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

NCT07266155 | Not Yet Recruiting Phase 2

• 1 other identifier: P10-LP005-05
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

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).

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Outcome Measures

Primary Outcomes (1)

• Incidence of adverse events (AEs)

  Up to approximately 2 years

Secondary Outcomes (7)

• 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

Study Arms (2)

LP-005 1200 mg

EXPERIMENTAL

Patients will receive intravenous infusion of LP-005 Injection at a dose of 1200 mg once every 4 weeks.

Biological: LP-005 Injection

LP-005 1500 mg

EXPERIMENTAL

Patients will receive intravenous infusion of LP-005 Injection at a dose of 1500 mg once every 4 weeks.

Biological: LP-005 Injection

Interventions

LP-005 Injection BIOLOGICAL

IV infusion, Q4W

LP-005 1200 mg; LP-005 1500 mg

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients have fully understood the trial, have voluntarily agreed to participate in this clinical trial, and have signed a written Informed Consent Form (ICF).
• 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.
• 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.
• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Longbio Pharma: lead

Study Sites (1)

The First Affiliated Hospital of Nanjing Medical University

Nanjing, China

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Study Officials

• Guangsheng He

  The First Affiliated Hospital with Nanjing Medical University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Tingyuan Yang

CONTACT

86-18611978535; yangty@longbiopharma.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 24, 2025
• First Posted: December 5, 2025
• Study Start: December 5, 2025
• Primary Completion (Estimated): December 30, 2027
• Study Completion (Estimated): May 1, 2028
• Last Updated: December 5, 2025

Record last verified: 2025-11

Locations

CN (1)