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).

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.