Clinical trial NCT06050226: A Study of MY008211A in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Official title: A Multi-center, Randomized, Open-label, Phase 2 Study to Evaluate the Efficacy and Safety of MY008211A Tablets in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria and Active Hemolysis.
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Paroxysmal Nocturnal Hemoglobinuria
Lead sponsor: Wuhan Createrna Science and Technology Co., Ltd
Sponsor class: INDUSTRY
First posted: 2023-09-22
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, in China.

Detailed description: The purpose of this study is to determine whether MY008211A is efficacious and safe for the treatment of PNH patients who are naive to complement inhibitor therapy, including anti-C5 antibody.

Eligibility criteria: Inclusion Criteria:

* Male and female participants ≥ 18 years of age, BMI≥18 kg/m2，with a diagnosis of PNH confirmed by laboratory tests, according to the PNH diagnostic criteria in the Chinese Guidelines for the Diagnosis and Treatment of Rare Diseases (2019 edition) , and 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: Proportion of participants achieving a sustained increase in hemoglobin levels of ≥ 20 g/L in the absence of red blood cell transfusion.
timeFrame: up to 84 days
Secondary outcomes:
measure: Proportion of participants achieving sustained hemoglobin levels ≥ 120 g/L in the absence of red blood cell transfusions.
timeFrame: up to 84 days
measure: Change from baseline in hemoglobin concentration.
timeFrame: up to 84 days
measure: Change from baseline in serum LDH levels.
timeFrame: up to 84 days
measure: Change from baseline in Reticulocyte count.
timeFrame: up to 84 days
measure: Changes from baseline in transfusion volume.
timeFrame: up to 84 days
measure: Change in the level of PNH red cell clones.
timeFrame: up to 84 days
measure: Occurrences of AEs occurring between Day 1 and Day 84.
timeFrame: up to 84 days
Other outcomes:
measure: Changes from baseline in alternative complement pathway activity.
timeFrame: up to 84 days
measure: Change from baseline in plasma levels of the Bb fragment.
timeFrame: up to 84 days
measure: Maximum Plasma Concentration (Cmax) Of MY008211A tablets
timeFrame: up to 84 days
measure: Area Under The Concentration Versus Time Curve (AUC) Of MY008211A
timeFrame: up to 84 days
Locations:
city: Tianjin
country: China
facility: Nstitute of Hematology & Blood Disease Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College
state: Tianjin
Study officials:
affiliation: Blood Disease Hospital, Chinese Academy of Medical Sciences
name: Fengkui Zhang, Ph.D
role: PRINCIPAL_INVESTIGATOR
References: