Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria

Phase 2

Recruiting

• Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Interventions: Drug: MY008211A tablets

• Registration Number: NCT06933914
• Lead Sponsor: Wuhan Createrna Science and Technology Co., Ltd

Brief Summary

This is a multicenter, single-arm, open-label study to characterize long-term safety and tolerability of MY008211A tablets and to provide access to MY008211A tablets to patients with PNH who have completed Phase 2 or 3 studies with MY008211A tablets.

Detailed Description

The purpose of this open-label, single arm, multicenter study is to evaluate the long-term safety, tolerability and efficacy of MY008211A tablets in patients with PNH and to provide access to patients who have completed (without tapering down) Phase 2 and Phase 3 trials and derived benefit from MY008211A treatment.

Study Timeline

• Study Start: 2024-11-30
• Status Verified: 2025-04
• Study First Submitted: 2025-04-11
• Study First Submitted QC: 2025-04-11
• Last Update Submitted: 2025-04-11
• Study First Posted: 2025-04-18
• Last Update Posted: 2025-04-18
• Primary Completion: 2026-12-30
• Study Completion: 2026-12-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 120

Inclusion Criteria

1. Patients who have previously received and completed MY008211A study treatment, and are judged by the investigator to have treatment benefit and may benefit from continued treatment of MY008211A.
2. Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections.

Exclusion Criteria

1. History of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.
2. Known or suspected hereditary complement deficiency.
3. Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the investigator, could put the subject at increased risk or potentially confound study data.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Proportion of participants with adverse events, safety laboratory parameters, vital signs, ECG.	About 100 weeks	Safety evaluations including but not limited to adverse events, laboratory parameters, vital signs, ECG through End of Study visit every 12 weeks.

Secondary Outcome Measures

Name	Time	Method
Proportion of participants achieving sustained hemoglobin levels ≥ 120 g/L in the absence of red blood cell transfusions	About 100 weeks	Proportion of participants achieving sustained hemoglobin levels ≥ 120 g/L in the absence of red blood cell transfusions evaluated every 12 weeks.
Change From Baseline in Hemoglobin	About 100 weeks	Change in hemoglobin concentration from baseline in patients without RBC transfusion every 12 weeks.
The proportion of patients without RBC transfusion.	About 100 weeks	The proportion of patients without RBC transfusion.
The Clinical BTH Rate	About 100 weeks	The Clinical BTH Rate
The Major Adverse Vascular Events Rate	About 100 weeks	The Major Adverse Vascular Events Rate

Trial Locations

Locations (1)

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