An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease
Trial Parameters
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.
Eligibility Criteria
Inclusion Criteria: 1. Males and females \>= 6 months of age at the time of Screening. 2. Have VWM disease defined as: 1. A clinical diagnosis by a physician experienced in the assessment of VWM disease; and 2. A molecular diagnosis of VWM disease, and 3. A magnetic resonance imaging (MRI) presentation consistent with VWM disease. 3. Have a designated caregiver who is able to complete the respective caregiver-centered assessments. 4. Signed and dated informed consent provided by the participant, or from a legally authorized representative (LAR) if participant is incapable to consent themselves. 5. Participants must meet criteria (a) and at least one of the following functional criteria (b or c): 1. Medical history of at least 1 neurological symptom that is assessed by the investigator as having a reasonable possibility of being related to VWM disease. 2. Motor criteria defined as inability to walk 10 or more steps with or without light support of 2 hands 3. Cognitive criteria as assess