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Antisense Oligonucleotide Therapy for ALS

Age: Any Age
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: n-Lorem Foundation
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

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.

Are You a Good Fit for This Trial?

This trial is for a single person diagnosed with ALS (Lou Gehrig's disease) who has a specific harmful change in the TARDBP gene. The treatment is personalized just for this individual.

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
I have a neurological disorder confirmed by genetic testing.

Exclusion Criteria

Participant has any condition that in the opinion of the Site Investigator would ultimately prevent the completion of study procedures
I have recently taken an investigational medication.

What Are the Treatments Tested in This Trial?

Interventions

  • nL-TARDB-002

Trial Overview

A custom-made antisense oligonucleotide drug called nL-TARDB-002, designed to target the participant’s unique genetic mutation causing their ALS, will be given and its effects monitored.

How Is the Trial Designed?

1

Treatment groups

Experimental Treatment

Group I: Open LabelExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

n-Lorem Foundation

Lead Sponsor

Trials
5
Recruited
5+

Thomas Jefferson University

Collaborator

Trials
475
Recruited
189,000+

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