Thalassemia
Conditions
Brief summary
This study is a multicenter study to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of treatment with SNH-119014 in adult participants with non-transfusion-dependent thalassemia. 30 participants with non-transfusion-dependent thalassemia were enrolled.
Interventions
DRUGSNH-119014 dose 1
Study Drug
DRUGSNH-119014 dose 2
Study Drug
DRUGPlacebo
Placebo
Sponsors
ScinnoHub Pharmaceutical Co., Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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;
Exclusion criteria
* Hemoglobin S forms of thalassemia; * 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; * Poorly controlled hypertension or diabetes; * Markedly abnormal QTcF interval; * Severe arrhythmia requiring treatment as assessed by the investigator; * Markedly abnormal left ventricular ejection fraction; * Diagnosis of any other congenital or acquired hematologic disorder, or any other hemolytic process; * 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; * History of neurological or psychiatric disorders that, in the investigator's assessment, may affect the participant's ability to participate in the clinical study; * Post-splenectomy sepsis; * Severe pulmonary disease or hepatobiliary disease; * Abnormal laboratory tests during screening; * 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; * Pregnant or lactating women; * Major surgery within 6 months prior to signing informed consent or planned during the study period; * Splenectomy scheduled during the study treatment period; * Participation in another clinical trial and receipt of investigational treatment within 3 months; * 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety evaluation | From signing the informed consent to 4 weeks after the last dose | The incidence of Abnormal Laboratory Values, Clinically Significant Physical Examination Findings, vital signs, electrocardiogram (ECG) and/or Adverse Events |
Countries
China
Contacts
CONTACTYongrong Lai, Ph.D
Outcome results
None listed