Paroxysmal Nocturnal Hemoglobinuria (PNH)Phase 2NCT06134414
Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
A Phase 2 study testing MY008211A tablets in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by Wuhan Createrna Science and Technology Co., Ltd.
Trial summary
The main purpose of this study is to evaluate the efficacy of MY008211A in adult patients with PNH, showing signs of active hemolysis. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- What is being tested?
- MY008211A tablets
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- No sites listed yet
- Study dates
- Dec 2025to Dec 2027
- Phase
- Phase 2
- 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.
- LDH > 1.5 x Upper Limit of Normal (ULN).
- 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.
- Were using a complement inhibitor before the first administration of MY008211A tablets or had discontinued a previous complement inhibitor for less than five half-lives or 120 days, whichever was the longest.
- 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
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Multi-center, Randomized, Parallel, Open-label Clinical Phase II Study, to Evaluate the Efficacy and Safety of MY008211A in Adult Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients With Signs of Active Hemolysis
The main purpose of this study is to evaluate the efficacy of MY008211A in adult patients with PNH, showing signs of active hemolysis.
Read the detailed description
The purpose of this study is to determine whether MY008211A is efficacious and safe for the treatment of PNH patients who are naïve to complement inhibitor therapy, including anti-C5 antibody.
- Being tested
- MY008211A tablets
- 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
Also called MY008211A
dose 1 (400 mg BID) and dose 2 (600 mg BID) in a 1:1 ratio by central randomization
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 MY008211A low dose | Experimental | Participants will receive MY008211A at a dose of 400 mg orally b.i.d Receives: Drug: MY008211A tablets |
| Arm 2 MY008211A high dose | Experimental | Participants will receive MY008211A at a dose of 600 mg orally b.i.d Receives: Drug: MY008211A tablets |
What is being measured
The primary outcome is the main question the study is designed to answer.
The proportion of subjects with an increase in hemoglobin concentration ≥ 20 g/L from baseline among subjects who do not receive RBC transfusion after 4 weeks of dosing
Proportion of participants achieving a sustained increase from baseline in hemoglobin levels of ≥ 20 g/L assessed , in the absence of red blood cell transfusions
Measured: Day 70
Secondary outcomes (14)
The proportion of patients with an increase in hemoglobin ≥ 20 g/L from baseline among those without RBC transfusion
The proportion of patients with an increase in hemoglobin ≥ 20 g/L from baseline among those without RBC transfusion
Measured: Day14, 21, 28, 42, 56
The proportion of patients with hemoglobin ≥ 120 g/L among those without RBC transfusion
The proportion of patients with hemoglobin ≥ 120 g/L among those without RBC transfusion
Measured: Day14, 21, 28, 42, 56 and 70
Change in hemoglobin concentration from baseline in patients without RBC transfusion
Change in hemoglobin concentration from baseline in patients without RBC transfusion
Measured: Day14, 21, 28, 42, 56 and 70
Change in LDH level from baseline
Change in LDH level from baseline
Measured: Day7, 14, 21, 28, 42, 56 and 70
The proportion of patients with hemolysis controlled
The proportion of patients with hemolysis controlled (defined as LDH < 1.5 ULN)
Measured: Day7, 14, 21, 28, 42, 56 and 70
Change in reticulocyte count from baseline in patients without RBC transfusion
Change in reticulocyte count from baseline in patients without RBC transfusion
Measured: Day7, 14, 21, 28, 42, 56 and 70
Change in indirect bilirubin level from baseline
Change in indirect bilirubin level from baseline
Measured: Day7, 14, 21, 28, 42, 56 and 70
The proportion of patients without RBC transfusion
The proportion of patients without RBC transfusion
Measured: Day14, 21, 28, 42, 56 and 70
Change in the average weekly amount of RBC transfused during the efficacy observation period
Change in the average weekly amount of RBC transfused during the efficacy observation period compared with that pre-dose
Measured: Day70
Change From Baseline in FACIT-Fatigue Questionnaire
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: Day7, 14, 21, 28, 42, 56 and 70
Changes from baseline in alternative complement pathway activity
Alternative complement pathway activity measured by the WIESLAB® kit.
Measured: Day14, 28, 56 and 70
Change in the amount of fragment Bb of CFB in plasma from baseline
Bb fragment cleaved by factor B of complement.
Measured: Day14, 28, 56 and 70
Change in the level of PNH RBC clones from baseline in patients without RBC transfusion.
Change from baseline in the level of PNH red cell clones.
Measured: Day70
Incidence of Adverse Events (AEs) between Day 1 and Day 70
Adverse Events (AEs)
Measured: Day 70
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2023
- Study startDec 2025 (estimated)
- Primary completionJul 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal Hemoglobinuria (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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 40 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
- NCT06134414
- Sponsor's protocol ID
- MY008211A-PNH-2-01-F
- Status last verified
- Oct 2024
- First submitted
- 10 Nov 2023
- Last updated
- 29 Jul 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 29 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center, Randomized, Parallel, Open-label Clinical Phase II Study, to Evaluate the Efficacy and Safety of MY008211A in Adult Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients With Signs of Active Hemolysis
- ClinicalTrials.gov ID
- NCT06134414
- Acronym / sponsor ID
- MY008211A-PNH-2-01-F
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Wuhan Createrna Science and Technology Co., LtdIndustry (pharma, biotech, devices)
- Study start
- Dec 2025 (estimated)
- Primary completion
- Jul 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 18 Nov 2023 (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 in adult patients with PNH, showing signs of active hemolysis.
Detailed description
The purpose of this study is to determine whether MY008211A is efficacious and safe for the treatment of PNH patients who are naïve to complement inhibitor therapy, including anti-C5 antibody.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm 1 MY008211A low dose | Experimental | Drug: MY008211A tabletsParticipants will receive MY008211A at a dose of 400 mg orally b.i.d |
| Arm 2 MY008211A high dose | Experimental | Drug: MY008211A tabletsParticipants will receive MY008211A at a dose of 600 mg orally b.i.d |
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.
- LDH > 1.5 x Upper Limit of Normal (ULN).
- 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 (7 listed)
- Patients with reticulocytes <100x10^9/L; platelets <30x10^9/L; neutrophils <0.5x10^9/L.
- Were using a complement inhibitor before the first administration of MY008211A tablets or had discontinued a previous complement inhibitor for less than five half-lives or 120 days, whichever was the longest.
- 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.
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 tabletsAlso: MY008211Adose 1 (400 mg BID) and dose 2 (600 mg BID) in a 1:1 ratio by central randomization | Drug | Arm 1 MY008211A low dose, Arm 2 MY008211A high dose |
Primary outcome
The proportion of subjects with an increase in hemoglobin concentration ≥ 20 g/L from baseline among subjects who do not receive RBC transfusion after 4 weeks of dosing
Time frame: Day 70
Proportion of participants achieving a sustained increase from baseline in hemoglobin levels of ≥ 20 g/L assessed , in the absence of red blood cell transfusions
14 secondary outcomes
The proportion of patients with an increase in hemoglobin ≥ 20 g/L from baseline among those without RBC transfusion
Time frame: Day14, 21, 28, 42, 56
The proportion of patients with hemoglobin ≥ 120 g/L among those without RBC transfusion
Time frame: Day14, 21, 28, 42, 56 and 70
Change in hemoglobin concentration from baseline in patients without RBC transfusion
Time frame: Day14, 21, 28, 42, 56 and 70
Change in LDH level from baseline
Time frame: Day7, 14, 21, 28, 42, 56 and 70
The proportion of patients with hemolysis controlled
Time frame: Day7, 14, 21, 28, 42, 56 and 70
Change in reticulocyte count from baseline in patients without RBC transfusion
Time frame: Day7, 14, 21, 28, 42, 56 and 70
Change in indirect bilirubin level from baseline
Time frame: Day7, 14, 21, 28, 42, 56 and 70
The proportion of patients without RBC transfusion
Time frame: Day14, 21, 28, 42, 56 and 70
Change in the average weekly amount of RBC transfused during the efficacy observation period
Time frame: Day70
Change From Baseline in FACIT-Fatigue Questionnaire
Time frame: Day7, 14, 21, 28, 42, 56 and 70
Changes from baseline in alternative complement pathway activity
Time frame: Day14, 28, 56 and 70
Change in the amount of fragment Bb of CFB in plasma from baseline
Time frame: Day14, 28, 56 and 70
Change in the level of PNH RBC clones from baseline in patients without RBC transfusion.
Time frame: Day70
Incidence of Adverse Events (AEs) between Day 1 and Day 70
Time frame: Day 70
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 29 Jul 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.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing MY008211A tablets in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by Wuhan Createrna Science and Technology Co., Ltd.
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 7 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: opening soon, as of the sponsor's last update on 29 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Xiaoni Li, Ph.D (021-51158605, lixiaoni@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 subjects with an increase in hemoglobin concentration ≥ 20 g/L from baseline among subjects who do not receive RBC transfusion after 4 weeks of dosing (time frame: Day 70). 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 29 Jul 2025.