Experimental gene therapy aims to halt devastating brain disease in infants
NCT ID NCT04771416
First seen Jun 27, 2026 · Last updated Jul 01, 2026 · Updated 2 times
Summary
This study tests a gene therapy called PBKR03 for babies with early infantile Krabbe disease, a severe genetic disorder that damages the brain and nerves. The treatment delivers a working copy of the GALC gene to the brain and body. Researchers will check safety and find the best dose in 24 infants up to 9 months old.
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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
Locations
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Amsterdam UMC
Amsterdam, Netherlands
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Ann & Robert Lurie
Chicago, Illinois, 60611, United States
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Hospital De Clinicas De Porto Alegre
Porto Alegre, Brazil
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Manchester University
Manchester, United Kingdom
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Montreal Children's Hospital
Montreal, Canada
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New York-Presbyterian
New York, New York, 10065, United States
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Shaare Zadek Medical Center
Jerusalem, Israel
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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University of Utah School of Medicine
Salt Lake City, Utah, 84132, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.