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NCT07226297

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

Enrolling by Invitation
PHASE1Ages 13+InterventionalTreatment
n-Lorem Foundation
~1 participants
Updated 2025-11-10 on ClinicalTrials.gov
What's tested:nL-GARS1-001

At a glance

Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Motor Skills
Measured over Baseline to 24 months
+4 more outcomes measured

Conditions

Charcot-Marie-Tooth Disease Type 2D

Explore related trials

Charcot-Marie-Tooth Disease Type 2DGARS1 Gene

Where it's being run

1 sites across 1 states
Texas1

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

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Eligibility criteria

Inclusion

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.

Exclusion

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

What this trial measures

  • Motor SkillsBaseline to 24 months

    Change in fine motor skills from baseline to 12- and 24-months post nL-GARS1-001 administration as measured by the annualized rate of change in the Nine-Hole Peg Test (9-HPT)

  • Motor SkillsBaseline to 24 months

    Change in fine motor skills from baseline to 12- and 24-months post nL-GARS1-001 administration as measured by the annualized rate of change in the Box and Block Test (BBT)

  • Motor SkillsBaseline to 24 months

    Change in gross motor skills from baseline to 12- and 24-months post nL-GARS1-001 administration as measured by the annualized rate of change on Revised Upper Limb Module (RULM)

  • Motor SkillsBaseline to 24 months

    Change in gross motor skills from baseline to 12- and 24-months post nL-GARS1-001 administration as measured by the annualized rate of change on Hammersmith Functional Motor Scale - Expanded (HFMSE)

  • Motor SkillsBaseline to 24 months

    Change in gross motor skills from baseline to 12- and 24-months post nL-GARS1-001 administration as measured by the annualized rate of change on home videography assessment