RECRUITING

An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease

Conditions

Vanishing White Matter Disease Neurodegenerative Diseases Nervous System Diseases Central Nervous System Brain Diseases Hereditary Central Nervous System Leukoencephalopathy with Vanishing White Matter

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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

Quick Facts

Study Start:2023-03-13

Study Completion:2029-11

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05757141

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:6 Months

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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 3. Cognitive criteria as assessed by the age-appropriate version of the Wechsler Intelligence Scale, with participants scoring \< 50 on specific indices; specific details can be provided by the Study physician. 6. Pediatric participants in Cohort 4 must meet both criteria a and b below, or criterion 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 as defined below: 7. All male participants who are sexually active and not surgically sterilized must agree to use an acceptable contraceptive method. Additionally, male participants must agree to not donate sperm during the study until 30 days after the final dose of study drug. 8. All female participants who are sexually active and of childbearing potential must agree to use a highly effective contraceptive method. Additionally, female participants must agree to not donate eggs during the study and for 30 days after the final dose of study drug.	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.

Inclusion Criteria

Exclusion 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
3. Cognitive criteria as assessed by the age-appropriate version of the Wechsler Intelligence Scale, with participants scoring \< 50 on specific indices; specific details can be provided by the Study physician.
6. Pediatric participants in Cohort 4 must meet both criteria a and b below, or criterion 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 as defined below:
7. All male participants who are sexually active and not surgically sterilized must agree to use an acceptable contraceptive method. Additionally, male participants must agree to not donate sperm during the study until 30 days after the final dose of study drug.
8. All female participants who are sexually active and of childbearing potential must agree to use a highly effective contraceptive method. Additionally, female participants must agree to not donate eggs during the study and for 30 days after the final dose of study drug.

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.

Contacts and Locations

Study Contact

Call Center - English

CONTACT

1-833-250-9660

vwminfo@mylocalstudy.com

Principal Investigator

ABBVIE INC.

STUDY_DIRECTOR

AbbVie

Study Locations (Sites)

Massachusetts General Hospital

Boston, Massachusetts, 02114

United States

University of Utah /ID# 255624

Salt Lake City, Utah, 84112-5339

United States

Collaborators and Investigators

Sponsor: AbbVie

ABBVIE INC., STUDY_DIRECTOR, AbbVie

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-03-13

Study Completion Date2029-11

Study Record Updates

Study Start Date2023-03-13

Study Completion Date2029-11

Terms related to this study

Keywords Provided by Researchers

Neurodegenerative Diseases
Nervous System Diseases
Central Nervous System Brain Diseases
Hereditary Central Nervous System
Leukoencephalopathy with Vanishing White Matter

Additional Relevant MeSH Terms

Vanishing White Matter Disease