Personalized Antisense Oligonucleotide Therapy for Participants With TARDBP ALS

Part of paid clinical trials in St Louis, Missouri.

Sponsor
n-Lorem Foundation
Study ID
NCT07743268
Phase
PHASE1/PHASE2
Status
Enrolling By Invitation

Conditions

  • Amyotrophic Lateral Sclerosis (ALS)

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • nL-TARDB-006 — DRUG
    Personalized antisense oligonucleotide

Study Details

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.

Key Dates

First listed
Aug 3, 2026
Start date
Feb 19, 2026
Status verified
Jul 2026
Primary completion
Feb 29, 2028
Completion
Feb 29, 2028

Study Design

Enrollment
6 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Open Label

Primary Outcome Measure

Clinical Functioning [ Time Frame: Baseline to 12 months ]

Locations (2)

FacilityCityStateZIPSite coordinators
Washington UniversitySt LouisMissouri63110-
Columbia University, Irving Medical CenterNew YorkNew York10032-

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By research site
Washington University· St Louis, MOColumbia University, Irving Medical Center· New York, NY

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