Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT ID: NCT06134414

Last Updated: 2025-07-29

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 40 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-12-31
• Study Completion Date: 2027-12-31

Brief Summary

The main purpose of this study is to evaluate the efficacy of MY008211A in adult patients with PNH, showing signs of active hemolysis.

Detailed Description

The purpose of this study is to determine whether MY008211A is efficacious and safe for the treatment of PNH patients who are naïve to complement inhibitor therapy, including anti-C5 antibody.

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Arm 1 MY008211A low dose

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

Group Type: EXPERIMENTAL

MY008211A tablets

Intervention Type: DRUG

dose 1 (400 mg BID) and dose 2 (600 mg BID) in a 1:1 ratio by central randomization

Arm 2 MY008211A high dose

Participants will receive MY008211A at a dose of 600 mg orally b.i.d

Group Type: EXPERIMENTAL

MY008211A tablets

Intervention Type: DRUG

dose 1 (400 mg BID) and dose 2 (600 mg BID) in a 1:1 ratio by central randomization

Interventions

MY008211A tablets

dose 1 (400 mg BID) and dose 2 (600 mg BID) in a 1:1 ratio by central randomization

Intervention Type: DRUG

Other Intervention Names

MY008211A

Eligibility Criteria

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.
• LDH > 1.5 x Upper Limit of Normal (ULN).
• 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.
• Were using a complement inhibitor before the first administration of MY008211A tablets or had discontinued a previous complement inhibitor for less than five half-lives or 120 days, whichever was the longest.
• 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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Wuhan Createrna Science and Technology Co., Ltd

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Central Contacts

Xiaoni Li, Ph.D

Role: CONTACT

lixiaoni@createrna.com

021-51158605

Other Identifiers

MY008211A-PNH-2-01-F

Identifier Type: -

Identifier Source: org_study_id