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
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…
Interventions
- DrugFosigotifator
Oral Use
Locations (5)
- Massachusetts General Hospital /ID# 270960Boston, Massachusetts
- Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania
- University of Utah /ID# 255624Salt Lake City, Utah
- McGill University Health Centre - Glen SiteMontreal, Quebec
- Amsterdam UMC, locatie VUmc /ID# 270955Amsterdam, North Holland