Amyotrophic Lateral Sclerosis (ALS)GeneticsRare diseaseDrugPhase 1, Phase 2
What this study is
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.
What's being tested
- nL-TARDB-006
Intervention · DRUG
Personalized antisense oligonucleotide
Study details
- Official title
- An Open-label Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to TARDBP (TDP-43) Genetic Mutation
- Sponsor
- n-Lorem Foundation
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Enrolling
- 6 people
- Starts
- 2026-02-19
- Est. completion
- 2028-02
- Design
- Open-label, single group, Phase 1, Phase 2
- Record
- View on ClinicalTrials.gov ↗
Who can join
Sex ALLHealthy volunteers Not acceptedYou may qualify if
- 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
You can't join if
- 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
Sign in and we'll check these against your records.Full criteria on ClinicalTrials.gov ↗
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1
Study locations (2)
2 openWashington University
St Louis, Missouri, United States
Columbia University, Irving Medical Center
New York, New York, United States
Study team
Sponsor
n-Lorem Foundation
Study ID NLF-TARDB-006-A
Have questions? The record lists no central contact. Use Consider me and the site team can reach you, or find them through ClinicalTrials.gov ↗.
Frequently asked questions
The sponsor hasn't published answers to common questions for this trial.