Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
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: 18 years — 75 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
- Center list to be confirmed — check the primary protocol.
- 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
A Multi-center, Randomized, Parallel, Open-label Clinical Phase II Study, to Evaluate the Efficacy and Safety of MY008211A in Adult Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients With Signs of Active Hemolysis
Overview
The main purpose of this study is to evaluate the efficacy of MY008211A in adult patients with PNH, showing signs of active hemolysis.
Detailed description
The purpose of this study is to determine whether MY008211A is efficacious and safe for the treatment of PNH patients who are naïve to complement inhibitor therapy, including anti-C5 antibody.
Interventions
- Drug MY008211A tablets
dose 1 (400 mg BID) and dose 2 (600 mg BID) in a 1:1 ratio by central randomization
Primary outcome measures
- The proportion of subjects with an increase in hemoglobin concentration ≥ 20 g/L from baseline among subjects who do not receive RBC transfusion after 4 weeks of dosing [Time frame: Day 70]
Secondary outcome measures (12)
- The proportion of patients with an increase in hemoglobin ≥ 20 g/L from baseline among those without RBC transfusion [Time frame: Day14, 21, 28, 42, 56]
- The proportion of patients with hemoglobin ≥ 120 g/L among those without RBC transfusion [Time frame: Day14, 21, 28, 42, 56 and 70]
- Change in hemoglobin concentration from baseline in patients without RBC transfusion [Time frame: Day14, 21, 28, 42, 56 and 70]
- Change in LDH level from baseline [Time frame: Day7, 14, 21, 28, 42, 56 and 70]
- The proportion of patients with hemolysis controlled [Time frame: Day7, 14, 21, 28, 42, 56 and 70]
- Change in reticulocyte count from baseline in patients without RBC transfusion [Time frame: Day7, 14, 21, 28, 42, 56 and 70]
- Change in indirect bilirubin level from baseline [Time frame: Day7, 14, 21, 28, 42, 56 and 70]
- The proportion of patients without RBC transfusion [Time frame: Day14, 21, 28, 42, 56 and 70]
- Change in the average weekly amount of RBC transfused during the efficacy observation period [Time frame: Day70]
- Change From Baseline in FACIT-Fatigue Questionnaire [Time frame: Day7, 14, 21, 28, 42, 56 and 70]
- Changes from baseline in alternative complement pathway activity [Time frame: Day14, 28, 56 and 70]
- Change in the amount of fragment Bb of CFB in plasma from baseline [Time frame: Day14, 28, 56 and 70]
Eligibility criteria
Inclusion criteria
- 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%.
- Mean hemoglobin level <100 g/L.
- LDH > 1.5 x Upper Limit of Normal (ULN).
- 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
- Patients with reticulocytes <100x10\^9/L; platelets <30x10\^9/L; neutrophils <0.5x10\^9/L.
- Were using a complement inhibitor before the first administration of MY008211A tablets or had discontinued a previous complement inhibitor for less than five half-lives or 120 days, whichever was the longest.
- History of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.
- Known or suspected hereditary complement deficiency.
- Previous bone marrow or hematopoietic stem cell transplantation.
- Previous splenectomy.
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Center list to be confirmed — check the primary protocol.
Identifiers
NCT: NCT06134414 · MY008211A-PNH-2-01-F