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Research Study for Single-Patient Treatment of Cree Leukoencephalopathy/Vanishing White Matter Disease

NCT ID: NCT07272525

Last Updated: 2025-12-30

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

EARLY_PHASE1

Total Enrollment

1 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-11-14

Study Completion Date

2027-12-31

Brief Summary

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Cree Leukoencephalopathy (CLE) is a rare and fatal neurodegenerative disorder predominantly affecting the Cree population in Northern Quebec. Characterized by progressive white matter degeneration, this condition leads to severe neurological impairment and decline, leading to premature death. Despite its significant impact on the affected population, there are currently no effective treatments for CLE. CLE is caused by a single founder pathogenic variant in the EIF2B5 gene and is therefore allelic to VWM.

Fosigotifator (FGT, ABBV-CLS-7262) has been developed and its safety and efficacy are currently being studied in a multi-center Phase 1b/2 clinical trial for Vanishing White Matter (VWM) by Calico in collaboration with AbbVie.

This study aims to provide under compassionate use program access to an investigational drug (FGT) for a patient diagnosed with CLE/VWM disease which has no treatment options currently available. The study will also evaluate the risk/benefit of FGT in slowing or halting the progression of white matter degeneration in a patient with CLE. By targeting the underlying pathophysiological mechanisms of white matter damage, FGT is expected to alleviate neurological symptoms and improve the quality of life for the patient. The outcomes of this study could provide critical insights into the disease's management and pave the way for the development of targeted therapies, ultimately offering hope to a population with limited treatment options.

Detailed Description

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Conditions

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Cree Leukoencephalopathy Vanishing White Matter

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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N=1 trial

N=1 trial

Group Type EXPERIMENTAL

Fosigotifator (FGT/ABBV-CLS-7262)

Intervention Type DRUG

This study aims to provide under compassionate use program access to an investigational drug (FGT) for a patient diagnosed with Cree Leukoencephalopathy (CLE)/Vanishing White Matter (VWM) which has no treatment options currently available. The study will also evaluate the risk/benefit of FGT in slowing or halting the progression of white matter degeneration in a patient with CLE. By targeting the underlying pathophysiological mechanisms of white matter damage, FGT is expected to alleviate neurological symptoms and improve the quality of life for the patient. The outcomes of this study could provide critical insights into the disease's management and pave the way for the development of targeted therapies, ultimately offering hope to a population with limited treatment options.

Interventions

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Fosigotifator (FGT/ABBV-CLS-7262)

This study aims to provide under compassionate use program access to an investigational drug (FGT) for a patient diagnosed with Cree Leukoencephalopathy (CLE)/Vanishing White Matter (VWM) which has no treatment options currently available. The study will also evaluate the risk/benefit of FGT in slowing or halting the progression of white matter degeneration in a patient with CLE. By targeting the underlying pathophysiological mechanisms of white matter damage, FGT is expected to alleviate neurological symptoms and improve the quality of life for the patient. The outcomes of this study could provide critical insights into the disease's management and pave the way for the development of targeted therapies, ultimately offering hope to a population with limited treatment options.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

The patient meets the following criteria:

* Molecularly confirmed diagnosis of CLE
* Pre-symptomatic or early symptomatic patient
* Signed informed consent from the Legal Guardians/caregivers (parents)

Exclusion Criteria

N/A
Minimum Eligible Age

4 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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McGill University Health Centre/Research Institute of the McGill University Health Centre

OTHER

Sponsor Role lead

Responsible Party

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Genevieve Bernard

MD, MSc, FRCPc

Responsibility Role PRINCIPAL_INVESTIGATOR

Locations

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McGill University Health Centre

Montreal, Quebec, Canada

Site Status

Countries

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Canada

Other Identifiers

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2025-10780 (CLE-ABBV-CLS-7262)

Identifier Type: -

Identifier Source: org_study_id