AA Amyloidosis
Conditions
Brief summary
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.
Interventions
DRUGnL-SAA1-01
Personalized antisense oligonucleotide
Sponsors
Nelson Leung, MD
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Adverse Events and/or serious adverse events | 1 year | Number of adverse events and/or serious adverse events experienced |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in quality of life - physical well-being | 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. |
| Change in quality of life - emotional well-being | 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. |
| Change in quality of life - spiritual well-being | 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. |
| Change in kidney function | Baseline, 1 year | The change in kidney function is measured by the glomerular filtration rate (GFR) |
| Change in quality of life - overall well-being | 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. |
| Initiation of dialysis | 1 year | The amount of time between the first dose of the investigational product and the need for dialysis |
| Initiation of kidney transplantation | 1 year | The amount of time between the first dose of the investigational product and the need for a kidney transplantation. |
| Change in quality of life - intellectual well-being | 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. |
Countries
United States
Outcome results
None listed