An Early Phase 1 interventional study of Fosigotifator (FGT/ABBV-CLS-7262) in Cree Leukoencephalopathy and Vanishing White Matter, sponsored by McGill University Health Centre/Research Institute of the McGill University Health Centre. Active, not recruiting at 1 site in Canada. Open to participants aged 4 Months and older. Per ClinicalTrials.gov, last updated 2025-12-30.
Sponsored by McGill University Health Centre/Research Institute of the McGill University Health Centre · Early Phase 1, Interventional, and Treatment
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.
39 studies on the registry are indexed under Leukoencephalopathies; 16 are open to participants now.
Browse Leukoencephalopathies studies →McGill University Health Centre/Research Institute of the McGill University Health Centre is the lead sponsor of 421 studies on the registry; 105 are open to participants now.
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Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
N=1 trial
Drug: Fosigotifator (FGT/ABBV-CLS-7262)
Drug page: Fosigotifator
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.
Patient survival or need for continuous ventilatory support at 2 years
Patient survival (yes/no) or need for continuous ventilatory support (yes/no) at 2 years
Time frame: 3 years
Plan to share: Undecided
No publications or documents are linked to this record.
Started Nov 2024 (actual). Primary completion is estimated for Nov 2027, 36 months after the start.
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This study is active, not recruiting, as verified in Dec 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Fosigotifator
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