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Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS

NCT06392126 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2025-08-05

No results posted yet for this study

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

  • Mayo Clinic

    collaborator OTHER

  • n-Lorem Foundation

    lead OTHER

Principal Investigators

  • Björn E. Oskarsson, M.D. · Mayo Clinic

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
48 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-04-16
Primary Completion
2026-04-30
Completion
2026-04-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06392126 on ClinicalTrials.gov