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OpenTrials
Completed

NCT Number: NCT06397001

Treatment of 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.

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Key information

Conditions

AA Amyloidosis

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

nL-SAA1-01

Drug

Personalized antisense oligonucleotide

Primary outcomes

  1. Adverse Events and/or serious adverse events

    Time frame: 1 year

    Number of adverse events and/or serious adverse events experienced

Secondary outcomes

  1. Change in kidney function

    Time frame: Baseline, 1 year

    The change in kidney function is measured by the glomerular filtration rate (GFR)

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. Initiation of dialysis

    Time frame: 1 year

    The amount of time between the first dose of the investigational product and the need for dialysis

  8. 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.

Sponsors and collaborators

Lead sponsor

Nelson Leung, MD

Other

Registry information

Official study title

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

Important dates

Study start
2023
Primary completion
2024
Study completion
2024
First posted
May 2, 2024
Registry last updated
Feb 19, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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