Rare disease drug safety tracked in new observational study
NCT ID NCT04476862
First seen Jun 24, 2026 · Last updated Jul 01, 2026 · Updated 3 times
Summary
This study follows 35 children with CLN2 disease (a rare, severe brain disorder) who are taking cerliponase alfa (Brineura). Researchers will monitor for side effects and serious reactions over time. The goal is to better understand the long-term safety of this enzyme replacement therapy.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- cerliponase alfa (Brineura)
- What this could lead to
- If successful, this study will provide important safety data on long-term use of cerliponase alfa for CLN2 disease, helping guide future treatment.
- What could go wrong
- This is an observational study, not a controlled trial, so it cannot prove the drug works. It only monitors safety in a small group of 35 patients.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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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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
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