NCT06397001

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Nov 2023

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

November 28, 2023

Completed
5 months until next milestone

First Submitted

Initial submission to the registry

April 30, 2024

Completed
2 days until next milestone

First Posted

Study publicly available on registry

May 2, 2024

Completed
7 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 2, 2024

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 2, 2024

Completed
Last Updated

February 19, 2025

Status Verified

January 1, 2025

Enrollment Period

1 year

First QC Date

April 30, 2024

Last Update Submit

February 16, 2025

Conditions

AA Amyloidosis

Outcome Measures

Primary Outcomes (1)

  • Adverse Events and/or serious adverse events

    Number of adverse events and/or serious adverse events experienced

    1 year

Secondary Outcomes (8)

  • Change in kidney function

    Baseline, 1 year

  • Change in quality of life - physical well-being

    Baseline, 1 year

  • Change in quality of life - emotional well-being

    Baseline, 1 year

  • Change in quality of life - spiritual well-being

    Baseline, 1 year

  • Change in quality of life - intellectual well-being

    Baseline, 1 year

  • +3 more secondary outcomes

Study Arms (1)

nL-SAA1-01

EXPERIMENTAL

Subject will receive subcutaneous injection of nL-SAA1-01

Drug: nL-SAA1-01

Interventions

nL-SAA1-01DRUG

Personalized antisense oligonucleotide

nL-SAA1-01

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

MeSH Terms

Conditions

AA amyloidosis

Study Officials

  • Nelson Leung, MD

    Mayo Clinic

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

April 30, 2024

First Posted

May 2, 2024

Study Start

November 28, 2023

Primary Completion

December 2, 2024

Study Completion

December 2, 2024

Last Updated

February 19, 2025

Record last verified: 2025-01

Data Sharing

IPD Sharing
Will not share

Locations

US(1)