Personalized Antisense Oligonucleotide Therapy for Participants With TARDBP ALS

Phase 2Enrolling By InvitationNCT07743268

n-Lorem Foundation6 enrolled

Overview

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.

This is an interventional study to evaluate the safety and efficacy of treatment with an individualized antisense oligonucleotide (ASO) treatment in participants with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Interventions

nL-TARDB-006DRUG

Personalized antisense oligonucleotide

Eligibility

Sex: ALL

Medical Language ↔ Plain English

Inclusion Criteria: * Informed consent/assent provided by the participant (when appropriate), and/or participants parent(s) or legally authorized representative(s) * Ability to travel to the study site and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records * Genetically confirmed neurological disorder Exclusion Criteria: * Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures * Use of an investigational medication within less than 5 half-lives of the drug at enrollment

Locations (2)

Washington University

St Louis, Missouri, United States

Columbia University, Irving Medical Center

New York, New York, United States

Outcomes

Primary Outcomes

Clinical Functioning

Change from baseline at 12-months post nL-TARDB-006 administration in scores on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R).