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

Korábbi kezelés

• 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;

Más betegség

• 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.

Laborértékek

• Hemoglobin S forms of thalassemia;
• Markedly abnormal left ventricular ejection fraction;
• Abnormal laboratory tests during screening;

Egyéb

• 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;

• 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;