New hope for kids with rare immune disease: leniolisib trial launches
NCT ID NCT05693129
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a drug called leniolisib in children aged 1 to 6 years who have a rare genetic immune disorder called APDS. The drug aims to control the overactive immune system and reduce swollen lymph nodes. Researchers will monitor safety and how well the drug works over time.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- leniolisib
- What this could lead to
- If successful, leniolisib could offer a targeted treatment option to control APDS in young children, reducing infections and lymph node problems.
- What could go wrong
- This is a small, early-phase trial with only 16 children, so results may not apply to all. Side effects and long-term safety are still being studied.
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
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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
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