clinical trial · NCT05757141
An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease
Vanishing White Matter DiseaseFosigotifator
brief summary
Fosigotifator is an investigational drug being researched for the treatment of Vanishing White Matter disease in adult, pediatric and infant participants. This is a 201-week, open-label, multiple cohort study enrolling adults, pediatric and infant participants with Vanishing White Matter disease. Participants will attend regular visits during the course of the study and complete medical assessments, blood tests, questionnaires, and be evaluated for side effects.
started
Mar 13, 2023
primary completion
Nov 1, 2027
completion
Oct 1, 2035
last updated
Jul 1, 2026
official title
A Phase 1b/2 Open-label Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Exploratory Efficacy Following Fosigotifator Administration in Adult and Pediatric Subjects With Vanishing White Matter Disease
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol