Clinical Trial

Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D)

Name: Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D) — clinical trial record (NCT07226297)
Creator: DataLookout
License: https://creativecommons.org/licenses/by/4.0/

Enrolling by Invitation Phase 1/2

View on ClinicalTrials.gov →

Summary

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Charcot-Marie-Tooth disease type 2D (CMT2D) due to a pathogenic, de novo deletion mutation in GARS1

Trial Details

NCT Number	NCT07226297
Lead Sponsor	n-Lorem Foundation Collaborators: The University of Texas Health Science Center, Houston
Conditions	Charcot-Marie-Tooth Disease Type 2D
Enrollment	1 participants
Start Date	2025-10-27
Primary Completion	2027-10 (estimated)
Study Completion	2027-10 (estimated)
Updated on ClinicalTrials.gov	2025-11-10