RecruitingPhase 1NCT06772766

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

A Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of 9MW3011 in Patients With Non-transfusion-dependent β- Thalassemia

Sponsor

Mabwell (Shanghai) Bioscience Co., Ltd.

Enrollment

40 participants

Start Date

Dec 30, 2024

Study Type

INTERVENTIONAL

Conditions

Beta-Thalassemia

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 .

Eligibility

Min Age: 18 YearsMax Age: 65 Years

Inclusion Criteria11

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
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

Exclusion Criteria11

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