Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: MY008211A tablets.
- Who it may be relevant to
- Registry conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH). Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official 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)
Overview
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.
Interventions
- Drug MY008211A tablets
Participants will receive MY008211A at a dose of 400 mg orally b.i.d
Primary outcome measures
- Proportion of participants with adverse events, safety laboratory parameters, vital signs, ECG. [Time frame: About 100 weeks]
Secondary outcome measures (5)
- Proportion of participants achieving sustained hemoglobin levels ≥ 120 g/L in the absence of red blood cell transfusions [Time frame: About 100 weeks]
- Change From Baseline in Hemoglobin [Time frame: About 100 weeks]
- The proportion of patients without RBC transfusion. [Time frame: About 100 weeks]
- The Clinical BTH Rate [Time frame: About 100 weeks]
- The Major Adverse Vascular Events Rate [Time frame: About 100 weeks]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 1 center
- Chinese Academy of Medical Sciences Hematology Hospital (Institute of Hematology, Chinese — Tianjin
Identifiers
NCT: NCT06933914 · MY008211A-PNH-2-03