Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D)
Public ClinicalTrials.gov record NCT07226297. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
An Open-label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Charcot-Marie-Tooth Type 2D Due to GARS1 Genetic Mutation
Study identification
- NCT ID
- NCT07226297
- Recruitment status
- Enrolling by invitation
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Lead sponsor
- n-Lorem Foundation
- Other
- Enrollment
- 1 participant
Conditions and interventions
Conditions
Interventions
- nL-GARS1-001 Drug
Drug
Eligibility (public fields only)
- Age range
- 13 Years and older
- Sex
- Female
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Oct 26, 2025
- Primary completion
- Sep 30, 2027
- Completion
- Sep 30, 2027
- Last update posted
- Nov 9, 2025
2025 – 2027
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| UTHealth Houston | Houston | Texas | 77030 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT07226297, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Nov 9, 2025 · Synced May 5, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT07226297 live on ClinicalTrials.gov.