Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS
NCT06977451 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 1
Last updated 2025-07-25
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
- DRUG
-
nL-CHCHD-001
Personalized Antisense Oligonucleotide
Sponsors & Collaborators
- collaborator OTHER
-
n-Lorem Foundation
lead OTHER
Study Design
- Allocation
- NA
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SINGLE_GROUP
Eligibility
- Min Age
- 63 Years
- Max Age
- 63 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2024-06-24
- Primary Completion
- 2026-06-30
- Completion
- 2026-06-30
- FDA Drug
- Yes
Countries
- United States
Study Locations
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