Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria

An Open Label, Multicenter Study to Evaluate the Long-term Safety and Tolerability of MY008211A Tablets in Patients With PNH Paroxysmal Nocturnal Hemoglobinuria (PNH)

This is a multicenter, single-arm, open-label study to characterize long-term safety and tolerability of MY008211A tablets and to provide access to MY008211A tablets to patients with PNH who have completed Phase 2 or 3 studies with MY008211A tablets.

Study Overview

• Status: Recruiting
• Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Intervention / Treatment: Drug: MY008211A tablets

Detailed Description

The purpose of this open-label, single arm, multicenter study is to evaluate the long-term safety, tolerability and efficacy of MY008211A tablets in patients with PNH and to provide access to patients who have completed (without tapering down) Phase 2 and Phase 3 trials and derived benefit from MY008211A treatment.

• Study Type: Interventional
• Enrollment (Estimated): 120
• Phase: Phase 2; Phase 3

Contacts and Locations

• Study Contact: Name: Wuhan Createrna Science and Technology Co.,Ltd; Phone Number: 027-68788900; Email: lcyxzx@createrna.com

Study Locations

• China
  • Tianjin
    • Tianjin, Tianjin, China, 300020
      • Recruiting
      • Chinese Academy of Medical Sciences Hematology Hospital (Institute of Hematology, Chinese Academy of Medical Sciences)
      • Contact: Fengkui Zhang, PhD; Phone Number: 022-23909095

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Patients who have previously received and completed MY008211A study treatment, and are judged by the investigator to have treatment benefit and may benefit from continued treatment of MY008211A.
2. Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections.

Exclusion Criteria:

1. History of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.
2. Known or suspected hereditary complement deficiency.
3. Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the investigator, could put the subject at increased risk or potentially confound study data.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: MY008211A tablets; MY008211A tablets 400mg BID	Drug: MY008211A tablets; Participants will receive MY008211A at a dose of 400 mg orally b.i.d

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of participants with adverse events, safety laboratory parameters, vital signs, ECG.	Safety evaluations including but not limited to adverse events, laboratory parameters, vital signs, ECG through End of Study visit every 12 weeks.	About 100 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 the absence of red blood cell transfusions evaluated every 12 weeks.	About 100 weeks
Change From Baseline in Hemoglobin	Change in hemoglobin concentration from baseline in patients without RBC transfusion every 12 weeks.	About 100 weeks
The proportion of patients without RBC transfusion.	The proportion of patients without RBC transfusion.	About 100 weeks
The Clinical BTH Rate	The Clinical BTH Rate	About 100 weeks
The Major Adverse Vascular Events Rate	The Major Adverse Vascular Events Rate	About 100 weeks

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): November 30, 2024
• Primary Completion (Estimated): December 30, 2026
• Study Completion (Estimated): December 30, 2026

Study Registration Dates

• First Submitted: April 11, 2025
• First Submitted That Met QC Criteria: April 11, 2025
• First Posted (Actual): April 18, 2025

Study Record Updates

• Last Update Posted (Actual): April 18, 2025
• Last Update Submitted That Met QC Criteria: April 11, 2025
• Last Verified: April 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urogenital Diseases; Male Urogenital Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urination Disorders; Urological Manifestations; Hematologic Diseases; Bone Marrow Diseases; Anemia, Hemolytic; Anemia; Myelodysplastic Syndromes; Proteinuria; Hemoglobinuria; Hemoglobinuria, Paroxysmal
• Other Study ID Numbers: MY008211A-PNH-2-03

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No