What this means
A new study is recruiting people with a rare brain disease to test a drug called fosigotifator. It is still enrolling, so no results have been posted yet.
Plain-English summary written by an AI model from the registry listing.
- Status
- Recruiting
- Who can join
- Ages 6 Months and over
- Where
- 5 sites, including Massachusetts, Pennsylvania, Utah
The science
- Registered title
- An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease
- Trial ID
- NCT05757141 ↗
- Phase
- PHASE 1 / PHASE 2
- Study type
- interventional
- Conditions
- Vanishing White Matter Disease
- What is tested
- Fosigotifator
- Sponsor
- Calico Life Sciences LLC
- Ages
- 6 Months to no maximum
- Dates
- First posted Mar 7, 2023 · updated Sep 24, 2026
Registry 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.
Sites (5)
- Massachusetts General Hospital /ID# 270960, Boston, Massachusetts, United States
- Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
- University of Utah /ID# 255624, Salt Lake City, Utah, United States
- McGill University Health Centre - Glen Site, Montreal, Quebec, Canada
- Amsterdam UMC, locatie VUmc /ID# 270955, Amsterdam, North Holland, Netherlands