Proof of concept study to assess the efficacy, safety and pharmacokinetics of LFG316 in patients with paroxysmal nocturnal hemoglobinuria (PNH)
- Conditions
- paroxysmal nocturnal hemoglobinuria
- Registration Number
- JPRN-jRCT2080223041
- Lead Sponsor
- ovartis Pharma K.K.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- completed
- Sex
- All
- Target Recruitment
- 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% 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
- 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
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method efficacy<br>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]<br>Changes in serum lactate dehydrogenase (LDH) levels
- Secondary Outcome Measures
Name Time Method safety<br>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] <br>Number and grading of adverse events and reporting of serious adverse events<br>pharmacokinetics<br>Area Under the Curve (AUC, Cmax, Tmax) - Pharmacokinetics parameter [Time Frame: Participants will be followed for the whole duration of the study]<br>Blood draw for pharmacokinetics evaluation