Cree LeukoencephalopathyNCT07300397
Single Patient Investigational Treatment for Cree Leukoencephalopathy
A study testing Fosigotifator in people with Cree Leukoencephalopathy, 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 very rare and severe brain disease that mainly affects members of the Cree communities in Northern Quebec. It causes the white matter of the brain-the part that helps nerves communicate-to slowly break down. Read more about this study
- Who is it for?
- Ages 1 month and older
- Conditions the study is for
- Cree Leukoencephalopathy
- What is being tested?
- Fosigotifator
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteCanada
- Study dates
- 3 Dec 2025to 3 Jan 2029
- Phase
- Interventional
- 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 1 month and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- • Molecularly confirmed diagnosis of CLE
- Pre-symptomatic or early symptomatic patient
- Signed informed consent from the Legal Guardians/caregivers (parents)
- Must not be eligible for any actively enrolling trial of fosigotifator in CLE/VWM
- Must be at least 1 month of age at the Baseline visit
- Must weigh at least 5kg at the Baseline visit
Who cannot join
- N/A
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Single Patient Investigational Treatment for Cree Leukoencephalopathy (Vanishing White Matter Disease)
Cree Leukoencephalopathy (CLE) is a very rare and severe brain disease that mainly affects members of the Cree communities in Northern Quebec. It causes the white matter of the brain-the part that helps nerves communicate-to slowly break down. As the disease progresses, children develop serious neurological problems that worsen over time and, sadly, lead to early death. At the moment, there are no effective treatments for CLE. The disease is caused by a single genetic change in the EIF2B5 gene, the same gene involved in another related condition called Vanishing White Matter (VWM).
A new medication called fosigotifator (FGT, ABBV-CLS-7262) is currently being tested in an international clinical trial for VWM.
The goal of this study is to provide access to this investigational medication (FGT) for a patient with CLE/VWM for whom no other treatment options exist. The study will also look at whether the potential benefits of FGT outweigh the risks, and whether the drug might slow down or stop the brain's white matter from deteriorating. By targeting the underlying cause of the disease, FGT may help reduce neurological symptoms and improve the patient's quality of life.
Read the detailed description
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.
This study aims to provide access to an investigational drug (FGT) for a patient diagnosed with CLE/VWM disease for whom there are no other treatment options available. The study will also evaluate the risk/benefit of FGT in slowing or halting the progression of white matter degeneration in this 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.
- Conditions
- Cree Leukoencephalopathy
- Being tested
- Fosigotifator
- Keywords
- N=1, Cree Leukoencephalopathy, Fosigotifator
- Medical subject headings
- Leukoencephalopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FosigotifatorDrug
Fosigotifator
| Study group | Role | What they receive |
|---|---|---|
| Single patient trial | Experimental | Fosigotifator Receives: Drug: Fosigotifator |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to death or permanent ventilation
Time to death or permanent ventilation
Measured: From enrollment to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Dec 2025
- Study start3 Dec 2025
- Primary completion3 Jan 2029 (estimated)
- Study completion3 Jan 2029 (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
- Phase N/A
- 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
- NCT07300397
- Sponsor's protocol ID
- CLE02-ABBV-CLS-7262
- Status last verified
- Jan 2026
- First submitted
- 26 Nov 2025
- Last updated
- 16 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Single Patient Investigational Treatment for Cree Leukoencephalopathy (Vanishing White Matter Disease)
- ClinicalTrials.gov ID
- NCT07300397
- Acronym / sponsor ID
- CLE02-ABBV-CLS-7262
- Conditions
- Cree Leukoencephalopathy
- Keywords
- N=1, Cree Leukoencephalopathy, Fosigotifator
- 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
- 3 Dec 2025 (actual)
- Primary completion
- 3 Jan 2029 (estimated)
- Study completion
- 3 Jan 2029 (estimated)
- First posted
- 23 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 very rare and severe brain disease that mainly affects members of the Cree communities in Northern Quebec. It causes the white matter of the brain-the part that helps nerves communicate-to slowly break down. As the disease progresses, children develop serious neurological problems that worsen over time and, sadly, lead to early death. At the moment, there are no effective treatments for CLE. The disease is caused by a single genetic change in the EIF2B5 gene, the same gene involved in another related condition called Vanishing White Matter (VWM).
A new medication called fosigotifator (FGT, ABBV-CLS-7262) is currently being tested in an international clinical trial for VWM.
The goal of this study is to provide access to this investigational medication (FGT) for a patient with CLE/VWM for whom no other treatment options exist. The study will also look at whether the potential benefits of FGT outweigh the risks, and whether the drug might slow down or stop the brain's white matter from deteriorating. By targeting the underlying cause of the disease, FGT may help reduce neurological symptoms and improve the patient's quality of life.
Detailed description
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.
This study aims to provide access to an investigational drug (FGT) for a patient diagnosed with CLE/VWM disease for whom there are no other treatment options available. The study will also evaluate the risk/benefit of FGT in slowing or halting the progression of white matter degeneration in this 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single patient trial | Experimental | Drug: FosigotifatorFosigotifator |
Who may qualify
- Ages
- 1 month and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- • Molecularly confirmed diagnosis of CLE
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 |
|---|---|---|
| FosigotifatorFosigotifator | Drug | Single patient trial |
Primary outcome
Time to death or permanent ventilation
Time frame: From enrollment to 2 years
Time to death or permanent ventilation
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 16 Jan 2026.
- Individual participant dataSharing planned: No
Related trials for Cree Leukoencephalopathy
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Research Study for Single-Patient Treatment of Cree Leukoencephalopathy/Vanishing White Matter Disease
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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 study testing Fosigotifator in people with Cree Leukoencephalopathy, 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 6 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 16 Jan 2026. 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.
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?
Time to death or permanent ventilation (time frame: From enrollment to 2 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 16 Jan 2026.