A Study of SNH-119014 in Adult Participants With Non-transfusion-dependent Thalassemia (NTDT)

Ki vehet részt

• Male or Female; -≥18 years
• Documented diagnosis of thalassemia
• Non-transfusion-dependent
• Hb concentration ≤10.0 grams per deciliter (g/dL) during the screening period
• For women of reproductive potential: negative serum pregnancy test during the screening period
• Agreement to use approved contraceptive measures
• Informed consent

Ki nem vehet részt

• History of Congestive Heart Failure Within 6 months prior to signed informed consent
• Myocardial Infarction/unstable angina Within 6 months prior to signed informed consent
• Deep Vein Thrombosis/stroke/thromboembolic events Within 6 months prior to signed informed consent
• Active infection requiring intravenous antibiotics or of Grade ≥3 severity within 4 weeks prior to the first dose
• History of primary malignancy, except for: cured non-melanoma skin cancer; curatively treated cervical or breast carcinoma in situ; or other primary tumors treated with curative intent with no known active disease and no treatment in the past 5 years
• Use of luspatercept, hydroxyurea, erythropoietin, strong CYP3A4 inhibitors, or strong CYP3A4 inducers within the prohibited period specified in the protocol
• Receipt of anticoagulant therapy prior to screening, or requirement for anticoagulant therapy during the study period
• Prior bone marrow or stem cell transplant
• Major surgery within 6 months prior to signing informed consent or planned during the study period
• Poorly controlled hypertension or diabetes
• Severe arrhythmia requiring treatment as assessed by the investigator
• Diagnosis of any other congenital or acquired hematologic disorder, or any other hemolytic process
• History of neurological or psychiatric disorders that, in the investigator's assessment, may affect the participant's ability to participate in the clinical study
• Severe pulmonary disease or hepatobiliary disease
• History of severe allergy, or allergy to SNH-119014 or its excipients
• Any other condition that, in the investigator's opinion, makes the participant unsuitable for enrollment
• Hemoglobin S forms of thalassemia
• Markedly abnormal left ventricular ejection fraction
• Abnormal laboratory tests during screening
• Markedly abnormal QTcF interval
• Post-splenectomy sepsis
• Pregnant or lactating women
• Splenectomy scheduled during the study treatment period
• Participation in another clinical trial and receipt of investigational treatment within 3 months