Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07223632 | Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient | PHASE1 | ACTIVE NOT_RECRUITING | 1 | — | — | May 13, 2025 | Mar 1, 2026 | Nov 3, 2025 | 1 | United States |
Safety will be assessed by determining the incidence, severity, and dose relationship of adverse events that are related to treatment with VCA-894A.
The Revised Upper Limb Module for Spinal Muscular Atrophy (RULM) scores range from a minimum of 0 to a maximum of 37 points, with higher scores indicating better upper limb function.
The Hammersmith Functional Motor Scale - Expanded (HFMSE) scores range from a minimum of 0 to a maximum of 66 points, with higher scores indicating greater motor functioning.
| Arm | Type | Description |
|---|---|---|
| VCA-894A | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| VCA-894A | DRUG | intrathecal antisense oligonucleotide injection |
Inclusion Criteria: * Ability and acceptance to provide written informed consent. * Genetically confirmed diagnosis of CMT2S with confirmed IGHMBP2 intronic cryptic splice variant c. 1235+894C\>A. Exclusion Criteria: * Significant clinical deterioration of the patient's neurologic status, as judg...