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 .
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 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
Sequential
9MW3011
DrugAscending IV doses administered per protocol
9MW3011 placebo
DrugAscending IV doses administered per protocol
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
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.
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.
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).
Concentration of 9MW3011 in serum
PD-parameters-hepcidin
Change from baseline in hepcidin levels
PD-parameters-serum iron
Change from baseline in serum iron levels
Anti-drug antibody(ADA)
The incidence of ADA
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