Personalized Antisense Oligonucleotide Therapy for Participants With TARDBP ALS
NCT07743268 · Status: ENROLLING_BY_INVITATION · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 6
Last updated 2026-08-03
Summary
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for participants with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP.
Conditions
- Amyotrophic Lateral Sclerosis (ALS)
Interventions
- DRUG
-
nL-TARDB-006
Personalized antisense oligonucleotide
Sponsors & Collaborators
- collaborator OTHER
-
Washington University School of Medicine
collaborator OTHER -
n-Lorem Foundation
lead OTHER
Study Design
- Allocation
- NA
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SINGLE_GROUP
Eligibility
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2026-02-19
- Primary Completion
- 2028-02-29
- Completion
- 2028-02-29
- FDA Drug
- Yes
Countries
- United States
Study Locations
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