Clinical Trial: Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients With Paroxysmal Nocturnal Hemoglobinuria
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
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 Hemoglobi
Brief Summary: To determine whether LFG316 can induce a hematological response, as measured by reduction in hemolytic activity, in patients with PNH.
Detailed Summary:
Sponsor: Novartis Pharmaceuticals
Current Primary Outcome: Serum lactate dehydrogenase (LDH) levels [ Time Frame: Screening, weekly for 4 weeks, and every 2 weeks from week 4 to week 52 and follow-up period ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- 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 60 weeks after the first treatment) ]Number and grading of adverse events and reporting of serious adverse events
- Area Under the Curve (AUC) - Pharmacokinetics parameter [ Time Frame: Participants will be followed for the whole duration of the study (i.e. up to 60 weeks after the first treatment, which includes a screening visit, weekly visits for 4 weeks, and every 4 weeks from week 4 to week 60) ]Blood draw for pharmacokinetics evaluation
- Maximum Plasma Concentration (Cmax) - Pharmacokinetics parameter [ Time Frame: Participants will be followed for the whole duration of the study (i.e. up to 60 weeks after the first treatment, which includes a screening visit, weekly visits for 4 weeks, and every 4 weeks from week 4 to week 60) ]Blood draw for pharmacokinetics evaluation
- Time to Maximum Concentration (Tmax) - Pharmacokinetics parameter [ Time Frame: Participants will be followed for the whole duration of the study (i.e. up to 60 weeks after the first treatment, which includes a screening visit, weekly visits for 4 weeks, and every 4 weeks from week 4 to week 60) ]Blood draw for pharmacokinetics evaluation
- Electrocardiogram [ Time Frame: Participants will be followed for the whole duration of the study (i.e. up to 60 weeks after the first treatment, which includes a screening visit, every two weeks for 4 weeks, and every 4 weeks from week 4 to week 60) ]12-Lead Electrocardiogram
- Vital signs [ Time Frame: Participants will be followed for the whole duration of the study (i.e. up to 60 weeks after the first treatment, which includes a screening visit, weekly visits for 4 weeks, and every 4 weeks from week 4 to week 60) ]Blood pressure and heart rate
- Safety laboratory evaluations [ Time Frame: Participants will be followed for the whole duration of the study (i.e. up to 60 weeks after the first treatment, which includes a screening visit, weekly visits for 4 weeks, and every 2 weeks from week 4 to week 52, and every 4 weeks to week 60) ]Blood drawing for safety laboratory evaluations
Original Secondary Outcome:
- 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 60 weeks after the first treatment) ]Number and grading of adverse events and reporting of serious adverse events
- Area Under the Curve (AUC) - Pharmacokinetics parameter [ Time Frame: Participants will be followed for the whole duration of the study (i.e. up to 60 weeks after the first treatment, which includes a screening visit, weekly visits for 4 weeks, and every 4 weeks from week 4 to week 60) ]Blood draw for pharmacokinetics evaluation
- Maximum Plasma Concentration (Cmax) - Pharmacokinetics parameter [ Time Frame: Participants will be followed for the whole duration of the study (i.e. up to 60 weeks after the first treatment, which includes a screening visit, weekly visits for 4 weeks, and every 4 weeks from week 4 to week 60) ]Blood draw for pharmacokinetics evaluation
- Time to Maximum Concentration (Tmax) - Pharmacokinetics parameter [ Time Frame: Participants will be followed for the whole duration of the study (i.e. up to 60 weeks after the first treatment, which includes a screening visit, weekly visits for 4 weeks, and every 4 weeks from week 4 to week 60) ]Blood draw for pharmacokinetics evaluation
- Electrocardiogram [ Time Frame: Participants will be followed for the whole duration of the study (i.e. up to 60 weeks after the first treatment, which includes a screening visit, every two weeks for 4 weeks, and every 4 weeks from week 4 to week 60) ]12-Lead Electrocardiogram
- Vital signs [ Time Frame: Participants will be followed for the whole duration of the study (i.e. up to 60 weeks after the first treatment, which includes a screening visit, weekly visits for 4 weeks, and every 4 weeks from week 4 to week 60) ]Blood pressure and heart rate
- Forced expiratory Volume (FEV1) and Forced Vital Capacity (FVC) [ Time Frame: Participants will be followed for the whole duration of the study (i.e. up to 60 weeks after the first treatment, which includes a screening visit, once within the initial 4 weeks, and every 4 weeks from week 4 to week 60) ]Spirometry
- Clinical chemistry and Hematology evaluations [ Time Frame: Participants will be followed for the whole duration of the study (i.e. up to 60 weeks after the first treatment, which includes a screening visit, weekly visits for 4 weeks, and every 2 weeks from week 4 to week 52, and every 4 weeks to week 60) ]Blood drawing for safety laboratory evaluations
Information By: Novartis
Dates:
Date Received: August 7, 2015
Date Started: September 10, 2015
Date Completion: December 19, 2023
Last Updated: May 2, 2017
Last Verified: May 2017