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

NCT06932744 | Recruiting Phase 3

• 1 other identifier: MY008211A-PNH-3-02
• Study Type: interventional
• Target: 66
• Locations: 1 country
• Sites: 2

Brief Summary

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

Conditions

Paroxysmal Nocturnal Haemoglobinuria (PNH)

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (8)

• 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

• The proportion of patients with LDH < 1.5 ULN among those without RBC transfusion.

  between Day 126 and Day 168

• Change (Expressed as Percentages) in LDH level from baseline

  between Day 126 and Day 168

• Change in reticulocyte count from baseline

  between Day 126 and Day 168

• The proportion of patients without RBC transfusion

  between Day 14 and Day 168

Study Arms (2)

MY008211A tablets

EXPERIMENTAL

MY008211A tablets 400mg BID

Drug: MY008211A tablets

Eculizumab

ACTIVE COMPARATOR

Eculizumab Injection

Drug: Eculizumab Injection

Interventions

MY008211A tablets DRUG

Participants will receive MY008211A at a dose of 400 mg orally b.i.d for 24 weeks

MY008211A tablets

Eculizumab Injection DRUG

Eculizumab Injection for 24 weeks

Eculizumab

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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%.
• Mean hemoglobin level \<100 g/L at screening.
• LDH \> 1.5 x Upper Limit of Normal (ULN) at screening.
• 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.

You may not qualify if:

• Patients with reticulocytes \<100x10\^9/L; platelets \<30x10\^9/L; neutrophils \<0.5x10\^9/L.
• 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.

Sponsors & Collaborators

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

Study Sites (2)

Peking Union Medical College Hospital, Chinese Academy of Medical Sciences

Beijing, Beijing Municipality, 100032, China

NOT YET RECRUITING

Chinese Academy of Medical Sciences Hematology Hospital

Tianjin, Tianjin Municipality, 300020, China

RECRUITING

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Interventions

eculizumab

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Central Study Contacts

Wuhan Createrna Science and Technology Co.,Ltd

CONTACT

027-68788900; lcyxzx@createrna.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 10, 2025
• First Posted: April 17, 2025
• Study Start: August 30, 2024
• Primary Completion: December 30, 2025
• Study Completion: December 30, 2025
• Last Updated: May 9, 2025

Record last verified: 2025-04

Locations

CN (2)