Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in PNH Patients.

A Phase II, Multicenter, Randomized, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in Patients With Paroxysmal Nocturnal Hemoglobinuria.

This is a multicenter, randomized, open-label, Phase II clinical study to evaluate the efficacy, safety, and pharmacokinetics of LP-005 injection in adult patients with paroxysmal nocturnal hemoglobinuria (PNH).

Study Overview

• Status: Active, not recruiting
• Conditions: PNH - Paroxysmal Nocturnal Hemoglobinuria
• Intervention / Treatment: Biological: LP-005 Injection

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

Contacts and Locations

Study Locations

• China
  • Nanjing, China
    • The First Affiliated Hospital of Nanjing Medical University

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

• Male or female, aged 18 to 65 years.
• Diagnosis of PNH based on flow cytometry with clone size > 10% by granulocytes.
• Presence of one or more PNH-related signs or symptoms within 3 months prior to screening or a history of transfusion due to PNH.
• LDH level ≥2.0×upper limit of the normal range(ULN).
• Hemoglobin level <100 g/L at screening.

Exclusion Criteria:

• Active or suspected active viral, bacterial, fungal, or parasitic infection within 14 days prior to screening.
• History of meningococcal infection.
• History of splenectomy or congenital asplenia.
• History of systemic autoimmune disease or known/suspected immunodeficiency.
• History of hematopoietic stem cell transplantation.
• Use of any complement inhibitor within 3 months prior to screening or within 5 drug half-lives (whichever is longer).
• Pregnant or breastfeeding women, or women planning to become pregnant during the study or follow-up period.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

3

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
Experimental: LP-005 900 mg; Patients will receive intravenous infusion of LP-005 Injection at a dose of 900 mg once every 4 weeks.	Biological: LP-005 Injection; IV infusion, Q4W

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change from Baseline in serum lactate dehydrogenase (LDH) levels.	Baseline and at Week 12.
Proportion of participants with ≥2 g/dL increase in hemoglobin level from Baseline in the absence of transfusion.	Baseline and at Week 24.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in serum complement hemolytic activity.		Observation from Predose on Day 1 through 337 days post-administration.
Percentage of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs).		Baseline up to approximately 13 months.
Serum concentrations of LP-005.		Observation from Predose on Day 1 through 337 days post-administration.
Number of Participants with Treatment-emergent Anti-Drug Antibodies (ADA) and neutralizing antibodies (Nab) Response to LP-005.		Observation from Predose on Day 1 through 337 days post-administration.
Proportion of participants with ≥2 g/dL increase in hemoglobin level from Baseline in the absence of transfusion.		Baseline and at Week 12 and 48
Proportion of participants who are transfusion-free.		Baseline to Week 12, 24 and 48
Change from Baseline in the number of red blood cell (RBC) transfusions.		Baseline to Week 24 and 48.
Change from Baseline in hemoglobin levels.		Baseline, Week 12, 24 and 48
Proportion of participants achieving hemoglobin levels ≥100 g/L in the absence of transfusion.		Baseline, Week 12, 24 and 48
Proportion of participants with LDH levels normalized.		Baseline, Week 12, 24 and 48
Change From Baseline in Fatigue as Measured by Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F)	FACIT-F is a 40-item measure that assesses self-reported fatigue and its impact upon daily activities and function. The higher the score, the better the QOL.	Baseline and at Week 12, 36, and 48.

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 (Actual): November 14, 2024
• Primary Completion (Estimated): May 6, 2026
• Study Completion (Estimated): March 31, 2028

Study Registration Dates

• First Submitted: September 30, 2025
• First Submitted That Met QC Criteria: September 30, 2025
• First Posted (Estimated): October 8, 2025

Study Record Updates

• Last Update Posted (Estimated): January 16, 2026
• Last Update Submitted That Met QC Criteria: January 14, 2026
• Last Verified: January 1, 2026

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; Complex Mixtures; Biological Products
• Other Study ID Numbers: P10-LP005-02

Drug and device information, study documents

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