Paroxysmal Nocturnal Haemoglobinuria (PNH)Phase 3NCT06932744
Study of Safety and Efficacy of MY008211A in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy
A Phase 3 study testing MY008211A tablets and Eculizumab Injection in people with Paroxysmal Nocturnal Haemoglobinuria (PNH), run by Wuhan Createrna Science and Technology Co., Ltd at 2 sites in China.
Trial summary
The main purpose of this study is to evaluate the efficacy of MY008211A compared to eculizumab in PNH patients. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Paroxysmal Nocturnal Haemoglobinuria (PNH)
- What is being tested?
- MY008211A tablets and Eculizumab Injection
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 66 participants
- Where
- 2 sitesChina
- Study dates
- 30 Aug 2024to 30 Dec 2025
- Phase
- Phase 3
- 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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male and female participants ≥ 18 years of age and BMI ≥ 18.0 kg/m2 with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with clone size ≥ 10%.
- Mean hemoglobin level <100 g/L at screening.
- LDH > 1.5 x Upper Limit of Normal (ULN) at screening.
- Vaccination against Neisseria meningitidis infection is required prior to the start of study treatment. If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given.
Who cannot join
- Patients with reticulocytes <100x10^9/L; platelets <30x10^9/L; neutrophils <0.5x10^9/L.
- History of recurrent invasive infections caused by encapsulated organisms,e.g. meningococcus or pneumococcus.
- Known or suspected hereditary complement deficiency.
- Previous bone marrow or hematopoietic stem cell transplantation.
- Previous splenectomy.
- A history of malignancy within 5 years before screening, except cured local basal cell carcinoma of the skin and carcinoma in situ of the cervix.
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
- Not yet recruiting
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Beijing, Beijing Municipality, 100032
Bing Han, PhD, 010-69155027, Hanbing_Li@sina.com.cn
- Recruiting
Chinese Academy of Medical Sciences Hematology Hospital
Tianjin, Tianjin Municipality, 300020
Fengkui Zhang, PhD, 022-23909095
About this study
Official title: A Randomized, Multicenter, Active-comparator Controlled, Open-label Trial to Evaluate Efficacy and Safety of MY008211A Tablets in PNH Patients Who Are Naive to Complement Inhibitor Therapy
The main purpose of this study is to evaluate the efficacy of MY008211A compared to eculizumab in PNH patients.
Read the detailed description
This is a multi-center, randomized, open-label, active comparator-controlled, parallel group study. The purpose of this study is to determine whether MY008211A compared to eculizumab is efficacious and safe for the treatment of PNH patients who were naive to complement inhibitor therapy.
- Being tested
- MY008211A tablets, Eculizumab Injection
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MY008211A tabletsDrug
Participants will receive MY008211A at a dose of 400 mg orally b.i.d for 24 weeks
Eculizumab InjectionDrug
Eculizumab Injection for 24 weeks
| Study group | Role | What they receive |
|---|---|---|
| MY008211A tablets | Experimental | MY008211A tablets 400mg BID Receives: Drug: MY008211A tablets |
| Eculizumab | Active comparator | Eculizumab Injection Receives: Drug: Eculizumab Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
The proportion of Participants With Sustained Hemoglobin Levels of ≥ 120 g/L in the Absence of Red Blood Cell Transfusions
The proportion of patients with sustained hemoglobin levels ≥ 120 g/L among those without RBC transfusion (defined as no red blood cell infusion after D14 to D168).
Measured: between Day 126 and Day 168
Secondary outcomes (8)
The proportion of subjects with an increase in hemoglobin concentration ≥ 20 g/L from baseline among subjects who do not receive RBC transfusion
Proportion of participants achieving a sustained increase from baseline in hemoglobin levels of ≥ 20 g/L assessed among those without RBC transfusion (defined as no red blood cell infusion after D14 to D168).
Measured: between Day 126 and Day 168
The proportion of patients with LDH < 1.5 ULN among those without RBC transfusion.
The proportion of patients with hemolysis controlled (defined as LDH < 1.5 ULN) among those without RBC transfusion
Measured: between Day 126 and Day 168
Change (Expressed as Percentages) in LDH level from baseline
Change (Expressed as Percentages) in LDH level from baseline
Measured: between Day 126 and Day 168
Change in reticulocyte count from baseline
Change in reticulocyte count from baseline
Measured: between Day 126 and Day 168
The proportion of patients without RBC transfusion
The proportion of patients without RBC transfusion
Measured: between Day 14 and Day 168
Change in FACIT-F score from baseline
Change from baseline in FACIT-Fatigue scores. The FACIT-Fatigue is a 13-item questionnaire with support for its validity and reliability in PNH that assesses patient self-reported fatigue and its impact on daily activities and function. All FACIT scales are scored so that a high score is better. As each of the 13 items of the FACIT-F Scale ranges from 0-4, the range of possible scores is 0-52, with 0 being the worst possible score and 52 the best.
Measured: between Day 126 and Day 168
The Clinical BTH Rate
The Clinical BTH Rate
Measured: between Day 1 and Day 168
The Major Adverse Vascular Events Rate
The Major Adverse Vascular Events Rate
Measured: between Day 1 and Day 168
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2025
- Study start30 Aug 2024
- Primary completion30 Dec 2025 (estimated)
- Study completion30 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal Haemoglobinuria (PNH)Studies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 66 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Wuhan Createrna Science and Technology Co., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06932744
- Sponsor's protocol ID
- MY008211A-PNH-3-02
- Status last verified
- Apr 2025
- First submitted
- 10 Apr 2025
- Last updated
- 9 May 2025
- 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 9 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Multicenter, Active-comparator Controlled, Open-label Trial to Evaluate Efficacy and Safety of MY008211A Tablets in PNH Patients Who Are Naive to Complement Inhibitor Therapy
- ClinicalTrials.gov ID
- NCT06932744
- Acronym / sponsor ID
- MY008211A-PNH-3-02
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 66 participants (estimated)
- Lead sponsor
- Wuhan Createrna Science and Technology Co., LtdIndustry (pharma, biotech, devices)
- Study start
- 30 Aug 2024 (actual)
- Primary completion
- 30 Dec 2025 (estimated)
- Study completion
- 30 Dec 2025 (estimated)
- First posted
- 17 Apr 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.
The main purpose of this study is to evaluate the efficacy of MY008211A compared to eculizumab in PNH patients.
Detailed description
This is a multi-center, randomized, open-label, active comparator-controlled, parallel group study. The purpose of this study is to determine whether MY008211A compared to eculizumab is efficacious and safe for the treatment of PNH patients who were naive to complement inhibitor therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| MY008211A tablets | Experimental | Drug: MY008211A tabletsMY008211A tablets 400mg BID |
| Eculizumab | Active comparator | Drug: Eculizumab InjectionEculizumab Injection |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Male and female participants ≥ 18 years of age and BMI ≥ 18.0 kg/m2 with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with clone size ≥ 10%.
- Mean hemoglobin level <100 g/L at screening.
- LDH > 1.5 x Upper Limit of Normal (ULN) at screening.
- Vaccination against Neisseria meningitidis infection is required prior to the start of study treatment. If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given.
Key exclusion criteria (6 listed)
- Patients with reticulocytes <100x10^9/L; platelets <30x10^9/L; neutrophils <0.5x10^9/L.
- History of recurrent invasive infections caused by encapsulated organisms,e.g. meningococcus or pneumococcus.
- Known or suspected hereditary complement deficiency.
- Previous bone marrow or hematopoietic stem cell transplantation.
- Previous splenectomy.
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 |
|---|---|---|
| MY008211A tabletsParticipants will receive MY008211A at a dose of 400 mg orally b.i.d for 24 weeks | Drug | MY008211A tablets |
| Eculizumab InjectionEculizumab Injection for 24 weeks | Drug | Eculizumab |
Primary outcome
The proportion of Participants With Sustained Hemoglobin Levels of ≥ 120 g/L in the Absence of Red Blood Cell Transfusions
Time frame: between Day 126 and Day 168
The proportion of patients with sustained hemoglobin levels ≥ 120 g/L among those without RBC transfusion (defined as no red blood cell infusion after D14 to D168).
8 secondary outcomes
The proportion of subjects with an increase in hemoglobin concentration ≥ 20 g/L from baseline among subjects who do not receive RBC transfusion
Time frame: between Day 126 and Day 168
The proportion of patients with LDH < 1.5 ULN among those without RBC transfusion.
Time frame: between Day 126 and Day 168
Change (Expressed as Percentages) in LDH level from baseline
Time frame: between Day 126 and Day 168
Change in reticulocyte count from baseline
Time frame: between Day 126 and Day 168
The proportion of patients without RBC transfusion
Time frame: between Day 14 and Day 168
Change in FACIT-F score from baseline
Time frame: between Day 126 and Day 168
The Clinical BTH Rate
Time frame: between Day 1 and Day 168
The Major Adverse Vascular Events Rate
Time frame: between Day 1 and Day 168
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 9 May 2025.
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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 3 study testing MY008211A tablets and Eculizumab Injection in people with Paroxysmal Nocturnal Haemoglobinuria (PNH), run by Wuhan Createrna Science and Technology 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 4 inclusion and 6 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 9 May 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 Wuhan Createrna Science and Technology Co.,Ltd (027-68788900 ext. +86, lcyxzx@createrna.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The proportion of Participants With Sustained Hemoglobin Levels of ≥ 120 g/L in the Absence of Red Blood Cell Transfusions (time frame: between Day 126 and Day 168). 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 9 May 2025.