Trials / Active Not Recruiting
Active Not RecruitingNCT06977451
Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS
An Open-Label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to an Arg15Leu Pathogenic Variant in CHCHD10
- Status
- Active Not Recruiting
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 1 (actual)
- Sponsor
- n-Lorem Foundation · Academic / Other
- Sex
- All
- Age
- 63 Years – 63 Years
- Healthy volunteers
- Not accepted
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 CHCHD10
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | nL-CHCHD-001 | Personalized Antisense Oligonucleotide |
Timeline
- Start date
- 2024-06-24
- Primary completion
- 2026-06-01
- Completion
- 2026-06-01
- First posted
- 2025-05-18
- Last updated
- 2025-07-25
Locations
1 site across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT06977451. Inclusion in this directory is not an endorsement.