ClinicalTrials.gov record NCT06134414
Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
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
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This Phase II randomized, open-label study compares two oral doses of MY008211A in adults with PNH who have signs of active hemolysis and who have not previously received complement inhibitor therapy. Participants are assigned to a low or high dose and followed with blood tests and symptom questionnaires through Day 70. The study measures hemoglobin changes, markers of hemolysis, transfusion needs, fatigue, and safety.
What it is studying. Whether two different oral doses of MY008211A affect hemoglobin, hemolysis markers, transfusion requirements, fatigue scores, and safety in adults with active PNH who are naïve to complement inhibitors.
What the researchers hope to learn. To evaluate the efficacy and safety of MY008211A in adult PNH patients naïve to complement inhibitors by measuring: increases in hemoglobin (including proportion with ≥20 g/L rise), lactate dehydrogenase (LDH) and control of hemolysis, transfusion use, reticulocyte and bilirubin changes, PNH red-cell clone levels, alternative complement pathway activity, fatigue (FACIT-Fatigue), and adverse events.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
The main purpose of this study is to evaluate the efficacy of MY008211A in adult patients with PNH, showing signs of active hemolysis.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- MY008211A tablets
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- 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.
Exclusion criteria
- 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.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
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.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
If enrolled, participants are randomly assigned (open‑label) to one of two oral doses of MY008211A taken twice daily (400 mg or 600 mg). They will attend regular clinic visits for blood tests and safety checks, with key assessments scheduled through about Day 70 (including hemoglobin, LDH, and other laboratory measures). Participants will complete the FACIT‑Fatigue questionnaire and be monitored for adverse events; transfusion needs and hemolysis control will also be tracked. Vaccination against certain bacteria must be done before starting study treatment if not already given.
How the study is structured. Multi-center, randomized (1:1), parallel, open-label Phase II drug trial. Adults are randomly assigned to a low or high oral dose of MY008211A and take tablets twice daily. Study staff will collect blood tests, monitor for side effects, and collect questionnaire data at scheduled visits through about 10 weeks (assessments to Day 70).
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- No sites are listed on the registry yet. Where will the study take place?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on paroxysmal nocturnal hemoglobinuria
Important dates
- Study start
- December 2025 (estimated)
- Primary completion
- July 2027 (estimated)
- Study completion
- December 2027 (estimated)
- Target enrollment
- 40 participants (estimated)
- Last updated on registry
- Jul 29, 2025
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Wuhan Createrna Science and Technology Co., Ltd
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Xiaoni Li, Ph.D 📞 021-51158605 ✉ lixiaoni@createrna.com
Participating sites
Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins. The central study contact above may be able to tell you about planned sites.
Terms used on this page · explanations by MedicalUpdates.ai
- Upper Limit of Normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- five half-lives
- The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
- before screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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