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

NCT ID: NCT04065438

Last Updated: 2024-10-09

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: NA
• Total Enrollment: 35 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-01-01
• Study Completion Date: 2028-06-01

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.

Detailed Description

This study will enroll up to 35 adult patients at 3-10 clinical sites. The duration of the treatment phase of the study will take approximately 9 weeks for a total of 12 apheresis treatments with the LIPOSORBER® LA-15 System; the treatments will be given twice weekly for 3 weeks followed by once weekly for 6 weeks. Patients will undergo follow-up at 1, 3, 6, 12, and 24 months after the final apheresis treatment.

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 primary FSGS and post Transplant recurrence.

Conditions

Focal Segmental Glomerulosclerosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

LIPOSORBER® LA-15

Intervention Type: 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).

Interventions

LIPOSORBER® LA-15

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).

Intervention Type: DEVICE

Eligibility Criteria

Inclusion Criteria

A patient is deemed suitable for inclusion in the study if the patient has nephrotic syndrome associated with primary FSGS when:

• 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.

or

• The patient is post renal transplantation.

Exclusion Criteria

1. Patient is greater than 75 years of age at the start of the treatment period or less than 22

2. The patient is unwilling or unable to sign and date the informed consent

3. 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.)

4. Unable or unwilling to comply with the follow-up schedule

5. Simultaneously participating in another investigational drug or device study

6. Body weight < 15 kg (33.1 lbs)

1. 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.)

2. 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

3. 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

4. Hypersensitivity to dextran sulfate, heparin, or ethylene oxide

5. Adequate anticoagulation cannot be achieved due to severe hemophilia, severe hemorrhage diathesis, severe gastrointestinal ulcers, or are recipients of vitamin K antagonist medications

6. 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.

7. Cardiac impairments such as uncontrolled arrhythmia, unstable angina, decompensated congestive heart failure, or valvular disease

8. Functional thyroid disease or liver abnormalities

9. Unresolved systemic or local infection that could affect the clinical study outcomes

• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Kaneka Medical America LLC

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Loma Linda University Children's Hospital

Loma Linda, California, United States

Site Status: RECRUITING

Loma Linda University Hospital

Loma Linda, California, United States

Site Status: RECRUITING

Nemours/Alfred I DuPont Hospital for Children

Wilmington, Delaware, United States

Site Status: RECRUITING

Helen DeVos Children's Hospital

Grand Rapids, Michigan, United States

Site Status: RECRUITING

Weill Cornell Medicine / NewYork-Presbyterian

New York, New York, United States

Site Status: RECRUITING

University of North Carolina

Chapel Hill, North Carolina, United States

Site Status: RECRUITING

Akron Children's Hospital

Akron, Ohio, United States

Site Status: RECRUITING

Medical University of South Carolina Children's Hospital

Charleston, South Carolina, United States

Site Status: RECRUITING

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status: RECRUITING

Children's Hospital of Richmond at VCU

Richmond, Virginia, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Ayaka Kitamura

Role: CONTACT

Ayaka.Kitamura1@kaneka.co.jp

+81-74431813933

Facility Contacts

Cheryl P Sanchez-Kazi, MD

Role: primary

Amir Abdipour, MD

Role: primary

Daisy Sekly

Role: backup

DSekly@llu.edu

Joshua J Zaritsly, MD

Role: primary

joshua.zaritsky@nemours.org

302-651-5527

Alejandro Quiroga, MD

Role: primary

Eduardo M Perelstein, MD

Role: primary

Koyal Jain, MD

Role: primary

Anne Froment

Role: backup

anne_froment@med.unc.edu

(919) 445-2622

Rupesh Raina, MD

Role: primary

apokelsek@akronchildrens.org

330-543-0702

Katherine E Twombley, MD

Role: primary

Linda Walker

Role: primary

walkerlp@musc.edu

843-792-6109

Megan M Lo, MD

Role: primary

Other Identifiers

KMA-FSGS-H170002

Identifier Type: -

Identifier Source: org_study_id