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

January 24, 2023 updated by: Novartis Pharmaceuticals

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)

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

10

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: LFG316 then LNP023
LFG316: Treatment periods 1-3 and first 4 weeks of period 4. LNP023: Treatment Period 4
Biological: LFG316

LFG316 will be administered to all patients enrolled in the study

LFG316: Treatment periods 1-3 and first 4 weeks of period 4. LNP023: Treatment Period 4

Other Names:
  • LFG316 then LNP023
Drug: LNP023
LNP023 will be administered in period 4 to patients who participate in period 3 of this study and are willing to join long term extension study with LNP023.
Other Names:
  • LFG316 then LNP023

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
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)
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)
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)
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)
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)
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)
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)
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)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 9, 2015

Primary Completion (Actual)

May 24, 2022

Study Completion (Actual)

May 24, 2022

Study Registration Dates

First Submitted

August 7, 2015

First Submitted That Met QC Criteria

August 27, 2015

First Posted (Estimate)

August 28, 2015

Study Record Updates

Last Update Posted (Estimate)

January 26, 2023

Last Update Submitted That Met QC Criteria

January 24, 2023

Last Verified

January 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CLFG316X2201
  • 2014-005338-74 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Yes

IPD Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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