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

Wuhan Createrna Science and Technology

Status and phase: Enrolling; Phase 3

Conditions: Paroxysmal Nocturnal Haemoglobinuria (PNH)

Treatments: Drug: Eculizumab Injection; Drug: MY008211A tablets

Study type: Interventional

Funder types: Industry

Identifiers

NCT06932744

MY008211A-PNH-3-02

Details and patient eligibility

About

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

Full description

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.

Enrollment: 66 estimated patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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66 participants in 2 patient groups

MY008211A tablets

Experimental group

Description:

MY008211A tablets 400mg BID

Treatment:

Drug: MY008211A tablets

Eculizumab

Active Comparator group

Description:

Eculizumab Injection

Treatment:

Drug: Eculizumab Injection