Treatment of AA Amyloidosis

February 16, 2025 updated by: Nelson Leung, MD

An Open-label Single Center, Single Patient Study of an Experimental Antisense Oligonucleotide (ASO) Treatment in AA Amyloidosis

The purpose of this study is to assess the safety and efficacy of a subcutaneous injection of nL-SAA1-01in a patient with AA Amyloidosis.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

1

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Mayo Clinic

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Biopsy proven AA amyloidosis
  • Measurable disease
  • Ability to travel to the study site and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records

Exclusion Criteria:

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: nL-SAA1-01
Subject will receive subcutaneous injection of nL-SAA1-01
Drug: nL-SAA1-01
Personalized antisense oligonucleotide

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Adverse Events and/or serious adverse events
Time Frame: 1 year
Number of adverse events and/or serious adverse events experienced
1 year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in kidney function
Time Frame: Baseline, 1 year
The change in kidney function is measured by the glomerular filtration rate (GFR)
Baseline, 1 year
Change in quality of life - physical well-being
Time Frame: Baseline, 1 year
Physical well-being will be measured using a Linear Analog Scale Assessment (LASA) The Likert scale runs from 0 (as bad as possible) to 10 (as good as possible) where a higher rating suggest a higher quality of life.
Baseline, 1 year
Change in quality of life - emotional well-being
Time Frame: Baseline, 1 year
Emotional well-being will be measured using a Linear Analog Scale Assessment (LASA) The Likert scale runs from 0 (as bad as possible) to 10 (as good as possible) where a higher rating suggest a higher quality of life.
Baseline, 1 year
Change in quality of life - spiritual well-being
Time Frame: Baseline, 1 year
Spiritual well-being will be measured using a Linear Analog Scale Assessment (LASA) The Likert scale runs from 0 (as bad as possible) to 10 (as good as possible) where a higher rating suggest a higher quality of life.
Baseline, 1 year
Change in quality of life - intellectual well-being
Time Frame: Baseline, 1 year
Intellectual well-being will be measured using a Linear Analog Scale Assessment (LASA) The Likert scale runs from 0 (as bad as possible) to 10 (as good as possible) where a higher rating suggest a higher quality of life.
Baseline, 1 year
Change in quality of life - overall well-being
Time Frame: Baseline, 1 year
Overall well-being will be measured using a Linear Analog Scale Assessment (LASA) The Likert scale runs from 0 (as bad as possible) to 10 (as good as possible) where a higher rating suggest a higher quality of life.
Baseline, 1 year
Initiation of dialysis
Time Frame: 1 year
The amount of time between the first dose of the investigational product and the need for dialysis
1 year
Initiation of kidney transplantation
Time Frame: 1 year
The amount of time between the first dose of the investigational product and the need for a kidney transplantation.
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Nelson Leung, MD

Investigators

  • Principal Investigator: Nelson Leung, MD, Mayo Clinic

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 28, 2023

Primary Completion (Actual)

December 2, 2024

Study Completion (Actual)

December 2, 2024

Study Registration Dates

First Submitted

April 30, 2024

First Submitted That Met QC Criteria

April 30, 2024

First Posted (Actual)

May 2, 2024

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

February 16, 2025

Last Verified

January 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 22-005092

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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