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

Calico Life Sciences LLC

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

Age range: 0+ years
Sex: All
Healthy volunteers: No

Detailed criteria

Inclusion Criteria: 1. 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. 2. Subject must have VWM disease defined as: * 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 mole…

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

Summary

Eligibility

Interventions

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