New hope for children with rare brain disease: drug safety tracked over time
NCT ID NCT04476862
First seen Jan 15, 2026 · Last updated May 16, 2026 · Updated 21 times
Summary
This study follows 35 children with CLN2 disease, a rare and severe brain disorder, who are receiving cerliponase alfa. Researchers are monitoring long-term safety, including allergic reactions and device problems. The goal is to ensure the treatment remains safe 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
Explore the condition pages connected to this study.