Treatment of AA Amyloidosis

Nelson Leung, MD1 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

AA Amyloidosis

Interventions

nL-SAA1-01DRUG

Personalized antisense oligonucleotide

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (1)

Mayo Clinic

Rochester, Minnesota, United States

Outcomes

Primary Outcomes

Adverse Events and/or serious adverse events

Number of adverse events and/or serious adverse events experienced

Time frame: 1 year

Secondary Outcomes

Change in kidney function

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

Time frame: Baseline, 1 year

Change in quality of life - physical well-being

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.

Time frame: Baseline, 1 year

Change in quality of life - emotional well-being

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.

Time frame: Baseline, 1 year

Change in quality of life - spiritual well-being

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.

Time frame: Baseline, 1 year

Change in quality of life - intellectual well-being

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.

Time frame: Baseline, 1 year

Data from ClinicalTrials.gov

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