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Completed Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for A Single Participant With TARDBP ALS

ClinicalTrials.gov ID: NCT07095712

Public ClinicalTrials.gov record NCT07095712. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Oct 22, 2026, 8:46 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

An Open-label Multicenter, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to TARDBP (TDP-43) Genetic Mutation

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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.

Study identification

NCT ID
NCT07095712
Recruitment status
Completed
Study type
Interventional
Phase
Phase 1, Phase 2
Lead sponsor
n-Lorem Foundation
Other
Enrollment
1 participant

Conditions and interventions

Interventions

Drug

Eligibility (public fields only)

Age range
49 Years to 49 Years
Sex
Female
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Nov 25, 2024 (Actual)
Primary completion
Dec 16, 2025 (Actual)
Completion
Dec 16, 2025 (Actual)
Last update posted
Apr 9, 2026

2024 – 2025

United States locations

U.S. sites
2
U.S. states
2
U.S. cities
2
Facility City State ZIP Site status
Columbia University, Irving Medical Center New York New York 10032 —
Houston Methodist Houston Texas 77030 —

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT07095712, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Apr 9, 2026 · Synced Oct 22, 2026

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Open the official record

The complete public registry record, listed eligibility criteria, and available contact information for NCT07095712 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →