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

NCT02235857 | Recruiting FDA Device

• 1 other identifier: KMA-FSGS-H120005
• Study Type: interventional
• Target: 35
• Locations: 1 country
• Sites: 12

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.

Conditions

Focal Segmental Glomerulosclerosis

Keywords

pediatric, renal transplantation, recurrence, drug-resistant

Outcome Measures

Primary Outcomes (2)

• The percent of patients who show complete or partial remission

  1 month after the final treatment

• the rate of device-related and procedure-related serious adverse events

  During the period in which the apheresis procedures are administered and up to at the 1-month follow-up visit

Secondary Outcomes (5)

• Nephrotic Condition

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

• The percent of patients who obtain complete or partial remission

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

• Incidence of adverse events

  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

  From 1 months to 24 months after the final aphresis

• Various laboratory values

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

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 System

Interventions

LIPOSORBER® LA-15 System 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.

Also known as: LDL apheresis, LDL adsorption, dextran sulfate column

Liposorber® LA-15 System

Eligibility Criteria

• Age: Up to 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Kaneka Medical America LLC: lead

Study Sites (12)

Loma Linda University Children's Hospital

Loma Linda, California, 92354, United States

RECRUITING

Cedars Sinai Medical Center

Los Angeles, California, 90048, United States

RECRUITING

Nemours/A.I. duPont Hospital for Children

Wilmington, Delaware, 19803, United States

RECRUITING

Nemours Children's Health

Orlando, Florida, 32827, United States

TERMINATED

Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States

RECRUITING

University of Minnesota

Minneapolis, Minnesota, 55454, United States

RECRUITING

Weill Cornell Medical Center / 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

St. Christopher's Hospital for Children

Philadelphia, Pennsylvania, 19134, United States

RECRUITING

Medical University of South Carolina Children's Hospital

Charleston, South Carolina, 29425, United States

RECRUITING

Children's Hospital of Richmond at VCU

Richmond, Virginia, 23219, United States

WITHDRAWN

Related Publications (7)

• 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.

  PMID: 17216262 BACKGROUND

• 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.

  PMID: 16481888 BACKGROUND

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

  PMID: 11104158 BACKGROUND

• 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.

  PMID: 8897562 BACKGROUND

• 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.

  PMID: 17186280 BACKGROUND

• 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.

  PMID: 19158356 BACKGROUND

• 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.

  PMID: 21310077 BACKGROUND

MeSH Terms

Conditions

Glomerulosclerosis, Focal Segmental; Recurrence

Condition Hierarchy (Ancestors)

Glomerulonephritis; Nephritis; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Jeffrey I Silberzweig, MD

  Weill Medical College of Cornell University

  PRINCIPAL INVESTIGATOR

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

Model Details: Patients with focal segmental glomerulosclerosis treated by LIPOSORBER® LA-15 system

Study Record Dates

• First Submitted: September 6, 2014
• First Posted: September 10, 2014
• Study Start: May 3, 2015
• Primary Completion: May 3, 2026
• Study Completion (Estimated): July 3, 2028
• Last Updated: April 30, 2025

Record last verified: 2024-10

Locations

US (12)