Treatment of AA Amyloidosis

AA Amyloidosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT06397001
• Other study ID #: 22-005092
• Status: Active, not recruiting
• Phase: Phase 1
• Start date: November 28, 2023
• Est. completion date: January 2025

Study information

• Verified date: April 2024
• Source: Mayo Clinic
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 1
• Est. completion date: January 2025
• Est. primary completion date: January 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• AA Amyloidosis
• Amyloidosis

Intervention

Drug:
• nL-SAA1-01
Subject will receive subcutaneous injection of starting at 40mg once every 4 weeks for two cycles. The dose will be increased to 80mg once every 4 weeks for three cycles. On cycle six the dose will be increased to 120mg once every four weeks.

Locations

Country	Name	City	State
United States	Mayo Clinic	Rochester	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Nelson Leung, MD

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Adverse Events and/or serious adverse events	Number of adverse events and/or serious adverse events experienced	1 year
Secondary	Change in kidney function	The change in kidney function is measured by the glomerular filtration rate (GFR)	Baseline, 1 year
Secondary	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.	Baseline, 1 year
Secondary	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.	Baseline, 1 year
Secondary	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.	Baseline, 1 year
Secondary	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.	Baseline, 1 year
Secondary	Change in quality of life - overall well-being	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
Secondary	Initiation of dialysis	The amount of time between the first dose of the investigational product and the need for dialysis	1 year
Secondary	Initiation of kidney transplantation	The amount of time between the first dose of the investigational product and the need for a kidney transplantation.	1 year