Clinical Trial: Post Approval Study of Liposorber LA-15 System for the Treatment of Focal Segmental Glomerulosclerosis in Children

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Treatment of Drug-resistant Pediatric Primary Focal Segmental Glomerulosclerosis Using the Liposorber® LA-15 System

Brief Summary:

Liposorber® LA-15 System is a blood purification therapy that selectively removes malignant lipoproteins including low density lipoprotein from circulating blood flow and rapidly reduces the plasma cholesterol level. The system was originally developed for the treatment of patients with serious dyslipidemia such as familial hypercholesterolemia and then applied to improve the dyslipidemia, a common complication of nephrotic syndrome and found to bring about improvement not only with the dyslipidemic condition but the nephrotic condition (e.g, proteinuria and hypoproteinemia).

Although the definitive mechanism by which the system may relieve nephrotic syndrome is unknown, it has been recognized as one of alternative therapies for refractory nephrotic syndrome including focal segmental glomerulosclerosis (FSGS) in Japan and referred in the Guidelines for the Treatment of Nephrotic Syndrome endorsed by The Japanese Society of Nephrology.

This study is conducted as a post approval study imposed by Humanitarian Device Exemption (HDE) order to confirm the safety and efficacy of the Liposorber® LA-15 System in the treatment of drug-resistant pediatric primary FSGS.


Detailed Summary:
Sponsor: Kaneka Pharma America LLC

Current Primary Outcome:

  • The percent of patients who show complete or partial remission [ Time Frame: 1 month after the final treatment ]
  • the rate of device-related and procedure-related serious adverse events [ Time Frame: During the period in which the apheresis procedures are administered and up to at the 1-month follow-up visit ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Nephrotic Condition [ Time Frame: 1, 3, 6, 12, and 24 months after the final treatment ]

    Nephrotic condition defined as follows:

    urine protein:creatinine ratio > 2.0 (g/g) with a first morning void urine sample

  • The percent of patients who obtain complete or partial remission [ Time Frame: 3, 6, 12, and 24 months after the final treatment ]
  • Incidence of adverse events [ Time Frame: From the initiation of the first apheresis session until the termination of the last (usually 12th) apheresis session, standad period of 9 weeks for a total of 12 aoheresis sessions ]

    The protocol indicates the standard treatment schedule as follows:

    2 sessions weekly for the first 3 weeks followed by 1 session weekly for 6 weeks

  • Incidence of adverse events and severe adverse events [ Time Frame: From 1 months to 24 months after the final aphresis ]
  • Various laboratory values [ Time Frame: 1,3, 6, 12, and 24 months after the final apheresis ]
    Various laboratory values include Urine protein, Urine creatinine, Serum creatinine, Estimated glomerular filtration rate (eGFR), serum total protein, Serum albumin, Serum vitamin E, Hematocrit, Red blood cell, White blood cell, Platelet, Total cholesterol, LDL cholesterol, HDL cholesterol, Triglycerides, Serum soluble urokinase plasminogen activator receptor.


Original Secondary Outcome:

  • Nephrotic Condition [ Time Frame: 1, 3, 6, 12, and 24 months after the final treatment ]
  • The percent of patients who obtain complete or partial remission [ Time Frame: 3, 6, 12, and 24 months after the final treatment ]
  • Incidence of adverse events [ Time Frame: The period in which apheresis treatments are given ]
  • Incidence of adverse events and severe adverse events [ Time Frame: From 1 months to 24 months after the final aphresis ]
  • Various laboratory values [ Time Frame: 1,3, 6, 12, and 24 months after the final apheresis ]
    Various laboratory values include Urine protein, Urine creatinine, Serum creatinine, Estimated glomerular filtration rate (eGFR), serum total protein, Serum albumin, Serum vitamin E, Hematocrit, Red blood cell, White blood cell, Platelet, Total cholesterol, LDL cholesterol, HDL cholesterol, Triglycerides, Serum soluble urokinase plasminogen activator receptor.


Information By: Kaneka Pharma America LLC

Dates:
Date Received: September 6, 2014
Date Started: March 2015
Date Completion: October 2020
Last Updated: October 3, 2016
Last Verified: October 2016