Actively Recruiting
An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease
Led by Calico Life Sciences LLC · Updated on 2025-11-12
50
Participants Needed
5
Research Sites
346 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Males and females 6 months of age or older at screening
- Clinical diagnosis of Vanishing White Matter (VWM) disease by an experienced physician
- Molecular diagnosis confirming VWM disease
- MRI findings consistent with VWM disease
- Have a designated caregiver able to complete caregiver assessments
- Signed informed consent from participant or legally authorized representative
- Have at least one neurological symptom possibly related to VWM disease, or
- Unable to walk 10 or more steps with or without light support of 2 hands, or
- Cognitive impairment with scores below 50 on age-appropriate intelligence scales
- Pediatric participants in Cohort 4 must have neurological symptoms and motor abilities as defined, or be presymptomatic but genetically confirmed with high-risk mutations
- Sexually active males not surgically sterilized must agree to use contraception and avoid sperm donation during and 30 days after treatment
- Sexually active females of childbearing potential must use highly effective contraception and avoid egg donation during and 30 days after treatment
You will not qualify if you...
- Pediatric participants aged 6 months to under 6 years must not be on any respiratory support at screening
- Medication changes for VWM symptoms within 4 weeks before screening
- Uncontrolled seizure disorder within 6 months before screening
- Inability to complete study visits and procedures as judged by investigator
- Pregnant or breastfeeding adult females
- Receipt of any investigational treatment within 30 days or 5 half-lives before baseline
- Clinically significant laboratory or imaging abnormalities at screening
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 5 locations
1
Massachusetts General Hospital /ID# 270960
Boston, Massachusetts, United States, 02114
Actively Recruiting
2
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
3
University of Utah /ID# 255624
Salt Lake City, Utah, United States, 84112-5339
Actively Recruiting
4
McGill University Health Centre - Glen Site
Montreal, Quebec, Canada, H3H2L9
Actively Recruiting
5
Amsterdam UMC, locatie VUmc /ID# 270955
Amsterdam, North Holland, Netherlands, 1081 HV
Actively Recruiting
Research Team
C
Call Center - English
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
5
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