Open-Label Phase 1b2 Study of Fosigotifator in Adults and Children With Vanishing White Matter Disease Evaluating Safety, Tolerability, and Effects of Oral Fosigotifator

What this Trial Is About

Researchers are evaluating Fosigotifator, an investigational oral drug, for treating Vanishing White Matter VWM disease in adult, pediatric, and infant participants. This open-label, multiple cohort Phase 1b2 study aims to assess the drugs safety, tolerability, pharmacokinetics, and exploratory efficacy over a 201-week period. Participants are grouped into cohorts based on age, ranging from adults 18 years and older to children as young as 6 months. All participants receive Fosigotifator orally throughout the study. The study includes multiple cohorts adults, adolescents, younger children, and infants. Each cohort undergoes treatment and monitoring according to the protocol. Throughout the study, participants attend regular visits for medical assessments, blood tests, questionnaires, and monitoring for side effects. Researchers evaluate treatment-emergent adverse events, vital signs, ECG changes, clinical lab tests, and changes in mental health using the Columbia-Suicide Severity Rating Scale. Pharmacokinetic measures of Fosigotifator plasma concentration are tracked up to approximately 96 weeks, and MRI changes are assessed up to about 192 weeks. The total study duration extends up to 201 weeks, with ongoing safety and efficacy monitoring.

An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease