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Personalized Antisense Oligonucleotide Therapy for a Participant With TARDBP ALS

NCT07703462 · Status: ENROLLING_BY_INVITATION · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2026-07-14

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

  • Amyotrophic Lateral Sclerosis (ALS)

Interventions

DRUG

nL-TARDB-002

Personalized antisense oligonucleotide

Sponsors & Collaborators

  • Thomas Jefferson University

    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
2026-12-31
Primary Completion
2028-12-31
Completion
2028-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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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 NCT07703462 on ClinicalTrials.gov