Long-term safety and tolerability of HRS-5965 for patients with Paroxysmal Nocturnal Hemoglobinuria

An Open Label, Multicenter Study to Evaluate the Long-term Safety and Tolerability of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria

This study is testing if taking HRS-5965 for a longer time can be safe and helpful for people with Paroxysmal Nocturnal Hemoglobinuria who have already used this treatment before.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 120 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Chengdu Suncadia Medicine Co., Ltd. (Industry-sponsored)
• Drugs / interventions: eculizumab
• Locations: 2 sites (Beijing, Beijing and 1 other locations)
• Trial ID: NCT06238544 on ClinicalTrials.gov

What this trial studies

This multicenter, single-arm, open-label study evaluates the long-term safety and tolerability of HRS-5965 in patients with Paroxysmal Nocturnal Hemoglobinuria who have previously completed treatment with HRS-5965. All eligible participants will receive HRS-5965 in tablet or capsule form until the end of the treatment period. The study aims to determine if continued treatment provides additional benefits to these patients.

Who should consider this trial

Good fit: Ideal candidates are patients who have previously completed HRS-5965 treatment and are deemed by investigators to potentially benefit from continued treatment.

Not a fit: Patients with known or suspected complement deficiencies or a history of infections with capsular bacteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a safe and effective long-term treatment option for patients with Paroxysmal Nocturnal Hemoglobinuria.

How similar studies have performed: While this approach is focused on long-term treatment, similar studies have shown promise in managing Paroxysmal Nocturnal Hemoglobinuria, indicating potential for success.

Eligibility criteria

Inclusion Criteria:

1. Patients who have previously received and completed HRS-5965 study treatment, and are judged by the investigator to have treatment benefit and may benefit from continued treatment of HRS-5965 and patients in the control group of who received eculizumab treatment.

Exclusion Criteria:

1. Known or suspected hereditary or acquired complement deficiency;
2. History of infection with capsular bacteria (e.g., meningococcus, pneumococcus, etc.)

Where this trial is running

Beijing, Beijing and 1 other locations

• Peking Union Medical College Hospital, Chinese Academy of Medical Sciences — Beijing, Beijing, China (Recruiting)
• The Blood Disease Hospital of the Chinese Academy of Medical Sciences — Tianjin, Tianjin, China (Recruiting)

Study contacts

• Study coordinator: Sheng Qi

Conditions Paroxysmal Nocturnal Hemoglobinuria