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

NCT ID: NCT02235857

Last Updated: 2025-04-30

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

NA

Total Enrollment

35 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-05-03

Study Completion Date

2028-07-03

Brief Summary

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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 Description

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Conditions

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Focal Segmental Glomerulosclerosis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Patients with focal segmental glomerulosclerosis treated by LIPOSORBER® LA-15 system
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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 System

Intervention Type DEVICE

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.

Interventions

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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.

Intervention Type DEVICE

Other Intervention Names

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LDL apheresis LDL adsorption dextran sulfate column

Eligibility Criteria

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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

* 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)

* 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
Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Kaneka Medical America LLC

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Jeffrey I Silberzweig, MD

Role: PRINCIPAL_INVESTIGATOR

Weill Medical College of Cornell University

Locations

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Loma Linda University Children's Hospital

Loma Linda, California, United States

Site Status RECRUITING

Cedars Sinai Medical Center

Los Angeles, California, United States

Site Status RECRUITING

Nemours/A.I. duPont Hospital for Children

Wilmington, Delaware, United States

Site Status RECRUITING

Nemours Children's Health

Orlando, Florida, United States

Site Status TERMINATED

Helen DeVos Children's Hospital

Grand Rapids, Michigan, United States

Site Status RECRUITING

University of Minnesota

Minneapolis, Minnesota, United States

Site Status RECRUITING

Weill Cornell Medical Center / 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

St. Christopher's Hospital for Children

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Medical University of South Carolina Children's Hospital

Charleston, South Carolina, United States

Site Status RECRUITING

Children's Hospital of Richmond at VCU

Richmond, Virginia, United States

Site Status WITHDRAWN

Countries

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United States

Central Contacts

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Ayaka Kitamura

Role: CONTACT

+81-74431813933

Facility Contacts

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Cheryl P Sanchez-Kazi, MD

Role: primary

909-651-1904

Ananth S Karumanchi

Role: primary

310-423-7608

Joshua Zaritsky, MD PhD

Role: primary

302-651-5527

Alejandro Quiroga, MD

Role: primary

616-267-2400

Michelle Rheault, MD

Role: primary

612-626-2922

Eduardo M Perelstein, MD

Role: primary

646-962-4324

Koyal Jain, MD

Role: primary

Anne Froment

Role: backup

(919) 445-2622

Rupesh Raina, MD

Role: primary

330-543-8950

Ann Pokelsek, BSN, RN,

Role: backup

330-543-0702

Joshua J Zaritsky, MD

Role: primary

215-427-5190

Katherine E Twombley, MD

Role: primary

843-792-8904

References

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Reidy K, Kaskel FJ. Pathophysiology of focal segmental glomerulosclerosis. Pediatr Nephrol. 2007 Mar;22(3):350-4. doi: 10.1007/s00467-006-0357-2. Epub 2007 Jan 10.

Reference Type BACKGROUND
PMID: 17216262 (View on PubMed)

Franceschini N, North KE, Kopp JB, McKenzie L, Winkler C. NPHS2 gene, nephrotic syndrome and focal segmental glomerulosclerosis: a HuGE review. Genet Med. 2006 Feb;8(2):63-75. doi: 10.1097/01.gim.0000200947.09626.1c.

Reference Type BACKGROUND
PMID: 16481888 (View on PubMed)

Korbet SM. The treatment of primary focal segmental glomerulosclerosis. Ren Fail. 2000 Nov;22(6):685-96. doi: 10.1081/jdi-100101956.

Reference Type BACKGROUND
PMID: 11104158 (View on PubMed)

Tarshish P, Tobin JN, Bernstein J, Edelmann CM Jr. Cyclophosphamide does not benefit patients with focal segmental glomerulosclerosis. A report of the International Study of Kidney Disease in Children. Pediatr Nephrol. 1996 Oct;10(5):590-3. doi: 10.1007/s004670050167.

Reference Type BACKGROUND
PMID: 8897562 (View on PubMed)

Fine RN. Recurrence of nephrotic syndrome/focal segmental glomerulosclerosis following renal transplantation in children. Pediatr Nephrol. 2007 Apr;22(4):496-502. doi: 10.1007/s00467-006-0361-6. Epub 2006 Dec 21.

Reference Type BACKGROUND
PMID: 17186280 (View on PubMed)

Schwartz GJ, Munoz A, Schneider MF, Mak RH, Kaskel F, Warady BA, Furth SL. New equations to estimate GFR in children with CKD. J Am Soc Nephrol. 2009 Mar;20(3):629-37. doi: 10.1681/ASN.2008030287. Epub 2009 Jan 21.

Reference Type BACKGROUND
PMID: 19158356 (View on PubMed)

Trachtman H, Vento S, Gipson D, Wickman L, Gassman J, Joy M, Savin V, Somers M, Pinsk M, Greene T. Novel therapies for resistant focal segmental glomerulosclerosis (FONT) phase II clinical trial: study design. BMC Nephrol. 2011 Feb 10;12:8. doi: 10.1186/1471-2369-12-8.

Reference Type BACKGROUND
PMID: 21310077 (View on PubMed)

Other Identifiers

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KMA-FSGS-H120005

Identifier Type: -

Identifier Source: org_study_id

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