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 .
A total of 40 subjects diagnosed with non-transfusion-dependent β-thalassemia will be enrolled in this study and assigned into four dosage cohorts. In each cohort, subjects will be randomized in a 4:1 ratio to receive 9MW3011 or placebo via intravenous infusion.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
40
Ascending IV doses administered per protocol
Ascending IV doses administered per protocol
The first Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, China
RECRUITINGHainan General Hospital
Haikou, Hainan, China
RECRUITINGAdverse 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
Time frame: 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.
Time frame: 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.
Time frame: 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).
Time frame: up to day 169
Number of subjects with abnormal clinically significant clinical laboratory results
Clinical laboratory tests include hematology, urinalysis, blood chemistry, coagulation function.
Time frame: up to day 169
Concentration of 9MW3011 in serum
Time frame: up to day 169
PD-parameters-hepcidin
Change from baseline in hepcidin levels
Time frame: up to day 169
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PD-parameters-serum iron
Change from baseline in serum iron levels
Time frame: up to day 169
Anti-drug antibody(ADA)
The incidence of ADA
Time frame: up to day 169
Liver iron concentration(LIC)
Change from baseline in LIC
Time frame: up to day 169