Hope for tots with rare immune disease in new drug trial
NCT ID NCT05693129
Summary
This study is testing a drug called leniolisib in very young children (ages 1 to 6) who have a rare genetic immune disorder called APDS. The goal is to see if the drug is safe, how the body processes it, and if it can help control the disease by reducing swollen lymph nodes and infections. Children will take the drug orally for a year or more to monitor its long-term effects.
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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Locations
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Great Ormond Street Hospital
London, WC1N3JH, United Kingdom
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Hospital Pediátrico de Coimbra da ULS Coimbra UNIDADE LOCAL DE SAÚDE DE COIMBRA
Coimbra, 3000-075, Portugal
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Hospital Universitario Virgen del Rocío
Seville, 41013, Spain
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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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Rainbow Childrens Hospital
Shaker Heights, Ohio, 44122, United States
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Texas Children's Hospital
Houston, Texas, 77030, United States
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University of California Los Angeles
Los Angeles, California, 90095, United States
Conditions
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