New hope for kids with rare immune disease: drug targets root cause

NCT ID NCT05438407

Ongoing Disease control Sponsor: Pharming Technologies B.V. Source: ClinicalTrials.gov ↗

First seen Nov 01, 2025 · Last updated May 17, 2026 · Updated 24 times

Summary

This study tests a drug called leniolisib in children aged 4 to 11 with APDS, a rare genetic condition that causes a faulty immune system. The goal is to see if the drug is safe and can control the disease by blocking an overactive immune signal. About 15 children will take the drug and be monitored for side effects and improvements in swollen lymph nodes and other symptoms.

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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

Children's Healthcare of Atlanta

Atlanta, Georgia, 30322, United States
Institute of Science Tokyo Hospital

Tokyo, 113-8519, Japan
Kyoto University Hospital

Kyoto, 606-8507, Japan
National Institutes of Health

Bethesda, Maryland, 20814, United States
Necker Hospital Paris

Paris, 75015, France
Stanford University

Standford, California, 94305-5366, United States
University of California Los Angeles

Los Angeles, California, 90095, United States

Conditions

Explore the condition pages connected to this study.

APDS