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Personalized Antisense Oligonucleotide Therapy for A Single Participant With TARDBP ALS

NCT07095712 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2026-04-09

No results posted yet for this study

Summary

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP.

Conditions

Interventions

DRUG

nL-TARD-001

Personalized antisense oligonucleotide

Sponsors & Collaborators

  • The Methodist Hospital Research Institute

    collaborator OTHER

  • Columbia University

    collaborator OTHER

  • n-Lorem Foundation

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
49 Years
Max Age
49 Years
Sex
FEMALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-11-25
Primary Completion
2025-12-16
Completion
2025-12-16
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07095712 on ClinicalTrials.gov