Clinical trial NCT06932744: Study of Safety and Efficacy of MY008211A in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy
Official title: A Randomized, Multicenter, Active-comparator Controlled, Open-label Trial to Evaluate Efficacy and Safety of MY008211A Tablets in PNH Patients Who Are Naive to Complement Inhibitor Therapy
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Paroxysmal Nocturnal Haemoglobinuria (PNH)
Lead sponsor: Wuhan Createrna Science and Technology Co., Ltd
Sponsor class: INDUSTRY
First posted: 2025-04-17
Enrollment: 66
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 compared to eculizumab in PNH patients.

Detailed description: This is a multi-center, randomized, open-label, active comparator-controlled, parallel group study. The purpose of this study is to determine whether MY008211A compared to eculizumab is efficacious and safe for the treatment of PNH patients who were naive to complement inhibitor therapy.

Eligibility criteria: 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. Mean hemoglobin level \<100 g/L at screening.
3. LDH \> 1.5 x Upper Limit of Normal (ULN) at screening.
4. 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.

Primary outcomes:
measure: The proportion of Participants With Sustained Hemoglobin Levels of ≥ 120 g/L in the Absence of Red Blood Cell Transfusions
timeFrame: between Day 126 and Day 168
Secondary 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
timeFrame: between Day 126 and Day 168
measure: The proportion of patients with LDH < 1.5 ULN among those without RBC transfusion.
timeFrame: between Day 126 and Day 168
measure: Change (Expressed as Percentages) in LDH level from baseline
timeFrame: between Day 126 and Day 168
measure: Change in reticulocyte count from baseline
timeFrame: between Day 126 and Day 168
measure: The proportion of patients without RBC transfusion
timeFrame: between Day 14 and Day 168
measure: Change in FACIT-F score from baseline
timeFrame: between Day 126 and Day 168
measure: The Clinical BTH Rate
timeFrame: between Day 1 and Day 168
measure: The Major Adverse Vascular Events Rate
timeFrame: between Day 1 and Day 168
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
state: Beijing
city: Tianjin
country: China
facility: Chinese Academy of Medical Sciences Hematology Hospital
state: Tianjin
Study officials:
References: