A Study of BL-M11D1 in Patients With Relapsed/Refractory Myelodysplastic Syndromes

A Phase Ib/II Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetic Characteristics, and Preliminary Efficacy of BL-M11D1 for Injection in Patients With Relapsed/Refractory Myelodysplastic Syndromes

This study is an open-label, multicenter, non-randomized Phase Ib/II clinical study to evaluate the safety, tolerability, and pharmacokinetic characteristics of BL-M11D1 for injection in patients with relapsed/refractory myelodysplastic syndromes.

Study Overview

• Status: Not yet recruiting
• Conditions: Myelodysplastic Syndromes
• Intervention / Treatment: Drug: BL-M11D1

Detailed Description

The study consists of two phases: a dose-exploration phase (Phase Ib) and a dose-expansion phase (Phase II).

• Study Type: Interventional
• Enrollment (Estimated): 92
• Phase: Phase 2; Phase 1

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
      • Contact: Zhijian Xiao; Phone Number: 022-23909083; Email: zjxiao@ihcams.ac.cn

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 and ≤75 years;
4. Expected survival time ≥3 months;
5. Relapsed/refractory CD33+ MDS;
6. Morphological assessment showing blasts in bone marrow ≥5% and <20%;
7. Eastern Cooperative Oncology Group (ECOG) performance status ≤2;
8. Toxicities from prior anti-tumor therapy must have recovered to ≤ Grade 1 as defined by NCI-CTCAE v6.0;
9. Meet the required organ function levels;
10. For premenopausal women of childbearing potential, a pregnancy test (serum/urine) must be negative within 7 days before starting treatment, and they must not be breastfeeding; all enrolled trial participants (regardless of gender) must practice adequate barrier contraception throughout the entire treatment period and for 6 months after treatment completion.

Exclusion Criteria:

1. Use of chemotherapy, biotherapy, immunotherapy, etc., within 4 weeks or 5 half-lives prior to the first dose;
2. Presence of uncorrected folate deficiency or vitamin B12 deficiency, etc.;
3. History of severe cardiovascular or cerebrovascular disease;
4. Thromboembolic events requiring therapeutic intervention within 6 months prior to screening;
5. Active autoimmune diseases and inflammatory diseases;
6. History of extensive bowel resection or presence of Crohn's disease, ulcerative colitis, chronic diarrhea, or intestinal obstruction;
7. Diagnosis of another malignancy within 5 years prior to the first dose;
8. Poorly controlled hypertension;
9. Poorly controlled hyperglycemia or diabetes mellitus;
10. Pulmonary diseases classified as Grade ≥3 according to CTCAE v6.0, etc.;
11. Trial participants with central nervous system involvement;
12. Trial participants with extramedullary involvement;
13. Trial participants with a history of allergy to recombinant humanized antibodies or chimeric human-mouse antibodies, or hypersensitivity to any excipient component of BL-M11D1;
14. Prior organ transplantation or hematopoietic stem cell transplantation;
15. Positive for human immunodeficiency virus antibody, active tuberculosis, active hepatitis B virus infection, or active hepatitis C virus infection;
16. Active fungal, bacterial, or viral infections;
17. History of severe neurological or psychiatric disorders;
18. Trial participants with clinically significant bleeding or obvious bleeding tendency within 4 weeks prior to signing informed consent;
19. Presence of clinically symptomatic pleural, peritoneal, or pericardial effusion requiring repeated drainage;
20. Participation in another clinical trial within 4 weeks or 5 half-lives prior to the first dose;
21. Pregnant or breastfeeding women;
22. Other conditions deemed by the investigator to make the participant 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; Participants receive BL-M11D1 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Phase II: 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
Phase Ib: 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
Phase Ib: 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
Phase II: Complete Response (CR)	Complete Response (CR) is defined as the disappearance of all target lesions, with any pathological lymph nodes (whether target or non-target) having a short axis diameter reduced to <10 mm.	Up to approximately 24 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
AUC0-t	AUC0-t is defined as area under the serum concentration-time curve from time 0 to the time of the last measurable concentration.	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
T1/2	Half-life (T1/2) of BL-M11D1 will be investigated.	Up to approximately 24 months
CL (Clearance)	CL in the serum of BL-M11D1 per unit of time will be investigated.	Up to approximately 24 months
Phase II: Duration of Response (DOR)	The DOR for a responder is defined as the time from the participant's initial objective response to the first date of either disease progression or death, whichever occurs first.	Up to approximately 24 months
Phase II: Hematologic Improvement (HI)	HI is defined as achieving a predefined threshold of improvement in the erythroid, platelet, or neutrophil lineages according to the IWG 2006 and IWG 2023 criteria, with a duration of no less than 8 weeks.	Up to approximately 24 months
Phase II: AML Transformation Rate	The AML transformation rate is defined as the first confirmed event of transformation to acute myeloid leukemia according to WHO criteria (bone marrow blasts ≥20% or presence of extramedullary infiltration).	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): May 1, 2026
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2028

Study Registration Dates

• First Submitted: May 9, 2026
• First Submitted That Met QC Criteria: May 9, 2026
• First Posted (Actual): May 15, 2026

Study Record Updates

• Last Update Posted (Actual): May 15, 2026
• Last Update Submitted That Met QC Criteria: May 9, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Bone Marrow Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes
• Other Study ID Numbers: BL-M11D1-102

Drug and device information, study documents

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