Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria

NCT ID: NCT06051357

Last Updated: 2024-10-15

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 26 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-11-15
• Study Completion Date: 2024-03-25

Brief Summary

This is a multicenter, randomized, open-label phase II clinical trial. A total of 24 patients with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy were included. Subjects were treated with HRS-5965 for 12 weeks.

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treatment group A

Group Type: EXPERIMENTAL

HRS-5965 tablets

Intervention Type: DRUG

HRS-5965 tablets for 12 weeks

Treatment group B

Group Type: EXPERIMENTAL

HRS-5965 tablets

Intervention Type: DRUG

HRS-5965 tablets for 12 weeks

Interventions

HRS-5965 tablets

HRS-5965 tablets for 12 weeks

Intervention Type: DRUG

Eligibility Criteria

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;

4. Hemoglobin level < 10 g/dL.

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.

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

Sponsors

Chengdu Suncadia Medicine Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Peking Union Medical College Hospital, Chinese Academy of Medical Sciences

Beijing, Beijing Municipality, China

The Blood Disease Hospital of the Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, China

Countries

China

Other Identifiers

HRS-5965-202

Identifier Type: -

Identifier Source: org_study_id