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

NCT ID: NCT06933914

Last Updated: 2025-04-18

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 120 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-11-30
• Study Completion Date: 2026-12-30

Brief Summary

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.

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.

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

MY008211A tablets

MY008211A tablets 400mg BID

Group Type: EXPERIMENTAL

MY008211A tablets

Intervention Type: DRUG

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

Interventions

MY008211A tablets

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

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Wuhan Createrna Science and Technology Co., Ltd

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Chinese Academy of Medical Sciences Hematology Hospital (Institute of Hematology, Chinese Academy of Medical Sciences)

Tianjin, Tianjin Municipality, China

Site Status: RECRUITING

Countries

China

Central Contacts

Wuhan Createrna Science and Technology Co.,Ltd

Role: CONTACT

lcyxzx@createrna.com

027-68788900

Facility Contacts

Fengkui Zhang, PhD

Role: primary

022-23909095

Other Identifiers

MY008211A-PNH-2-03

Identifier Type: -

Identifier Source: org_study_id