Cree LeukoencephalopathyEarly Phase 1NCT07272525
Research Study for Single-Patient Treatment of Cree Leukoencephalopathy/Vanishing White Matter Disease
A Early Phase 1 study testing Fosigotifator (FGT/ABBV-CLS-7262) in people with Cree Leukoencephalopathy and Vanishing White Matter, run by McGill University Health Centre/Research Institute of the McGill University Health Centre at 1 site in Canada.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 4 months and older
- Conditions the study is for
- Cree Leukoencephalopathy, Vanishing White Matter
- What is being tested?
- Fosigotifator (FGT/ABBV-CLS-7262)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteCanada
- Study dates
- 14 Nov 2024to 31 Dec 2027
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- N/A
Trial locations and contacts
1 site in 1 country.
About this study
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.
- Conditions
- Cree Leukoencephalopathy, Vanishing White Matter
- Being tested
- Fosigotifator (FGT/ABBV-CLS-7262)
- Medical subject headings
- Leukoencephalopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fosigotifator (FGT/ABBV-CLS-7262)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.
| Study group | Role | What they receive |
|---|---|---|
| N=1 trial | Experimental | N=1 trial Receives: Drug: Fosigotifator (FGT/ABBV-CLS-7262) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2025
- Study start14 Nov 2024
- Primary completion30 Nov 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cree LeukoencephalopathyStudies of the same condition, open and under way.
- More trials run by McGill University Health Centre/Research Institute of the McGill University Health CentreThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (estimated)
Sponsor and investigators
- Lead sponsor
- McGill University Health Centre/Research Institute of the McGill University Health Centre
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Genevieve Bernard, McGill University Health Centre/Research Institute of the McGill University Health Centre
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07272525
- Sponsor's protocol ID
- 2025-10780 (CLE-ABBV-CLS-7262)
- Other IDs
- 2025
- Status last verified
- Dec 2025
- First submitted
- 11 Oct 2024
- Last updated
- 30 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 30 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Research Study for Single-Patient Treatment of Cree Leukoencephalopathy/Vanishing White Matter Disease
- ClinicalTrials.gov ID
- NCT07272525
- Acronym / sponsor ID
- 2025-10780 (CLE-ABBV-CLS-7262)
- Conditions
- Cree Leukoencephalopathy, Vanishing White Matter
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 1 participants (estimated)
- Lead sponsor
- McGill University Health Centre/Research Institute of the McGill University Health CentreUniversity, hospital or other
- Study start
- 14 Nov 2024 (actual)
- Primary completion
- 30 Nov 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 9 Dec 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| N=1 trial | Experimental | Drug: Fosigotifator (FGT/ABBV-CLS-7262)N=1 trial |
Who may qualify
- Ages
- 4 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Molecularly confirmed diagnosis of CLE
- Pre-symptomatic or early symptomatic patient
- Signed informed consent from the Legal Guardians/caregivers (parents)
Key exclusion criteria (1 listed)
- N/A
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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. | Drug | N=1 trial |
Primary outcome
Patient survival or need for continuous ventilatory support at 2 years
Time frame: 3 years
Patient survival (yes/no) or need for continuous ventilatory support (yes/no) at 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Dec 2025.
- Individual participant dataSharing planned: Undecided
Related trials for Cree Leukoencephalopathy
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Single Patient Investigational Treatment for Cree Leukoencephalopathy
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Fosigotifator (FGT/ABBV-CLS-7262) in people with Cree Leukoencephalopathy and Vanishing White Matter, run by McGill University Health Centre/Research Institute of the McGill University Health Centre at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 30 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Patient survival or need for continuous ventilatory support at 2 years (time frame: 3 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Dec 2025.