An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease
Recruiting
Phase 1
Phase 2
Interventional Study
Vanishing White Matter Disease
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 6 Months and older
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 50 (estimated)
- Sponsor
- Calico Life Sciences LLC · Industry
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About This Trial
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 assessmen…
Trial Locations
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Eligibility Criteria
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…
Contacts