Liposorber LA-15 Therapy for Focal Segmental Glomerulosclerosis

(FSGS pediatric Trial)

Recruiting at 9 trial locations

Overseen By Ayaka Kitamura

Age: < 65

Sex: Any

Trial Phase: Academic

Sponsor: Kaneka Medical America LLC

Must not be taking: Ace inhibitors, Arbs, Vitamin K antagonists

Disqualifiers: Age > 21, Pregnancy, Cardiac impairments, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 13

Duration: 9 weeks

35 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the Liposorber® LA-15 System, a blood purification therapy. The goal is to determine if it can help children with focal segmental glomerulosclerosis (FSGS), particularly when other treatments have failed or are not tolerated. Participants should be children with FSGS who have not responded well to standard treatments or have had a kidney transplant and still face issues. The study aims to confirm the safety and effectiveness of this therapy for these young patients. As an unphased trial, it offers a unique opportunity for patients to access potentially beneficial treatment options not yet widely available.

Will I have to stop taking my current medications?

The trial requires that certain medications, like ACE inhibitors and some antihypertensive drugs, be stopped at least 24 hours before each treatment session. If you're taking these medications, you may need to pause them temporarily for the trial.

What prior data suggests that the Liposorber® LA-15 System is safe for treating focal segmental glomerulosclerosis?

Research has shown that the Liposorber® LA-15 System is a safe treatment for people with nephrotic syndrome, including those with focal segmental glomerulosclerosis (FSGS). This device is approved for use in both adults and children with this condition. Used in Japan, it is considered a well-tolerated alternative therapy. The treatment removes harmful substances from the blood, which can help improve kidney function. Although the exact mechanism remains unclear, it serves as a helpful option for those who do not respond to standard treatments. Overall, no major safety concerns have been reported for its use in FSGS. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Unlike the standard treatments for Focal Segmental Glomerulosclerosis (FSGS) that often involve immunosuppressive drugs like corticosteroids and calcineurin inhibitors, the LIPOSORBER® LA-15 System offers a unique approach by physically removing harmful substances directly from the blood. This extracorporeal therapy works by targeting and reducing specific proteins and lipids that contribute to kidney damage, which is different from the traditional method of suppressing the immune system. Researchers are excited about this treatment because it may provide a non-drug alternative that could be effective for patients who do not respond well to standard therapies, potentially leading to fewer side effects and a new way to manage FSGS.

What evidence suggests that the Liposorber® LA-15 System is effective for focal segmental glomerulosclerosis?

Research shows that the Liposorber® LA-15 System can aid individuals with focal segmental glomerulosclerosis (FSGS), a kidney disease. Studies have found that it removes harmful fats from the blood, improving symptoms such as protein in the urine and low protein levels in the blood. Some patients with medication-resistant FSGS have even achieved full remission using this system. In Japan, the device is recognized as an alternative treatment for challenging cases of nephrotic syndrome, including FSGS. Although the exact mechanism by which it helps with nephrotic syndrome remains unclear, it is considered beneficial for those unresponsive to other treatments. Participants in this trial will receive treatment with the Liposorber® LA-15 System to evaluate its effectiveness in this context. ^{1 3 6 7 8}

Who Is on the Research Team?

Jeffrey I Silberzweig, MD

Principal Investigator

Weill Medical College of Cornell University

Are You a Good Fit for This Trial?

This trial is for children with a kidney condition called Focal Segmental Glomerulosclerosis (FSGS) who haven't responded to standard treatments. They should have a certain level of kidney function and not be on specific blood thinners or blood pressure medications that can't be paused. The study excludes those over 21, under 33.1 lbs, pregnant, or with severe heart issues among other criteria.

Inclusion Criteria

My kidney condition hasn't improved after 8 weeks of standard treatment.

My kidney condition treatment causes severe side effects without much benefit.

I am a child with FSGS and my kidney function is good.

Exclusion Criteria

I cannot undergo Liposorber® LA-15 treatment due to severe heart or blood pressure issues.

I weigh less than 33.1 lbs.

I am over 21 years old.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants undergo extracorporeal treatment using the Liposorber® LA-15 System, with 2 sessions weekly for the first 3 weeks followed by 1 session weekly for 6 weeks

9 weeks

12 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, with various laboratory values assessed

24 months

Visits at 1, 3, 6, 12, and 24 months

What Are the Treatments Tested in This Trial?

Interventions

• LIPOSORBER® LA-15 System

Trial Overview The Liposorber® LA-15 System is being tested in this study. It's a device that filters out harmful lipoproteins from the blood which might help improve conditions related to FSGS in kids who don’t respond well to usual drugs.

How Is the Trial Designed?

Group I: Liposorber® LA-15 System Experimental Treatment 1 Intervention

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.

LIPOSORBER® LA-15 System is already approved in United States, Japan for the following indications:

Approved in United States as LIPOSORBER LA-15 System for:

• Familial Hypercholesterolemia
• Focal Segmental Glomerulosclerosis (FSGS) in children

Approved in Japan as LIPOSORBER LA-15 System for:

• Familial Hypercholesterolemia
• Refractory nephrotic syndrome including focal segmental glomerulosclerosis (FSGS)

Who Is Running the Clinical Trial?

Kaneka Medical America LLC

Lead Sponsor

Trials

13

Recruited

720+

Published Research Related to This Trial

In a multicenter study involving 17 patients with drug-resistant primary FSGS, lipoprotein apheresis using the Liposorber® LA-15 system showed promising results, with partial or complete remission of nephrotic syndrome observed in 66.7% of patients at the 6-month follow-up.

All patients demonstrated improved or stable kidney function (eGFR) over the follow-up period, indicating that lipoprotein apheresis may enhance the effectiveness of standard treatments for resistant cases of primary FSGS.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dextran-Sulfate Plasma Adsorption Lipoprotein Apheresis in Drug Resistant Primary Focal Segmental Glomerulosclerosis Patients: Results From a Prospective, Multicenter, Single-Arm Intervention Study. Raina, R., Krishnappa, V., Sanchez-Kazi, C., et al.[2020]

In a multicenter trial involving 120 patients with severe hypercholesterolemia, LDL apheresis using the Liposorber LA-15 system resulted in significant reductions in total cholesterol (TC) by 52.6% and LDL-cholesterol (LDL-C) by 63.1% during treatment, demonstrating its efficacy for patients unresponsive to diet and maximum drug therapy.

The treatment was generally well tolerated, with adverse events primarily related to hypotension occurring in only 1% of treatments, indicating that LDL apheresis is a safe option for managing severe hypercholesterolemia, although caution is advised for patients on ACE inhibitors due to potential severe reactions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Low-density lipoprotein apheresis for prevention and regression of atherosclerosis: clinical results. Bambauer, R., Olbricht, CJ., Schoeppe, E.[2019]

Liposorber effectively purifies plasma by selectively adsorbing low-density lipoproteins, making it useful for treating conditions like familial hypercholesterolemia, peripheral arterial disease, and focal segmental glomerulosclerosis.

Selesorb targets and removes specific antibodies and immune complexes, providing a treatment option for patients with systemic lupus erythematosus, especially those who do not respond to standard medications.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Blood purification therapies using dextran sulfate cellulose columns Liposorber and Selesorb. Asahi, T., Yamamoto, T., Kutsuki, H.[2022]

LDL apheresis using the Liposorber system effectively removes LDL from the plasma of patients with high cholesterol who do not respond to diet or medication, acting as a pseudoreceptor for LDL.

Clinical benefits include a 70% reduction in cardiac events compared to drug therapy alone, and improvements in endothelial function and potential benefits for conditions like arteriosclerosis obliterans and focal glomerular sclerosis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Low-density lipoprotein apheresis using the Liposorber system: features of the system and clinical benefits. Yamamoto, T., Yamashita, T.[2019]

LDL-apheresis therapy using the Liposorber LA-15 System significantly reduces LDL cholesterol levels in patients with familial hypercholesterolemia (FH), achieving mean acute reductions of 76% in heterozygous FH and 81% in homozygous FH patients.

The procedure is generally safe, with hypotension being the most common side effect occurring in only 3% of treatments, and it is FDA-approved for specific patient groups with high LDL cholesterol levels.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Low-density lipoprotein apheresis using the Liposorber dextran sulfate cellulose system for patients with hypercholesterolemia refractory to medical therapy. Gordon, BR., Saal, SD.[2005]

The Liposorber LA-40 is an effective treatment for familial hypercholesterolemia, selectively adsorbing low-density and very low-density lipoproteins, which helps lower cholesterol levels significantly, as demonstrated in two clinical cases.

After seven months of bi-weekly treatments, patients maintained total cholesterol levels under 300 mg/dl without the need for replacement fluids, indicating both the efficacy and safety of this apheresis system.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Adsorption of lipoprotein containing apolipoprotein-B through plasma separation for treatment of familial hypercholesterolemia. Odaka, M., Kobayashi, H., Soeda, K., et al.[2017]

Citations

1. clinicaltrials.gov clinicaltrials.gov/ct2/show/NCT02235857?term=02235857&rank=1

Post Approval Study of Liposorber LA-15 System for the ... Liposorber® LA-15 System is a blood purification therapy that selectively removes malignant lipoproteins including low density lipoprotein from circulating ...

2. accessdata.fda.gov accessdata.fda.gov/cdrh_docs/pdf17/H170002D.pdf

LIPOSORBER® LA-15 The LIPOSORBER® LA-15 System is indicated for use in the treatment of adult and pediatric patients with nephrotic syndrome associated with primary focal ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT04065438

Post Approval Study for Treatment of Drug-resistant Adult ... This multicenter, prospective, single-arm clinical study will evaluate the probable benefit and safety of the LIPOSORBER® LA-15 System for the treatment of ...

4. fda.gov fda.gov/media/157895/download

Executive Summary H120005 Liposorber ... Liposorber LA-15 System for the treatment of pediatric patients who have FSGS with an estimated Glomerular Filtration Rate (eGFR) ≥60 ml/min/1.73 m2 ...

5. journals.lww.com journals.lww.com/jasn/fulltext/2024/10001/successful_treatment_of_primary_fsgs_with_ldl.947.aspx

Successful Treatment of Primary FSGS with LDL Apheresis:... All 4 of our patients were able to achieve complete remission of primary FSGS despite a lengthy history of multi-drug resistance.

6. accessdata.fda.gov accessdata.fda.gov/cdrh_docs/pdf12/H120005c.pdf

LIPOSORBER LA-15 SYSTEM This Operator's Manual contains the information needed to operate the. LIPOSORBER® LA-15 System correctly and safely. It is essential that you read.

7. clinicaltrials.cedars-sinai.edu clinicaltrials.cedars-sinai.edu/view/H170002

Treatment of Adult and Pediatric FSGS Using the ... The purpose of the study is to evaluate a device called the LIPOSORBER LA-15 System in relieving kidney problems associated with primary FSGS or post- ...

8. liposorber.com liposorber.com/kidney-disease/liposorber-for-fsgs/

FSGS Treatment | LIPOSORBER® The LIPOSORBER® LA-15 System is indicated for use in the treatment of adult and pediatric patients with nephrotic syndrome associated with primary focal ...