Post Approval Study of Liposorber LA-15 System for the Treatment of Focal Segmental Glomerulosclerosis in Children (FSGS pediatric)

March 28, 2022 updated by: Kaneka Medical America LLC

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

35

Phase

  • Not Applicable

Contacts and Locations

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

Study Contact

Study Locations

  • United States
    • California
      • Loma Linda, California, United States, 92354
        • Recruiting
        • Loma Linda University Children'S Hospital
        • Contact:
          • Cheryl P Sanchez-Kazi, MD
          • Phone Number: 909-651-1904
      • Los Angeles, California, United States, 90048
    • Delaware
      • Wilmington, Delaware, United States, 19803
        • Recruiting
        • Nemours/A.I. duPont Hospital for Children
        • Contact:
    • Florida
      • Orlando, Florida, United States, 32827
        • Terminated
        • Nemours Children's Health
    • Michigan
      • Grand Rapids, Michigan, United States, 49503
        • Recruiting
        • Helen DeVos Children's Hospital
        • Contact:
          • Alejandro Quiroga, MD
          • Phone Number: 616-267-2400
    • Minnesota
      • Minneapolis, Minnesota, United States, 55454
        • Recruiting
        • University of Minnesota
    • New York
      • New York, New York, United States, 10065
        • Recruiting
        • Weill Cornell Medical Center / NewYork-Presbyterian
        • Contact:
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • Recruiting
        • University of North Carolina
    • Ohio
      • Akron, Ohio, United States, 44308
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19134
        • Recruiting
        • St. Christopher's Hospital for Children
        • Contact:
          • Joshua J Zaritsky, MD
          • Phone Number: 215-427-5190
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Recruiting
        • Medical University of South Carolina Children's Hospital
        • Contact:
    • Virginia
      • Richmond, Virginia, United States, 23219
        • Withdrawn
        • Children's Hospital of Richmond at VCU

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

No older than 21 years (ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • A pediatric patient is deemed suitable for inclusion in the study if the patient has FSGS with a GFR ≥ 45 ml/min/1.73 m 2 and any of the following:

    • Refractory nephrotic syndrome in which standard treatment options are unsuccessful (i.e., patient is unresponsive to standard corticosteroid and/or calcineurin inhibitor therapy for at least 8 weeks resulting in failure to achieve complete or partial remission);
    • Refractory nephrotic syndrome in which standard treatment options are not well tolerated (i.e., patients intolerant to standard therapies due to severe side effects that negatively affect quality of life without providing an acceptable level of clinical benefit);
    • Refractory or recurrent nephrotic syndrome in which standard therapy is contraindicated.

or

- Pediatric post renal transplant patients with nephrotic syndrome associated with primary FSGS.

Exclusion Criteria:

  • General Exclusion Criteria

    • Patient is greater than 21 years of age
    • Parent or patient is unwilling or unable to sign and date the informed consent (Note: Only patients 18-21 years of age may sign the informed consent on their own behalf)
    • Pregnant, lactating, or planning to become pregnant prior to completing the study (Note: The safety of the use of Liposorber® in pregnant women has not been studied. There may be unknown risks to an embryo/fetus. Sexually active women of child bearing potential should avoid pregnancy during the use of the Liposorber device and throughout the study duration.)
    • Unable or unwilling to comply with the follow-up schedule
    • Simultaneously participating in another investigational drug or device study
    • Body weight < 15 kg (33.1 lbs)

  • Medical Exclusion Criteria

    • Currently being administered ACE inhibitors that cannot be withheld for at least 24 hours prior to each apheresis treatment (Note: The time period to withhold ACE inhibitors should be prolonged, if determined by the treating physician, considering each individual's renal function and the biological half-life of the ACE-inhibitor currently in use.)
    • Currently being administered antihypertensive drugs other than ACE inhibitors (e.g., Angiotensin II receptor blockers (ARBs) that cannot be withheld on the day of apheresis until after the procedure
    • Medical condition or disorder that would limit life expectancy to less than the primary clinical study endpoint or that may cause noncompliance with the study plan or confound the data analysis
    • Hypersensitivity to dextran sulfate, heparin, or ethylene oxide
    • Adequate anticoagulation cannot be achieved due to severe hemophilia, severe hemorrhage diathesis, severe gastrointestinal ulcers, or are recipients of vitamin K antagonist medications
    • Extracorporeal circulation therapy with Liposorber® LA-15 System cannot be tolerated due to severe cardiac insufficiency, acute myocardial infarction, severe cardiac arrhythmia, acute apoplexy, severe uncontrollable hypertension, or severe uncontrollable hypotension
    • Cardiac impairments such as uncontrolled arrhyth¬mia, unstable angina, decompensated congestive heart failure, or valvular disease
    • Functional thyroid disease or liver abnormalities
    • Unresolved systemic or local infection that could affect the clinical study outcomes

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: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: Liposorber® LA-15 System
All study patients who meet the study eligibility criteria will undergo the extracorporeal treatment using Liposorber® LA-15 System. The participants are to be treated with the system twice weekly for the 3weeks and then once weekly for the following 6 weeks.
Device: LIPOSORBER® LA-15 System
LIPOSORBER® LA-15 System is an extracorporeal blood purification system. Approximately 3 to 4 L of plasma is treated in a single treatment session and it takes 2 to 3 hours. Recommended frequency of the treatment is twice weekly for 3 weeks followed by once weekly for 6 weeks, thus it takes 9 weeks for a total of 12 treatment sessions.
Other Names:
  • LDL apheresis
  • LDL adsorption
  • dextran sulfate column

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
The percent of patients who show complete or partial remission
Time Frame: 1 month after the final treatment
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
During the period in which the apheresis procedures are administered and up to at the 1-month follow-up visit

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
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
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
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
Incidence of adverse events and severe adverse events
Time Frame: From 1 months to 24 months after the final aphresis
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.
1,3, 6, 12, and 24 months after the final apheresis

Collaborators and Investigators

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

Sponsor

Kaneka Medical America LLC

Investigators

  • Principal Investigator: Jeffrey I Silberzweig, MD, Weill Medical College of Cornell University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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

March 1, 2015

Primary Completion (ANTICIPATED)

October 1, 2022

Study Completion (ANTICIPATED)

October 1, 2024

Study Registration Dates

First Submitted

September 6, 2014

First Submitted That Met QC Criteria

September 9, 2014

First Posted (ESTIMATE)

September 10, 2014

Study Record Updates

Last Update Posted (ACTUAL)

April 6, 2022

Last Update Submitted That Met QC Criteria

March 28, 2022

Last Verified

April 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • KMA-FSGS-H120005

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

Yes

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.

Clinical Trials on Focal Segmental Glomerulosclerosis

Clinical Trials on LIPOSORBER® LA-15 System

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Ukraine

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe