Personalized Antisense Oligonucleotide Therapy for A Single Patient With CHCHD10 ALS (nL18576)

Part of paid clinical trials in Rochester, Minnesota.

Sponsor
n-Lorem Foundation
Study ID
NCT07423494
Phase
PHASE1/PHASE2
Status
Not Yet Recruiting

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Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • nL-CHCHD-001 — DRUG
    Personalized antisense oligonucleotide

Study Details

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

Key Dates

Start date
Mar 31, 2026
Status verified
Feb 2026
Primary completion
Mar 31, 2027
Completion
Mar 31, 2027

Study Design

Enrollment
1 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Open Label

Primary Outcome Measure

Clinical Functioning [ Time Frame: Baseline to 12 Months ]

Locations (1)

FacilityCityStateZIPSite coordinators
Mayo ClinicRochesterMinnesota55905-

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By research site
Mayo Clinic· Rochester, MN

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