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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 | Experimental | Study Drug |
|
| Cohort 2 | Experimental | Study Drug |
|
| Cohort 3 | Placebo Comparator | Placebo |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SNH-119014 dose 1 | Drug | Study Drug |
| |
| SNH-119014 dose 2 |
| Measure | Description | Time Frame |
|---|---|---|
| Safety evaluation | The incidence of Abnormal Laboratory Values, Clinically Significant Physical Examination Findings, vital signs, electrocardiogram (ECG) and/or Adverse Events | From signing the informed consent to 4 weeks after the last dose |
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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:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Yongrong Lai, Ph.D | Contact | 0771-6356510 | laiyongrong@263.net |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Foshan First People's Hospital | Foshan | Guangdong | China |
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| ID | Term |
|---|---|
| D013789 | Thalassemia |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| Drug |
Study Drug |
|
| Placebo | Drug | Placebo |
|
| Nanfang Hospital, Southern Medical University | Guangzhou | Guangdong | China |
|
| Maoming People's Hospital | Maoming | Guangdong | China |
|
| Liuzhou People's Hospital | Liuzhou | Guangxi | China |
|
| 923rd Hospital of the Joint Logistics Support Force of the People's Liberation Army of China | Nanning | Guangxi | China |
|
| The First Affiliated Hospital of Guangxi Medical University | Nanning | Guangxi | China |
|
| The First Affiliated Hospital of Hainan Medical University | Haikou | Hunan | China |
|
| Sichuan Academy of Medical Sciences & Sichuan Provincial People's Hospital | Chengdu | Sichuan | China |
|
| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |