New hope for kids with rare immune disease
NCT ID NCT05438407
Summary
This study is testing a medication called leniolisib in children aged 4 to 11 who have a rare genetic immune disorder called APDS. The main goals are to see if the drug is safe for children, how their bodies process it, and if it can help reduce swollen lymph nodes and infections. The trial has two parts: a 12-week initial study and a longer one-year follow-up to monitor safety over time.
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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Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
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Institute of Science Tokyo Hospital
Tokyo, 113-8519, Japan
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Kyoto University Hospital
Kyoto, 606-8507, Japan
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National Institutes of Health
Bethesda, Maryland, 20814, United States
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Necker Hospital Paris
Paris, 75015, France
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Stanford University
Standford, California, 94305-5366, United States
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University of California Los Angeles
Los Angeles, California, 90095, United States
Conditions
Explore the condition pages connected to this study.