Paroxysmal Nocturnal HemoglobinuriaPhase 2NCT06050226
A Study of MY008211A in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
A Phase 2 study testing MY008211A tablets in people with Paroxysmal Nocturnal Hemoglobinuria, run by Wuhan Createrna Science and Technology Co., Ltd at 1 site in China.
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, in China. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- 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
- 34 participants
- Where
- 1 siteChina
- Study dates
- 6 Jul 2023to 28 Nov 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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, BMI≥18 kg/m2，with a diagnosis of PNH confirmed by laboratory tests, according to the PNH diagnostic criteria in the Chinese Guidelines for the Diagnosis and Treatment of Rare Diseases (2019 edition) , and 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
1 site in 1 country.
China1 site
Institute of Hematology & Blood Disease Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College
Tianjin, Tianjin Municipality
About this study
Official title: A Multi-center, Randomized, Open-label, Phase 2 Study to Evaluate the Efficacy and Safety of MY008211A Tablets in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria and 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, in China.
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 naive to complement inhibitor therapy, including anti-C5 antibody.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- 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
The first 10 participants will be received low-dose MY008211A tablets, and the next 30 participants will be randomized to low-dose or high-dose treatment arms in a 1:2 ratio.
| Study group | Role | What they receive |
|---|---|---|
| Arm1：low MY008211A dose | Experimental | Participants will receive low MY008211A dose orally b.i.d Receives: Drug: MY008211A tablets |
| Arm2：high MY008211A dose | Experimental | Participants will receive high MY008211A dose 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.
Proportion of participants achieving a sustained increase in hemoglobin levels of ≥ 20 g/L in the absence of red blood cell transfusion.
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: up to 84 days
Secondary outcomes (7)
Proportion of participants achieving sustained hemoglobin levels ≥ 120 g/L in the absence of red blood cell transfusions.
Proportion of participants achieving sustained hemoglobin levels ≥ 120 g/L in absence of red blood cell transfusion
Measured: up to 84 days
Change from baseline in hemoglobin concentration.
Change from baseline in hemoglobin concentration (g/L) in absence of red blood cell transfusion
Measured: up to 84 days
Change from baseline in serum LDH levels.
Change from baseline in serum LDH levels (U/L)
Measured: up to 84 days
Change from baseline in Reticulocyte count.
Change from baseline in Reticulocyte count (×10^9/L)
Measured: up to 84 days
Changes from baseline in transfusion volume.
The average number of red blood cells transfused per week
Measured: up to 84 days
Change in the level of PNH red cell clones.
Change from baseline in the level of PNH red cell clones.
Measured: up to 84 days
Occurrences of AEs occurring between Day 1 and Day 84.
Adverse Events (AEs)
Measured: up to 84 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2023
- Study start6 Jul 2023
- Primary completion30 Apr 2024
- Study completion28 Nov 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies 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
- 34 participants (actual)
Sponsor and investigators
- Lead sponsor
- Wuhan Createrna Science and Technology Co., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Fengkui Zhang, Ph.D, Principal investigator, Blood Disease Hospital, Chinese Academy of Medical Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06050226
- Sponsor's protocol ID
- MY008211A-PNH-2-01
- Status last verified
- Sep 2023
- First submitted
- 11 Sep 2023
- Last updated
- 30 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 30 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center, Randomized, Open-label, Phase 2 Study to Evaluate the Efficacy and Safety of MY008211A Tablets in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria and Active Hemolysis.
- ClinicalTrials.gov ID
- NCT06050226
- Acronym / sponsor ID
- MY008211A-PNH-2-01
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 34 participants (actual)
- Lead sponsor
- Wuhan Createrna Science and Technology Co., LtdIndustry (pharma, biotech, devices)
- Study start
- 6 Jul 2023 (actual)
- Primary completion
- 30 Apr 2024 (actual)
- Study completion
- 28 Nov 2024 (actual)
- First posted
- 22 Sep 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, in China.
Detailed description
The purpose of this study is to determine whether MY008211A is efficacious and safe for the treatment of PNH patients who are naive 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 |
|---|---|---|
| Arm1：low MY008211A dose | Experimental | Drug: MY008211A tabletsParticipants will receive low MY008211A dose orally b.i.d |
| Arm2：high MY008211A dose | Experimental | Drug: MY008211A tabletsParticipants will receive high MY008211A dose 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, BMI≥18 kg/m2，with a diagnosis of PNH confirmed by laboratory tests, according to the PNH diagnostic criteria in the Chinese Guidelines for the Diagnosis and Treatment of Rare Diseases (2019 edition) , and 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: MY008211AThe first 10 participants will be received low-dose MY008211A tablets, and the next 30 participants will be randomized to low-dose or high-dose treatment arms in a 1:2 ratio. | Drug | Arm1：low MY008211A dose, Arm2：high MY008211A dose |
Primary outcome
Proportion of participants achieving a sustained increase in hemoglobin levels of ≥ 20 g/L in the absence of red blood cell transfusion.
Time frame: up to 84 days
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
7 secondary outcomes
Proportion of participants achieving sustained hemoglobin levels ≥ 120 g/L in the absence of red blood cell transfusions.
Time frame: up to 84 days
Change from baseline in hemoglobin concentration.
Time frame: up to 84 days
Change from baseline in serum LDH levels.
Time frame: up to 84 days
Change from baseline in Reticulocyte count.
Time frame: up to 84 days
Changes from baseline in transfusion volume.
Time frame: up to 84 days
Change in the level of PNH red cell clones.
Time frame: up to 84 days
Occurrences of AEs occurring between Day 1 and Day 84.
Time frame: up to 84 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 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. This study is not recruiting at the moment.
Fengkui Zhang, Ph.D principal investigator
Blood Disease Hospital, Chinese Academy of Medical Sciences
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing MY008211A tablets in people with Paroxysmal Nocturnal Hemoglobinuria, run by Wuhan Createrna Science and Technology Co., Ltd at 1 site 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 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: finished, as of the sponsor's last update on 30 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of participants achieving a sustained increase in hemoglobin levels of ≥ 20 g/L in the absence of red blood cell transfusion. (time frame: up to 84 days). 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 30 Jul 2025.