Lixelle® for Dialysis Amyloidosis

Overseen By Joanne McLaughlin

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Kaneka Medical America LLC

Disqualifiers: Rheumatoid arthritis, Osteoporosis, Osteoarthritis, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 104 weeks

40 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment called Lixelle® for individuals with dialysis-related amyloidosis (DRA), a condition that can occur in long-term dialysis patients. DRA arises when the protein β2-microglobulin accumulates in the blood and forms deposits in bones and joints, causing pain and other symptoms. Lixelle® filters this protein from the blood during dialysis sessions, aiming to relieve symptoms and improve daily life. Individuals who undergo dialysis three times a week and experience issues like joint pain or carpal tunnel syndrome might be suitable candidates for this trial. The trial will compare two groups: one receiving Lixelle® treatment and another under observation. As an unphased trial, this study offers a unique opportunity to contribute to understanding and potentially improving treatment for DRA.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What prior data suggests that this device is safe for dialysis-related amyloidosis patients?

Lixelle® is a treatment designed for individuals with dialysis-related amyloidosis (DRA), a condition that can develop in long-term dialysis patients. Used in Japan since 1996, the treatment employs a special filter to remove the protein β2-microglobulin (β2M) from the blood, which can accumulate over time in these patients.

Research has shown that Lixelle® can improve symptoms of DRA. Some patients have reported enhanced daily activities and reduced joint pain. Although Lixelle® is generally safe, some individuals have experienced temporary low blood pressure and a decrease in red blood cell count during treatment.

Overall, Lixelle® is usually well-tolerated, but like any treatment, it can have side effects. Consulting with a healthcare provider is important to determine if this treatment is suitable. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about Lixelle® for dialysis-related amyloidosis because it offers a novel approach that is not available with standard treatments like symptomatic management and supportive therapies. Unlike these approaches, Lixelle® directly targets and removes amyloid proteins from the bloodstream through a unique filtering process. This method has the potential to address the root cause of the condition rather than just alleviating symptoms, which could lead to significant improvements in patient outcomes.

What is the effectiveness track record for Lixelle® in treating dialysis-related amyloidosis?

Research has shown that Lixelle® effectively treats dialysis-related amyloidosis (DRA). A nationwide survey found that 84.9-96.5% of patients experienced symptom relief after using Lixelle®. In this trial, participants will either receive Lixelle® treatment or be observed under natural history conditions. Studies indicate that adding Lixelle® to regular dialysis significantly improves symptoms. The treatment removes a protein called β2-microglobulin, which can accumulate in the blood of individuals with kidney issues. This removal helps prevent harmful deposits that cause pain and inflammation. Since 1996, Lixelle® has been used successfully in Japan, demonstrating its long-term effectiveness in managing this condition. ^{2 3 6 7 8}

Who Is on the Research Team?

Jeffrey Silberzweig, MD

Principal Investigator

The Rogosin Institute

Are You a Good Fit for This Trial?

This trial is for patients on thrice-weekly hemodialysis diagnosed with Dialysis-Related Amyloidosis (DRA), showing specific symptoms like joint pain, carpal tunnel syndrome, or bone cysts. It's not for those with rheumatoid arthritis, osteoporosis, planning pregnancy or transplant, or who can't do extracorporeal therapy due to severe heart issues.

Inclusion Criteria

I am on thrice-weekly hemodialysis and diagnosed with DRA.

My biopsy showed amyloid deposits consistent with β2M.

I have been diagnosed with two or more of the following: joint pain, carpal tunnel, trigger finger, dialysis-related back issues, or bone cysts not caused by other diseases.

Exclusion Criteria

Patient planning to become pregnant, pregnant, or breast-feeding

I am planning to have a kidney transplant during the study.

I have been diagnosed with rheumatoid arthritis.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Lixelle® treatment for dialysis-related amyloidosis over a period of 2 years

104 weeks

Regular visits during each dialysis session

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Lixelle® treatment

Trial Overview The study tests Lixelle®, a blood-cleansing treatment designed to remove β2-microglobulin from the blood of DRA patients during dialysis. The goal is to alleviate symptoms and prevent disease progression by using a special column in the dialysis circuit.

How Is the Trial Designed?

Group I: Lixelle® treatment Experimental Treatment 1 Intervention

2 years of Lixelle® treatment in the patients with dialysis related amyloidosis (DRA)

Group II: natural history Active Control 1 Intervention

2 years of natural history in the patients with dialysis related amyloidosis (DRA)

Who Is Running the Clinical Trial?

Kaneka Medical America LLC

Lead Sponsor

Trials

13

Recruited

720+

Published Research Related to This Trial

A nationwide survey involving 345 patients treated with Lixelle for dialysis-related amyloidosis showed that 84.9-96.5% of patients reported that their symptoms worsened less frequently, indicating significant therapeutic efficacy.

Physicians also supported these findings, with 72.8% evaluating Lixelle treatment as effective or partially effective, suggesting that Lixelle can be a valuable treatment option for managing this serious complication of long-term hemodialysis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Survey of the effects of a column for adsorption of β2-microglobulin in patients with dialysis-related amyloidosis in Japan. Gejyo, F., Amano, I., Ando, T., et al.[2015]

In a study of 39 hemodialysis patients treated with the β(2)-microglobulin adsorption column (Lixelle), there was a significant reduction in the number and area of bone cysts in the wrist joints, indicating its efficacy in treating dialysis-related amyloidosis (DRA).

Clinical symptoms associated with DRA also improved significantly in patients receiving Lixelle treatment, highlighting its potential benefits beyond just radiographic changes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effectiveness of β(2)-microglobulin adsorption column in treating dialysis-related amyloidosis: a multicenter study. Kuragano, T., Inoue, T., Yoh, K., et al.[2016]

Lixelle is an effective treatment for dialysis-related amyloidosis (DRA), significantly reducing levels of beta2-microglobulin and inflammatory cytokines, which helps improve symptoms like joint pain and daily activities in hemodialysis patients.

In vitro studies show that Lixelle can eliminate over 1 mg of beta2-microglobulin per milliliter of its adsorbent beads, demonstrating its strong capacity for selective adsorption.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

beta(2)-Microglobulin-selective direct hemoperfusion column for the treatment of dialysis-related amyloidosis. Kutsuki, H.[2016]

The Lixelle column effectively removes beta2-microglobulin from the blood of patients with dialysis-related amyloidosis, leading to significant improvements in symptoms like reduced nocturnal awakenings and joint pain.

While the treatment shows promise, hypotension is a common side effect, highlighting the need for further research to understand both its efficacy and potential adverse effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

New adsorption column (Lixelle) to eliminate beta2-microglobulin for direct hemoperfusion. Furuyoshi, S., Nakatani, M., Taman, J., et al.[2019]

Lixelle S-35 effectively removes beta2-microglobulin from the blood, improving symptoms of dialysis-related amyloidosis (DRA) such as joint pain and nocturnal awakening, based on its larger column volume of 350 mL.

Lixelle S-15, with a smaller column volume of 150 mL, can achieve similar beta2-microglobulin removal when paired with a high-clearance dialysis membrane, and it has fewer adverse effects like hypotension and decreased hematocrit compared to S-35, while still improving joint pain over a 6-month treatment period.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Beta2-microglobulin-selective adsorbent column (Lixelle) for the treatment of dialysis-related amyloidosis. Suzuki, K., Shimazaki, M., Kutsuki, H.[2019]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT02952144

Post Approval Study of Lixelle for the Treament of Dialysis ... Lixelle® is a whole-blood β2M apheresis column developed to adsorb and eliminate β2M selectively from the blood of DRA patients. The treatment is performed with ...

2. sciencedirect.com sciencedirect.com/science/article/pii/S008525381549492X

Effect of β2-microglobulin adsorption column on dialysis- ... These results suggest that the addition of Lixelle™ to the high-flux dialyzer is associated with a significant clinical improvement in DRA patients. Previous ...

3. withpower.com withpower.com/trial/phase-amyloidosis-9-2015-56caa

Lixelle® for Dialysis Amyloidosis A nationwide survey involving 345 patients treated with Lixelle for dialysis-related amyloidosis showed that 84.9-96.5% of patients reported that their symptoms ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8384206/

The Kyushu Dialysis-Related Amyloidosis Study - PMC As Lixelle® has been reported to be effective in improving DRA ... Dialysis-Related Amyloidosis: Results from the Japanese Society for Dialysis ...

5. accessdata.fda.gov accessdata.fda.gov/cdrh_docs/pdf13/H130001d.pdf

Lixelle® Beta 2-microglobulin Apheresis Column Long term efficacy and safety of the small-sized β2- microglobulin adsorption column for dialysis-related amyloidosis. Therap. Apheresis Dialy.; 2011; 15(5):.

6. accessdata.fda.gov accessdata.fda.gov/cdrh_docs/pdf13/H130001b.pdf

SUMMARY OF SAFETY AND PROBABLE BENEFIT (SSPB) The Lixelle® β2-microglobulin apheresis column is indicated for the treatment of patients with clinically-diagnosed dialysis-related amyloidosis.

7. healio.com healio.com/news/primary-care/20150306/fda-authorizes-lixelle-column-for-dialysisrelated-amyloidosis

FDA authorizes Lixelle column for dialysis-related ... Both studies showed improvement in dialysis-related amyloidosis symptoms with Lixelle Column use. Temporary hypotension and a decrease in red ...

8. sciencedirect.com sciencedirect.com/science/article/abs/pii/S1570963905002517

β2-Microglobulin-selective direct hemoperfusion column ... Lixelle was developed as a β2-microglobulin adsorption column for patients with dialysis-related amyloidosis. The adsorption spectra in vitro showed that ...