This study is currently recruiting participants.
Number
001573-I
Sponsoring Institute
National Institute of Allergy and Infectious Diseases (NIAID)
Recruitment Detail
Type: Participants currently recruited/enrolled Gender: Male & Female Min Age: 4 Years Max Age: 11 Years
Referral Letter Required
Yes
Population Exclusion(s)
None
Keywords
Leniolisib
Recruitment Keyword(s)
Condition(s)
Activated Phosphoinositide 3-Kinase Delta Syndrome
Investigational Drug(s)
CDZ 173 (leniolisib)
Investigational Device(s)
Intervention(s)
Drug: Leniolisib
Supporting Site
National Institute of Allergy and Infectious Diseases
Activated phosphoinositide 3-kinase delta syndrome (APDS) is a rare, inherited disease that affects how the immune system works. Symptoms often first appear in children younger than 3 years. Symptoms can include frequent respiratory infections, severe viral illnesses, and slowed growth and development.
Objective:
To test a drug (leniolisib) in children with APDS. (This drug is approved to treat APDS in people older than 12 years.)
Eligibility:
People aged 4 to 11 years with APDS.
Design:
Participants will have 11 study visits in 15 to 18 months.
Screening will take up to 44 days. Participants will have a physical exam, with tests of the blood, urine, and stool. They will have imaging scans and tests of their heart function. They will be tested for HIV, tuberculosis, COVID-19, and hepatitis. Their body will be examined to determine their level of sexual maturity.
Leniolisib is a tablet taken by mouth. It must be swallowed whole. Participants will take the tablet twice a day, every day, for about 15 months. Their caregiver will record doses in a diary; they will also record any changes in their child s health.
Blood and urine tests will be repeated at study visits; heart tests and imaging scans will be repeated on some visits. Participants (and/or their caregivers) will fill in questionnaires. They will answer questions about their symptoms and any changes since they began taking the drug.
Participants will have a follow-up visit about 28 days after they stop taking the drug.
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INCLUSION CRITERIA: Participants seen at the NIH must be co-enrolled on NIH IRB-reviewed protocol #14I0206 (Novel Genetic Disorders of the Immune System/Omnibus).
Participants seen at the NIH must be co-enrolled on NIH IRB-reviewed protocol #14I0206 (Novel Genetic Disorders of the Immune System/Omnibus).
Principal Investigator
Referral Contact
For more information: