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Clinical Trial Details — Status: Not yet recruiting

Administrative data

NCT number NCT06134414
Other study ID # MY008211A-PNH-2-01-F
Secondary ID
Status Not yet recruiting
Phase Phase 2
First received
Last updated
Start date December 2024
Est. completion date December 2025

Study information

Verified date October 2023
Source Wuhan Createrna Science and Technology Co., Ltd
Contact Xiaoni Li, Ph.D
Phone 021-51158605
Email lixiaoni@createrna.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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


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.


Recruitment information / eligibility

Status Not yet recruiting
Enrollment 40
Est. completion date December 2025
Est. primary completion date July 2025
Accepts healthy volunteers No
Gender All
Age group 18 Years to 75 Years
Eligibility 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.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
MY008211A tablets
dose 1 (400 mg BID) and dose 2 (600 mg BID) in a 1:1 ratio by central randomization

Locations

Country Name City State
n/a

Sponsors (1)

Lead Sponsor Collaborator
Wuhan Createrna Science and Technology Co., Ltd

Outcome

Type Measure Description Time frame Safety issue
Primary 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 Day 70
Secondary The proportion of patients with an increase in hemoglobin = 20 g/L from baseline among those without RBC transfusion The proportion of patients with an increase in hemoglobin = 20 g/L from baseline among those without RBC transfusion Day14, 21, 28, 42, 56
Secondary The proportion of patients with hemoglobin = 120 g/L among those without RBC transfusion The proportion of patients with hemoglobin = 120 g/L among those without RBC transfusion Day14, 21, 28, 42, 56 and 70
Secondary Change in hemoglobin concentration from baseline in patients without RBC transfusion Change in hemoglobin concentration from baseline in patients without RBC transfusion Day14, 21, 28, 42, 56 and 70
Secondary Change in LDH level from baseline Change in LDH level from baseline Day7, 14, 21, 28, 42, 56 and 70
Secondary The proportion of patients with hemolysis controlled The proportion of patients with hemolysis controlled (defined as LDH < 1.5 ULN) Day7, 14, 21, 28, 42, 56 and 70
Secondary Change in reticulocyte count from baseline in patients without RBC transfusion Change in reticulocyte count from baseline in patients without RBC transfusion Day7, 14, 21, 28, 42, 56 and 70
Secondary Change in indirect bilirubin level from baseline Change in indirect bilirubin level from baseline Day7, 14, 21, 28, 42, 56 and 70
Secondary The proportion of patients without RBC transfusion The proportion of patients without RBC transfusion Day14, 21, 28, 42, 56 and 70
Secondary Change in the average weekly amount of RBC transfused during the efficacy observation period Change in the average weekly amount of RBC transfused during the efficacy observation period compared with that pre-dose Day70
Secondary Change From Baseline in FACIT-Fatigue Questionnaire Change from baseline in FACIT-Fatigue scores. The FACIT-Fatigue is a 13-item questionnaire with support for its validity and reliability in PNH that assesses patient self-reported fatigue and its impact on daily activities and function. All FACIT scales are scored so that a high score is better. As each of the 13 items of the FACIT-F Scale ranges from 0-4, the range of possible scores is 0-52, with 0 being the worst possible score and 52 the best. Day7, 14, 21, 28, 42, 56 and 70
Secondary Changes from baseline in alternative complement pathway activity Alternative complement pathway activity measured by the WIESLAB® kit. Day14, 28, 56 and 70
Secondary Change in the amount of fragment Bb of CFB in plasma from baseline Bb fragment cleaved by factor B of complement. Day14, 28, 56 and 70
Secondary Change in the level of PNH RBC clones from baseline in patients without RBC transfusion. Change from baseline in the level of PNH red cell clones. Day70
Secondary Incidence of Adverse Events (AEs) between Day 1 and Day 70 Adverse Events (AEs) Day 70
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