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

NCT06134414 | Not Yet Recruiting Phase 2

• 1 other identifier: MY008211A-PNH-2-01-F
• Study Type: interventional
• Target: 40
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (14)

• The proportion of patients with an increase in hemoglobin ≥ 20 g/L from baseline among those without RBC transfusion

  Day14, 21, 28, 42, 56

• The proportion of patients with hemoglobin ≥ 120 g/L among those without RBC transfusion

  Day14, 21, 28, 42, 56 and 70

• Change in hemoglobin concentration from baseline in patients without RBC transfusion

  Day14, 21, 28, 42, 56 and 70

• Change in LDH level from baseline

  Day7, 14, 21, 28, 42, 56 and 70

• The proportion of patients with hemolysis controlled

  Day7, 14, 21, 28, 42, 56 and 70

Study Arms (2)

Arm 1 MY008211A low dose

EXPERIMENTAL

Participants will receive MY008211A at a dose of 400 mg orally b.i.d

Drug: MY008211A tablets

Arm 2 MY008211A high dose

EXPERIMENTAL

Participants will receive MY008211A at a dose of 600 mg orally b.i.d

Drug: MY008211A tablets

Interventions

MY008211A tablets DRUG

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

Also known as: MY008211A

Arm 1 MY008211A low dose; Arm 2 MY008211A high dose

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Wuhan Createrna Science and Technology Co., Ltd: lead

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Central Study Contacts

Xiaoni Li, Ph.D

CONTACT

021-51158605; lixiaoni@createrna.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 10, 2023
• First Posted: November 18, 2023
• Study Start: December 1, 2025
• Primary Completion (Estimated): July 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: July 29, 2025

Record last verified: 2024-10