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

Phase 2Active Not RecruitingNCT07212426

Longbio Pharma30 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

PNH - Paroxysmal Nocturnal Hemoglobinuria

Interventions

LP-005 InjectionBIOLOGICAL

IV infusion, Q4W

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

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.

Locations (1)

The First Affiliated Hospital of Nanjing Medical University

Nanjing, China

Outcomes

Primary Outcomes

Change from Baseline in serum lactate dehydrogenase (LDH) levels.

Time frame: Baseline and at Week 12.

Proportion of participants with ≥2 g/dL increase in hemoglobin level from Baseline in the absence of transfusion.

Time frame: Baseline and at Week 24.

Secondary Outcomes

Proportion of participants with ≥2 g/dL increase in hemoglobin level from Baseline in the absence of transfusion.

Time frame: Baseline and at Week 12 and 48

Proportion of participants who are transfusion-free.

Time frame: Baseline to Week 12, 24 and 48

Change from Baseline in the number of red blood cell (RBC) transfusions.

Time frame: Baseline to Week 24 and 48.

Change from Baseline in hemoglobin levels.

Time frame: Baseline, Week 12, 24 and 48

Proportion of participants achieving hemoglobin levels ≥100 g/L in the absence of transfusion.

Time frame: Baseline, Week 12, 24 and 48

Proportion of participants with LDH levels normalized.

Time frame: Baseline, Week 12, 24 and 48

Changes in serum complement hemolytic activity.

Time frame: Observation from Predose on Day 1 through 337 days post-administration.

Change From Baseline in Fatigue as Measured by Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F)

Data from ClinicalTrials.gov

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