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
- 6
- Locations
- 2
- Primary Endpoint
- Clinical Functioning
Study Overview
Brief Summary
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.
Detailed Description
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 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
Outcomes
Primary Outcomes
Clinical Functioning
Time Frame: Baseline to 12 months
Change from baseline at 12-months post nL-TARDB-006 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-006 administration in survival status
Disease Biomarkers
Time Frame: Baseline to 12 months
Change from baseline at 12-months post nL-TARDB-006 administration in serum/plasma and CSF neurofilament light chain levels
Secondary Outcomes
No secondary outcomes reported