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VCA-894A · 1 trial · 2 indications
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...
VCA-894A is an investigational small molecule being developed for the treatment of Charcot Marie Tooth Disease (CMT), a group of inherited neurological disorders affecting the peripheral nerves. It is specifically being studied for axonal type 2S (CMT2S), a subtype of the disease. The drug is currently in Phase 1 clinical development.
VCA-894A targets IGHMBP2, a protein involved in DNA and RNA metabolism. Mutations in the IGHMBP2 gene are associated with CMT2S, a form of Charcot Marie Tooth Disease. By targeting this protein, the drug aims to address the underlying genetic cause of the condition.
VCA-894A is being developed by Vanda Pharmaceuticals Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol VNDA. The company is conducting clinical research on this drug for the treatment of Charcot Marie Tooth Disease.
VCA-894A is in Phase 1 clinical development. It is an investigational drug and has not yet been approved by regulatory authorities. The drug has received Orphan Drug and Rare Pediatric Disease designations from the FDA for its intended use in Charcot Marie Tooth Disease.
VCA-894A is being studied in a Phase 1 clinical trial with the identifier NCT07223632. The trial is titled "Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient" and is active but not recruiting. It is an uncontrolled, open-label study enrolling one female patient in the United States.