Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

A Phase Ⅱ Extension Clinical Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

This is an extension study for patients who have completed a prior P10-LP005-02 clinical study. The aim of this study is to evaluate the long-term safety, efficacy, and pharmacokinetics of LP-005 injection in adult patients with paroxysmal nocturnal hemoglobinuria (PNH).

Study Overview

• Status: Not yet recruiting
• Conditions: Paroxysmal Nocturnal Hemoglobinuria
• Intervention / Treatment: Biological: LP-005 Injection

• Study Type: Interventional
• Enrollment (Estimated): 30
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Tingyuan Yang; Phone Number: 86-18611978535; Email: yangty@longbiopharma.com

Study Locations

• China
  • Nanjing, China
    • The First Affiliated Hospital of Nanjing Medical University
    • Contact: Guangsheng He; Phone Number: +86 153 1205 2798; Email: heguangsheng1972@sina.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Patients have fully understood the trial, have voluntarily agreed to participate in this clinical trial, and have signed a written Informed Consent Form (ICF).
2. Patients who have completed the treatment of the Phase Ⅱ clinical study of LP-005 Injection and are assessed by the investigator as eligible for continued treatment with LP-005 Injection.
3. Patients who have received Neisseria meningitidis vaccine and Streptococcus pneumoniae vaccine in accordance with the requirements of previous studies; if the vaccine protection period does not cover the treatment duration of this study, patients must agree to receive booster vaccination in a timely manner in accordance with the vaccine administration guidelines and the requirements of local vaccination institutions.
4. Females and males of childbearing potential (including male subjects with female partners) must agree to use effective contraceptive measures from the start of the trial until 3 months after the end of the trial.

Exclusion Criteria:

1. Patients who have not completed the treatment of the Phase II clinical trial (P10-LP005-02) of the study drug.
2. Patients who have completed the Phase II treatment phase but are unwilling to continue receiving the study drug treatment.
3. Patients for whom the investigator does not recommend the continued use of LP-005 Injection after comprehensive assessment.
4. Patients who did not participate in the Phase II clinical trial (P10-LP005-02) of the study drug.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: LP-005 1200 mg; Patients will receive intravenous infusion of LP-005 Injection at a dose of 1200 mg once every 4 weeks.	Biological: LP-005 Injection; IV infusion, Q4W
Experimental: LP-005 1500 mg; Patients will receive intravenous infusion of LP-005 Injection at a dose of 1500 mg once every 4 weeks.	Biological: LP-005 Injection; IV infusion, Q4W

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence of adverse events (AEs)	Up to approximately 2 years

Secondary Outcome Measures

Outcome Measure	Time Frame
Change from baseline in serum lactate dehydrogenase (LDH) levels.	Up to approximately 2 years
Change from baseline in hemoglobin levels.	Up to approximately 2 years
Proportion of patients who are transfusion-free.	Up to approximately 2 years
Proportion of patients achieving hemoglobin levels ≥120 g/L	Up to approximately 2 years
Proportion of patients with breakthrough hemolysis	Up to approximately 2 years
Serum concentrations of LP-005.	Up to approximately 2 years
Number of patients with anti-drug antibodies (ADA)	Up to approximately 2 years

Collaborators and Investigators

• Sponsor: Longbio Pharma
• Investigators: Principal Investigator: Guangsheng He, The First Affiliated Hospital with Nanjing Medical University

Study record dates

Study Major Dates

• Study Start (Estimated): December 5, 2025
• Primary Completion (Estimated): December 30, 2027
• Study Completion (Estimated): May 1, 2028

Study Registration Dates

• First Submitted: November 24, 2025
• First Submitted That Met QC Criteria: November 24, 2025
• First Posted (Actual): December 5, 2025

Study Record Updates

• Last Update Posted (Actual): December 5, 2025
• Last Update Submitted That Met QC Criteria: November 24, 2025
• Last Verified: November 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Bone Marrow Diseases; Anemia, Hemolytic; Anemia; Myelodysplastic Syndromes; Hemic and Lymphatic Diseases; Hemoglobinuria, Paroxysmal
• Other Study ID Numbers: P10-LP005-05

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No