Trials / Recruiting
RecruitingNCT05757141
An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease
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
- Status
- Recruiting
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 50 (estimated)
- Sponsor
- Calico Life Sciences LLC · Industry
- Sex
- All
- Age
- 6 Months
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Fosigotifator | Oral Use |
Timeline
- Start date
- 2023-03-13
- Primary completion
- 2027-11-01
- Completion
- 2029-11-01
- First posted
- 2023-03-07
- Last updated
- 2025-11-12
Locations
5 sites across 3 countries: United States, Canada, Netherlands
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT05757141. Inclusion in this directory is not an endorsement.