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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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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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