Chinese Academy of Medical Sciences Hematology Hospital (Institute of Hematology, Chinese Academy of Medical Sciences)
Tianjin, Tianjin Municipality, 300020, China
Location status: Recruiting
Recruiting
NCT Number: NCT06933914
Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria
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.
Recruiting
Interested in participating?
Request InfoKey information
Conditions
Age range
18 year and older
Sex eligibility
All sexes
Study type
Interventional
Phase
Phase 2 / Phase 3
Primary location
About this study
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.
Who can participate
Healthy volunteers accepted: No
Only the study team can determine whether someone qualifies for participation.
Inclusion criteria
- 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.
Exclusion criteria
- 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.
Treatment and study plan
MY008211A tablets
DrugParticipants will receive MY008211A at a dose of 400 mg orally b.i.d
Primary outcomes
-
Proportion of participants with adverse events, safety laboratory parameters, vital signs, ECG.
Time frame: 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 outcomes
-
Proportion of participants achieving sustained hemoglobin levels ≥ 120 g/L in the absence of red blood cell transfusions
Time frame: 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
Time frame: About 100 weeks
Change in hemoglobin concentration from baseline in patients without RBC transfusion every 12 weeks.
-
The proportion of patients without RBC transfusion.
Time frame: About 100 weeks
The proportion of patients without RBC transfusion.
-
The Clinical BTH Rate
Time frame: About 100 weeks
The Clinical BTH Rate
-
The Major Adverse Vascular Events Rate
Time frame: About 100 weeks
The Major Adverse Vascular Events Rate
Study contacts
Contact information is provided by the study sponsor or research team.
Sponsors and collaborators
Lead sponsor
Wuhan Createrna Science and Technology Co., Ltd
Industry
Registry information
Official study title
An Open Label, Multicenter Study to Evaluate the Long-term Safety and Tolerability of MY008211A Tablets in Patients With PNH Paroxysmal Nocturnal Hemoglobinuria (PNH)
Important dates
- Study start
- 2024
- Primary completion
- 2026
- Study completion
- 2026
- First posted
- Apr 18, 2025
- Registry last updated
- Apr 18, 2025
OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.
View the official ClinicalTrials.gov record (opens in a new tab)This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.
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