Testing 9MW3011 for patients with non-transfusion-dependent beta-thalassemia

A Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of 9MW3011 in Patients With Non-transfusion-dependent β- Thalassemia

This study is testing a new drug called 9MW3011 to see if it is safe and effective for people with non-transfusion-dependent beta-thalassemia.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 40 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Mabwell (Shanghai) Bioscience Co., Ltd. (Industry-sponsored)
• Locations: 2 sites (Nanning, Guangxi and 1 other locations)
• Trial ID: NCT06772766 on ClinicalTrials.gov

What this trial studies

This phase Ib study evaluates the safety, tolerability, pharmacokinetics, pharmacodynamics, and immunogenicity of 9MW3011 in patients diagnosed with non-transfusion-dependent beta-thalassemia. A total of 40 subjects will be enrolled and assigned to four dosage cohorts, with participants randomized in a 4:1 ratio to receive either 9MW3011 or a placebo via intravenous infusion. The study aims to gather comprehensive data on the effects of the drug over multiple ascending doses.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 to 65 with a documented genetic diagnosis of non-transfusion-dependent beta-thalassemia and specific hemoglobin levels.

Not a fit: Patients with alpha-thalassemia or transfusion-dependent beta-thalassemia will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with non-transfusion-dependent beta-thalassemia, potentially improving their quality of life.

How similar studies have performed: While this approach is novel, similar studies targeting beta-thalassemia have shown promise in the past.

Eligibility criteria

Key Inclusion Criteria:

1. Male and female subjects aged 18 to 65 years (inclusive)
2. Subject must have a documented genetic diagnosis of β-thalassemia or hemoglobin E/ β-thalassemia
3. Subjects must meet the criteria for non-transfusion-dependent thalassemia
4. Subjects must have a baseline hemoglobin level between 70-100 g/L(inclusive), based on 2 consecutive measurements taken at least 1 week apart within 4 weeks before randomization
5. Subjects must have evidence of iron overload during screening
6. Subject must have performance status: Eastern Cooperative Oncology Group (ECOG) performance score of 0 to 1
7. Subjects must fully understand the study procedures and methods, voluntarily participate in the trial, and sign an informed consent form

Key Exclusion Criteria:

1. Subjects diagnosed with alpha-thalassemia
2. Subjects diagnosed with HbS/beta-thalassemia or transfusion-dependent beta-thalassemia
3. Subjects exhibit severe iron overload at the time of screening
4. In addition to thalassemia, subjects have any other forms of anemia and hematological disorders that the investigator assesses may compromise safety or influence study outcomes
5. Combined with any significant systemic diseases or psychiatric disorders
6. Subjects have New York Heart Association (NYHA) Class III-IV heart failure and other cardiovascular diseases within 6 months prior to screening or currently present
7. During the screening or baseline period, subjects exhibiting a QTcF interval of ≥450ms for males and ≥470ms for females on a 12-lead electrocardiogram (ECG), or presenting an abnormal 12-lead ECG with clinical significance
8. Uncontrolled hypertension before screening
9. A history of malignant neoplasm occurring within the last five years
10. Severe infection requiring hospitalization or intravenous antimicrobial therapy, or uncontrolled systemic bacterial, fungal, or viral active infection
11. Subject have received concomitant treatment that was not permitted by the protocol
12. Subjects whose hematological parameters did not meet the inclusion criteria during screening
13. Subjects with a history of substance abuse, as well as those who yield positive results on substance abuse screening
14. Subjects who are unable to undergo MRI scans
15. Pregnant or lactating women
16. Subjects presenting any other factors deemed unsuitable for participation assessed by the investigator

Where this trial is running

Nanning, Guangxi and 1 other locations

• The first Affiliated Hospital of Guangxi Medical University — Nanning, Guangxi, China (Recruiting)
• Hainan General Hospital — Haikou, Hainan, China (Recruiting)

Study contacts

• Study coordinator: Yongrong Lai

Conditions Beta-Thalassemia