Proof of concept study to assess the efficacy, safety and pharmacokinetics of LFG316 in patients with paroxysmal nocturnal hemoglobinuria (PNH)

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)

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2080223041

Enrollment

Registered

2015-12-11

Start date

2015-09-09

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

paroxysmal nocturnal hemoglobinuria

Interventions

investigational material(s) Generic name etc : LFG316 INN of investigational material : - Therapeutic category code : 399 Agents affecting metabolism, n.e.c. Dosage and Administration for Investigatio

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 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% or more 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. Other protocol-defined inclusion criteria may apply

Exclusion criteria

Exclusion criteria: 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. Other protocol-defined exclusion criteria may apply

Design outcomes

Primary

Measure	Time frame
efficacy Serum lactate dehydrogenase (LDH) levels [Time Frame: Screening, weekly for 4 weeks, and every 2 weeks from week 4 to week 208, every 8 weeks from week 210 to week 312, follow-up and EoS] Changes in serum lactate dehydrogenase (LDH) levels	—

Secondary

Measure	Time frame
safety Adverse Events (AEs) and Serious Adverse Events (SAEs) [Time Frame: Participants will be monitored for AEs and SAEs for the whole duration of the study] Number and grading of adverse events and reporting of serious adverse events pharmacokinetics Area Under the Curve (AUC, Cmax, Tmax) - Pharmacokinetics parameter [Time Frame: Participants will be followed for the whole duration of the study] Blood draw for pharmacokinetics evaluation	—

Measure

Time frame

safety Adverse Events (AEs) and Serious Adverse Events (SAEs) [Time Frame: Participants will be monitored for AEs and SAEs for the whole duration of the study] Number and grading of adverse events and reporting of serious adverse events pharmacokinetics Area Under the Curve (AUC, Cmax, Tmax) - Pharmacokinetics parameter [Time Frame: Participants will be followed for the whole duration of the study] Blood draw for pharmacokinetics evaluation

—

Countries

Asia except Japan, Europe, Japan

Contacts

Public ContactHideki Maruyama

Novartis Pharma K. K.

rinshoshiken.toroku@novartis.com+81-120-003-293

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026