An Open-label Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to TARDBP (TDP-43) Genetic Mutation
Trial Snapshot
- Phase
- Phase 1
- Status
- Enrolling By Invitation
- Sponsor
- n-Lorem Foundation
- Enrollment
- 1
- Locations
- 1
- Primary Endpoint
- Clinical Functioning
Study Overview
Brief Summary
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.
Detailed Description
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 Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Sex
- All
- Accepts Healthy Volunteers
- No
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
Arms & Interventions
Open Label
Intervention: nL-TARDB-002 (Drug)
Outcomes
Primary Outcomes
Clinical Functioning
Time Frame: Baseline to 12 months
Change from baseline at 12-months post nL-TARDB-002 administration in scores on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R).
Survival
Time Frame: Baseline to 12 months
Change from baseline at 12-months post nL-TARDB-002 administration in survival status
Disease Biomarkers
Time Frame: Baseline to 12 months
Change from baseline at 12-months post nL-TARDB-002 administration in serum/plasma and CSF neurofilament light chain levels
Secondary Outcomes
No secondary outcomes reported