Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Phase 2Not Yet RecruitingNCT06134414

Wuhan Createrna Science and Technology Co., Ltd40 enrolled

Overview

The main purpose of this study is to evaluate the efficacy of MY008211A in adult patients with PNH, showing signs of active hemolysis.

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Interventions

MY008211A tabletsDRUG

dose 1 (400 mg BID) and dose 2 (600 mg BID) in a 1:1 ratio by central randomization

Eligibility

Sex: ALLMin age: 18 YearsMax age: 75 Years

Medical Language ↔ Plain English

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.

Outcomes

Primary Outcomes

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

Proportion of participants achieving a sustained increase from baseline in hemoglobin levels of ≥ 20 g/L assessed , in the absence of red blood cell transfusions

Time frame: Day 70

Secondary Outcomes

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

The proportion of patients with hemolysis controlled (defined as LDH \< 1.5 ULN)

Time frame: Day7, 14, 21, 28, 42, 56 and 70

Change in reticulocyte count from baseline in patients without RBC transfusion

Central Contacts

Xiaoni Li, Ph.D

CONTACT

021-51158605 lixiaoni@createrna.com

Data from ClinicalTrials.gov

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