Paroxysmal Nocturnal Hemoglobinuria
Conditions
Brief summary
The main purpose of this study is to evaluate the efficacy of MY008211A in PNH patients with residual anemia despite treatment with anti-C5 antibody.
Detailed description
This is a multicenter, single-arm, open-label phase III clinical trial. The purpose of this study is to determine whether MY008211A is efficacious and safe for the treatment of PNH patients Who Are Still Anemia After Anti-C5 Antibody Treatment.
Interventions
MY008211A tablets 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 75 Years
Healthy volunteers
No
Inclusion criteria
1. Male and female participants ≥ 18 years of age and BMI ≥ 18.0 kg/m2 with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with clone size ≥ 10%. 2. Stable regimen of anti-C5 antibody treatment for at least 6 months before treatment, and Hb was still \< 100 g/L. 3. The average hemoglobin level of at least two tests in 4 months before screening \< 100 g/L. 4. The average hemoglobin level of two tests in the central laboratory during screening \< 100 g/L. 5. Vaccination against Neisseria meningitidis infection is required prior to the start of study treatment. If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given.
Exclusion criteria
1. Patients with reticulocytes \<100x10\^9/L; platelets \<30x10\^9/L; neutrophils \<0.5x10\^9/L. 2. History of recurrent invasive infections caused by encapsulated organisms,e.g. meningococcus or pneumococcus. 3. Known or suspected hereditary complement deficiency. 4. Previous bone marrow or hematopoietic stem cell transplantation. 5. Previous splenectomy. 6. A history of malignancy within 5 years before screening, except cured local basal cell carcinoma of the skin and carcinoma in situ of the cervix.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The proportion of Participants With Sustained Hemoglobin Levels of ≥ 120 g/L in the Absence of Red Blood Cell Transfusions (defined as no red blood cell infusion after D14 to D168) | between Day 126 and Day 168 | The proportion of patients with sustained hemoglobin levels ≥ 120 g/L among those without RBC transfusion. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Hemoglobin | between Day 126 and Day 168 | Change in hemoglobin concentration from baseline in patients without RBC transfusion. |
| The proportion of patients with LDH < 1.5 ULN among those without RBC transfusion. | between Day 126 and Day 168 | The proportion of patients with hemolysis controlled (defined as LDH \< 1.5 ULN) among those without RBC transfusion. |
| Change (Expressed as Percentages) in LDH level from baseline | between Day 126 and Day 168 | Change (Expressed as Percentages) in LDH level from baseline |
| Change in reticulocyte count from baseline | between Day 126 and Day 168 | Change in reticulocyte count from baseline |
| The proportion of subjects with an increase in hemoglobin concentration ≥ 20 g/L from baseline among subjects who do not receive RBC transfusion (defined as no red blood cell infusion after D14 to D168) | between Day 126 and Day 168 | Proportion of participants achieving a sustained increase from baseline in hemoglobin levels of ≥ 20 g/L assessed among those without RBC transfusion. |
| Change in FACIT-F score from baseline | between Day 126 and Day 168 | Change in FACIT-Fatigue score from baseline. The FACIT-Fatigue is a 13-item questionnaire with support for its validity and reliability in PNH that assesses patient self-reported fatigue and its impact on daily activities and function. All FACIT scales are scored so that a high score is better. As each of the 13 items of the FACIT-F Scale ranges from 0-4, the range of possible scores is 0-52, with 0 being the worst possible score and 52 the best. |
| The Clinical BTH Rate | between Day 1 and Day 168 | The Clinical BTH Rate |
| The Major Adverse Vascular Events Rate | between Day 1 and Day 168 | The Major Adverse Vascular Events Rate |
| The proportion of patients without RBC transfusion | between Day 14 and Day 168 | The proportion of patients without RBC transfusion. |
Countries
China
Contacts
Primary ContactWuhan Createrna Science and Technology Co.,Ltd
Outcome results
None listed