Single Patient Investigational Treatment for Cree Leukoencephalopathy

NCT07300397 | Active Not Recruiting DMC

• 1 other identifier: CLE02-ABBV-CLS-7262
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Cree Leukoencephalopathy

Keywords

N=1; Cree Leukoencephalopathy; Fosigotifator

Outcome Measures

Primary Outcomes (1)

• Time to death or permanent ventilation

  Time to death or permanent ventilation

  From enrollment to 2 years

Study Arms (1)

Single patient trial

EXPERIMENTAL

Fosigotifator

Drug: Fosigotifator

Interventions

Fosigotifator DRUG

Fosigotifator

Single patient trial

Eligibility Criteria

• Age: 1 Month+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• N/A

Sponsors & Collaborators

• McGill University Health Centre/Research Institute of the McGill University Health Centre: lead

Study Sites (1)

McGill University Health Centre

Montreal, Quebec, H4A3J1, Canada

Location

MeSH Terms

Conditions

Leukoencephalopathies

Condition Hierarchy (Ancestors)

Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD, MSc, FRCPc

Study Record Dates

• First Submitted: November 26, 2025
• First Posted: December 23, 2025
• Study Start: December 3, 2025
• Primary Completion (Estimated): January 3, 2029
• Study Completion (Estimated): January 3, 2029
• Last Updated: January 16, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1)