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

Phase 3

Recruiting

• Conditions: Paroxysmal Nocturnal Haemoglobinuria (PNH)
• Interventions: Drug: MY008211A tablets; Drug: Eculizumab Injection

• Registration Number: NCT06932744
• Lead Sponsor: Wuhan Createrna Science and Technology Co., Ltd

Brief Summary

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

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 Timeline

• Study Start: 2024-08-30
• Status Verified: 2025-04
• Study First Submitted: 2025-04-10
• Study First Submitted QC: 2025-04-16
• Study First Posted: 2025-04-17
• Last Update Submitted: 2025-05-05
• Last Update Posted: 2025-05-09
• Primary Completion: 2025-12-30
• Study Completion: 2025-12-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 66

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
MY008211A tablets	MY008211A tablets	MY008211A tablets 400mg BID
Eculizumab	Eculizumab Injection	Eculizumab Injection

Primary Outcome Measures

Name	Time	Method
The proportion of Participants With Sustained Hemoglobin Levels of ≥ 120 g/L in the Absence of Red Blood Cell Transfusions	between Day 126 and Day 168	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).

Secondary Outcome Measures

Name	Time	Method
Change (Expressed as Percentages) in LDH level from baseline	between Day 126 and Day 168	Change (Expressed as Percentages) in LDH level from baseline
Change in reticulocyte count from baseline	between Day 126 and Day 168	Change in reticulocyte count from baseline
The proportion of patients without RBC transfusion	between Day 14 and Day 168	The proportion of patients without RBC transfusion
The Clinical BTH Rate	between Day 1 and Day 168	The Clinical BTH Rate
The Major Adverse Vascular Events Rate	between Day 1 and Day 168	The Major Adverse Vascular Events Rate
The proportion of subjects with an increase in hemoglobin concentration ≥ 20 g/L from baseline among subjects who do not receive RBC transfusion	between Day 126 and Day 168	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).
The proportion of patients with LDH < 1.5 ULN among those without RBC transfusion.	between Day 126 and Day 168	The proportion of patients with hemolysis controlled (defined as LDH \< 1.5 ULN) among those without RBC transfusion
Change in FACIT-F score from baseline	between Day 126 and Day 168	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.

Trial Locations

Locations (2)

Peking Union Medical College Hospital, Chinese Academy of Medical Sciences; 🇨🇳 Beijing, Beijing, China

Chinese Academy of Medical Sciences Hematology Hospital; 🇨🇳 Tianjin, Tianjin, China