Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D)
Part of paid clinical trials in Houston, Texas.
- Sponsor
- n-Lorem Foundation
- Study ID
- NCT07226297
- Phase
- PHASE1/PHASE2
- Status
- Enrolling By Invitation
Conditions
- Charcot-Marie-Tooth Disease Type 2D
Eligibility Criteria
- Sex
- FEMALE
- Age
- 13 Years - N/A
- Healthy Volunteers
- Not accepted
Interventions
- nL-GARS1-001 — DRUGPersonalized antisense oligonucleotide
Study Details
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 Dates
- First listed
- Nov 10, 2025
- Start date
- Oct 27, 2025
- Status verified
- Oct 2025
- Primary completion
- Oct 31, 2027
- Completion
- Oct 31, 2027
Study Design
- Enrollment
- 1 participants (estimated)
- Allocation
- NA
- Intervention model
- SINGLE_GROUP
- Primary purpose
- TREATMENT
Arms
- Experimental: Open Label
Primary Outcome Measure
Motor Skills [ Time Frame: Baseline to 24 months ]
Locations (1)
| Facility | City | State | ZIP | Site coordinators |
|---|---|---|---|---|
| UTHealth Houston | Houston | Texas | 77030 | - |
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