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NCT Number: NCT06932744

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

The main purpose of this study is to evaluate the efficacy of MY008211A compared to eculizumab in PNH patients.

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Key information

Conditions

Paroxysmal Nocturnal Haemoglobinuria (PNH) Anemia Anemia, Hemolytic Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinuria, Paroxysmal Myelodysplastic Syndromes

Age range

18 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Beijing, Beijing Municipality, China

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

MY008211A tablets

Drug

Participants will receive MY008211A at a dose of 400 mg orally b.i.d for 24 weeks

Eculizumab Injection

Drug

Eculizumab Injection for 24 weeks

Primary outcomes

  1. The proportion of Participants With Sustained Hemoglobin Levels of ≥ 120 g/L in the Absence of Red Blood Cell Transfusions

    Time frame: between Day 126 and Day 168

    The proportion of patients with sustained hemoglobin levels ≥ 120 g/L among those without RBC transfusion (defined as no red blood cell infusion after D14 to D168).

Secondary outcomes

  1. The proportion of subjects with an increase in hemoglobin concentration ≥ 20 g/L from baseline among subjects who do not receive RBC transfusion

    Time frame: between Day 126 and Day 168

    Proportion of participants achieving a sustained increase from baseline in hemoglobin levels of ≥ 20 g/L assessed among those without RBC transfusion (defined as no red blood cell infusion after D14 to D168).

  2. The proportion of patients with LDH < 1.5 ULN among those without RBC transfusion.

    Time frame: between Day 126 and Day 168

    The proportion of patients with hemolysis controlled (defined as LDH < 1.5 ULN) among those without RBC transfusion

  3. Change (Expressed as Percentages) in LDH level from baseline

    Time frame: between Day 126 and Day 168

    Change (Expressed as Percentages) in LDH level from baseline

  4. Change in reticulocyte count from baseline

    Time frame: between Day 126 and Day 168

    Change in reticulocyte count from baseline

  5. The proportion of patients without RBC transfusion

    Time frame: between Day 14 and Day 168

    The proportion of patients without RBC transfusion

  6. Change in FACIT-F score from baseline

    Time frame: between Day 126 and Day 168

    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.

  7. The Clinical BTH Rate

    Time frame: between Day 1 and Day 168

    The Clinical BTH Rate

  8. The Major Adverse Vascular Events Rate

    Time frame: between Day 1 and Day 168

    The Major Adverse Vascular Events Rate

Study contacts

Contact information is provided by the study sponsor or research team.

Wuhan Createrna Science and Technology Co.,Ltd

CONTACT

[email protected]

027-68788900 ext. +86

Sponsors and collaborators

Lead sponsor

Wuhan Createrna Science and Technology Co., Ltd

Industry

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2025
Study completion
2025
First posted
Apr 17, 2025
Registry last updated
May 9, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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