Single Patient Investigational Treatment for Cree Leukoencephalopathy

Condition(s) studied

Investigational drug(s) / intervention(s)

Fosigotifator: Fosigotifator

Study 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.

Eligibility

Primary outcome measure(s)

• Time to death or permanent ventilation — From enrollment to 2 years
  Time to death or permanent ventilation

Trial sites (1)

More McGill University Health Centre/Research Institute of the McGill University Health Centre trials in Canada

Other trials for the same condition