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

• Register: ClinicalTrials.gov
• Last refreshed on: 6 February 2023
• Main ID: NCT02534909
• Date of registration: 07/08/2015
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharmaceuticals
• Public title: Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients With Paroxysmal Nocturnal Hemoglobinuria
• Scientific title: 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)
• Date of first enrolment: September 9, 2015
• Target sample size: 10
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02534909
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Czech Republic, Czechia, Japan, Lithuania, Moldova, Republic of

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Paroxysmal Nocturnal Hemoglobinuria PNH

Intervention(s)

Biological: LFG316

Drug: LNP023

Primary Outcome(s)

Serum lactate dehydrogenase (LDH) levels [Time Frame: Screening, weekly for 4 weeks, every 2 weeks from week 4 to week 208, every 8 weeks from week 210 to 312 in periods 1-3, every 1 or 2 weeks from day 1 to day 57, every 4 or 8 weeks from day 85 to day 141 in period 4, and EoS]

Secondary Outcome(s)

AUC (0-t) = Area under the serum concentration versus time curve from time zero (pre-dose) to time of last quantifiable concentration (0-t) - Pharmacokinetics parameter [Time Frame: 320 weeks: screening visit, weekly visits for 4 weeks, and every 4 weeks from week 4 to week 54, visit on weeks 80, 106 132, 158, 184 and EOS)]

Maximum Plasma Concentration (Cmax) - Pharmacokinetics parameter [Time Frame: 320 weeks: screening visit, weekly visits for 4 weeks, and every 4 weeks from week 4 to week 54, visit on weeks 80, 106 132, 158, 184 and EOS)]

Number of Participants with 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 (i.e. up to 312 weeks after the first treatment for periods 1-3 and up to day 148 for period 4)]

Time to Maximum Concentration (Tmax) - Pharmacokinetics parameter [Time Frame: 320 weeks: screening visit, weekly visits for 4 weeks, and every 4 weeks from week 4 to week 54, visit on weeks 80, 106 132, 158, 184 and EOS)]

Secondary ID(s)

CLFG316X2201

2014-005338-74

Source(s) of Monetary Support

Please refer to primary and secondary sponsors