Last hope: experimental drug tested in single child with rare brain disease
NCT ID NCT07272525
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study gives one patient with Cree Leukoencephalopathy, a rare and fatal brain disease, access to an experimental drug called fosigotifator. The drug aims to slow or stop the brain damage that causes severe disability and early death. Researchers will track whether the patient survives or needs a breathing machine after two years.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Fosigotifator (FGT/ABBV-CLS-7262)
What this could lead to
If it works, this could point toward a treatment that slows or halts brain damage in Cree Leukoencephalopathy, offering hope for a disease with no current options.
What could go wrong
This is a very early study in just one patient, so results may not apply to others. The drug is still investigational and its safety and effectiveness are not yet proven.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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McGill University Health Centre
Montreal, Quebec, H4A3J1, Canada