A Study to Evaluate the Efficacy and Safety of Oral HRS-5965 in Adult Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy

Chengdu Suncadia Medicine

Status and phase: Active, not recruiting; Phase 3

Conditions: Paroxysmal Nocturnal Hemoglobinuria

Treatments: Drug: Eculizumab Injection; Drug: HRS-5965 capsule

Study type: Interventional

Funder types: Industry

Identifiers

NCT06593938

HRS-5965-301

Details and patient eligibility

About

A study of the efficacy and safety of HRS-5965 capsules compared to eculizumab for 24 weeks in patients with PNH.

Enrollment: 76 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Diagnosis of PNH confirmed by flow cytometry with clone size > 10%.
2. Have not received complement inhibitor therapy;
3. LDH > 1.5*ULN at screening.
4. Hemoglobin level < 10 g/dL at screening.

Exclusion criteria

1. Known or suspected hereditary or acquired complement deficiency;
2. Patients with laboratory evidence of bone marrow failure (reticulocytes <100x109/L; platelets <30x109/L; neutrophils <0.5x109/L);
3. Presence or suspicion of a systemic active bacterial, viral, or fungal infection (based on judgment of the investigator) within 2 weeks prior to the first dose of HRS-5965;
4. History of infection with capsular bacteria (e.g., meningococcus, pneumococcus, etc.)
5. Positive of HIV, HBsAg or HCVAb.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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76 participants in 2 patient groups

HRS-5965 capsule

Experimental group

Treatment:

Drug: HRS-5965 capsule

Eculizumab Injection

Experimental group

Treatment:

Drug: Eculizumab Injection