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An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease | Clinical Trials | Clareo Health

RECRUITINGPHASE1/PHASE2

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

NCT05757141•Calico Life Sciences LLC

View on ClinicalTrials.gov

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.

Eligibility

Age

0 - No limit years

Sex

ALL

Healthy Volunteers

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) presentation consistent with VWM disease.
•3. Have a designated caregiver who is able to complete the respective caregiver-centered assessments.
•4. Signed and dated informed consent provided by the participant, or from a legally authorized representative (LAR) if participant is incapable to consent themselves.
•5. Participants must meet criteria (a) and at least one of the following functional criteria (b or c):
•1. Medical history of at least 1 neurological symptom that is assessed by the investigator as having a reasonable possibility of being related to VWM disease.
•2. Motor criteria defined as inability to walk 10 or more steps with or without light support of 2 hands
+8 more criteria

Exclusion Criteria

•1. Pediatric participants \>= 6 months and \< 6 years of age must not be on any form of respiratory support at the time of Screening.
•2. Changes in medication use for the management of VWM disease symptoms within the 4 weeks preceding Screening.
•3. Seizure disorder not considered adequately controlled by the investigator within the 6 months preceding Screening.
•4. Participant who, in the opinion of the investigator, is incapable of completing study-required visits and procedures to assess primary and secondary endpoints.
•5. Adult female participants who are pregnant, breastfeeding or providing breast milk.
•6. Treatment with any other investigational treatment within 30 days or 5 half-lives (whichever is longer) prior to Baseline.
•7. Any clinically significant laboratory or imaging findings at Screening.

Locations (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

Key Dates

Start Date

March 13, 2023

Primary Completion Date

November 1, 2027

Completion Date

November 1, 2029

Last Updated

November 12, 2025

Enrollment

ESTIMATED participants

Conditions

Vanishing White Matter Disease

Interventions

Fosigotifator

DRUG

Contacts

Call Center - English

CONTACT

1-833-250-9660 vwminfo@mylocalstudy.com

The Myelin Disorders Biorepository Project

NCT03047369

LeukodystrophyWhite Matter Disease+64 more

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: November 12, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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