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

A Multi-center, Randomized, Open-label, Phase 2 Study to Evaluate the Efficacy and Safety of MY008211A Tablets in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria and Active Hemolysis.

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.

Study Overview

• Status: Completed
• Conditions: Paroxysmal Nocturnal Hemoglobinuria
• Intervention / Treatment: Drug: MY008211A tablets

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.

• Study Type: Interventional
• Enrollment (Actual): 34
• Phase: Phase 2

Contacts and Locations

Study Locations

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

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Arm1：low MY008211A dose; Participants will receive low MY008211A dose orally b.i.d	Drug: 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.; Other Names: MY008211A
Experimental: Arm2：high MY008211A dose; Participants will receive high MY008211A dose orally b.i.d	Drug: 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.; Other Names: MY008211A

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of participants achieving a sustained increase in hemoglobin levels of ≥ 20 g/L in the absence of red blood cell transfusion.	Proportion of participants achieving a sustained increase from baseline in hemoglobin levels of ≥ 20 g/L assessed , in the absence of red blood cell transfusions	up to 84 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of participants achieving sustained hemoglobin levels ≥ 120 g/L in the absence of red blood cell transfusions.	Proportion of participants achieving sustained hemoglobin levels ≥ 120 g/L in absence of red blood cell transfusion	up to 84 days
Change from baseline in hemoglobin concentration.	Change from baseline in hemoglobin concentration (g/L) in absence of red blood cell transfusion	up to 84 days
Change from baseline in serum LDH levels.	Change from baseline in serum LDH levels (U/L)	up to 84 days
Change from baseline in Reticulocyte count.	Change from baseline in Reticulocyte count (×10^9/L)	up to 84 days
Changes from baseline in transfusion volume.	The average number of red blood cells transfused per week	up to 84 days
Change in the level of PNH red cell clones.	Change from baseline in the level of PNH red cell clones.	up to 84 days
Occurrences of AEs occurring between Day 1 and Day 84.	Adverse Events (AEs)	up to 84 days

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes from baseline in alternative complement pathway activity.	Alternative complement pathway activity measured by the WIESLAB® kit.	up to 84 days
Change from baseline in plasma levels of the Bb fragment.	Bb fragment cleaved by factor B of complement.	up to 84 days
Maximum Plasma Concentration (Cmax) Of MY008211A tablets	PK parameters	up to 84 days
Area Under The Concentration Versus Time Curve (AUC) Of MY008211A	PK parameters	up to 84 days

Collaborators and Investigators

• Sponsor: Wuhan Createrna Science and Technology Co., Ltd
• Investigators: Principal Investigator: Fengkui Zhang, Ph.D, Blood disease hospital, Chinese academy of medical sciences

Study record dates

Study Major Dates

• Study Start (Actual): July 6, 2023
• Primary Completion (Actual): April 30, 2024
• Study Completion (Actual): November 28, 2024

Study Registration Dates

• First Submitted: September 11, 2023
• First Submitted That Met QC Criteria: September 17, 2023
• First Posted (Actual): September 22, 2023

Study Record Updates

• Last Update Posted (Actual): July 30, 2025
• Last Update Submitted That Met QC Criteria: July 27, 2025
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urogenital Diseases; Male Urogenital Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urination Disorders; Urological Manifestations; Hematologic Diseases; Bone Marrow Diseases; Anemia, Hemolytic; Anemia; Myelodysplastic Syndromes; Proteinuria; Hemoglobinuria; Hemoglobinuria, Paroxysmal
• Other Study ID Numbers: MY008211A-PNH-2-01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No