Actively Recruiting
An Open Label, Multicenter Study to Evaluate the Long-term Safety and Tolerability of MY008211A Tablets in Patients With PNH Paroxysmal Nocturnal Hemoglobinuria (PNH)
Led by Wuhan Createrna Science and Technology Co., Ltd · Updated on 2025-04-18
120
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to evaluate the long-term safety, tolerability, and effectiveness of MY008211A tablets in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). It provides continued access to MY008211A for patients who have previously completed Phase 2 or Phase 3 trials of the drug and have derived benefit from it. This open-label, single-arm, multicenter study focuses on understanding the ongoing impact of MY008211A treatment in this patient group. Participants in this study will take MY008211A tablets at a dose of 400 mg twice daily. The study allows patients who completed earlier trials without tapering down their treatment to continue receiving MY008211A. This single treatment group design means all participants receive the study drug openly without a placebo or comparator. During the study, participants will be monitored for adverse events, laboratory safety parameters, vital signs, and ECG over about 100 weeks. Researchers will assess outcomes such as sustained hemoglobin levels without transfusions, changes in hemoglobin from baseline, rates of red blood cell transfusion avoidance, clinical breakthrough hemolysis, and major adverse vascular events. The study provides long-term safety and efficacy information while ensuring ongoing treatment access for eligible patients.
CONDITIONS
Brief Title
Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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 will not qualify if you...
- 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.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - About 100 weeks
Participants receive MY008211A tablets orally twice daily to evaluate long-term safety, tolerability, and efficacy.
Trial Site Locations
Total: 1 location
1
Chinese Academy of Medical Sciences Hematology Hospital (Institute of Hematology, Chinese Academy of Medical Sciences)
Tianjin, Tianjin Municipality, China, 300020
Actively Recruiting
Research Team
W
Wuhan Createrna Science and Technology Co.,Ltd
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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