An Open-label Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316, an Anti-C5 Monoclonal Antibody in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

CLFG316X2201

To determine whether LFG316 can induce a hematological response, as measured by reduction in hemolytic activity, in patients with PNH.

2015-09-09

2022-05-24

2022-05-24

Completed

Early phase I

10

Inclusion Criteria: * Male and female patients between the age of 18-75 (inclusive), or 18-65 (applicable in Czech Republic) with a diagnosis of PNH prior to screening * A documented PNH clone size of ≥10% by RBCs and/or granulocytes * Serum LDH levels at least 1.5-fold above the upper limit of normal (ULN) at screening * Negative pregnancy test for women of child bearing potential at screening * Previous vaccination against Neisseria meningitidis is required at least 2 weeks prior to first dosing. Exclusion Criteria: * Known or suspected hereditary complement deficiency * History of recurrent meningitis, history of meningococcal meningitis despite vaccination * Presence or suspicion (based on judgment of the investigator) of active bacterial infection within 2 weeks prior to first dose of LFG316, or recurrent bacterial infections * Under active therapy with other agents interfering with the complement system * Severe concurrent co-morbidities that are a likely caused by underlying autoimmune diseases other than PNH * Women of child-bearing potential, unless they are using highly effective methods of contraception during dosing and for 50 days after the last dose of LFG316.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 10, 'type': 'ACTUAL'}}

2023-01-26