Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients With Paroxysmal Nocturnal Hemoglobinuria

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)

ClinicalTrials.gov Identifier: NCT02534909

Novartis Reference Number: CLFG316X2201

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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

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

Condition 
Paroxysmal Nocturnal Hemoglobinuria PNH
Phase 
Phase 2
Overall status 
Active, not recruiting
Enrollment count 
10 participants
Start date 
Sep 09, 2015
Completion date 
Feb 25, 2022
Gender 
All
Age(s)
18 Years and older (Adult, Older Adult)

Interventions

Biological
LFG316
LFG316 will be administered to all patients enrolled in the study

Eligibility 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% 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.

Study Locations

Czechia
Novartis Investigative Site
-
Brno Bohunice, 625 00
Czech Republic
Czechia
Japan
Novartis Investigative Site
-
Fukushima city, 960 1295
Fukushima
Japan
Novartis Investigative Site
-
Isehara, 259-1193
Kanagawa
Japan
Novartis Investigative Site
-
Suita city, 565 0871
Osaka
Japan
Novartis Investigative Site
-
Shinjuku-ku, 160-0023
Tokyo
Japan
Novartis Investigative Site
-
Niigata, 951 8520
-
Japan
Lithuania
Novartis Investigative Site
-
Vilnius, LT-08661
-
Lithuania

Have a question?

Call 1-999-669-6682 or email [email protected]