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

Trial Summary

What is the purpose of this trial?

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.

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 data supports the effectiveness of the Liposorber LA-15 treatment for Focal Segmental Glomerulosclerosis?

The Liposorber LA-15 treatment showed that some patients with drug-resistant Focal Segmental Glomerulosclerosis experienced partial or complete remission of symptoms, such as reduced protein in urine, and maintained stable kidney function over time. Additionally, this system has been effective in lowering cholesterol levels in patients with severe hypercholesterolemia, suggesting its potential in managing other conditions. ^{1 2 3 4 5}

How does the Liposorber LA-15 treatment differ from other treatments for focal segmental glomerulosclerosis?

The Liposorber LA-15 treatment is unique because it uses a process called apheresis to filter the blood and remove harmful substances, which is different from standard drug treatments. This approach is particularly useful for patients who do not respond to traditional medications or have drug-resistant forms of the condition. ^{1 2 3 4 6}

Research Team

Jeffrey I Silberzweig, MD

Principal Investigator

Weill Medical College of Cornell University

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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]

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 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]

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]

References

1. Switzerland 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. [2020]

2. United States pubmed.ncbi.nlm.nih.gov

Low-density lipoprotein apheresis for prevention and regression of atherosclerosis: clinical results. [2019]

3. United States pubmed.ncbi.nlm.nih.gov

Adsorption of lipoprotein containing apolipoprotein-B through plasma separation for treatment of familial hypercholesterolemia. [2017]

4. Australia pubmed.ncbi.nlm.nih.gov

Blood purification therapies using dextran sulfate cellulose columns Liposorber and Selesorb. [2022]

5. United States pubmed.ncbi.nlm.nih.gov

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

6. United States pubmed.ncbi.nlm.nih.gov

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