A Study of MY008211A in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal Nocturnal Hemoglobinuria Clinical Trial

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.

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06050226
• Other study ID #: MY008211A-PNH-2-01
• Status: Recruiting
• Phase: Phase 2
• Start date: July 6, 2023
• Est. completion date: July 30, 2024

Study information

• Verified date: September 2023
• Source: Wuhan Createrna Science and Technology Co., Ltd
• Contact: Zhen Fu, Ph.D
• Phone: 13477072640
• Email: fuzhen[@]createrna.com
• Is FDA regulated: No
• 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, in China.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 40
• Est. completion date: July 30, 2024
• Est. primary completion date: January 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

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.

Related Conditions & MeSH terms

• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Paroxysmal Nocturnal Hemoglobinuria

Intervention

Drug:
• MY008211A tablets
The first 10 participants will be received low-dose MY008211A tablets, and the next 30 participants will be randomized to low-dose or high-dose treatment arms in a 1:2 ratio.

Locations

Country	Name	City	State
China	Nstitute of Hematology & Blood Disease Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College	Tianjin	Tianjin

Sponsors (1)

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

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Changes from baseline in alternative complement pathway activity.	Alternative complement pathway activity measured by the WIESLAB® kit.	up to 84 days
Other	Change from baseline in plasma levels of the Bb fragment.	Bb fragment cleaved by factor B of complement.	up to 84 days
Other	Maximum Plasma Concentration (Cmax) Of MY008211A tablets	PK parameters	up to 84 days
Other	Area Under The Concentration Versus Time Curve (AUC) Of MY008211A	PK parameters	up to 84 days
Primary	Proportion of participants achieving a sustained increase in hemoglobin levels of = 20 g/L in the absence of red blood cell transfusion.	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	up to 84 days
Secondary	Proportion of participants achieving sustained hemoglobin levels = 120 g/L in the absence of red blood cell transfusions.	Proportion of participants achieving sustained hemoglobin levels = 120 g/L in absence of red blood cell transfusion	up to 84 days
Secondary	Change from baseline in hemoglobin concentration.	Change from baseline in hemoglobin concentration (g/L) in absence of red blood cell transfusion	up to 84 days
Secondary	Change from baseline in serum LDH levels.	Change from baseline in serum LDH levels (U/L)	up to 84 days
Secondary	Change from baseline in Reticulocyte count.	Change from baseline in Reticulocyte count (×10^9/L)	up to 84 days
Secondary	Changes from baseline in transfusion volume.	The average number of red blood cells transfused per week	up to 84 days
Secondary	Change in the level of PNH red cell clones.	Change from baseline in the level of PNH red cell clones.	up to 84 days
Secondary	Occurrences of AEs occurring between Day 1 and Day 84.	Adverse Events (AEs)	up to 84 days