11D1; Administration by intravenous infusion for a cycle of 4 weeks.; Drug: Cytarabine; Administration in 4-week cycles.; Drug: Daunorubicin; Administration in 4-week cycles.; Drug: Venetoclax; Administration in 4-week cycles.; Drug: Azacitidine; Administration in 4-week cycles.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Objective Response Rate (ORR)	ORR is defined as the percentage of participants, who has a CR (disappearance of all target lesions) or PR (at least a 30% decrease in the sum of diameters of target lesions). The percentage of participants who experiences a confirmed CR or PR is according to RECIST 1.1.	Up to approximately 24 months
Composite Response Rate（CRc）	CRc is defined as the proportion of patients who achieve any one of several pre-defined types of treatment response.	Up to approximately 24 months
Phase IIa: Recommended Phase II Dose (RP2D)	The RP2D is defined as the dose level chosen by the sponsor (in consultation with the investigators) for phase II study, based on safety, tolerability, efficacy, PK, and PD data collected during the dose escalation study of BL-M11D1.	Up to approximately 24 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Disease Control Rate (DCR)	The DCR is defined as the percentage of participants who has a CR, PR, or Stable Disease (SD: neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease [PD: at least a 20% increase in the sum of diameters of target lesions and an absolute increase of at least 5 mm. The appearance of one or more new lesions is also considered PD]).	Up to approximately 24 months
Treatment-Emergent Adverse Event (TEAE)	TEAE is defined as any unfavorable and unintended change in the structure, function, or chemistry of the body temporally emerging, or any worsening (i.e., any clinically significant adverse change in frequency and/or intensity) of a pre-existing condition during the treatment of BL-M11D1. The type, frequency and severity of TEAE will be evaluated during the treatment of BL-M11D1.	Up to approximately 24 months
Progression-free survival (PFS)	Progression-free survival (PFS) as assessed by BICR is defined as the time between the date subjects were randomized and the first observation of disease progression (based on BICR's image-based assessment) or death.	Up to approximately 24 months
Relapse-Free Survival (RFS)	Relapse-Free Survival (RFS) is defined as the time from randomization (or from the start of treatment for non-randomized studies) until the first occurrence of disease recurrence or death from any cause.	Up to approximately 24 months
Cmax	Maximum serum concentration (Cmax) of BL-M11D1 will be investigated.	Up to approximately 24 months
Tmax	Time to maximum serum concentration (Tmax) of BL-M11D1 will be investigated.	Up to approximately 24 months
Ctrough	Ctrough is defined as the lowest serum concentration of BL-M11D1 prior to the next dose will be administered.	Up to approximately 24 months
ADA (anti-drug antibody)	Frequency of anti-BL-M11D1 antibody (ADA) will be investigated.	Up to approximately 24 months

Collaborators and Investigators

• Sponsor: Sichuan Baili Pharmaceutical Co., Ltd.
• Collaborators: Baili-Bio (Chengdu) Pharmaceutical Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Estimated): March 1, 2026
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027

Study Registration Dates

• First Submitted: November 20, 2025
• First Submitted That Met QC Criteria: November 20, 2025
• First Posted (Actual): December 1, 2025

Study Record Updates

• Last Update Posted (Actual): March 5, 2026
• Last Update Submitted That Met QC Criteria: March 3, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Histologic Type; Hematologic Diseases; Leukemia, Myeloid; Leukemia; Hemic and Lymphatic Diseases; Leukemia, Myeloid, Acute; Organic Chemicals; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleic Acids, Nucleotides, and Nucleosides; Hydrocarbons; Hydrocarbons, Cyclic; Carbohydrates; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Polycyclic Compounds; Glycosides; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Aza Compounds; Nucleosides; Ribonucleosides; Arabinonucleosides; Anthracyclines; Naphthacenes; Aminoglycosides; Cytarabine; Azacitidine; Daunorubicin; venetoclax
• Other Study ID Numbers: BL-M11D1-201

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No