Trials / Active Not Recruiting
Active Not RecruitingNCT07223632
Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient
The Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient With Confirmed IGHMBP2 Intronic Cryptic Splice Variant C. 1235+894C>A That is Amenable to Antisense Oligonucleotide (ASO)-Mediated Correction of IGHMBP2 Splicing
- Status
- Active Not Recruiting
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 1 (actual)
- Sponsor
- Vanda Pharmaceuticals · Industry
- Sex
- Female
- Age
- —
- Healthy volunteers
- Not accepted
Summary
This is an 'N of 1', open-label, single center study to evaluate the safety of therapy with VCA-894A, an ASO designed to rescue and restore the activity of IGHMBP2, when administered by intrathecal injection.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | VCA-894A | intrathecal antisense oligonucleotide injection |
Timeline
- Start date
- 2025-05-13
- Primary completion
- 2026-03-01
- Completion
- 2026-03-01
- First posted
- 2025-11-03
- Last updated
- 2025-11-03
Locations
1 site across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT07223632. Inclusion in this directory is not an endorsement.