Long-term Clinical Study of CN128 in Thalassemia With Sever Liver Iron Overloaded Patients

Hangzhou Zede Pharma-Tech Co., Ltd.16 enrolled

Overview

The safety and efficacy of CN128 is studied in thalassaemia with sever liver iron overloaded patients.

1. The study is designed as a single arm and opened phase IIb clinical trial, so as to investigate the safety and efficacy of CN128. 2. A total of 50 eligible subjects are planned to be enrolled, and orally administration of CN128 for 52 weeks according to the administration plan. 3. 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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Thalassemia Iron Overload

Interventions

CN128 TabletsDRUG

Iron chelator, oral tablets

Eligibility

Sex: ALLMin age: 16 YearsMax age: 60 Years

Medical Language ↔ Plain English

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.

Locations (1)

The First Affiliated Hospital Of Guangxi Medical University

Nanning, Guangxi, China

Outcomes

Primary Outcomes

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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: Baseline, 2, 4, 6, 8, 12, 16, 20, 26, 39 and 52 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.

Data from ClinicalTrials.gov

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Time frame: 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.

Time frame: 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.