Comparing 5 approaches for vanishing white matter disease
Official title: 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
- Phase 1
- 5 groups
- One site, in Montreal
- Recruiting
Interventions
- Medication
Fosigotifator
Oral Use
Canadian Sites (1)
1 of 1 recruiting
- Recruiting
McGill University Health Centre - Glen Site
Montreal, Quebec
Eligibility Criteria
See who this study is looking for25 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +Males and females \>= 6 months of age at the time of ScreeningScreeningThe checks done before joining, to see whether a study fits.Read more →.
- +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 consentInformed consentThe process of being told what taking part involves, then choosing freely.Read more → 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 investigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more → 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 CohortCohortA group of participants sharing a characteristic, followed together.Read more → 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 controlControl groupThe group a new treatment is measured against.Read more → 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 armsArmOne of the groups in a study, each receiving something different.Read more → actively to weight bearing in that position.
- +c. Presymptomatic and homozygous for Cree Leukoencephalopathy (EIF2B5 R195H) or other mutation with known imminent risk of significant clinical decline or death (sponsorSponsorThe organisation responsible for the study overall.Read more → must be notified and provide approval prior to screening and enrolling a participant that meets eligibilityEligibility criteriaThe full list of requirements for taking part in a study.Read more → with only this criterion).
- +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.
- +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.
Exclusion
- −Pediatric participants \>= 6 months and \< 6 years of age must not be on any form of respiratory support at the time of ScreeningScreeningThe checks done before joining, to see whether a study fits.Read more →.
- −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 investigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more → 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 endpointsSecondary endpointAdditional things measured alongside the main question.Read more →.
- −Treatment with any other investigational treatment within 30 days or 5 half-lives (whichever is longer) prior to BaselineBaselineYour starting measurements, taken before treatment begins.Read more →.
- −Any clinically significant laboratory or imaging findings at Screening.
- −Adult female participants who are pregnant, breastfeeding or providing breast milk.
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 1Phase 1The earliest stage of human testing, in a small group, focused on safety.Read more →/2 — a combined study that starts with early safety and continues into what the treatment does.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
Who receives what
There are 5 groups in this study.
Groups A, B, C, D and E receive Fosigotifator.
Registry label: A: Fosigotifator - Cohort 1 · B: Fosigotifator - Cohort 1b · C: Fosigotifator - Cohort 2 · D: Fosigotifator - Cohort 3 · E: Fosigotifator - Cohort 4
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].label
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
How the study is run
Groups are assigned by the study team using set rules, rather than by chance.
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
Who the study is looking for
The study lists a minimum age of 6 months, with no upper limit given.
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 50 people.
The study is currently expected to finish around October 2035.
The main measurement is taken over: BaselineBaselineYour starting measurements, taken before treatment begins.Read more → up to Approximately Day 28.
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Incidence of Treatment-Emergent Adverse EventsAdverse eventAny medical problem that happens during a study, whether or not the treatment caused it.Read more → — measured over BaselineBaselineYour starting measurements, taken before treatment begins.Read more → up to Approximately Day 28.
Number of Participants with Change in Vital Signs — measured over Baseline up to Approximately Day 28.
Number of Participants with Change in ECG — measured over Baseline up to Approximately Day 28.
Number of Participants with Change in Clinical Laboratory Tests — measured over Baseline up to Approximately Day 28.
The study lists 6 further main measurements.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT05757141 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About FosigotifatorDrug
Oral Use
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: a minimum age of 6 months.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
Groups are assigned by the study team using set rules, rather than by chance.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists a minimum age of 6 months, with no upper limit given.
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: BaselineBaselineYour starting measurements, taken before treatment begins.Read more → up to Approximately Day 28.
The study as a whole is currently expected to finish around 2035-10.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 50 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists one location: Montreal, Quebec, Canada.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
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 assessments, blood tests, questionnaires, and be evaluated for side effects.
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See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.