New hope for children with rare fatal brain disease: drug safety tracked
NCT ID NCT04476862
First seen Jan 15, 2026 · Last updated May 21, 2026 · Updated 22 times
Summary
This study tracks the long-term safety of cerliponase alfa in 35 children with CLN2 disease, a rare and fatal genetic disorder that causes seizures, loss of movement, and speech. Participants are already receiving or will start the treatment. Researchers monitor for serious side effects and how the drug affects motor and language skills over time.
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This is a summary of
the original study
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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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Contacts and locations
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Locations
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Advent Health
Orlando, Florida, 32803, United States
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Arkansas Children's Hospital
Little Rock, Arkansas, 72205, United States
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Boston Children's Hospital,
Boston, Massachusetts, 02115, United States
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Children's Healthcare of Atlanta
Atlanta, Georgia, 30329, United States
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Children's Hospital Minnesota
Minneapolis, Minnesota, 55404, United States
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Children's Hospital of Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital of Orange County
Orange, California, 92868, United States
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Children's National Hospital
Washington D.C., District of Columbia, 20010, United States
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Mt. Sinai School of Medicine
New York, New York, 10029, United States
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NYU Langone Medical Center
New York, New York, 10017, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43130, United States
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Rush University Medical Center
Chicago, Illinois, 60612, United States
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Seattle Children's Hospital
Seattle, Washington, 98145-5005, United States
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Texas Children's Hospital
Houston, Texas, 77030, United States
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University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104, United States
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University of Rochester Medical Center
Rochester, New York, 14642, United States
Conditions
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