Back to resultsLong-term Clinical Study of CN128 in Thalassemia With Sever Liver Iron Overloaded Patients
Primary Purpose
Thalassemia, Iron Overload
Status
Recruiting
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
CN128 Tablets
Sponsored by
About this trial
This is an interventional treatment trial for Thalassemia
Eligibility Criteria
Inclusion Criteria:
- Thalassemia patients.
- The number of blood transfusion per month ≥1. Or hemoglobin can not be maintained at 90g/L above, if blood transfusions is less than once per month.
- Severe iron overloaded patients (serum ferritin>2500 µg/L or liver iron concentration (LIC)>15 mg/g dw) despite prior treatment with at least one iron chelator.
- Patients aged 16 and above.
- Volunteer for the trial and sign the informed consent.
Exclusion Criteria:
- Active hepatitis B (HBsAg positive, HBsAb negative) or hepatitis C (HCV antibody positive, detectable HCV RNA, and Alkaline Phosphatase (ALT) beyond normal range)
- Active gastrointestinal disease history (including: gastric ulcer, duodenal ulcer, stomach or esophageal varices, ulcerative colitis, Crohn's disease, gastrointestinal cancer, familial genetic multiple intestinal polyps), and History of gastrointestinal perforation, gastrointestinal surgery that influence drug absorption, and other potential intestinal complications considered by researchers.
- ALT or Aspartate Aminotransferase (AST) > 2.5 × upper limit of normal (ULN), or serum creatinine > 1.5 × ULN.
- Neutropenia patient (neutrophil count < 1.5 × 10^9 / L).
- Active infection uncontrolled.
- Autoimmune hemolytic anemia patients.
- The patients who are currently taking CYP3A strong inducer or strong inhibitor drugs, or the drug that may extend the QT interval, or the drug that may decrease neutrophil count, but can not temporarily interrupt the use of such drugs.
- Cardiac iron overloaded patients, cardiac magnetic resonance MRI T2*<10 ms
- The patients who are allergic or contraindicated to the main ingredients or excipients of CN128 tablets.
- Congenital long QT syndrome or known family history of long QT syndrome, QTc interval>480 ms, clinically significant ventricular or atrial fast arrhythmia.
- The patients who can not accept MRI as detection means, such as claustrophobic for MRI, pacemaker, and those using ferromagnetic metal implants.
- Birth planner (including male subjects) within or within 3 months after the end of the trial.
- Participated in other clinical trials in the four weeks before taking the test preparation, except for non-interventional studies.
- Pregnant or lactating women.
- Unsuitable to participate in the trial considered by the researchers.
Sites / Locations
- The First Affiliated Hospital Of Guangxi Medical UniversityRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
CN128 Group
Arm Description
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.
Outcomes
Primary Outcome Measures
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.
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.
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.
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.
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.
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.
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.
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.
Change in Auditory Function From Baseline Over Time
The patient's auditory function will be determined by otorhinolaryngology.
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.
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.
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.
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.
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.
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.
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.
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.
Absolute Change in Hematocrit (Unit:%) From Baseline Over Time
The patient's hematocrit will be determined, and it's one kind of laboratory test.
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.
Absolute Change in Thrombocytocrit (Unit: %) From Baseline Over Time
The patient's thrombocytocrit will be determined, and it's one kind of laboratory test.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
Secondary Outcome Measures
Absolute Change in Liver iron content (Unit: mg/g dw) From Baseline Over Time
Liver iron content will be determined by liver magnetic resonance.
Absolute Change in Serum ferritin (Unit: µg/L) From Baseline Over Time
Serum ferritin will be determined, and it's one kind of laboratory test.
Full Information
NCT ID
NCT05355766
First Posted
April 22, 2022
Last Updated
June 8, 2023
Sponsor
Hangzhou Zede Pharma-Tech Co., Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT05355766
Brief Title
Long-term Clinical Study of CN128 in Thalassemia With Sever Liver Iron Overloaded Patients
Official Title
Phase IIb Clinical Study to Assess the Safety and Efficacy of CN128 Tablets in the Treatment of Iron Overload in Transfusion Dependent Thalassemia With Sever Liver Iron Overloaded Patients Aged 16 and Above
Study Type
Interventional
2. Study Status
Record Verification Date
June 2023
Overall Recruitment Status
Recruiting
Study Start Date
June 2, 2022 (Actual)
Primary Completion Date
December 31, 2023 (Anticipated)
Study Completion Date
March 31, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Hangzhou Zede Pharma-Tech Co., Ltd.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
The safety and efficacy of CN128 is studied in thalassaemia with sever liver iron overloaded patients.
Detailed Description
The study is designed as a single arm and opened phase IIb clinical trial, so as to investigate the safety and efficacy of CN128.
A total of 50 eligible subjects are planned to be enrolled, and orally administration of CN128 for 52 weeks according to the administration plan.
Administration plan:
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Thalassemia, Iron Overload
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
50 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
CN128 Group
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
CN128 Tablets
Other Intervention Name(s)
CN128
Intervention Description
Iron chelator, oral tablets
Primary Outcome Measure Information:
Title
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.
Description
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.
Time Frame
Up to 52 weeks
Title
Absolute Change in Weight (Unit: kg) From Baseline Over Time
Description
The patient's weight will be determined, and it's one kind of Physical examination.
Time Frame
Baseline, 26, 39 and 52 weeks.
Title
Absolute Change in Height (Unit: m) From Baseline Over Time
Description
The patient's height will be determined, and it's one kind of Physical examination.
Time Frame
Baseline, 26, 39 and 52 weeks.
Title
Absolute Change in Temperature (Unit: ℃）From Baseline Over Time
Description
The patient's temperature will be determined, and it's one kind of vital signs checks.
Time Frame
Baseline, 2, 4, 6, 8, 12, 16, 20, 26, 39 and 52 weeks.
Title
Absolute Change in Blood pressure (Unit: mmHg ) From Baseline Over Time
Description
Both patient's systolic and diastolic blood pressure will be measured, and it's one kind of vital signs checks.
Time Frame
Baseline, 2, 4, 6, 8, 12, 16, 20, 26, 39 and 52 weeks.
Title
Absolute Change in Heart rate (Unit: bpm) From Baseline Over Time
Description
The patient's heart rate will be determined, and it's one kind of vital signs checks.
Time Frame
Baseline, 2, 4, 6, 8, 12, 16, 20, 26, 39 and 52 weeks.
Title
Absolute Change in Respiration (Unit: bpm) From Baseline Over Time
Description
The patient's respiration will be determined, and it's one kind of vital signs checks.
Time Frame
Baseline, 2, 4, 6, 8, 12, 16, 20, 26, 39 and 52 weeks.
Title
Absolute Change in Electrocardiogram (P-R (Unit: ms), QRS (Unit: ms), QTc interval (Unit: ms), etc) From Baseline Over Time
Description
The patient's electrocardiogram will be measured, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 4, 6, 8, 12, 16, 20, 26, 39 and 52 weeks.
Title
Change in Auditory Function From Baseline Over Time
Description
The patient's auditory function will be determined by otorhinolaryngology.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in White Blood Count (Unit: 10^9/L) From Baseline Over Time
Description
The patient's white blood count will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Neutrophil Count (Unit: 10^9/L) From Baseline Over Time
Description
The patient's neutrophil count will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Lymphocyte Count (Unit: 10^9/L) From Baseline Over Time
Description
The patient's lymphocyte count will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Monocyte Count (Unit: 10^9/L) From Baseline Over Time
Description
The patient's monocyte count will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Eosinophilic Count (Unit: 10^9/L) From Baseline Over Time
Description
The patient's eosinophilic count will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Basophilic Count (Unit: 10^9/L) From Baseline Over Time
Description
The patient's basophilic count will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Red Blood Count (Unit: 10^9/L) From Baseline Over Time
Description
The patient's red blood count will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Hemoglobin (Unit: g/L) From Baseline Over Time
Description
The patient's hemoglobin will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Hematocrit (Unit:%) From Baseline Over Time
Description
The patient's hematocrit will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Blood Platelet Count (Unit: 10^9/L) From Baseline Over Time
Description
The patient's blood platelet count will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Thrombocytocrit (Unit: %) From Baseline Over Time
Description
The patient's thrombocytocrit will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Alanine Aminotransferase (Unit: U/L) From Baseline Over Time
Description
The patient's alanine aminotransferase will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Total Bilirubin (Unit:µmol/L) From Baseline Over Time
Description
The patient's total bilirubin will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Total Protein (Unit:g/L) From Baseline Over Time
Description
The patient's total protein will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Albumin (Unit: g/L) From Baseline Over Time
Description
The patient's albumin will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Globulin (Unit: g/L) From Baseline Over Time
Description
The patient's globulin will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Alkaline Phosphatase (Unit: U/L) From Baseline Over Time
Description
The patient's alkaline phosphatase will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Aspartate Aminotransferase (Unit: U/L) From Baseline Over Time
Description
The patient's aspartate aminotransferase will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Lactate Dehydrogenase (Unit: IU/L) From Baseline Over Time
Description
The patient's lactate dehydrogenase will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Urea (Unit: mmol/L) From Baseline Over Time
Description
The patient's urea will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Creatinine (Unit:µmol/L) From Baseline Over Time
Description
The patient's creatinine will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Glucose (Unit: mmol/L) From Baseline Over Time [ Time
Description
The patient's glucose will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Potassium (Unit: mmol/L) From Baseline Over Time
Description
The patient's potassium will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Sodium (Unit: mmol/L) From Baseline Over Time
Description
The patient's sodium will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Chlorine (Unit: mmol/L) From Baseline Over Time
Description
The patient's chlorine will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Calcium (Unit: mmol/L) From Baseline Over Time
Description
The patient's calcium will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Fibrinogen (Unit: g/L) From Baseline Over Time
Description
The patient's fibrinogen will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Prothrombin time (Unit: s) From Baseline Over Time
Description
The patient's prothrombin time will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Activated Partial Thromboplastin Time (APTT, Unit: s) From Baseline Over Time
Description
The patient's APTT will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Serum Total Thyroxine (Unit: nmol/L) From Baseline Over Time
Description
The patient's serum total thyroxine will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Parathyroid Hormone (Unit: pg/mL) From Baseline Over Time
Description
The patient's parathyroid hormone will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Total Triiodothyronine (Unit: nmol/L) From Baseline Over Time
Description
The patient's total triiodothyronine will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Thyrotropin (Unit: mIU/L) From Baseline Over Time
Description
The patient's thyrotropin will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Urine Glucose (Unit: mmol/L) From Baseline Over Time
Description
The patient's urine glucose will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Urine Protein (Unit: g/L)) From Baseline Over Time
Description
The patient's urine protein will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 12, 26, 39 and 52 weeks.
Title
Absolute Change in Total Testosterone (Unit: ng/ml) in men From Baseline Over Time
Description
The patient's total testosterone will be determined in men, and it's one kind of laboratory test.
Time Frame
Baseline, 26, 39 and 52 weeks.
Title
Absolute Change in Free Testosterone (Unit: nmol/L) in men From Baseline Over Time
Description
The patient's free testosterone will be determined in men, and it's one kind of laboratory test.
Time Frame
Baseline, 26, 39 and 52 weeks.
Title
Absolute Change in Follicle-generating Hormone (Unit: mIU/mL) in women From Baseline Over Time
Description
The patient's follicle-generating hormone will be determined in women, and it's one kind of laboratory test.
Time Frame
Baseline, 26, 39 and 52 weeks.
Title
Absolute Change in Luteinizing Hormon (Unit: mIU/mL) in women From Baseline Over Time
Description
The patient's luteinizing hormonwill be determined in women, and it's one kind of laboratory test.
Time Frame
Baseline, 26, 39 and 52 weeks.
Secondary Outcome Measure Information:
Title
Absolute Change in Liver iron content (Unit: mg/g dw) From Baseline Over Time
Description
Liver iron content will be determined by liver magnetic resonance.
Time Frame
Baseline, 12, 26,39 and 52 weeks.
Title
Absolute Change in Serum ferritin (Unit: µg/L) From Baseline Over Time
Description
Serum ferritin will be determined, and it's one kind of laboratory test.
Time Frame
Baseline, 2, 4, 6, 8, 12, 16, 20, 26, 39 and 52 weeks.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
16 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Thalassemia patients.
The number of blood transfusion per month ≥1. Or hemoglobin can not be maintained at 90g/L above, if blood transfusions is less than once per month.
Severe iron overloaded patients (serum ferritin>2500 µg/L or liver iron concentration (LIC)>15 mg/g dw) despite prior treatment with at least one iron chelator.
Patients aged 16 and above.
Volunteer for the trial and sign the informed consent.
Exclusion Criteria:
Active hepatitis B (HBsAg positive, HBsAb negative) or hepatitis C (HCV antibody positive, detectable HCV RNA, and Alkaline Phosphatase (ALT) beyond normal range)
Active gastrointestinal disease history (including: gastric ulcer, duodenal ulcer, stomach or esophageal varices, ulcerative colitis, Crohn's disease, gastrointestinal cancer, familial genetic multiple intestinal polyps), and History of gastrointestinal perforation, gastrointestinal surgery that influence drug absorption, and other potential intestinal complications considered by researchers.
ALT or Aspartate Aminotransferase (AST) > 2.5 × upper limit of normal (ULN), or serum creatinine > 1.5 × ULN.
Neutropenia patient (neutrophil count < 1.5 × 10^9 / L).
Active infection uncontrolled.
Autoimmune hemolytic anemia patients.
The patients who are currently taking CYP3A strong inducer or strong inhibitor drugs, or the drug that may extend the QT interval, or the drug that may decrease neutrophil count, but can not temporarily interrupt the use of such drugs.
Cardiac iron overloaded patients, cardiac magnetic resonance MRI T2*<10 ms
The patients who are allergic or contraindicated to the main ingredients or excipients of CN128 tablets.
Congenital long QT syndrome or known family history of long QT syndrome, QTc interval>480 ms, clinically significant ventricular or atrial fast arrhythmia.
The patients who can not accept MRI as detection means, such as claustrophobic for MRI, pacemaker, and those using ferromagnetic metal implants.
Birth planner (including male subjects) within or within 3 months after the end of the trial.
Participated in other clinical trials in the four weeks before taking the test preparation, except for non-interventional studies.
Pregnant or lactating women.
Unsuitable to participate in the trial considered by the researchers.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Jianmin Luo, PhD
Phone
+86-13517667021
Email
jmluo@aliyun.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jianmin Luo, PhD
Organizational Affiliation
First Affiliated Hospital of Guangxi Medical University
Official's Role
Principal Investigator
Facility Information:
Facility Name
The First Affiliated Hospital Of Guangxi Medical University
City
Nanning
State/Province
Guangxi
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Yanwen Zhou
Email
gxzywjg@163.com
12. IPD Sharing Statement
Learn more about this trial
Long-term Clinical Study of CN128 in Thalassemia With Sever Liver Iron Overloaded Patients
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