Beta-ThalassemiaPhase 1NCT06772766
A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia
A Phase 1 study testing 9MW3011 in people with Beta-Thalassemia, run by Mabwell (Shanghai) Bioscience Co., Ltd. at 2 sites in China.
Trial summary
This is a phase Ib, randomized, double-blind, placebo-controlled, multiple ascending dose study . The objectives of the study are to evaluate the safety , tolerability, pharmacokinetics(PK), pharmacodynamics(PD), and immunogenicity of 9MW3011 in patients with non-transfusion-dependent β- thalassemia . Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Beta-Thalassemia
- What is being tested?
- 9MW3011
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 2 sitesChina
- Study dates
- 30 Dec 2024to Oct 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male and female subjects aged 18 to 65 years (inclusive)
- Subject must have a documented genetic diagnosis of β-thalassemia or hemoglobin E/ β-thalassemia
- Subjects must meet the criteria for non-transfusion-dependent thalassemia
- 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
- Subjects must have evidence of iron overload during screening
- Subject must have performance status: Eastern Cooperative Oncology Group (ECOG) performance score of 0 to 1
- Subjects must fully understand the study procedures and methods, voluntarily participate in the trial, and sign an informed consent form
Who cannot join
- Subjects diagnosed with alpha-thalassemia
- Subjects diagnosed with HbS/beta-thalassemia or transfusion-dependent beta-thalassemia
- Subjects exhibit severe iron overload at the time of screening
- 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
- Combined with any significant systemic diseases or psychiatric disorders
- 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
Show all 16Show fewer
- 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
- Uncontrolled hypertension before screening
- A history of malignant neoplasm occurring within the last five years
- Severe infection requiring hospitalization or intravenous antimicrobial therapy, or uncontrolled systemic bacterial, fungal, or viral active infection
- Subject have received concomitant treatment that was not permitted by the protocol
- Subjects whose hematological parameters did not meet the inclusion criteria during screening
- Subjects with a history of substance abuse, as well as those who yield positive results on substance abuse screening
- Subjects who are unable to undergo MRI scans
- Pregnant or lactating women
- Subjects presenting any other factors deemed unsuitable for participation assessed by the investigator
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China2 sites, 2 recruiting
- Recruiting
Hainan General Hospital
Haikou, Hainan, 570311
Guyun Wang, +86089868642117, wangguyun@126.com
- Recruiting
The first Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, 530021
Yongrong Lai, +8607715356304, laiyongrong@hotmail.com
About this study
Official title: 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 is a phase Ib, randomized, double-blind, placebo-controlled, multiple ascending dose study . The objectives of the study are to evaluate the safety , tolerability, pharmacokinetics(PK), pharmacodynamics(PD), and immunogenicity of 9MW3011 in patients with non-transfusion-dependent β- thalassemia .
Read the detailed description
A total of 40 subjects diagnosed with non-transfusion-dependent β-thalassemia will be enrolled in this study and assigned into four dosage cohorts. In each cohort, subjects will be randomized in a 4:1 ratio to receive 9MW3011 or placebo via intravenous infusion.
- Conditions
- Beta-Thalassemia
- Being tested
- 9MW3011, 9MW3011 placebo
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
9MW3011Drug
Ascending IV doses administered per protocol
9MW3011 placeboDrug
Ascending IV doses administered per protocol
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | 9MW3011 or placebo (Randomized 4:1) Receives: Drug: 9MW3011; Drug: 9MW3011 placebo |
| Cohort 2 | Experimental | 9MW3011 or placebo (Randomized 4:1) Receives: Drug: 9MW3011; Drug: 9MW3011 placebo |
| Cohort 3 | Experimental | 9MW3011 or placebo (Randomized 4:1) Receives: Drug: 9MW3011; Drug: 9MW3011 placebo |
| Cohort 4 | Experimental | 9MW3011 or placebo (Randomized 4:1) Receives: Drug: 9MW3011; Drug: 9MW3011 placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Event(including serious adverse event)
The incidence of Adverse Events(AEs)and Serious Adverse Events(SAEs)from treatment until the last scheduled follow-up visit
Measured: up to day 169
Number of subjects with abnormal vital signs
Vital signs measurements will include pulse rate, respiration rate, blood pressure (systolic and diastolic blood pressure) and body temperature.
Measured: up to day 169
Number of subjects with abnormal clinically significant results from physical examination
The physical examinations will include examination of the following: skin and mucous membranes, lymph nodes, head and neck, chest, abdomen, musculoskeletal, nervous system, and other sites of note elicited from the subject.
Measured: up to day 169
Number of subjects with abnormal clinically significant 12-lead electrocardiogram (ECG) parameters
The examination indicators include heart rate, PR, QRS, uncorrected QT, and QTcF(corrected by Fridericia formula).
Measured: up to day 169
Number of subjects with abnormal clinically significant clinical laboratory results
Clinical laboratory tests include hematology, urinalysis, blood chemistry, coagulation function.
Measured: up to day 169
Secondary outcomes (5)
Concentration of 9MW3011 in serum
Measured: up to day 169
PD-parameters-hepcidin
Change from baseline in hepcidin levels
Measured: up to day 169
PD-parameters-serum iron
Change from baseline in serum iron levels
Measured: up to day 169
Anti-drug antibody(ADA)
The incidence of ADA
Measured: up to day 169
Liver iron concentration(LIC)
Change from baseline in LIC
Measured: up to day 169
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2025
- Study start30 Dec 2024
- Primary completionOct 2026 (estimated)
- Study completionOct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Beta-ThalassemiaStudies of the same condition, open and under way.
- More trials run by Mabwell (Shanghai) Bioscience Co., Ltd.The sponsor's other registered studies.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Mabwell (Shanghai) Bioscience Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06772766
- Sponsor's protocol ID
- 9MW3011-C04
- Status last verified
- Jan 2025
- First submitted
- 9 Jan 2025
- Last updated
- 14 Jan 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06772766
- Acronym / sponsor ID
- 9MW3011-C04
- Conditions
- Beta-Thalassemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Mabwell (Shanghai) Bioscience Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 30 Dec 2024 (actual)
- Primary completion
- Oct 2026 (estimated)
- Study completion
- Oct 2026 (estimated)
- First posted
- 14 Jan 2025 (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.
This is a phase Ib, randomized, double-blind, placebo-controlled, multiple ascending dose study . The objectives of the study are to evaluate the safety , tolerability, pharmacokinetics(PK), pharmacodynamics(PD), and immunogenicity of 9MW3011 in patients with non-transfusion-dependent β- thalassemia .
Detailed description
A total of 40 subjects diagnosed with non-transfusion-dependent β-thalassemia will be enrolled in this study and assigned into four dosage cohorts. In each cohort, subjects will be randomized in a 4:1 ratio to receive 9MW3011 or placebo via intravenous infusion.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind (participant, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Drug: 9MW3011, Drug: 9MW3011 placebo9MW3011 or placebo (Randomized 4:1) |
| Cohort 2 | Experimental | Drug: 9MW3011, Drug: 9MW3011 placebo9MW3011 or placebo (Randomized 4:1) |
| Cohort 3 | Experimental | Drug: 9MW3011, Drug: 9MW3011 placebo9MW3011 or placebo (Randomized 4:1) |
| Cohort 4 | Experimental | Drug: 9MW3011, Drug: 9MW3011 placebo9MW3011 or placebo (Randomized 4:1) |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Male and female subjects aged 18 to 65 years (inclusive)
- Subject must have a documented genetic diagnosis of β-thalassemia or hemoglobin E/ β-thalassemia
- Subjects must meet the criteria for non-transfusion-dependent thalassemia
- 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
- Subjects must have evidence of iron overload during screening
Key exclusion criteria (16 listed)
- Subjects diagnosed with alpha-thalassemia
- Subjects diagnosed with HbS/beta-thalassemia or transfusion-dependent beta-thalassemia
- Subjects exhibit severe iron overload at the time of screening
- 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
- Combined with any significant systemic diseases or psychiatric disorders
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 |
|---|---|---|
| 9MW3011Ascending IV doses administered per protocol | Drug | Cohort 1, Cohort 2, Cohort 3, Cohort 4 |
| 9MW3011 placeboAscending IV doses administered per protocol | Drug | Cohort 1, Cohort 2, Cohort 3, Cohort 4 |
Primary outcome
Adverse Event(including serious adverse event)
Time frame: up to day 169
The incidence of Adverse Events(AEs)and Serious Adverse Events(SAEs)from treatment until the last scheduled follow-up visit
Number of subjects with abnormal vital signs
Time frame: up to day 169
Vital signs measurements will include pulse rate, respiration rate, blood pressure (systolic and diastolic blood pressure) and body temperature.
Number of subjects with abnormal clinically significant results from physical examination
Time frame: up to day 169
The physical examinations will include examination of the following: skin and mucous membranes, lymph nodes, head and neck, chest, abdomen, musculoskeletal, nervous system, and other sites of note elicited from the subject.
Number of subjects with abnormal clinically significant 12-lead electrocardiogram (ECG) parameters
Time frame: up to day 169
The examination indicators include heart rate, PR, QRS, uncorrected QT, and QTcF(corrected by Fridericia formula).
Number of subjects with abnormal clinically significant clinical laboratory results
Time frame: up to day 169
Clinical laboratory tests include hematology, urinalysis, blood chemistry, coagulation function.
5 secondary outcomes
Concentration of 9MW3011 in serum
Time frame: up to day 169
PD-parameters-hepcidin
Time frame: up to day 169
PD-parameters-serum iron
Time frame: up to day 169
Anti-drug antibody(ADA)
Time frame: up to day 169
Liver iron concentration(LIC)
Time frame: up to day 169
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jan 2025.
- Individual participant dataSharing planned: No
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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.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing 9MW3011 in people with Beta-Thalassemia, run by Mabwell (Shanghai) Bioscience Co., Ltd. at 2 sites 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 7 inclusion and 16 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: looking for participants now, as of the sponsor's last update on 14 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yongrong Lai (+8607715356304, laiyongrong@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse Event(including serious adverse event) (time frame: up to day 169). The study lists 5 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 14 Jan 2025.