Long-term Safety and Tolerability of HRS-5965 in 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

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT06238544

Enrollment

132

Registered

2024-02-02

Start date

2024-02-06

Completion date

2027-12-31

Last updated

2025-12-24

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Brief summary

This is a multicenter, single-arm, open-label study. Patients with Paroxysmal Nocturnal Hemoglobinuria who had previously received and completed the HRS-5965 study well included. All eligible subjects received HRS-5965 tablets or capsules until the end of treatment in this study.

Interventions

DRUGHRS-5965 tablets

HRS-5965 tablets until the end of study treatment

DRUGHRS-5965 Capsules

HRS-5965 capsules.

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

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.)

Design outcomes

Primary

Measure	Time frame
Incidence and severity of adverse events.	Every six months,for about 3 years

Secondary

Measure	Time frame
Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions was assessed every half a year during treatment..	Every six months ,for about 3 years
Proportion of participants who remain free from transfusions was assessed every half a year during treatment..	Every six months,for about 3 years
Rate of breakthrough hemolysis was assessed every half a year during treatment	Every six months,for about 3 years
Proportion of participants with Major Adverse Vascular Events MAVEs.	Every six months,for about 3 years
Plasma concentration of HRS-5965.	Start of Treatment to end of study，for about 3 years

Countries

China

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026