ClinicalTrials.gov record NCT06933914
Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria
An Open Label, Multicenter Study to Evaluate the Long-term Safety and Tolerability of MY008211A Tablets in Patients With PNH Paroxysmal Nocturnal Hemoglobinuria (PNH)
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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 open-label, single-arm, multicenter study follows people with paroxysmal nocturnal hemoglobinuria (PNH) who receive MY008211A tablets to evaluate long-term safety and tolerability. Participants take MY008211A 400 mg orally twice daily. The study also provides continued access to MY008211A for patients who completed prior Phase 2 or Phase 3 trials.
What it is studying. The study is collecting long-term safety and tolerability data for MY008211A tablets given at 400 mg twice daily in people with PNH. It will record adverse events, laboratory safety tests, vital signs, and ECGs, and it will track blood hemoglobin levels and transfusion-related outcomes over the study period (about 100 weeks).
What the researchers hope to learn. To characterize the long-term safety and tolerability of MY008211A in people with PNH. To collect safety data (adverse events, laboratory parameters, vital signs, ECG). To evaluate hemoglobin changes, the proportion of participants avoiding red blood cell transfusion, clinical breakthrough hemolysis rate, and major adverse vascular events over about 100 weeks.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
This is a multicenter, single-arm, open-label study to characterize long-term safety and tolerability of MY008211A tablets and to provide access to MY008211A tablets to patients with PNH who have completed Phase 2 or 3 studies with MY008211A tablets.
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
- Patients who have previously received and completed MY008211A study treatment, and are judged by the investigator to have treatment benefit and may benefit from continued treatment of MY008211A.
- Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections.
Exclusion criteria
- History of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.
- Known or suspected hereditary complement deficiency.
- Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the investigator, could put the subject at increased risk or potentially confound study data.
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 open-label, single arm, multicenter study is to evaluate the long-term safety, tolerability and efficacy of MY008211A tablets in patients with PNH and to provide access to patients who have completed (without tapering down) Phase 2 and Phase 3 trials and derived benefit from MY008211A treatment.
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.
Participants will take MY008211A tablets 400 mg by mouth twice daily. They will attend regular clinic visits (about every 12 weeks) for safety monitoring—reporting adverse events, blood tests, vital signs, ECGs—and for assessments of hemoglobin levels and transfusion status. The study follows participants to approximately 100 weeks. The registry does not list particularly invasive procedures (for example, biopsies or spinal taps).
How the study is structured. Open-label (no blinding), single-group interventional trial. All participants receive MY008211A tablets 400 mg twice daily. Safety and outcome assessments are performed regularly (about every 12 weeks) over roughly 100 weeks at multiple study centers.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
- 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
- Nov 30, 2024
- Primary completion
- Dec 30, 2026 (estimated)
- Study completion
- Dec 30, 2026 (estimated)
- Target enrollment
- 120 participants (estimated)
- Last updated on registry
- Apr 18, 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.
- Wuhan Createrna Science and Technology Co.,Ltd 📞 027-68788900 ✉ lcyxzx@createrna.com
Participating sites (1)
- Chinese Academy of Medical Sciences Hematology Hospital (Institute of Hematology, Chinese Academy of Medical Sciences)Recruiting
Tianjin, Tianjin Municipality, 300020 · China
- Fengkui Zhang, PhD · 022-23909095
Terms used on this page · explanations by MedicalUpdates.ai
- single-arm
- All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- Phase 2
- A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
- 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.
- Phase 3
- A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.
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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