Single Patient Investigational Treatment for Cree Leukoencephalopathy (NCT07300397)
Single Patient Investigational Treatment for Cree Leukoencephalopathy (Vanishing White Matter Disease)
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: McGill University Health Centre — Montreal, Quebec
Age range: 1 Month
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (1 Sites)
- McGill University Health Centre — Montreal, Quebec
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Cree Leukoencephalopathy
Explore by condition: All Cree Leukoencephalopathy clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.