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A Phase 1b/2 Open-label Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Exploratory Efficacy of Fosigotifator in Adults and Children With Vanishing White Matter Disease
Led by Calico Life Sciences LLC · Updated on 2026-06-02
50
Participants Needed
5
Research Sites
104 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating Fosigotifator, an investigational oral drug, for treating Vanishing White Matter disease in adults, children, and infants. This is an open-label study lasting 201 weeks, including multiple groups based on age. The study aims to assess safety, tolerability, pharmacokinetics, and exploratory efficacy for participants with this disease. Participants are divided into cohorts by age: adults 18 years and older, children aged 12 to less than 18 years, children 6 to less than 12 years, and children 6 months to less than 6 years. All participants receive Fosigotifator orally. The study includes regular visits and ongoing assessments over nearly four years. During the study, participants will have medical exams, blood tests, questionnaires, and evaluations for side effects. Researchers will monitor vital signs, ECGs, lab tests, and brain MRI changes. Drug levels in the blood and cognitive safety measures are also tracked. The study involves close monitoring from baseline to about week 197 to understand the drug's effects and participant safety.
CONDITIONS
Brief 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 aged 6 months or older at screening
- Diagnosed with Vanishing White Matter disease by a physician experienced with this condition
- Molecular diagnosis and MRI findings consistent with Vanishing White Matter disease
- Have a caregiver able to complete caregiver assessments
- Signed informed consent by participant or legally authorized representative
- Meet one of the following: history of at least one neurological symptom related to the disease, inability to walk 10 or more steps with or without light support, or cognitive score below 50 on specific tests
- Pediatric participants in youngest cohort must meet additional specific motor or genetic risk criteria
- Sexually active males and females must agree to use contraceptive methods and avoid sperm or egg donation during and for 30 days after the study
You will not qualify if you...
- Pediatric participants aged 6 months to less than 6 years who use any form of respiratory support at screening
- Changes in medication for Vanishing White Matter disease symptoms within 4 weeks before screening
- Seizure disorder not well controlled within 6 months prior to screening
- Inability to complete study visits and procedures as judged by investigator
- Adult females who are pregnant, breastfeeding, or providing breast milk
- Treatment with other investigational therapies 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
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to approximately 96 weeks
Participants receive Fosigotifator orally as the study treatment.
Visits scheduled regularly during treatment up to Week 96
Duration - Up to approximately Week 197 from baseline
Participants continue to be monitored for safety and efficacy after treatment ends.
Visits scheduled periodically up to Week 197
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
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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
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5
Amsterdam UMC, locatie VUmc /ID# 270955
Amsterdam, North Holland, Netherlands, 1081 HV
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Research Team
C
Call Center - English
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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