NCT05757141

Brief Summary

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.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
50

participants targeted

Target at P50-P75 for phase_1

Timeline
43mo left

Started Mar 2023

Longer than P75 for phase_1

Geographic Reach
3 countries

5 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress48%
Mar 2023Nov 2029

First Submitted

Initial submission to the registry

February 23, 2023

Completed
12 days until next milestone

First Posted

Study publicly available on registry

March 7, 2023

Completed
6 days until next milestone

Study Start

First participant enrolled

March 13, 2023

Completed
4.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2027

Expected
2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2029

Last Updated

November 12, 2025

Status Verified

November 1, 2025

Enrollment Period

4.6 years

First QC Date

February 23, 2023

Last Update Submit

November 10, 2025

Conditions

Vanishing White Matter Disease

Keywords

Neurodegenerative DiseasesNervous System DiseasesCentral Nervous System Brain DiseasesHereditary Central Nervous SystemLeukoencephalopathy with Vanishing White Matter

Outcome Measures

Primary Outcomes (10)

  • 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

  • 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

Secondary Outcomes (6)

  • Incidence of Treatment-Emergent Adverse Events

    Baseline up to Approximately Week 197

  • Number of Participants with Change in Vital Signs

    Baseline up to approximately Week 197

  • Number of Participants with Change in ECG

    Baseline up to approximately Week 197

  • Number of Participants with Change in Clinical Laboratory Tests

    Baseline up to approximately Week 197

  • Change from Baseline in Columbia-Suicide Severity Rating Scale (C-SSRS)

    Baseline up to approximately Week 197

  • +1 more secondary outcomes

Study Arms (5)

Fosigotifator - Cohort 1

EXPERIMENTAL

Cohort 1: VWM adults \>= 18 years.

Drug: Fosigotifator

Fosigotifator - Cohort 1b

EXPERIMENTAL

Cohort 1b: VWM adults \>= 18 years.

Drug: Fosigotifator

Fosigotifator - Cohort 2

EXPERIMENTAL

Cohort 2: VWM children\>= 12 y and \<18 years.

Drug: Fosigotifator

Fosigotifator - Cohort 3

EXPERIMENTAL

Cohort 3: VWM children \>= 6 y and \<12 years.

Drug: Fosigotifator

Fosigotifator - Cohort 4

EXPERIMENTAL

Cohort 4: VWM children \>= 6 months and \<6 years.

Drug: Fosigotifator

Interventions

FosigotifatorDRUG

Oral Use

Also known as: ABBV-CLS-7262
Fosigotifator - Cohort 1Fosigotifator - Cohort 1bFosigotifator - Cohort 2Fosigotifator - Cohort 3Fosigotifator - Cohort 4

Eligibility Criteria

Age6 Months+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify 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.
  • 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 actively to weight bearing in that position.
  • +3 more criteria

You may not qualify 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 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

Massachusetts General Hospital /ID# 270960

Boston, Massachusetts, 02114, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

University of Utah /ID# 255624

Salt Lake City, Utah, 84112-5339, United States

RECRUITING

McGill University Health Centre - Glen Site

Montreal, Quebec, H3H2L9, Canada

RECRUITING

Amsterdam UMC, locatie VUmc /ID# 270955

Amsterdam, North Holland, 1081 HV, Netherlands

RECRUITING

Related Links

MeSH Terms

Conditions

LeukoencephalopathiesNeurodegenerative DiseasesNervous System Diseases

Condition Hierarchy (Ancestors)

Brain DiseasesCentral Nervous System Diseases

Study Officials

  • ABBVIE INC.

    AbbVie

    STUDY DIRECTOR

Central Study Contacts

Call Center - English

CONTACT

1-833-250-9660vwminfo@mylocalstudy.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 23, 2023

First Posted

March 7, 2023

Study Start

March 13, 2023

Primary Completion (Estimated)

November 1, 2027

Study Completion (Estimated)

November 1, 2029

Last Updated

November 12, 2025

Record last verified: 2025-11

Data Sharing

IPD Sharing
Will share

AbbVie is committed to responsible clinical trial data sharing. This includes access to anonymized, individual and trial-level data (analysis data sets), as well as other information.

Shared Documents
STUDY PROTOCOL, SAP
Time Frame
For details on when studies are available for sharing, visit https://vivli.org/ourmember/abbvie/
Access Criteria
To learn more about the process, or to submit a request, visit the following link https://www.abbvieclinicaltrials.com/hcp/data-sharing/
More information

Locations

CA(1)US(3)NL(1)