Post Approval Study for Treatment of Drug-resistant Adult and Pediatric Primary FSGS Using the LIPOSORBER® LA-15 System

NCT04065438 | Recruiting FDA Device

• 1 other identifier: KMA-FSGS-H170002
• Study Type: interventional
• Target: 35
• Locations: 1 country
• Sites: 10

Brief Summary

This multicenter, prospective, single-arm clinical study will evaluate the probable benefit and safety of the LIPOSORBER® LA-15 System for the treatment of adult patients with nephrotic syndrome associated with primary focal segmental glomerulosclerosis, when the standard treatment options, including corticosteroid and/or calcineurin inhibitors treatments, have been unsuccessful or not well tolerated, and the patient has a GFR ≥ 45 ml/min/1.73m2, or the patient has post-renal transplant recurrence. Treatment for FSGS is considered unsuccessful if the patient is unresponsive to standard therapy (e.g., at least 8 weeks of corticosteroids) and fails to achieve complete or partial remission. A standard treatment is considered not well tolerated if the patient experiences severe side effects without providing an acceptable level of clinical benefit.

Conditions

Focal Segmental Glomerulosclerosis

Keywords

FSGS; Liposorber

Outcome Measures

Primary Outcomes (1)

• Evaluate the safety and probable benefit of the LIPOSORBER® LA-15 System in relieving nephrotic syndrome associated with primary FSGS at 1 month after the final apheresis treatment. partial remission at 1 month after the final apheresis treatment.

  The primary probable benefit endpoint will be assessed by calculating the percent reduction in Up/c values at screening and at 1 month after the final apheresis.

  1 month after the final Apheresis treatment.

Secondary Outcomes (1)

• Nephrotic condition at 1, 3, 6, 12, and 24 months after the final apheresis treatment, including the percentage of patients who obtain complete or partial remission at 3, 6, 12, and 24 months.

  1, 3, 6, 12, and 24 months after the final treatment

Study Arms (1)

LIPOSORBER® LA-15 System

EXPERIMENTAL

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

Interventions

LIPOSORBER® LA-15 DEVICE

LIPOSORBER® LA-15 is a blood purification therapy; selectively removes apoprotein B-containing lipoproteins such as LDL from circulating blood and rapidly reduces the plasma cholesterol level. It was originally developed for prevention of coronary atherosclerosis progression in patients with serious hyperlipidemia such as familial hypercholesterolemia. In the late 1980's, LDL-A was used to improve dyslipidemia in Nephrotic Syndrome, initially to prevent organ damage. LDL-A was found to improve both the dyslipidemic condition and clinical symptoms (proteinuria and hypoproteinemia).

LIPOSORBER® LA-15 System

Eligibility Criteria

• Age: Up to 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Standard treatment options, including corticosteroid and/or calcineurin inhibitors, are unsuccessful or not well tolerated and the patient's glomerular filtration rate (GFR) ≥ 45 ml/min/1.73 m2.
• The patient is post renal transplantation.

You may not qualify if:

• Patient is greater than 75 years of age at the start of the treatment period or less than 22
• The patient is unwilling or unable to sign and date the informed consent
• 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 childbearing 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)
• 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., 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 Note: Severe uncontrollable hypotension/hypertension indicates the cases with systolic and/or diastolic blood pressure ≤ 5th percentile for age, gender, and height.
• Cardiac impairments such as uncontrolled arrhythmia, 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

Sponsors & Collaborators

• Kaneka Medical America LLC: lead

Study Sites (10)

Loma Linda University Children's Hospital

Loma Linda, California, 92354, United States

RECRUITING

Loma Linda University Hospital

Loma Linda, California, 92354, United States

RECRUITING

Nemours/Alfred I DuPont Hospital for Children

Wilmington, Delaware, 19803, United States

RECRUITING

Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States

RECRUITING

Weill Cornell Medicine / NewYork-Presbyterian

New York, New York, 10065, United States

RECRUITING

University of North Carolina

Chapel Hill, North Carolina, 27599, United States

RECRUITING

Akron Children's Hospital

Akron, Ohio, 44308, United States

RECRUITING

Medical University of South Carolina Children's Hospital

Charleston, South Carolina, 29425, United States

RECRUITING

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

RECRUITING

Children's Hospital of Richmond at VCU

Richmond, Virginia, 23298, United States

RECRUITING

MeSH Terms

Conditions

Glomerulosclerosis, Focal Segmental

Condition Hierarchy (Ancestors)

Glomerulonephritis; Nephritis; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases

Central Study Contacts

Ayaka Kitamura

CONTACT

+81-74431813933; Ayaka.Kitamura1@kaneka.co.jp

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 20, 2019
• First Posted: August 22, 2019
• Study Start: January 1, 2020
• Primary Completion: April 1, 2026
• Study Completion (Estimated): June 1, 2028
• Last Updated: October 9, 2024

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (10)