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

Enrolling by invitation · Phase 1/Phase 2

Conditions studied: Charcot-Marie-Tooth Disease Type 2D

In brief

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

Key facts

Study ID

NCT07226297

Run by

n-Lorem Foundation

People needed

1

Starts

2025-10-27

Expected to finish

2027-10-01

Last updated by the study team

2025-11-10

Who can join

Age: 13 and older. Sex: female. Healthy volunteers: not accepted.

You may qualify if…

• Informed consent/assent provided by the participant (when appropriate), and/or the participant's parent(s) or legally authorized representative(s).
• Ability to travel to the study site and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records.
• Genetically confirmed GARS1 genetic variant

You may not qualify if…

• Participant has any condition that, in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures.

Where it is running

• UTHealth Houston — Houston, Texas, United States

Full record on ClinicalTrials.gov

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