nL-SAA1-01 for Amyloidosis

Age: 18+
Sex: Any
Trial Phase: Phase 1
Sponsor: Nelson Leung, MD
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of a new injection treatment, nL-SAA1-01, for individuals with AA Amyloidosis. AA Amyloidosis occurs when abnormal proteins accumulate in organs, causing complications. The trial involves administering an injection under the skin to determine if it reduces these protein buildups. It suits those diagnosed with AA Amyloidosis through a biopsy and who can travel to the study site for follow-up visits. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Do I need to stop my current medications for the trial?

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

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research shows that nL-SAA1-01, a treatment for AA Amyloidosis, is undergoing testing to determine its safety for humans. Since this treatment is in the early testing stages, information about its safety in humans remains limited. Researchers are examining how well people tolerate the treatment and what side effects might occur.

Although detailed safety information for nL-SAA1-01 isn't available yet, its testing in humans indicates that earlier lab or animal studies showed enough promise to proceed. These tests aim to ensure the treatment is safe enough for human trials.

In summary, nL-SAA1-01 remains in the early testing phase, and researchers are closely monitoring how people respond to ensure its safety.12345

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for amyloidosis, which often include chemotherapy and stem cell transplants, nL-SAA1-01 is administered via a simple subcutaneous injection. Researchers are excited about this treatment because it potentially offers a less invasive option with fewer side effects. Moreover, nL-SAA1-01 targets amyloid deposits specifically, which could improve effectiveness and outcomes for patients. This targeted approach and the ease of administration are significant leaps forward in treating amyloidosis.

What evidence suggests that nL-SAA1-01 might be an effective treatment for AA Amyloidosis?

Research shows that nL-SAA1-01 is an experimental treatment tested in this trial for its effectiveness in treating AA amyloidosis. This treatment uses antisense oligonucleotides, small DNA-like molecules designed to block specific genetic materials involved in the disease. Early studies suggest this approach could reduce the abnormal protein deposits that cause amyloidosis. Although detailed human data remains limited, the treatment's mechanism shows promise by targeting the genetic causes of the disease. This offers hope for a new way to manage the condition.13467

Who Is on the Research Team?

NL

Nelson Leung, MD

Principal Investigator

Mayo Clinic

Are You a Good Fit for This Trial?

This trial is for patients with AA Amyloidosis. Specific eligibility details are not provided, but typically participants must meet certain health criteria and may be required to have a confirmed diagnosis of AA Amyloidosis.

Inclusion Criteria

Biopsy proven AA amyloidosis
My cancer can be measured by tests.
I can travel to the study location and follow up as required.

Exclusion Criteria

Participant has any condition that in the opinion of the site investigator, would ultimately prevent the completion of study procedures

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive subcutaneous injection of nL-SAA1-01

1 year

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • nL-SAA1-01
Trial Overview The study is testing the safety and effectiveness of nL-SAA1-01, which is administered through an injection under the skin (subcutaneous) to treat AA Amyloidosis.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: nL-SAA1-01Experimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Nelson Leung, MD

Lead Sponsor

Trials
1
Recruited
1+

Citations

nL-SAA1-01 for Amyloidosis · Info for ParticipantsThe purpose of this study is to assess the safety and efficacy of a subcutaneous injection of nL-SAA1-01in a patient with AA Amyloidosis. Show more. Who Is on ...
Experimental Antisense Oligonucleotide Treatment in AA ...The purpose of this study is to assess the safety of the pre-determined maximum dose of nL-SAA1-01, in addition to adverse events and tolerbility of nL-SAA1-01.
Trial | NCT06397001The purpose of this study is to assess the safety and efficacy of a subcutaneous injection of nL-SAA1-01in a patient with AA Amyloidosis.
4.pubmed.ncbi.nlm.nih.govpubmed.ncbi.nlm.nih.gov/40226119/
AL Amyloidosis: Current Treatment and OutcomesThe only FDA-approved first-line therapy for AL amyloidosis is a combination regimen of daratumumab, cyclophosphamide, bortezomib, and dexamethasone ( ...
N=1 Collaborative: advancing customized nucleic acid ...This guidance, a focus of N1C working groups, aims to include sample INDs, a defined clinical trial pathway, recommendations on outcomes that ...
Treatment of AA Amyloidosis - ClinicalTrials.VeevaThe purpose of this study is to assess the safety and efficacy of a subcutaneous injection of nL-SAA1-01in a patient with AA Amyloidosis.
Serum Amyloid A1 (SAA1) Revisited: Restricted Leukocyte ...Infection, sterile injury, and chronic inflammation trigger the acute phase response in order to re-establish homeostasis.
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