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

NCT06050226 | Completed Phase 2

• 1 other identifier: MY008211A-PNH-2-01
• Study Type: interventional
• Target: 34
• Locations: 1 country
• Sites: 1

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.

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (7)

• Proportion of participants achieving sustained hemoglobin levels ≥ 120 g/L in the absence of red blood cell transfusions.

  up to 84 days

• Change from baseline in hemoglobin concentration.

  up to 84 days

• Change from baseline in serum LDH levels.

  up to 84 days

• Change from baseline in Reticulocyte count.

  up to 84 days

• Changes from baseline in transfusion volume.

  up to 84 days

Other Outcomes (4)

• Changes from baseline in alternative complement pathway activity.

  up to 84 days

• Change from baseline in plasma levels of the Bb fragment.

  up to 84 days

• Maximum Plasma Concentration (Cmax) Of MY008211A tablets

  up to 84 days

Study Arms (2)

Arm1：low MY008211A dose

EXPERIMENTAL

Participants will receive low MY008211A dose orally b.i.d

Drug: MY008211A tablets

Arm2：high MY008211A dose

EXPERIMENTAL

Participants will receive high MY008211A dose orally b.i.d

Drug: MY008211A tablets

Interventions

MY008211A tablets DRUG

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.

Also known as: MY008211A

Arm1：low MY008211A dose; Arm2：high MY008211A 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, 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.

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

Study Sites (1)

Institute of Hematology & Blood Disease Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College

Tianjin, Tianjin Municipality, China

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

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

Study Officials

• Fengkui Zhang, Ph.D

  Blood Disease Hospital, Chinese Academy of Medical Sciences

  PRINCIPAL INVESTIGATOR

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: September 11, 2023
• First Posted: September 22, 2023
• Study Start: July 6, 2023
• Primary Completion: April 30, 2024
• Study Completion: November 28, 2024
• Last Updated: July 30, 2025

Record last verified: 2023-09

Locations

CN (1)