Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria
An Open Label, Multicenter Study to Evaluate the Long-term Safety and Tolerability of MY008211A Tablets in Patients With PNH Paroxysmal Nocturnal Hemoglobinuria (PNH)
1 other identifier
interventional
120
1 country
1
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Nov 2024
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
November 30, 2024
CompletedFirst Submitted
Initial submission to the registry
April 11, 2025
CompletedFirst Posted
Study publicly available on registry
April 18, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 30, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 30, 2026
April 18, 2025
April 1, 2025
2.1 years
April 11, 2025
April 11, 2025
Conditions
Outcome Measures
Primary Outcomes (1)
Proportion of participants with adverse events, safety laboratory parameters, vital signs, ECG.
Safety evaluations including but not limited to adverse events, laboratory parameters, vital signs, ECG through End of Study visit every 12 weeks.
About 100 weeks
Secondary Outcomes (5)
Proportion of participants achieving sustained hemoglobin levels ≥ 120 g/L in the absence of red blood cell transfusions
About 100 weeks
Change From Baseline in Hemoglobin
About 100 weeks
The proportion of patients without RBC transfusion.
About 100 weeks
The Clinical BTH Rate
About 100 weeks
The Major Adverse Vascular Events Rate
About 100 weeks
Study Arms (1)
MY008211A tablets
EXPERIMENTALMY008211A tablets 400mg BID
Interventions
Participants will receive MY008211A at a dose of 400 mg orally b.i.d
Eligibility Criteria
You may qualify if:
- 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.
- Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections.
You may not qualify if:
- History of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.
- Known or suspected hereditary complement deficiency.
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Chinese Academy of Medical Sciences Hematology Hospital (Institute of Hematology, Chinese Academy of Medical Sciences)
Tianjin, Tianjin Municipality, 300020, China
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 11, 2025
First Posted
April 18, 2025
Study Start
November 30, 2024
Primary Completion (Estimated)
December 30, 2026
Study Completion (Estimated)
December 30, 2026
Last Updated
April 18, 2025
Record last verified: 2025-04