Clinical trial NCT06134414: Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
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
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE2
Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH)
Lead sponsor: Wuhan Createrna Science and Technology Co., Ltd
Sponsor class: INDUSTRY
First posted: 2023-11-18
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.

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.

Primary outcomes:
measure: 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
timeFrame: Day 70
Secondary outcomes:
measure: The proportion of patients with an increase in hemoglobin ≥ 20 g/L from baseline among those without RBC transfusion
timeFrame: Day14, 21, 28, 42, 56
measure: The proportion of patients with hemoglobin ≥ 120 g/L among those without RBC transfusion
timeFrame: Day14, 21, 28, 42, 56 and 70
measure: Change in hemoglobin concentration from baseline in patients without RBC transfusion
timeFrame: Day14, 21, 28, 42, 56 and 70
measure: Change in LDH level from baseline
timeFrame: Day7, 14, 21, 28, 42, 56 and 70
measure: The proportion of patients with hemolysis controlled
timeFrame: Day7, 14, 21, 28, 42, 56 and 70
measure: Change in reticulocyte count from baseline in patients without RBC transfusion
timeFrame: Day7, 14, 21, 28, 42, 56 and 70
measure: Change in indirect bilirubin level from baseline
timeFrame: Day7, 14, 21, 28, 42, 56 and 70
measure: The proportion of patients without RBC transfusion
timeFrame: Day14, 21, 28, 42, 56 and 70
measure: Change in the average weekly amount of RBC transfused during the efficacy observation period
timeFrame: Day70
measure: Change From Baseline in FACIT-Fatigue Questionnaire
timeFrame: Day7, 14, 21, 28, 42, 56 and 70
measure: Changes from baseline in alternative complement pathway activity
timeFrame: Day14, 28, 56 and 70
measure: Change in the amount of fragment Bb of CFB in plasma from baseline
timeFrame: Day14, 28, 56 and 70
measure: Change in the level of PNH RBC clones from baseline in patients without RBC transfusion.
timeFrame: Day70
measure: Incidence of Adverse Events (AEs) between Day 1 and Day 70
timeFrame: Day 70
Other outcomes:
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