Tracking safety of rare disease treatment in children
NCT ID NCT04476862
Summary
This study monitors the long-term safety of cerliponase alfa, an approved treatment for CLN2 disease, a rare and severe childhood genetic disorder. It follows 35 children who are already receiving or planning to start this treatment. Researchers collect safety data during regular clinic visits to better understand how patients tolerate the treatment over time.
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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.