Paroxysmal Nocturnal Hemoglobinuria PNH Clinical Trial
Official 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)
| Verified date | January 2023 |
| Source | Novartis |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
To determine whether LFG316 can induce a hematological response, as measured by reduction in hemolytic activity, in patients with PNH.
| Status | Completed |
| Enrollment | 10 |
| Est. completion date | May 24, 2022 |
| Est. primary completion date | May 24, 2022 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years and older |
| Eligibility | 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. |
| Country | Name | City | State |
|---|---|---|---|
| Czechia | Novartis Investigative Site | Brno Bohunice | Czech Republic |
| Japan | Novartis Investigative Site | Fukushima city | Fukushima |
| Japan | Novartis Investigative Site | Isehara | Kanagawa |
| Japan | Novartis Investigative Site | Niigata | |
| Japan | Novartis Investigative Site | Shinjuku-ku | Tokyo |
| Japan | Novartis Investigative Site | Suita | Osaka |
| Lithuania | Novartis Investigative Site | Vilnius |
| Lead Sponsor | Collaborator |
|---|---|
| Novartis Pharmaceuticals |
Czechia, Japan, Lithuania,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Serum lactate dehydrogenase (LDH) levels | Changes in serum lactate dehydrogenase (LDH) levels after treatment with LFG316 | 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 | Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) | Including any clinically relevant findings related with ECG, vital signs, laboratory data after treatment with LFG316 | 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) | |
| Secondary | 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 | Blood draw for pharmacokinetics evaluation after treatment with LFG316 | 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 | Maximum Plasma Concentration (Cmax) - Pharmacokinetics parameter | Blood draw for pharmacokinetics evaluation after treatment with LFG316 | 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 | Time to Maximum Concentration (Tmax) - Pharmacokinetics parameter | Blood draw for pharmacokinetics evaluation after treatment with LFG316 | 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) |