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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
50
Oral Use
Massachusetts General Hospital /ID# 270960
Boston, Massachusetts, United States
RECRUITINGChildren's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
RECRUITINGUniversity of Utah /ID# 255624
Salt Lake City, Utah, United States
RECRUITINGMcGill University Health Centre - Glen Site
Montreal, Quebec, Canada
RECRUITINGAmsterdam UMC, locatie VUmc /ID# 270955
Amsterdam, North Holland, Netherlands
RECRUITINGIncidence of Treatment-Emergent Adverse Events
Number of participants with treatment-related adverse events as assessed by CTCAE v4.03
Time frame: Baseline up to Approximately Day 28
Number of Participants with Change in Vital Signs
Number of Participants with Change in Vital Signs will be assessed.
Time frame: Baseline up to Approximately Day 28
Number of Participants with Change in ECG
Number of Participants with Change in ECG will be assessed.
Time frame: Baseline up to Approximately Day 28
Number of Participants with Change in Clinical Laboratory Tests
Number of participants with change in clinical laboratory tests will be assessed.
Time frame: Baseline up to Approximately Day 28
Change from Baseline in Columbia-Suicide Severity Rating Scale (C-SSRS)
The C-SSRS is a systematically administered instrument that reports the severity of both suicidal ideation and behavior, with a higher score denoting more severe suicidal ideation and behavior.
Time frame: Baseline up to Approximately Day 28
Plasma Concentration of Fosigotifator
Maximum Plasma Concentration \[Cmax\]
Time frame: Baseline up to approximately Week 96
Time to Cmax (Tmax) of Fosigotifator
Tmax of Fosigotifator
Time frame: Baseline up to approximately Week 96
Area Under the Plasma Concentration-Time Curve (AUC0-24h) of Fosigotifator
AUC0-24h of Fosigotifator
Time frame: Baseline up to approximately Week 96
Trough Concentration (Ctrough) of Fosigotifator
Ctrough of Fosigotifator
Time frame: Baseline up to approximately Week 96
Terminal Elimination Half-Life (t1/2) of Fosigotifator
t1/2 of Fosigotifator
Time frame: Baseline up to approximately Week 96
Incidence of Treatment-Emergent Adverse Events
Number of patients with treatment-related adverse events as assessed by CTCAE v4.03
Time frame: Baseline up to Approximately Week 197
Number of Participants with Change in Vital Signs
Number of Participants with Change in Vital Signs will be assessed.
Time frame: Baseline up to approximately Week 197
Number of Participants with Change in ECG
Number of Participants with Change in ECG will be assessed.
Time frame: Baseline up to approximately Week 197
Number of Participants with Change in Clinical Laboratory Tests
Number of participants with change in clinical laboratory tests will be assessed.
Time frame: Baseline up to approximately Week 197
Change from Baseline in Columbia-Suicide Severity Rating Scale (C-SSRS)
The C-SSRS is a systematically administered instrument that reports the severity of both suicidal ideation and behavior, with a higher score denoting more severe suicidal ideation and behavior.
Time frame: Baseline up to approximately Week 197
Number of Participants with Change in Magnetic Resonance Imaging (MRI)
Change in Brain Magnetic Resonance Imaging (MRI) associated with adverse events.
Time frame: Baseline up to approximately Week 192
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