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

NCT07226297 · Status: ENROLLING_BY_INVITATION · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2025-11-10

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 Charcot-Marie-Tooth disease type 2D (CMT2D) due to a pathogenic, de novo deletion mutation in GARS1

Conditions

  • Charcot-Marie-Tooth Disease Type 2D

Interventions

DRUG

nL-GARS1-001

Personalized antisense oligonucleotide

Sponsors & Collaborators

  • The University of Texas Health Science Center, Houston

    collaborator OTHER

  • n-Lorem Foundation

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
13 Years
Sex
FEMALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-10-27
Primary Completion
2027-10-31
Completion
2027-10-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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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 NCT07226297 on ClinicalTrials.gov