A Study of MY008211A in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT ID: NCT06050226

Last Updated: 2025-07-30

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 34 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-07-06
• Study Completion Date: 2024-11-28

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, in China.

Detailed Description

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

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Study Design

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

Study Groups

Arm1：low MY008211A dose

Participants will receive low MY008211A dose orally b.i.d

Group Type: EXPERIMENTAL

MY008211A tablets

Intervention Type: DRUG

The first 10 participants will be received low-dose MY008211A tablets, and the next 30 participants will be randomized to low-dose or high-dose treatment arms in a 1:2 ratio.

Arm2：high MY008211A dose

Participants will receive high MY008211A dose orally b.i.d

Group Type: EXPERIMENTAL

MY008211A tablets

Intervention Type: DRUG

The first 10 participants will be received low-dose MY008211A tablets, and the next 30 participants will be randomized to low-dose or high-dose treatment arms in a 1:2 ratio.

Interventions

MY008211A tablets

The first 10 participants will be received low-dose MY008211A tablets, and the next 30 participants will be randomized to low-dose or high-dose treatment arms in a 1:2 ratio.

Intervention Type: DRUG

Other Intervention Names

MY008211A

Eligibility Criteria

Inclusion Criteria

• Male and female participants ≥ 18 years of age, BMI≥18 kg/m2，with a diagnosis of PNH confirmed by laboratory tests, according to the PNH diagnostic criteria in the Chinese Guidelines for the Diagnosis and Treatment of Rare Diseases (2019 edition) , and 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

Principal Investigators

Fengkui Zhang, Ph.D

Role: PRINCIPAL_INVESTIGATOR

Blood Disease Hospital, Chinese Academy of Medical Sciences

Locations

Institute of Hematology & Blood Disease Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College

Tianjin, Tianjin Municipality, China

Countries

China

Other Identifiers

MY008211A-PNH-2-01

Identifier Type: -

Identifier Source: org_study_id