ThalassemiaPhase 2NCT04614779
Long-term Clinical Study of CN128 in Thalassemia Patients
A Phase 2 study testing CN128 Tablets in people with Thalassemia and Iron Overload, run by Hangzhou Zede Pharma-Tech Co., Ltd. at 1 site in China.
Trial summary
1. Primary objectives: To evaluate the safety and efficacy of long-term orally administration of CN128 in thalassaemia patients with blood transfusion dependent and aged 16 and above. 2. Read more about this study
- Who is it for?
- Ages 16 to 60 years
- Conditions the study is for
- Thalassemia, Iron Overload
- What is being tested?
- CN128 Tablets
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteChina
- Study dates
- 30 Sep 2020to 18 Aug 2022
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
- Serum ferritin ≥ 500 µg/L
- Patients aged 16 and above
- Volunteer for the trial and sign the informed consent.
Who cannot join
- Active hepatitis B (HBsAg positive, HBsAb negative) or hepatitis C (HCV antibody positive, detectable HCV RNA, and 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 AST > 2.5 × ULN, or serum creatinine > 1.5 × ULN;
- Neutropenia patient (neutrophil count < 1.5 × 109 / L);
- Active infection uncontrolled;
- 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;
Show all 13Show fewer
- 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 > 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 three months before taking the test preparation, except for non-interventional studies;
- Pregnant or lactating women;
- Unsuitable to participate in the trial considered by the researchers.
Trial locations and contacts
1 site in 1 country.
China1 site
The First Affiliated Hospital Of Guangxi Medical University
Nanning, Guangxi, 530021
About this study
Official title: Phase IIa Clinical Study to Assess the Safety and Efficacy of CN128 Tablets in the Treatment of Iron Overload in Transfusion Dependent Thalassemia Patients Aged 16 and Above
- Primary objectives:
- To evaluate the safety and efficacy of long-term orally administration of CN128 in thalassaemia patients with blood transfusion dependent and aged 16 and above.
Read more
- Design:
- The study is designed as a single arm and opened phase IIa 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 24 weeks or 48 weeks according to the administration plan. The treatment period is from day 0 to 24 weeks, and the extended treatment period was from 25 weeks to 96 weeks.
- Subjects' medication status, uncomfortable symptoms, concomitant medication or non-drug therapy were recorded daily.
- Subject 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.
- Serum ferritin ≥ 500 µg/L
- Patients aged 16 and above
- Volunteer for the trial and sign the informed consent.
- Subject exclusion criteria:
- Active hepatitis B (HBsAg positive, HBsAb negative) or hepatitis C (HCV antibody positive, detectable HCV RNA, and alanine transaminase (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 transaminase (AST) > 2.5 × Upper limit of normal (ULN), or serum creatinine > 1.5 × ULN;
- Neutropenia patient (neutrophil count < 1.5 × 109 / L);
- Active infection uncontrolled;
- 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;
- Congenital long QT syndrome or known family history of long QT syndrome; QTc > 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 three months before taking the test preparation, except for non-interventional studies;
- Pregnant or lactating women;
- Unsuitable to participate in the trial considered by the researchers.
- Usage:
- All subjects will be given the lower (10 mg/kg bw, bid) or higher dose (15 mg/kg bw, bid) for 24 or 48 weeks, according to the administration plan.
- All subjects will be given the lower (15 mg/kg bw, bid) or higher dose (20 mg/kg bw, bid) for 49 or 96 weeks, according to the administration plan.
- Safety assessments: Safety evaluations include adverse events, adverse reactions, severe adverse events, and severe adverse reactions; growth (weight, height); total and free testosterone in men, follicle-generating hormone and luteinizing hormone in women; vital signs and electrocardiogram; hearing, laboratory tests (blood routine analytes, blood biochemistry, coagulation function, thyroid and para-thyroid function, urine routine analytes.), urine pregnancy test (women of childbearing age),Levels of drug exposure during the study.
- Efficacy assessments: Efficacy evaluations include serum ferritin, liver iron content (MRI R2) and cardiac iron content (MRI T2*).
- Statistics:
- Subject characteristic distribution Demographic characteristics, general conditions, and baseline conditions (pre-treatment) of enrolled subjects were analyzed.The measurement data are described by means, standard deviation, minimum value and maximum value, while the qualitative data list frequency and percentage.
- Safety analysis Descriptive statistical analysis was used for safety endpoints.
- Effectiveness analysis Mean, standard deviation, median, minimum and maximum values were described and 95% confidence intervals were calculated. Paired T-test was used to compare each time point with the baseline if necessary. The 95% confidence interval was calculated by using Clopper-Pearson method for the proportion of patients.
Read the detailed description
Clinical Trial - IIa - Study Description - Detailed Description
- Primary objectives:
- To evaluate the adverse events, adverse reactions, severe adverse events and severe adverse reactions during the study period, so as to investigate the safety;
- To evaluate the changes of serum ferritin over time after orally administration of CN128 in thalassaemia patients with blood transfusion dependent and aged 16 and above, so as to investigate the efficacy;
- To evaluate the effect on iron excretion in liver after orally administration of CN128 for 12, 24 weeks in thalassaemia patients with blood transfusion dependent and aged 16 and above, so as to investigate the efficacy;
- To evaluate the effect on iron excretion in heart after orally administration of CN128 for 12, 24 weeks in thalassaemia patients with blood transfusion dependent and aged 16 and above, so as to investigate the efficacy;
- To evaluate the proportion of patients with decreased or unchanged liver iron content after orally administration of CN128 for 12, 24 weeks in thalassaemia patients with blood transfusion dependent and aged 16 and above, so as to investigate the efficacy;
- To evaluate the safety and efficacy of long-term orally administration of CN128 in thalassaemia patients with blood transfusion dependent and aged 16 and above.
- Design:
- The study is designed as a single arm and opened phase IIa 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 24 weeks or 48 weeks according to the administration plan. The treatment period is from day 0 to 24 weeks, and the extended treatment period was from 25 weeks to 96 weeks.
- Administration plan: (1)0 day~48 weeks： The trial will start with the lower dose of CN128 (10 mg/kg body weight [bw], bid) for two weeks, then the subjects will return to the study center. If no unacceptable toxicity associated with CN128 is found, the subjects will be given the higher dose (15 mg/kg body weight [bw], bid). If unacceptable toxicity associated with CN128 is found, the subjects will be suspended or stopped administration. If the adverse event turns to normal or abnormal but no clinical significance after suspension administration, the subjects will be given the lower dose of CN128 (10 mg/kg body weight [bw], bid). After taking CN128 at 15 mg/kg, if unacceptable toxicity associated with CN128 is found, the dosage will be reduce to 10 mg/kg. If no symptoms appear, the dose can be increased to 15 mg/kg. If the unacceptable toxicity related to CN128 still occurs, the subjects will be suspended or stopped administration. If the adverse event turns to normal or abnormal but no clinical significance after suspension administration, the subjects will be given the lower dose of CN128 (10 mg/kg body weight [bw], bid). The dose will be assessed once every two or four weeks. Subjects' medication status, uncomfortable symptoms, concomitant medication or non-drug therapy were recorded daily. (2)49 weeks~96 weeks： Subjects who have completed 48 weeks of treatment may continue dosing at the original dose until the end of the study or withdraw early if they do not experience intolerable toxicity associated with CN128 tablets and have fair efficacy (≥20% elevation on MRI T2*) and if, in the judgment of the investigator, the benefits outweigh the risks. Subjects who have completed 48 weeks of treatment, who have not experienced intolerable toxicity associated with CN128 tablets, but who have been assessed by the investigator as having poor efficacy (<20% elevation of MRI T2*) and in the judgment of the investigator the benefit outweighs the risk, may, with the subject's consent, have the dosage increased at the additional visit or the established most recent visit to 15 mg/kg in the morning and 20 mg/kg in the evening, with an assessment made at 2 weeks of dosing： If no relevant intolerable toxicity occurs, the dose may continue to be increased to 20 mg/kg bid and assessed after 2 weeks of dosing:
- If no relevant intolerable toxicity occurs, the 20 mg/kg bid dose may be administered until the end of the study or early withdrawal (daily dose 40 mg/kg/d);
- If relevant intolerable toxicity occurs, the dose may be reduced to 15 mg/kg in the morning and 20 mg/kg bid in the evening until the end of the study or early withdrawal (daily dose 35 mg/kg/d). If relevant intolerable toxicity occurs, the dose may be reduced to 15 mg/kg bid until the end of the study or early withdrawal (daily dose 30 mg/kg/d).
- Subject 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.
- Serum ferritin ≥ 500 µg/L
- Patients aged 16 and above
- Volunteer for the trial and sign the informed consent.
- Subject exclusion criteria:
- Active hepatitis B (HBsAg positive, HBsAb negative) or hepatitis C (HCV antibody positive, detectable HCV RNA, and 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 AST > 2.5 × ULN, or serum creatinine > 1.5 × ULN;
- Neutropenia patient (neutrophil count < 1.5 × 109 / L);
- Active infection uncontrolled;
- 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;
- 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 > 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 three months before taking the test preparation, except for non-interventional studies;
- Pregnant or lactating women;
- Unsuitable to participate in the trial considered by the researchers.
- Usage: All subjects will be given the lower (10 mg/kg bw, bid) or higher dose (15 mg/kg bw, bid) for 24 or 48 weeks, according to the administration plan. All subjects will be given the lower (15 mg/kg bw, bid) or higher dose (20 mg/kg bw, bid) for 49 or 96 weeks, according to the administration plan.
- Safety assessments: Safety evaluations include adverse events, adverse reactions, severe adverse events, and severe adverse reactions; growth (weight, height); total and free testosterone in men, follicle-generating hormone and luteinizing hormone in women; vital signs and electrocardiogram; hearing, laboratory tests (blood routine analytes, blood biochemistry, coagulation function, thyroid and para-thyroid function, urine routine analytes.), urine pregnancy test (women of childbearing age),Levels of drug exposure during the study.
- Efficacy assessments: Efficacy evaluations include serum ferritin, liver iron content (MRI R2) and cardiac iron content (MRI T2*).
- Statistics:
- Subject characteristic distribution Demographic characteristics, general conditions, and baseline conditions (pre-treatment) of enrolled subjects were analyzed.The measurement data are described by means, standard deviation, minimum value and maximum value, while the qualitative data list frequency and percentage.
- Safety analysis Descriptive statistical analysis was used for safety endpoints. Summarize the incidence of adverse events, adverse reactions, adverse events leading to withdrawal from the trial, adverse events leading to death, severe adverse events, and severe adverse reactions. The incidence is calculated by subsystem, symptom/sign. Severity of adverse events and adverse reactions: if multiple adverse events occur in the same subject, the most serious one is included in the analysis; if different adverse events occurred in the same subject, the most severe adverse events were counted in the analysis. Drug exposure during the study: describe medication compliance during the study, actual dose, administration adjustments during the study, whether the study was discontinued, and reasons for the suspension.
- Effectiveness analysis Mean, standard deviation, median, minimum and maximum values were described and 95% confidence intervals were calculated. Paired T-test was used to compare each time point with the baseline if necessary. The 95% confidence interval was calculated by using Clopper-Pearson method for the proportion of patients.
- Conditions
- Thalassemia, Iron Overload
- Being tested
- CN128 Tablets
- Medical subject headings
- Thalassemia, Iron Overload
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CN128 TabletsDrug
Also called CN128
Iron chelator, oral tablets
| Study group | Role | What they receive |
|---|---|---|
| CN128 Group | Experimental | All subjects will be given the lower (10 mg/kg bw, bid) or higher dose (15 mg/kg bw, bid) for 24 or 48 weeks, according to the administration plan.
All subjects will be given the lower (15 mg/kg bw, bid) or higher dose (20 mg/kg bw, bid) for 49 or 96 weeks, according to the administration plan.
The dosage form is tablets. Receives: Drug: CN128 Tablets |
What is being measured
The primary outcome is the main question the study is designed to answer.
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 96 weeks.
Measured: up to 96 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.
Measured: Baseline, 24, 48, 72 and 96 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.
Measured: Baseline,24, 48, 72 and 96 weeks.
Absolute Change in Hormon (total and free testosterone in men, follicle-generating hormone and luteinizing hormon in women) From Baseline Over Time
Hormon will be determined, and it's one kind of laboratory test.
Measured: Baseline, 24, 48, 72 and 96 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.
Measured: Baseline, 2, 4, 8, 12, 16, 20, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 4, 8, 12, 16, 20, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 4, 8, 12, 16, 20, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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.
Measured: Baseline, 2, 4, 8, 12, 16, 20, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
Absolute Change in Electrocardiogram (P-R (Unit: ms), QRS (Unit: ms), QTc (Unit: ms), etc) From Baseline Over Time
The patient's electrocardiogram will be measured, and it's one kind of laboratory test.
Measured: Baseline, 2, 4, 8, 12, 16, 20, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
Change in Auditory Function From Baseline Over Time
The patient's auditory function will be determined by otorhinolaryngology.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
Absolute Change in White Blood Count (Unit: 10E9/L) From Baseline Over Time
The patient's white blood count will be determined, and it's one kind of laboratory test.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
Absolute Change in Neutrophil Count (Unit: 10E9/L) From Baseline Over Time
The patient's neutrophil count will be determined, and it's one kind of laboratory test.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
Absolute Change in Lymphocyte Count (Unit: 10E9/L) From Baseline Over Time
The patient's lymphocyte count will be determined, and it's one kind of laboratory test.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
Absolute Change in Monocyte Count (Unit: 10E9/L) From Baseline Over Time
The patient's monocyte count will be determined, and it's one kind of laboratory test.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
Absolute Change in Eosinophilic Count (Unit: 10E9/L) From Baseline Over Time
The patient's eosinophilic count will be determined, and it's one kind of laboratory test.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
Absolute Change in Basophilic Count (Unit: 10E9/L) From Baseline Over Time
The patient's basophilic count will be determined, and it's one kind of laboratory test.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
Absolute Change in Red Blood Count (Unit: 10E9/L) From Baseline Over Time
The patient's red blood count will be determined, and it's one kind of laboratory test.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
Absolute Change in Blood Platelet Count (Unit: 10E9/L) From Baseline Over Time
The patient's blood platelet count will be determined, and it's one kind of laboratory test.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
Absolute Change in Glucose (Unit: mmol/L) From Baseline Over Time
The patient's glucose will be determined, and it's one kind of laboratory test.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
Absolute Change in Thyroid and para-gland function (Serum total thyroxine, parathyroid hormone, total triiodothyronine and thyrotropin) From Baseline Over Time
The patient's thyroid and para-gland function will be determined, and it's one kind of laboratory test.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 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.
Measured: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
Absolute Change in Dosage（mg/kg）during the study period
The dosages will be investigated up to 96 weeks.
Measured: Up to 96 weeks
Duration of Administration during the study period
The duration of administration will be investigated up to 96 weeks.
Measured: Up to 96 weeks
Number of Participants during the study period
The number of participants will be investigated up to 96 weeks.
Measured: Up to 96 weeks
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.
Measured: Baseline, 2, 4, 8, 12, 16, 20, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
Absolute Change in Liver iron content (MRI R2) From Baseline Over Time
Liver iron content will be determined by liver magnetic resonance.
Measured: Baseline, 12, 24, 48, 72 and 96 weeks.
Absolute Change in Heart iron content (MRI T2*) From Baseline Over Time
Heart iron content will be determined by cardio magnetic resonance.
Measured: Baseline, 12, 24, 48, 72 and 96 weeks.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Nov 2020
- Study start30 Sep 2020
- Primary completion18 Aug 2022
- Study completion18 Aug 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ThalassemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hangzhou Zede Pharma-Tech Co., Ltd.
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jianmin Jianmin, PhD, Principal investigator, First Affiliated Hospital of Guangxi Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04614779
- Sponsor's protocol ID
- A160605
- Status last verified
- Nov 2023
- First submitted
- 23 Sep 2020
- Last updated
- 22 Nov 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase IIa Clinical Study to Assess the Safety and Efficacy of CN128 Tablets in the Treatment of Iron Overload in Transfusion Dependent Thalassemia Patients Aged 16 and Above
- ClinicalTrials.gov ID
- NCT04614779
- Acronym / sponsor ID
- A160605
- Conditions
- Thalassemia, Iron Overload
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Hangzhou Zede Pharma-Tech Co., Ltd.University, hospital or other
- Study start
- 30 Sep 2020 (actual)
- Primary completion
- 18 Aug 2022 (actual)
- Study completion
- 18 Aug 2022 (actual)
- First posted
- 4 Nov 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
- Primary objectives:
- To evaluate the safety and efficacy of long-term orally administration of CN128 in thalassaemia patients with blood transfusion dependent and aged 16 and above.
- Design:
- The study is designed as a single arm and opened phase IIa 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 24 weeks or 48 weeks according to the administration plan. The treatment period is from day 0 to 24 weeks, and the extended treatment period was from 25 weeks to 96 weeks.
- Subjects' medication status, uncomfortable symptoms, concomitant medication or non-drug therapy were recorded daily.
- Subject 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.
- Serum ferritin ≥ 500 µg/L
- Patients aged 16 and above
- Volunteer for the trial and sign the informed consent.
- Subject exclusion criteria:
- Active hepatitis B (HBsAg positive, HBsAb negative) or hepatitis C (HCV antibody positive, detectable HCV RNA, and alanine transaminase (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 transaminase (AST) > 2.5 × Upper limit of normal (ULN), or serum creatinine > 1.5 × ULN;
- Neutropenia patient (neutrophil count < 1.5 × 109 / L);
- Active infection uncontrolled;
- 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;
- Congenital long QT syndrome or known family history of long QT syndrome; QTc > 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 three months before taking the test preparation, except for non-interventional studies;
- Pregnant or lactating women;
- Unsuitable to participate in the trial considered by the researchers.
- Usage:
- All subjects will be given the lower (10 mg/kg bw, bid) or higher dose (15 mg/kg bw, bid) for 24 or 48 weeks, according to the administration plan.
- All subjects will be given the lower (15 mg/kg bw, bid) or higher dose (20 mg/kg bw, bid) for 49 or 96 weeks, according to the administration plan.
- Safety assessments: Safety evaluations include adverse events, adverse reactions, severe adverse events, and severe adverse reactions; growth (weight, height); total and free testosterone in men, follicle-generating hormone and luteinizing hormone in women; vital signs and electrocardiogram; hearing, laboratory tests (blood routine analytes, blood biochemistry, coagulation function, thyroid and para-thyroid function, urine routine analytes.), urine pregnancy test (women of childbearing age),Levels of drug exposure during the study.
- Efficacy assessments: Efficacy evaluations include serum ferritin, liver iron content (MRI R2) and cardiac iron content (MRI T2*).
- Statistics:
- Subject characteristic distribution Demographic characteristics, general conditions, and baseline conditions (pre-treatment) of enrolled subjects were analyzed.The measurement data are described by means, standard deviation, minimum value and maximum value, while the qualitative data list frequency and percentage.
- Safety analysis Descriptive statistical analysis was used for safety endpoints.
- Effectiveness analysis Mean, standard deviation, median, minimum and maximum values were described and 95% confidence intervals were calculated. Paired T-test was used to compare each time point with the baseline if necessary. The 95% confidence interval was calculated by using Clopper-Pearson method for the proportion of patients.
Detailed description
Clinical Trial - IIa - Study Description - Detailed Description
- Primary objectives:
- To evaluate the adverse events, adverse reactions, severe adverse events and severe adverse reactions during the study period, so as to investigate the safety;
- To evaluate the changes of serum ferritin over time after orally administration of CN128 in thalassaemia patients with blood transfusion dependent and aged 16 and above, so as to investigate the efficacy;
- To evaluate the effect on iron excretion in liver after orally administration of CN128 for 12, 24 weeks in thalassaemia patients with blood transfusion dependent and aged 16 and above, so as to investigate the efficacy;
- To evaluate the effect on iron excretion in heart after orally administration of CN128 for 12, 24 weeks in thalassaemia patients with blood transfusion dependent and aged 16 and above, so as to investigate the efficacy;
- To evaluate the proportion of patients with decreased or unchanged liver iron content after orally administration of CN128 for 12, 24 weeks in thalassaemia patients with blood transfusion dependent and aged 16 and above, so as to investigate the efficacy;
- To evaluate the safety and efficacy of long-term orally administration of CN128 in thalassaemia patients with blood transfusion dependent and aged 16 and above.
- Design:
- The study is designed as a single arm and opened phase IIa 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 24 weeks or 48 weeks according to the administration plan. The treatment period is from day 0 to 24 weeks, and the extended treatment period was from 25 weeks to 96 weeks.
- Administration plan: (1)0 day~48 weeks： The trial will start with the lower dose of CN128 (10 mg/kg body weight [bw], bid) for two weeks, then the subjects will return to the study center. If no unacceptable toxicity associated with CN128 is found, the subjects will be given the higher dose (15 mg/kg body weight [bw], bid). If unacceptable toxicity associated with CN128 is found, the subjects will be suspended or stopped administration. If the adverse event turns to normal or abnormal but no clinical significance after suspension administration, the subjects will be given the lower dose of CN128 (10 mg/kg body weight [bw], bid). After taking CN128 at 15 mg/kg, if unacceptable toxicity associated with CN128 is found, the dosage will be reduce to 10 mg/kg. If no symptoms appear, the dose can be increased to 15 mg/kg. If the unacceptable toxicity related to CN128 still occurs, the subjects will be suspended or stopped administration. If the adverse event turns to normal or abnormal but no clinical significance after suspension administration, the subjects will be given the lower dose of CN128 (10 mg/kg body weight [bw], bid). The dose will be assessed once every two or four weeks. Subjects' medication status, uncomfortable symptoms, concomitant medication or non-drug therapy were recorded daily. (2)49 weeks~96 weeks： Subjects who have completed 48 weeks of treatment may continue dosing at the original dose until the end of the study or withdraw early if they do not experience intolerable toxicity associated with CN128 tablets and have fair efficacy (≥20% elevation on MRI T2*) and if, in the judgment of the investigator, the benefits outweigh the risks. Subjects who have completed 48 weeks of treatment, who have not experienced intolerable toxicity associated with CN128 tablets, but who have been assessed by the investigator as having poor efficacy (<20% elevation of MRI T2*) and in the judgment of the investigator the benefit outweighs the risk, may, with the subject's consent, have the dosage increased at the additional visit or the established most recent visit to 15 mg/kg in the morning and 20 mg/kg in the evening, with an assessment made at 2 weeks of dosing： If no relevant intolerable toxicity occurs, the dose may continue to be increased to 20 mg/kg bid and assessed after 2 weeks of dosing:
- If no relevant intolerable toxicity occurs, the 20 mg/kg bid dose may be administered until the end of the study or early withdrawal (daily dose 40 mg/kg/d);
- If relevant intolerable toxicity occurs, the dose may be reduced to 15 mg/kg in the morning and 20 mg/kg bid in the evening until the end of the study or early withdrawal (daily dose 35 mg/kg/d). If relevant intolerable toxicity occurs, the dose may be reduced to 15 mg/kg bid until the end of the study or early withdrawal (daily dose 30 mg/kg/d).
- Subject 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.
- Serum ferritin ≥ 500 µg/L
- Patients aged 16 and above
- Volunteer for the trial and sign the informed consent.
- Subject exclusion criteria:
- Active hepatitis B (HBsAg positive, HBsAb negative) or hepatitis C (HCV antibody positive, detectable HCV RNA, and 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 AST > 2.5 × ULN, or serum creatinine > 1.5 × ULN;
- Neutropenia patient (neutrophil count < 1.5 × 109 / L);
- Active infection uncontrolled;
- 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;
- 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 > 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 three months before taking the test preparation, except for non-interventional studies;
- Pregnant or lactating women;
- Unsuitable to participate in the trial considered by the researchers.
- Usage: All subjects will be given the lower (10 mg/kg bw, bid) or higher dose (15 mg/kg bw, bid) for 24 or 48 weeks, according to the administration plan. All subjects will be given the lower (15 mg/kg bw, bid) or higher dose (20 mg/kg bw, bid) for 49 or 96 weeks, according to the administration plan.
- Safety assessments: Safety evaluations include adverse events, adverse reactions, severe adverse events, and severe adverse reactions; growth (weight, height); total and free testosterone in men, follicle-generating hormone and luteinizing hormone in women; vital signs and electrocardiogram; hearing, laboratory tests (blood routine analytes, blood biochemistry, coagulation function, thyroid and para-thyroid function, urine routine analytes.), urine pregnancy test (women of childbearing age),Levels of drug exposure during the study.
- Efficacy assessments: Efficacy evaluations include serum ferritin, liver iron content (MRI R2) and cardiac iron content (MRI T2*).
- Statistics:
- Subject characteristic distribution Demographic characteristics, general conditions, and baseline conditions (pre-treatment) of enrolled subjects were analyzed.The measurement data are described by means, standard deviation, minimum value and maximum value, while the qualitative data list frequency and percentage.
- Safety analysis Descriptive statistical analysis was used for safety endpoints. Summarize the incidence of adverse events, adverse reactions, adverse events leading to withdrawal from the trial, adverse events leading to death, severe adverse events, and severe adverse reactions. The incidence is calculated by subsystem, symptom/sign. Severity of adverse events and adverse reactions: if multiple adverse events occur in the same subject, the most serious one is included in the analysis; if different adverse events occurred in the same subject, the most severe adverse events were counted in the analysis. Drug exposure during the study: describe medication compliance during the study, actual dose, administration adjustments during the study, whether the study was discontinued, and reasons for the suspension.
- Effectiveness analysis Mean, standard deviation, median, minimum and maximum values were described and 95% confidence intervals were calculated. Paired T-test was used to compare each time point with the baseline if necessary. The 95% confidence interval was calculated by using Clopper-Pearson method for the proportion of patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CN128 Group | Experimental | Drug: CN128 TabletsAll subjects will be given the lower (10 mg/kg bw, bid) or higher dose (15 mg/kg bw, bid) for 24 or 48 weeks, according to the administration plan. All subjects will be given the lower (15 mg/kg bw, bid) or higher dose (20 mg/kg bw, bid) for 49 or 96 weeks, according to the administration plan. The dosage form is tablets. |
Who may qualify
- Ages
- 16 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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.
- Serum ferritin ≥ 500 µg/L
- Patients aged 16 and above
- Volunteer for the trial and sign the informed consent.
Key exclusion criteria (13 listed)
- Active hepatitis B (HBsAg positive, HBsAb negative) or hepatitis C (HCV antibody positive, detectable HCV RNA, and 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 AST > 2.5 × ULN, or serum creatinine > 1.5 × ULN;
- Neutropenia patient (neutrophil count < 1.5 × 109 / L);
- Active infection uncontrolled;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CN128 TabletsAlso: CN128Iron chelator, oral tablets | Drug | CN128 Group |
Primary outcome
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
Time frame: up to 96 weeks
To determine the incidence, type and severity of adverse events, adverse reactions, severe adverse events and severe adverse reactions in patients up to 96 weeks.
Absolute Change in Weight (Unit: kg) From Baseline Over Time
Time frame: Baseline, 24, 48, 72 and 96 weeks.
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
Time frame: Baseline,24, 48, 72 and 96 weeks.
The patient's height will be determined, and it's one kind of Physical examination.
Absolute Change in Hormon (total and free testosterone in men, follicle-generating hormone and luteinizing hormon in women) From Baseline Over Time
Time frame: Baseline, 24, 48, 72 and 96 weeks.
Hormon will be determined, and it's one kind of laboratory test.
Absolute Change in Temperature (Unit: ℃）From Baseline Over Time
Time frame: Baseline, 2, 4, 8, 12, 16, 20, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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
Time frame: Baseline, 2, 4, 8, 12, 16, 20, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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
Time frame: Baseline, 2, 4, 8, 12, 16, 20, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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
Time frame: Baseline, 2, 4, 8, 12, 16, 20, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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 (Unit: ms), etc) From Baseline Over Time
Time frame: Baseline, 2, 4, 8, 12, 16, 20, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
The patient's electrocardiogram will be measured, and it's one kind of laboratory test.
Change in Auditory Function From Baseline Over Time
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
The patient's auditory function will be determined by otorhinolaryngology.
Absolute Change in White Blood Count (Unit: 10E9/L) From Baseline Over Time
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
The patient's white blood count will be determined, and it's one kind of laboratory test.
Absolute Change in Neutrophil Count (Unit: 10E9/L) From Baseline Over Time
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
The patient's neutrophil count will be determined, and it's one kind of laboratory test.
Absolute Change in Lymphocyte Count (Unit: 10E9/L) From Baseline Over Time
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
The patient's lymphocyte count will be determined, and it's one kind of laboratory test.
Absolute Change in Monocyte Count (Unit: 10E9/L) From Baseline Over Time
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
The patient's monocyte count will be determined, and it's one kind of laboratory test.
Absolute Change in Eosinophilic Count (Unit: 10E9/L) From Baseline Over Time
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
The patient's eosinophilic count will be determined, and it's one kind of laboratory test.
Absolute Change in Basophilic Count (Unit: 10E9/L) From Baseline Over Time
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
The patient's basophilic count will be determined, and it's one kind of laboratory test.
Absolute Change in Red Blood Count (Unit: 10E9/L) From Baseline Over Time
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
The patient's hemoglobin will be determined, and it's one kind of laboratory test.
Absolute Change in Hematocrit (Unit:%) From Baseline Over Time
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
The patient's hematocrit will be determined, and it's one kind of laboratory test.
Absolute Change in Blood Platelet Count (Unit: 10E9/L) From Baseline Over Time
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
The patient's alkaline phosphatase will be determined, and it's one kind of laboratory test.
Absolute Change in Lactate Dehydrogenase (Unit: IU/L) From Baseline Over Time
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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 frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
The patient's calcium will be determined, and it's one kind of laboratory test.
Absolute Change in Aspartate Aminotransferase (Unit: U/L) From Baseline Over Time
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
The patient's aspartate aminotransferase will be determined, and it's one kind of laboratory test.
Absolute Change in Fibrinogen (Unit: g/L) From Baseline Over Time
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
The patient's APTT will be determined, and it's one kind of laboratory test.
Absolute Change in Thyroid and para-gland function (Serum total thyroxine, parathyroid hormone, total triiodothyronine and thyrotropin) From Baseline Over Time
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
The patient's thyroid and para-gland function will be determined, and it's one kind of laboratory test.
Absolute Change in Urine Glucose (Unit: mmol/L) From Baseline Over Time
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
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
Time frame: Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
The patient's urine protein will be determined, and it's one kind of laboratory test.
Absolute Change in Dosage（mg/kg）during the study period
Time frame: Up to 96 weeks
The dosages will be investigated up to 96 weeks.
Duration of Administration during the study period
Time frame: Up to 96 weeks
The duration of administration will be investigated up to 96 weeks.
Number of Participants during the study period
Time frame: Up to 96 weeks
The number of participants will be investigated up to 96 weeks.
Absolute Change in Serum ferritin (Unit: µg/L) From Baseline Over Time
Time frame: Baseline, 2, 4, 8, 12, 16, 20, 24, 32, 40, 48, 60, 72, 84 and 96 weeks.
Serum ferritin will be determined, and it's one kind of laboratory test.
Absolute Change in Liver iron content (MRI R2) From Baseline Over Time
Time frame: Baseline, 12, 24, 48, 72 and 96 weeks.
Liver iron content will be determined by liver magnetic resonance.
Absolute Change in Heart iron content (MRI T2*) From Baseline Over Time
Time frame: Baseline, 12, 24, 48, 72 and 96 weeks.
Heart iron content will be determined by cardio magnetic resonance.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Nov 2023.
Related trials for Thalassemia
Active, not recruiting
Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
Transfusion-dependent Beta-Thalassemia
RecruitingObservational study
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Transfusion-dependent Beta-Thalassemia, Gene Therapy
Not yet recruiting
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
Beta-Thalassemia
RecruitingPhase 1/2
β-Thalassemia Treatment With KL003 Cell Injection
Transfusion-dependent Beta-Thalassemia
Active, not recruitingPhase 3
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jianmin Jianmin, PhD principal investigator
First Affiliated Hospital of Guangxi Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing CN128 Tablets in people with Thalassemia and Iron Overload, run by Hangzhou Zede Pharma-Tech Co., Ltd. at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 (time frame: up to 96 weeks). The study lists 48 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Nov 2023.