Personalized Antisense Oligonucleotide for Participants With CHCHD10 ALS

Part of paid clinical trials in New York, New York.

Sponsor
n-Lorem Foundation
Study ID
NCT07095686
Phase
PHASE1/PHASE2
Status
Enrolling By Invitation

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 individual participants with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in CHCHD10

Key Dates

First listed
Jul 31, 2025
Start date
Oct 2, 2024
Status verified
Jul 2026
Primary completion
Apr 30, 2027
Completion
Apr 30, 2027

Study Design

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

Arms

  • Experimental: Open Label nL-CHCHD-001

Primary Outcome Measure

Clinical Functioning [ Time Frame: Baseline to 12 months ]

Locations (1)

FacilityCityStateZIPSite coordinators
Columbia University, Irving Medical CenterNew YorkNew York10032-

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Columbia University, Irving Medical Center· New York, NY

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