Evaluating MY008211A for treating PNH patients new to complement inhibitors

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

This study is testing a new medication called MY008211A to see if it works better than the standard treatment for people with Paroxysmal Nocturnal Hemoglobinuria who haven't used complement inhibitors before.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 66 (estimated)
• Ages: 18 Years to 75 Years
• Sex: All
• Sponsor: Wuhan Createrna Science and Technology Co., Ltd (Industry-sponsored)
• Drugs / interventions: eculizumab
• Locations: 2 sites (Beijing, Beijing Municipality and 1 other locations)
• Trial ID: NCT06932744 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to assess the safety and efficacy of MY008211A compared to eculizumab in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have not previously received complement inhibitor therapy. It is a multi-center, randomized, open-label study that will involve parallel groups receiving either MY008211A tablets or eculizumab injections. The study will measure various health outcomes to determine the effectiveness of MY008211A in improving patient conditions compared to the standard treatment.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 and older with a confirmed diagnosis of PNH and specific laboratory criteria.

Not a fit: Patients with severe blood cell deficiencies or a history of certain infections or malignancies may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new effective option for PNH patients who have not yet been treated with complement inhibitors.

How similar studies have performed: Other studies have shown success with complement inhibitors in treating PNH, but the efficacy of MY008211A is being specifically evaluated in this novel approach.

Eligibility criteria

Inclusion Criteria:

1. 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%.
2. Mean hemoglobin level \<100 g/L at screening.
3. LDH \> 1.5 x Upper Limit of Normal (ULN) at screening.
4. 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:

1. Patients with reticulocytes \<100x10\^9/L; platelets \<30x10\^9/L; neutrophils \<0.5x10\^9/L.
2. History of recurrent invasive infections caused by encapsulated organisms,e.g. meningococcus or pneumococcus.
3. Known or suspected hereditary complement deficiency.
4. Previous bone marrow or hematopoietic stem cell transplantation.
5. Previous splenectomy.
6. 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.

Where this trial is running

Beijing, Beijing Municipality and 1 other locations

• Peking Union Medical College Hospital, Chinese Academy of Medical Sciences — Beijing, Beijing Municipality, China (Not_yet_recruiting)
• Chinese Academy of Medical Sciences Hematology Hospital — Tianjin, Tianjin Municipality, China (Recruiting)

Study contacts

• Study coordinator: Wuhan Createrna Science and Technology Co.,Ltd

Conditions Paroxysmal Nocturnal Haemoglobinuria