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NCT07223632 · ClinicalTrials.gov registry record · Phase 1
Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient
A Phase 1 study of Neuromuscular Diseases (NMD) and Charcot Marie Tooth Disease (CMT), sponsored by Vanda Pharmaceuticals.
- Active
- Registry status
- Phase 1
- Development phase
- 1
- Enrollment target
- 1
- Study location
NCT07223632 is a Phase 1 study of Neuromuscular Diseases (NMD) and Charcot Marie Tooth Disease (CMT) that is active but no longer recruiting, run by Vanda Pharmaceuticals. The registered enrollment target is 1 participants, below the 858-participant average among 3 other Neuromuscular Diseases (NMD) trials with a reported enrollment target (100% lower). The trial reports 1 study location across 1 state.
The verdict
NCT07223632, a Phase 1 study of Neuromuscular Diseases (NMD) and Charcot Marie Tooth Disease (CMT), is active but no longer recruiting, sponsored by Vanda Pharmaceuticals.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 1 participants
- Enrollment target
- 1
- Study location
Study 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 Studied
Interventions
- DRUG VCA-894A
Study Locations (1)
Wisconsin
- Vanda Investigational Site - Madison
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 1 participants |
| Start Date | 2025-05-13 |
| Est. Completion | 2026-03 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT07223632
The ClinicalTrials.gov registry entry for NCT07223632 describes a study currently listed as active not recruiting, categorized as Phase 1. The registered enrollment target is 1 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 858-participant average among 3 other Neuromuscular Diseases (NMD) trials with a reported enrollment target (100% lower). The listed sponsor is Vanda Pharmaceuticals, which has 68 total studies on file at ClinicalTrials.gov.
The record links to 2 conditions, with Neuromuscular Diseases (NMD) appearing as the primary indexed condition, and to 1 intervention - of which VCA-894A is the first listed.
NCT07223632 reports 1 study location spanning 1 distinct geographic area - top geographies include Wisconsin.
Frequently Asked Questions
What is clinical trial NCT07223632 about?
NCT07223632 is a clinical study titled "Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient". 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.
What is the current status of trial NCT07223632?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 1 participants. The study started on 2025-05-13. Estimated completion is 2026-03.
What conditions does trial NCT07223632 study?
This clinical trial studies the following conditions: Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT).
What interventions are being tested in trial NCT07223632?
The interventions under investigation include: VCA-894A (DRUG).
Who is sponsoring clinical trial NCT07223632?
This trial is sponsored by Vanda Pharmaceuticals, which has 68 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT07223632 being conducted?
This trial has 1 study location across Wisconsin. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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