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| ID | Type | Description | Link |
|---|---|---|---|
| 2023-505704-30-00 | Other Identifier | EU CT |
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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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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Fosigotifator - Cohort 1 | Experimental | Cohort 1: VWM adults >= 18 years. |
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| Fosigotifator - Cohort 1b | Experimental | Cohort 1b: VWM adults >= 18 years. |
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| Fosigotifator - Cohort 2 | Experimental | Cohort 2: VWM children>= 12 y and <18 years. |
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| Fosigotifator - Cohort 3 | Experimental | Cohort 3: VWM children >= 6 y and <12 years. |
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| Fosigotifator - Cohort 4 | Experimental | Cohort 4: VWM children >= 6 months and <6 years. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Fosigotifator | Drug | Oral Use |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Treatment-Emergent Adverse Events | Number of participants with treatment-related adverse events as assessed by CTCAE v4.03 | Baseline up to Approximately Day 28 |
| Number of Participants with Change in Vital Signs | Number of Participants with Change in Vital Signs will be assessed. | Baseline up to Approximately Day 28 |
| Number of Participants with Change in ECG | Number of Participants with Change in ECG will be assessed. | Baseline up to Approximately Day 28 |
| Number of Participants with Change in Clinical Laboratory Tests | Number of participants with change in clinical laboratory tests will be assessed. | Baseline up to Approximately Day 28 |
| Change from Baseline in Columbia-Suicide Severity Rating Scale (C-SSRS) | The C-SSRS is a systematically administered instrument that reports the severity of both suicidal ideation and behavior, with a higher score denoting more severe suicidal ideation and behavior. | Baseline up to Approximately Day 28 |
| Plasma Concentration of Fosigotifator | Maximum Plasma Concentration [Cmax] | Baseline up to approximately Week 96 |
| Time to Cmax (Tmax) of Fosigotifator | Tmax of Fosigotifator | Baseline up to approximately Week 96 |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Treatment-Emergent Adverse Events | Number of patients with treatment-related adverse events as assessed by CTCAE v4.03 | Baseline up to Approximately Week 197 |
| Number of Participants with Change in Vital Signs |
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Inclusion Criteria:
Males and females >= 6 months of age at the time of Screening.
Have VWM disease defined as:
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):
Pediatric participants in Cohort 4 must meet both criteria a and b below, or criterion c:
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 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 (sponsor must be notified and provide approval prior to screening and enrolling a participant that meets eligibility 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 Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Call Center - English | Contact | 1-833-250-9660 | vwminfo@mylocalstudy.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Massachusetts General Hospital /ID# 270960 | Recruiting | Boston | Massachusetts | 02114 | United States | |
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| ID | Term |
|---|---|
| D056784 | Leukoencephalopathies |
| D019636 | Neurodegenerative Diseases |
| D009422 | Nervous System Diseases |
| ID | Term |
|---|---|
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| Area Under the Plasma Concentration-Time Curve (AUC0-24h) of Fosigotifator | AUC0-24h of Fosigotifator | Baseline up to approximately Week 96 |
| Trough Concentration (Ctrough) of Fosigotifator | Ctrough of Fosigotifator | Baseline up to approximately Week 96 |
| Terminal Elimination Half-Life (t1/2) of Fosigotifator | t1/2 of Fosigotifator | Baseline up to approximately Week 96 |
Number of Participants with Change in Vital Signs will be assessed.
| Baseline up to approximately Week 197 |
| Number of Participants with Change in ECG | Number of Participants with Change in ECG will be assessed. | Baseline up to approximately Week 197 |
| Number of Participants with Change in Clinical Laboratory Tests | Number of participants with change in clinical laboratory tests will be assessed. | Baseline up to approximately Week 197 |
| Change from Baseline in Columbia-Suicide Severity Rating Scale (C-SSRS) | The C-SSRS is a systematically administered instrument that reports the severity of both suicidal ideation and behavior, with a higher score denoting more severe suicidal ideation and behavior. | Baseline up to approximately Week 197 |
| Number of Participants with Change in Magnetic Resonance Imaging (MRI) | Change in Brain Magnetic Resonance Imaging (MRI) associated with adverse events. | Baseline up to approximately Week 192 |
| Children's Hospital of Philadelphia |
| Recruiting |
| Philadelphia |
| Pennsylvania |
| 19104 |
| United States |
| University of Utah /ID# 255624 | Recruiting | Salt Lake City | Utah | 84112-5339 | United States |
| McGill University Health Centre - Glen Site | Recruiting | Montreal | Quebec | H3H2L9 | Canada |
| Amsterdam UMC, locatie VUmc /ID# 270955 | Recruiting | Amsterdam | North Holland | 1081 HV | Netherlands |