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

Longbio Pharma

Status and phase: Active, not recruiting; Phase 2

Conditions: PNH - Paroxysmal Nocturnal Hemoglobinuria

Treatments: Biological: LP-005 Injection

Study type: Interventional

Funder types: Industry

Identifiers

NCT07212426

P10-LP005-02

Details and patient eligibility

About

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).

Enrollment: 30 patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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30 participants in 3 patient groups

LP-005 900 mg

Experimental group

Description:

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

Treatment:

Biological: LP-005 Injection

LP-005 1200 mg

Experimental group

Description:

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

Treatment:

Biological: LP-005 Injection

LP-005 1500 mg

Experimental group

Description:

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

Treatment:

Biological: LP-005 Injection