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

Last updated: March 13, 2025
Sponsor: Hangzhou Zede Pharma-Tech Co., Ltd.
Overall Status: Completed

Phase

2

Condition

Thalassemia

Treatment

CN128 Tablets

Clinical Study ID

NCT05355766
A160605-202
  • Ages 16-60
  • All Genders

Study Summary

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

Eligibility Criteria

Inclusion

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 ironconcentration (LIC)>15 mg/g dw) despite prior treatment with at least one ironchelator.

  • Patients aged 16 and above.

  • Volunteer for the trial and sign the informed consent.

Exclusion

Exclusion Criteria:

  • Active hepatitis B (HBsAg positive, HBsAb negative) or hepatitis C (HCV antibodypositive, 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, gastrointestinalcancer, familial genetic multiple intestinal polyps), and History ofgastrointestinal perforation, gastrointestinal surgery that influence drugabsorption, and other potential intestinal complications considered by researchers.

  • ALT or Aspartate Aminotransferase (AST) > 2.5 × upper limit of normal (ULN), orserum 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 inhibitordrugs, or the drug that may extend the QT interval, or the drug that may decreaseneutrophil 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 orexcipients of CN128 tablets.

  • Congenital long QT syndrome or known family history of long QT syndrome, QTcinterval>480 ms, clinically significant ventricular or atrial fast arrhythmia.

  • The patients who can not accept MRI as detection means, such as claustrophobic forMRI, pacemaker, and those using ferromagnetic metal implants.

  • Birth planner (including male subjects) within or within 3 months after the end ofthe trial.

  • Participated in other clinical trials in the four weeks before taking the testpreparation, except for non-interventional studies.

  • Pregnant or lactating women.

  • Unsuitable to participate in the trial considered by the researchers.

Study Design

Total Participants: 16
Treatment Group(s): 1
Primary Treatment: CN128 Tablets
Phase: 2
Study Start date:
June 13, 2022
Estimated Completion Date:
March 06, 2025

Study Description

  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.

Connect with a study center

  • The First Affiliated Hospital Of Guangxi Medical University

    Nanning, Guangxi
    China

    Site Not Available

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.