Paroxysmal Nocturnal Hemoglobinuria (PNH)
Conditions
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.
Interventions
Participants will receive MY008211A at a dose of 400 mg orally b.i.d
Sponsors
Wuhan Createrna Science and Technology Co., Ltd
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Countries
China
Contacts
Primary ContactWuhan Createrna Science and Technology Co.,Ltd
Outcome results
None listed