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

Paroxysmal Nocturnal Hemoglobinuria Clinical Trial

Official title:

An Multicenter, Randomized, Open-label Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT06051357
• Other study ID #: HRS-5965-202
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: November 15, 2023
• Est. completion date: October 2024

Study information

• Verified date: September 2023
• Source: Chengdu Suncadia Medicine Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 26
• Est. completion date: October 2024
• Est. primary completion date: October 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Paroxysmal Nocturnal Hemoglobinuria

Intervention

Drug:
• HRS-5965 tablets
HRS-5965 tablets for 12 weeks

Locations

Country	Name	City	State
China	Peking Union Medical College Hospital, Chinese Academy of Medical Sciences	Beijing	Beijing
China	The Blood Disease Hospital of the Chinese Academy of Medical Sciences	Tianjin	Tianjin

Sponsors (1)

Lead Sponsor	Collaborator
Chengdu Suncadia Medicine Co., Ltd.

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Changes in hemoglobin.		12 weeks
Secondary	Changes in LDH.		12 weeks
Secondary	Changes in haptoglobin.		12 weeks
Secondary	Changes in bilirubin.		12 weeks
Secondary	Changes in reticulocyte counts.		12weeks
Secondary	Changes in C3 complement fragment deposition.		12 week
Secondary	Percentage of patients who did not receive a blood transfusion.		12 weeks
Secondary	Number of RBC units of transfused.		12weeks
Secondary	Incidence and severity of adverse events		16 weeks
Secondary	Incidence of thromboembolic events.		16 weeks
Secondary	Plasma concentration of HRS-5965.		12 weeks