Personalized Antisense Oligonucleotide Therapy for a Participant With TARDBP ALS

Phase 2Enrolling By InvitationNCT07703462

n-Lorem Foundation1 enrolled

Overview

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.

This is an interventional study to evaluate the safety and efficacy of treatment with an individualized antisense oligonucleotide (ASO) treatment in a single participant 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-002DRUG

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 (1)

Jefferson Health

Philadelphia, Pennsylvania, United States

Outcomes

Primary Outcomes

Clinical Functioning

Change from baseline at 12-months post nL-TARDB-002 administration in scores on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R).