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

Start date
Oct 2, 2024
Status verified
May 2025
Primary completion
Apr 30, 2026
Completion
Apr 30, 2026

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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By research site
Columbia University, Irving Medical Center· New York, NY

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