Study of Safety and Efficacy of MY008211A in Patients With Residual Anemia Despite Anti-C5 Antibody Treatment

Wuhan Createrna Science and Technology

Status and phase: Enrolling; Phase 3

Conditions: Paroxysmal Nocturnal Hemoglobinuria

Treatments: Drug: MY008211A tablets

Study type: Interventional

Funder types: Industry

Identifiers

NCT06932471

MY008211A-PNH-3-01

Details and patient eligibility

About

The main purpose of this study is to evaluate the efficacy of MY008211A in PNH patients with residual anemia despite treatment with anti-C5 antibody.

Full description

This is a multicenter, single-arm, open-label phase III clinical trial. The purpose of this study is to determine whether MY008211A is efficacious and safe for the treatment of PNH patients Who Are Still Anemia After Anti-C5 Antibody Treatment.

Enrollment: 20 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. Stable regimen of anti-C5 antibody treatment for at least 6 months before treatment, and Hb was still < 100 g/L.
3. The average hemoglobin level of at least two tests in 4 months before screening < 100 g/L.
4. The average hemoglobin level of two tests in the central laboratory during screening < 100 g/L.
5. 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: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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20 participants in 1 patient group

MY008211A tablets

Experimental group

Description:

MY008211A tablets 400mg BID

Treatment:

Drug: MY008211A tablets