NCT05757141
Brief summary
Fosigotifator is an investigational drug being researched for the treatment of Vanishing White Matter disease (VWM) in adult and pediatric subjects. This is a 96-week, open-label, multiple cohort study enrolling adults and pediatric subjects with Vanishing White Matter disease who are 6 years or older. Subjects will attend regular visits during the course of the study and complete medical assessments, blood tests, checking for side effects, and completing questionnaires.
Interventional study
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Please note, participation in the clinical trial is open to patients with the disease or condition under investigation. |
Age:
6 Years and older.
Inclusion Criteria:
1. Males and females ≥6 years 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 subject, or from a legally
authorized representative (LAR) if subject is incapable to consent themselves.
5. Subjects must meet at least one of the following functional criteria:
1. Motor criteria defined as inability to walk 10 or more steps with or without
light support of 2 hands
2. Cognitive criteria as assessed by the age-appropriate version of the Wechsler
Intelligence Scale, with subjects scoring < 50 on specific indices; specific
details can be provided by the Study physician.
6. All male subjects who are sexually active and not surgically sterilized must agree
to use an acceptable contraceptive method. Additionally, male subjects must agree to
not donate sperm during the study until 30 days after the final dose of study drug.
7. All female subjects who are sexually active and of childbearing potential must agree
to use a highly effective contraceptive method. Additionally, female subjects must
agree to not donate eggs during the study and for 30 days after the final dose of
study drug.
Exclusion Criteria:
1. Changes in medication use for the management of VWM disease symptoms within the 4
weeks preceding Screening.
2. Seizure disorder not considered adequately controlled by the investigator within the
6 months preceding Screening.
3. Subject who, in the opinion of the investigator, is incapable of completing
study-required visits and procedures to assess primary and secondary endpoints.
4. Pregnant or breastfeeding.
5. Treatment with any other investigational treatment within 30 days or 5 half-lives
(whichever is longer) prior to Baseline.
6. Any clinically significant laboratory or imaging findings at Screening
