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The safety and efficacy of CN128 is studied in thalassaemia with sever liver iron overloaded patients.
The trial will start with the lower dose of CN128 (10 mg/kg body weight [bw], bid) for one week. If no unacceptable toxicity associated with CN128 is found, the subjects will be given 15 mg/kg body weight [bw], bid for one week. If no unacceptable toxicity associated with CN128 is found, the subjects will be given 20 mg/kg body weight [bw], bid for two weeks. If no unacceptable toxicity associated with CN128 is found, the subjects will be given 25 mg/kg body weight [bw], bid for two weeks. If no unacceptable toxicity associated with CN128 is found, the subjects will be given 30 mg/kg body weight [bw], bid for two weeks. If no unacceptable toxicity associated with CN128 is found, the subjects will be given 30 mg/kg body weight [bw], bid to Week 52. Dosage will be adjusted according to subject status and study plan.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| CN128 Group | Experimental | All subjects will be given the lower (10 mg/kg bw, bid) to higher dose (30 mg/kg bw, bid) for 52 weeks, according to the administration plan. The dosage form is tablets. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CN128 Tablets | Drug | Iron chelator, oral tablets |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with adverse events, adverse reactions, severe adverse events and severe adverse reactions as a measure of safety and tolerability during the study period. | To determine the incidence, type and severity of adverse events, adverse reactions, severe adverse events and severe adverse reactions in patients up to 52 weeks. | Up to 52 weeks |
| Absolute Change in Weight (Unit: kg) From Baseline Over Time | The patient's weight will be determined, and it's one kind of Physical examination. | Baseline, 26, 39 and 52 weeks. |
| Absolute Change in Height (Unit: m) From Baseline Over Time | The patient's height will be determined, and it's one kind of Physical examination. | Baseline, 26, 39 and 52 weeks. |
| Absolute Change in Temperature (Unit: ℃）From Baseline Over Time | The patient's temperature will be determined, and it's one kind of vital signs checks. | Baseline, 2, 4, 6, 8, 12, 16, 20, 26, 39 and 52 weeks. |
| Absolute Change in Blood pressure (Unit: mmHg ) From Baseline Over Time | Both patient's systolic and diastolic blood pressure will be measured, and it's one kind of vital signs checks. | Baseline, 2, 4, 6, 8, 12, 16, 20, 26, 39 and 52 weeks. |
| Absolute Change in Heart rate (Unit: bpm) From Baseline Over Time | The patient's heart rate will be determined, and it's one kind of vital signs checks. | Baseline, 2, 4, 6, 8, 12, 16, 20, 26, 39 and 52 weeks. |
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change in Liver iron content (Unit: mg/g dw) From Baseline Over Time | Liver iron content will be determined by liver magnetic resonance. | Baseline, 12, 26,39 and 52 weeks. |
| Absolute Change in Serum ferritin (Unit: µg/L) From Baseline Over Time |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Jianmin Luo, PhD | First Affiliated Hospital of Guangxi Medical University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The First Affiliated Hospital Of Guangxi Medical University | Nanning | Guangxi | China |
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| ID | Term |
|---|---|
| D013789 | Thalassemia |
| D019190 | Iron Overload |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| Absolute Change in Respiration (Unit: bpm) From Baseline Over Time | The patient's respiration will be determined, and it's one kind of vital signs checks. | Baseline, 2, 4, 6, 8, 12, 16, 20, 26, 39 and 52 weeks. |
| Absolute Change in Electrocardiogram (P-R (Unit: ms), QRS (Unit: ms), QTc interval (Unit: ms), etc) From Baseline Over Time | The patient's electrocardiogram will be measured, and it's one kind of laboratory test. | Baseline, 2, 4, 6, 8, 12, 16, 20, 26, 39 and 52 weeks. |
| Change in Auditory Function From Baseline Over Time | The patient's auditory function will be determined by otorhinolaryngology. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in White Blood Count (Unit: 10^9/L) From Baseline Over Time | The patient's white blood count will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Neutrophil Count (Unit: 10^9/L) From Baseline Over Time | The patient's neutrophil count will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Lymphocyte Count (Unit: 10^9/L) From Baseline Over Time | The patient's lymphocyte count will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Monocyte Count (Unit: 10^9/L) From Baseline Over Time | The patient's monocyte count will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Eosinophilic Count (Unit: 10^9/L) From Baseline Over Time | The patient's eosinophilic count will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Basophilic Count (Unit: 10^9/L) From Baseline Over Time | The patient's basophilic count will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Red Blood Count (Unit: 10^9/L) From Baseline Over Time | The patient's red blood count will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Hemoglobin (Unit: g/L) From Baseline Over Time | The patient's hemoglobin will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Hematocrit (Unit:%) From Baseline Over Time | The patient's hematocrit will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Blood Platelet Count (Unit: 10^9/L) From Baseline Over Time | The patient's blood platelet count will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Thrombocytocrit (Unit: %) From Baseline Over Time | The patient's thrombocytocrit will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Alanine Aminotransferase (Unit: U/L) From Baseline Over Time | The patient's alanine aminotransferase will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Total Bilirubin (Unit:µmol/L) From Baseline Over Time | The patient's total bilirubin will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Total Protein (Unit:g/L) From Baseline Over Time | The patient's total protein will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Albumin (Unit: g/L) From Baseline Over Time | The patient's albumin will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Globulin (Unit: g/L) From Baseline Over Time | The patient's globulin will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Alkaline Phosphatase (Unit: U/L) From Baseline Over Time | The patient's alkaline phosphatase will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Aspartate Aminotransferase (Unit: U/L) From Baseline Over Time | The patient's aspartate aminotransferase will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Lactate Dehydrogenase (Unit: IU/L) From Baseline Over Time | The patient's lactate dehydrogenase will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Urea (Unit: mmol/L) From Baseline Over Time | The patient's urea will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Creatinine (Unit:µmol/L) From Baseline Over Time | The patient's creatinine will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Glucose (Unit: mmol/L) From Baseline Over Time [ Time | The patient's glucose will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Potassium (Unit: mmol/L) From Baseline Over Time | The patient's potassium will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Sodium (Unit: mmol/L) From Baseline Over Time | The patient's sodium will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Chlorine (Unit: mmol/L) From Baseline Over Time | The patient's chlorine will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Calcium (Unit: mmol/L) From Baseline Over Time | The patient's calcium will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Fibrinogen (Unit: g/L) From Baseline Over Time | The patient's fibrinogen will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Prothrombin time (Unit: s) From Baseline Over Time | The patient's prothrombin time will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Activated Partial Thromboplastin Time (APTT, Unit: s) From Baseline Over Time | The patient's APTT will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Serum Total Thyroxine (Unit: nmol/L) From Baseline Over Time | The patient's serum total thyroxine will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Parathyroid Hormone (Unit: pg/mL) From Baseline Over Time | The patient's parathyroid hormone will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Total Triiodothyronine (Unit: nmol/L) From Baseline Over Time | The patient's total triiodothyronine will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Thyrotropin (Unit: mIU/L) From Baseline Over Time | The patient's thyrotropin will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Urine Glucose (Unit: mmol/L) From Baseline Over Time | The patient's urine glucose will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Urine Protein (Unit: g/L)) From Baseline Over Time | The patient's urine protein will be determined, and it's one kind of laboratory test. | Baseline, 2, 12, 26, 39 and 52 weeks. |
| Absolute Change in Total Testosterone (Unit: ng/ml) in men From Baseline Over Time | The patient's total testosterone will be determined in men, and it's one kind of laboratory test. | Baseline, 26, 39 and 52 weeks. |
| Absolute Change in Free Testosterone (Unit: nmol/L) in men From Baseline Over Time | The patient's free testosterone will be determined in men, and it's one kind of laboratory test. | Baseline, 26, 39 and 52 weeks. |
| Absolute Change in Follicle-generating Hormone (Unit: mIU/mL) in women From Baseline Over Time | The patient's follicle-generating hormone will be determined in women, and it's one kind of laboratory test. | Baseline, 26, 39 and 52 weeks. |
| Absolute Change in Luteinizing Hormon (Unit: mIU/mL) in women From Baseline Over Time | The patient's luteinizing hormonwill be determined in women, and it's one kind of laboratory test. | Baseline, 26, 39 and 52 weeks. |
Serum ferritin will be determined, and it's one kind of laboratory test. |
| Baseline, 2, 4, 6, 8, 12, 16, 20, 26, 39 and 52 weeks. |
| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D019189 | Iron Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |