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New treatment option for Vanishing White Matter Disease
Official title An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease
ClinicalTrials.gov ID: NCT05757141
What this study is testing
What is Fosigotifator?
Fosigotifator is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for vanishing white matter disease.
Also referred to as ABBV-CLS-7262.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 and older
You may be able to join if
- Males and females \>= 6 months of age at the time of Screening.
- Have VWM disease defined as:
- A clinical diagnosis by a physician experienced in the assessment of VWM disease; and
- A molecular diagnosis of VWM disease, and
- A magnetic resonance imaging (MRI) presentation consistent with VWM disease.
You likely can't join if
- Pediatric participants \>= 6 months and \< 6 years of age must not be on any form of respiratory support at the time of Screening.
- Changes in medication use for the management of VWM disease symptoms within the 4 weeks preceding Screening.
- Seizure disorder not considered adequately controlled by the investigator within the 6 months preceding Screening.
- Participant who, in the opinion of the investigator, is incapable of completing study-required visits and procedures to assess primary and secondary...
- Adult female participants who are pregnant, breastfeeding or providing breast milk.
- Treatment with any other investigational treatment within 30 days or 5 half-lives (whichever is longer) prior to Baseline.
See the full eligibility criteria
- Males and females \>= 6 months of age at the time of Screening.
- Have VWM disease defined as:
- A clinical diagnosis by a physician experienced in the assessment of VWM disease; and
- A molecular diagnosis of VWM disease, and
- A magnetic resonance imaging (MRI) presentation consistent with VWM disease.
- Have a designated caregiver who is able to complete the respective caregiver-centered assessments.
- Signed and dated informed consent provided by the participant, or from a legally authorized representative (LAR) if participant is incapable to consent themselves.
- Participants must meet criteria (a) and at least one of the following functional criteria (b or c):
- 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.
- Motor criteria defined as inability to walk 10 or more steps with or without light support of 2 hands
- 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.
- Pediatric participants in Cohort 4 must meet both criteria a and b below, or criterion c:
- 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.
- Motor criteria as defined below: i. More than minimal head control as demonstrated by: While in prone position, the participant can lift his/her head and sustain the position for 10 seconds and bring his/her arms...
- 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...
- 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...
- Pediatric participants \>= 6 months and \< 6 years of age must not be on any form of respiratory support at the time of Screening.
- Changes in medication use for the management of VWM disease symptoms within the 4 weeks preceding Screening.
- Seizure disorder not considered adequately controlled by the investigator within the 6 months preceding Screening.
- Participant who, in the opinion of the investigator, is incapable of completing study-required visits and procedures to assess primary and secondary endpoints.
- Adult female participants who are pregnant, breastfeeding or providing breast milk.
- Treatment with any other investigational treatment within 30 days or 5 half-lives (whichever is longer) prior to Baseline.
- Any clinically significant laboratory or imaging findings at Screening.
The study team makes the final eligibility decision.
Where it's taking place
- Boston, Massachusetts, United States
- Philadelphia, Pennsylvania, United States
- Salt Lake City, Utah, United States
- Montreal, Quebec, Canada
- Amsterdam, North Holland, Netherlands
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 6 months and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Boston, Massachusetts, United States; Philadelphia, Pennsylvania, United States; Salt Lake City, Utah, United States; Montreal, Quebec, Canada; Amsterdam, North Holland, Netherlands. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.