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

A Multicenter, Single-arm, Open-label Phase III Study to Evaluate Efficacy and Safety of MY008211A Tablets in Patients With PNH and Residual Anemia, Despite Treatment With Anti-C5 Antibody.

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.

Study Overview

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

Detailed 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.

• Study Type: Interventional
• Enrollment (Estimated): 20
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Wuhan Createrna Science and Technology Co.,Ltd; Phone Number: 027-68788900; Email: lcyxzx@createrna.com

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 100032
      • Recruiting
      • Peking Union Medical College Hospital (PUMCH).
      • Contact: Bing Han, PhD; Phone Number: 010-69155027; Email: Hanbing_Li@sina.com.cn

Participation Criteria

Eligibility Criteria

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

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: MY008211A tablets; MY008211A tablets 400mg BID	Drug: MY008211A tablets; MY008211A tablets Participants will receive MY008211A at a dose of 400 mg orally b.i.d; Other Names: MY008211A

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The proportion of Participants With Sustained Hemoglobin Levels of ≥ 120 g/L in the Absence of Red Blood Cell Transfusions (defined as no red blood cell infusion after D14 to D168)	The proportion of patients with sustained hemoglobin levels ≥ 120 g/L among those without RBC transfusion.	between Day 126 and Day 168

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change (Expressed as Percentages) in LDH level from baseline	Change (Expressed as Percentages) in LDH level from baseline	between Day 126 and Day 168
Change in reticulocyte count from baseline	Change in reticulocyte count from baseline	between Day 126 and Day 168
The Clinical BTH Rate	The Clinical BTH Rate	between Day 1 and Day 168
The Major Adverse Vascular Events Rate	The Major Adverse Vascular Events Rate	between Day 1 and Day 168
The proportion of subjects with an increase in hemoglobin concentration ≥ 20 g/L from baseline among subjects who do not receive RBC transfusion (defined as no red blood cell infusion after D14 to D168)	Proportion of participants achieving a sustained increase from baseline in hemoglobin levels of ≥ 20 g/L assessed among those without RBC transfusion.	between Day 126 and Day 168
Change From Baseline in Hemoglobin	Change in hemoglobin concentration from baseline in patients without RBC transfusion.	between Day 126 and Day 168
The proportion of patients with LDH < 1.5 ULN among those without RBC transfusion.	The proportion of patients with hemolysis controlled (defined as LDH < 1.5 ULN) among those without RBC transfusion.	between Day 126 and Day 168
The proportion of patients without RBC transfusion	The proportion of patients without RBC transfusion.	between Day 14 and Day 168
Change in FACIT-F score from baseline	Change in FACIT-Fatigue score from baseline. The FACIT-Fatigue is a 13-item questionnaire with support for its validity and reliability in PNH that assesses patient self-reported fatigue and its impact on daily activities and function. All FACIT scales are scored so that a high score is better. As each of the 13 items of the FACIT-F Scale ranges from 0-4, the range of possible scores is 0-52, with 0 being the worst possible score and 52 the best.	between Day 126 and Day 168

Collaborators and Investigators

• Sponsor: Wuhan Createrna Science and Technology Co., Ltd

Study record dates

Study Major Dates

• Study Start (Actual): August 30, 2024
• Primary Completion (Estimated): December 30, 2025
• Study Completion (Estimated): December 30, 2025

Study Registration Dates

• First Submitted: April 9, 2025
• First Submitted That Met QC Criteria: April 9, 2025
• First Posted (Actual): April 17, 2025

Study Record Updates

• Last Update Posted (Actual): May 9, 2025
• Last Update Submitted That Met QC Criteria: May 5, 2025
• Last Verified: April 1, 2025

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-3-01

Drug and device information, study documents

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