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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02534909
Recruitment Status : Active, not recruiting
First Posted : August 28, 2015
Last Update Posted : July 19, 2019
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

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

Condition or disease Intervention/treatment Phase
Paroxysmal Nocturnal Hemoglobinuria PNH Biological: LFG316 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date : September 9, 2015
Actual Primary Completion Date : June 7, 2017
Estimated Study Completion Date : February 24, 2022


Arm Intervention/treatment
Experimental: LFG316
During the treatment period, all patients will receive LFG316
Biological: LFG316
LFG316 will be administered to all patients enrolled in the study




Primary Outcome Measures :
  1. 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 312, follow-up and EoS ]
    Changes in serum lactate dehydrogenase (LDH) levels


Secondary Outcome Measures :
  1. 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 320 weeks after the first treatment) ]
    Including any clinically relevant findings related with ECG, vital signs, laboratory data

  2. Area Under the Curve (AUC) - 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

  3. 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

  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) ]
    Blood draw for pharmacokinetics evaluation



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02534909


Locations
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Czechia
Novartis Investigative Site
Brno Bohunice, Czech Republic, Czechia, 625 00
Novartis Investigative Site
Praha 2, Czech Republic, Czechia, 128 20
Japan
Novartis Investigative Site
Fukushima city, Fukushima, Japan, 960 1295
Novartis Investigative Site
Isehara, Kanagawa, Japan, 259-1193
Novartis Investigative Site
Suita city, Osaka, Japan, 565 0871
Novartis Investigative Site
Shinjuku-ku, Tokyo, Japan, 160-0023
Novartis Investigative Site
Niigata, Japan, 951 8520
Lithuania
Novartis Investigative Site
Vilnius, Lithuania, LT-08661
Sponsors and Collaborators
Novartis Pharmaceuticals

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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02534909     History of Changes
Other Study ID Numbers: CLFG316X2201
2014-005338-74 ( EudraCT Number )
First Posted: August 28, 2015    Key Record Dates
Last Update Posted: July 19, 2019
Last Verified: July 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
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


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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
PNH
Additional relevant MeSH terms:
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Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases