Study of Safety and Efficacy of MY008211A in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy

A Randomized, Multicenter, Active-comparator Controlled, Open-label Trial to Evaluate Efficacy and Safety of MY008211A Tablets in PNH Patients Who Are Naive to Complement Inhibitor Therapy

The main purpose of this study is to evaluate the efficacy of MY008211A compared to eculizumab in PNH patients.

Study Overview

• Status: Recruiting
• Conditions: Paroxysmal Nocturnal Haemoglobinuria (PNH)
• Intervention / Treatment: Drug: Eculizumab Injection; Drug: MY008211A tablets

Detailed Description

This is a multi-center, randomized, open-label, active comparator-controlled, parallel group study. The purpose of this study is to determine whether MY008211A compared to eculizumab is efficacious and safe for the treatment of PNH patients who were naive to complement inhibitor therapy.

• Study Type: Interventional
• Enrollment (Estimated): 66
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Wuhan Createrna Science and Technology Co.,Ltd; Phone Number: +86 027-68788900; Email: lcyxzx@createrna.com

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 100032
      • Not yet recruiting
      • Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
      • Contact: Bing Han, PhD; Phone Number: 010-69155027; Email: Hanbing_Li@sina.com.cn
  • Tianjin
    • Tianjin, Tianjin, China, 300020
      • Recruiting
      • Chinese Academy of Medical Sciences Hematology Hospital
      • Contact: Fengkui Zhang, PhD; Phone Number: 022-23909095

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. Mean hemoglobin level <100 g/L at screening.
3. LDH > 1.5 x Upper Limit of Normal (ULN) at screening.
4. 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: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: MY008211A tablets; MY008211A tablets 400mg BID	Drug: MY008211A tablets; Participants will receive MY008211A at a dose of 400 mg orally b.i.d for 24 weeks
Active Comparator: Eculizumab; Eculizumab Injection	Drug: Eculizumab Injection; Eculizumab Injection for 24 weeks

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	The proportion of patients with sustained hemoglobin levels ≥ 120 g/L among those without RBC transfusion (defined as no red blood cell infusion after D14 to D168).	between Day 126 and Day 168

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The proportion of subjects with an increase in hemoglobin concentration ≥ 20 g/L from baseline among subjects who do not receive RBC transfusion	Proportion of participants achieving a sustained increase from baseline in hemoglobin levels of ≥ 20 g/L assessed among those without RBC transfusion (defined as no red blood cell infusion after D14 to D168).	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
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 proportion of patients without RBC transfusion	The proportion of patients without RBC transfusion	between Day 14 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
Change in FACIT-F score from baseline	Change from baseline in FACIT-Fatigue scores. 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 10, 2025
• First Submitted That Met QC Criteria: April 16, 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; Immunosuppressive Agents; Immunologic Factors; Physiological Effects of Drugs; Complement Inactivating Agents; Eculizumab
• Other Study ID Numbers: MY008211A-PNH-3-02

Drug and device information, study documents

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