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

NCT07212426 | Active Not Recruiting Phase 2

• 1 other identifier: P10-LP005-02
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

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).

Conditions

PNH - Paroxysmal Nocturnal Hemoglobinuria

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (11)

• 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

Study Arms (3)

LP-005 900 mg

EXPERIMENTAL

Patients will receive intravenous infusion of LP-005 Injection at a dose of 900 mg once every 4 weeks.

Biological: LP-005 Injection

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; LP-005 900 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:

• 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.

You may not qualify if:

• 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.

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

Study Design

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

Study Record Dates

• First Submitted: September 30, 2025
• First Posted: October 8, 2025
• Study Start: November 14, 2024
• Primary Completion: May 6, 2026
• Study Completion (Estimated): March 31, 2028
• Last Updated: January 16, 2026

Record last verified: 2026-01

Locations

CN (1)