ClinicalTrials.gov record NCT06932744

Study of Safety and Efficacy of MY008211A in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

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

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 3A 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.
Age18 years old to 75 years old
SexAll
LocationChina
AI summaryWhat this study is about, 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.

This randomized, open-label trial compares oral MY008211A tablets with eculizumab injections in adults with PNH who are naive to complement inhibitor therapy. Participants receive either MY008211A 400 mg twice daily or eculizumab for 24 weeks. The main measure is the proportion who sustain hemoglobin ≥120 g/L without red blood cell transfusion late in treatment.

What it is studying. The study is testing whether oral MY008211A (400 mg twice daily) is efficacious and safe compared with eculizumab injections in adults with PNH who have not previously received complement inhibitor therapy. It focuses on blood outcomes (sustained hemoglobin without transfusion, hemolysis measured by LDH, reticulocytes), fatigue scores, breakthrough hemolysis events, and major vascular events.

What the researchers hope to learn. To evaluate the efficacy and safety of MY008211A compared with eculizumab in PNH. Specific scientific goals include measuring the proportion achieving sustained hemoglobin ≥120 g/L without transfusion, changes in LDH and reticulocyte counts, change in FACIT‑Fatigue score, clinical breakthrough hemolysis rate, and major adverse vascular 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 compared to eculizumab in PNH patients.

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
  • Eculizumab Injection

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 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.

Exclusion criteria

  • 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.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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.

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 are randomized to receive either oral MY008211A tablets (400 mg twice daily) or eculizumab injections for 24 weeks. They will have clinic visits for treatment, safety checks and blood tests to monitor hemoglobin, LDH, reticulocyte count and other labs, plus assessments of transfusion use and fatigue (FACIT‑F questionnaire). Vaccination against meningococcus (and pneumococcus/H. influenzae if needed) is required before starting treatment. The study includes scheduled visits between Day 1 and Day 168 to collect these measurements and monitor for side effects.

How the study is structured. A randomized, open-label, parallel-group drug trial. Participants are assigned to one of two arms for 24 weeks: MY008211A tablets given orally twice daily, or eculizumab given by injection; key outcomes are assessed through Day 168.

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

Clinical trials44 trials listed for paroxysmal nocturnal hemoglobinuriaEvery trial the registry lists for paroxysmal nocturnal hemoglobinuria, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in paroxysmal nocturnal hemoglobinuria researchLatest monthly update: September 2026. Includes: Treatment access. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Aug 30, 2024
Primary completion
Dec 30, 2025 (estimated)
Study completion
Dec 30, 2025 (estimated)
Target enrollment
66 participants (estimated)
Last updated on registry
May 9, 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.

Participating sites (2)

  • Peking Union Medical College Hospital, Chinese Academy of Medical SciencesNot Yet Recruiting

    Beijing, Beijing Municipality, 100032 · China

  • Chinese Academy of Medical Sciences Hematology HospitalRecruiting

    Tianjin, Tianjin Municipality, 300020 · China

Terms used on this page · explanations by MedicalUpdates.ai

at screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
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.
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.
randomized
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
open-label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
active comparator
An existing treatment used as the comparison group instead of a placebo, so the study asks whether the new treatment does as well as or better than current care.
parallel group
Participants are divided into two or more groups, and each group receives a different intervention at the same time, so the groups can be compared.

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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