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.
Males and females ≥18 y of age (Cohort 1 and Cohort 1b - adults), ≥12 y and <18 y of age (Cohort 2 - adolescents), ≥6 y and <12 y of age (Cohort 3 - children), and ≥6 mos and <6 y of age (Cohort 4 - children [≥1 y and <6 y of age] and infants [≥6 mos and <1 y of age]) at the time of Screening.
A clinical diagnosis by a physician experienced in the assessment of VWM disease, AND
A molecular diagnosis of VWM disease (Note: Subjects who can provide a verified laboratory report of previously performed VWM molecular diagnosis, including the specific mutation(s), will not be required to reconfirm diagnosis before Screening), AND
A magnetic resonance imaging (MRI) presentation consistent with VWM disease, as assessed by a neuroradiologist or by a physician experienced in the assessment of VWM disease except for presymptomatic homozygous carriers of Cree leukoencephalopathy (EIF2B5 R195H) or other mutation with known imminent risk of significant clinical decline or death (requires approval from Sponsor).
Pediatric subjects ≥6 mos and <6 y of age must not be on any form of respiratory support at the time of Screening.
Subject who has any clinically significant electrocardiogram (ECG) abnormalities, including QT interval corrected for heart rate using Fridericia's correction formula (QTcF) of >450 msec for adult males, >470 msec for adult females, or >450 msec for adolescents ≥12 y and <18 y of age (Cohort 2) and children ≥6 y and <12 y of age (Cohort 3), or >414 msec for infants and children ≥0.5 y and <6 y of age (Cohort 4).
Subject with current or history of abnormal screening laboratory or imaging results that, in the opinion of the Investigator, are indicative of any significant cardiac, endocrinologic, hematologic, hepatic, immunologic, infectious, metabolic, urologic, pulmonary, gastrointestinal, dermatologic, psychiatric, renal, neurologic, and/or other major disease (other than VWM) that would preclude administration of FGT or safe participation in the study Abnormal adrenal function, defined as confirmed abnormal random cortisol or ACTH at Screening (see Section 7.2.8 for cortisol and ACTH thresholds) Participants may undergo repeat cortisol and ACTH testing once, in order to establish normal adrenal function. If repeat cortisol and/or ACTH testing is abnormal, at the Investigator's discretion, the participant may undergo cosyntropin stimulation testing to establish normal adrenal function to meet eligibility criteria.
Subject who has suicidal ideation at the Screening Visit (V1). Prior medical history and/or Columbia-Suicide Severity Rating Scale (C-SSRS) may be used to inform the Investigator's decision.
Parallel
Fosigotifator
DrugOral Use
6
Treatment groupsSee each treatment group below.
Incidence of Treatment-Emergent Adverse Events
Number of participants with treatment-related adverse events as assessed by CTCAE v4.03
Number of Participants with Change in Vital Signs
Number of Participants with Change in Vital Signs will be assessed.
Number of Participants with Change in ECG
Number of Participants with Change in ECG will be assessed.
Number of Participants with Change in Clinical Laboratory Tests
Number of participants with change in clinical laboratory tests will be assessed.
Incidence of Treatment-Emergent Adverse Events
Number of patients with treatment-related adverse events as assessed by CTCAE v4.03
Number of Participants with Change in Vital Signs
Number of Participants with Change in Vital Signs will be assessed.
Number of Participants with Change in ECG
Number of Participants with Change in ECG will be assessed.
Number of Participants with Change in Clinical Laboratory Tests
Number of participants with change in clinical laboratory tests will be assessed.
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