A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia

NCT06772766 | Recruiting Phase 1

• 1 other identifier: 9MW3011-C04
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 2

Brief Summary

This is a phase Ib, randomized, double-blind, placebo-controlled, multiple ascending dose study . The objectives of the study are to evaluate the safety , tolerability, pharmacokinetics(PK), pharmacodynamics(PD), and immunogenicity of 9MW3011 in patients with non-transfusion-dependent β- thalassemia .

Conditions

Beta-Thalassemia

Outcome Measures

Primary Outcomes (5)

• Adverse Event(including serious adverse event)

  The incidence of Adverse Events(AEs)and Serious Adverse Events(SAEs)from treatment until the last scheduled follow-up visit

  up to day 169

• Number of subjects with abnormal vital signs

  Vital signs measurements will include pulse rate, respiration rate, blood pressure (systolic and diastolic blood pressure) and body temperature.

  up to day 169

• Number of subjects with abnormal clinically significant results from physical examination

  The physical examinations will include examination of the following: skin and mucous membranes, lymph nodes, head and neck, chest, abdomen, musculoskeletal, nervous system, and other sites of note elicited from the subject.

  up to day 169

• Number of subjects with abnormal clinically significant 12-lead electrocardiogram (ECG) parameters

  The examination indicators include heart rate, PR, QRS, uncorrected QT, and QTcF(corrected by Fridericia formula).

  up to day 169

• Number of subjects with abnormal clinically significant clinical laboratory results

  Clinical laboratory tests include hematology, urinalysis, blood chemistry, coagulation function.

  up to day 169

Secondary Outcomes (5)

• Concentration of 9MW3011 in serum

  up to day 169

• PD-parameters-hepcidin

  up to day 169

• PD-parameters-serum iron

  up to day 169

• Anti-drug antibody(ADA)

  up to day 169

• Liver iron concentration(LIC)

  up to day 169

Study Arms (4)

Cohort 1

EXPERIMENTAL

9MW3011 or placebo (Randomized 4:1)

Drug: 9MW3011; Drug: 9MW3011 placebo

Cohort 2

EXPERIMENTAL

9MW3011 or placebo (Randomized 4:1)

Drug: 9MW3011; Drug: 9MW3011 placebo

Cohort 3

EXPERIMENTAL

9MW3011 or placebo (Randomized 4:1)

Drug: 9MW3011; Drug: 9MW3011 placebo

Cohort 4

EXPERIMENTAL

9MW3011 or placebo (Randomized 4:1)

Drug: 9MW3011; Drug: 9MW3011 placebo

Interventions

9MW3011 DRUG

Ascending IV doses administered per protocol

Cohort 1; Cohort 2; Cohort 3; Cohort 4

9MW3011 placebo DRUG

Ascending IV doses administered per protocol

Cohort 1; Cohort 2; Cohort 3; Cohort 4

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male and female subjects aged 18 to 65 years (inclusive)
• Subject must have a documented genetic diagnosis of β-thalassemia or hemoglobin E/ β-thalassemia
• Subjects must meet the criteria for non-transfusion-dependent thalassemia
• Subjects must have a baseline hemoglobin level between 70-100 g/L(inclusive), based on 2 consecutive measurements taken at least 1 week apart within 4 weeks before randomization
• Subjects must have evidence of iron overload during screening
• Subject must have performance status: Eastern Cooperative Oncology Group (ECOG) performance score of 0 to 1
• Subjects must fully understand the study procedures and methods, voluntarily participate in the trial, and sign an informed consent form

You may not qualify if:

• Subjects diagnosed with alpha-thalassemia
• Subjects diagnosed with HbS/beta-thalassemia or transfusion-dependent beta-thalassemia
• Subjects exhibit severe iron overload at the time of screening
• In addition to thalassemia, subjects have any other forms of anemia and hematological disorders that the investigator assesses may compromise safety or influence study outcomes
• Combined with any significant systemic diseases or psychiatric disorders
• Subjects have New York Heart Association (NYHA) Class III-IV heart failure and other cardiovascular diseases within 6 months prior to screening or currently present
• During the screening or baseline period, subjects exhibiting a QTcF interval of ≥450ms for males and ≥470ms for females on a 12-lead electrocardiogram (ECG), or presenting an abnormal 12-lead ECG with clinical significance
• Uncontrolled hypertension before screening
• A history of malignant neoplasm occurring within the last five years
• Severe infection requiring hospitalization or intravenous antimicrobial therapy, or uncontrolled systemic bacterial, fungal, or viral active infection
• Subject have received concomitant treatment that was not permitted by the protocol
• Subjects with a history of substance abuse, as well as those who yield positive results on substance abuse screening
• Subjects who are unable to undergo MRI scans
• Pregnant or lactating women
• Subjects presenting any other factors deemed unsuitable for participation assessed by the investigator

Sponsors & Collaborators

• Mabwell (Shanghai) Bioscience Co., Ltd.: lead

Study Sites (2)

The first Affiliated Hospital of Guangxi Medical University

Nanning, Guangxi, 530021, China

RECRUITING

Hainan General Hospital

Haikou, Hainan, 570311, China

RECRUITING

MeSH Terms

Conditions

beta-Thalassemia

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Yongrong Lai

CONTACT

+8607715356304; laiyongrong@hotmail.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 9, 2025
• First Posted: January 14, 2025
• Study Start: December 30, 2024
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): October 1, 2026
• Last Updated: January 14, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

CN (2)