Pediatric Patients Aged 1 to 6 Years With APDS
An Open-label, Single Arm Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Leniolisib in Pediatric Patients (Aged 1 to 6 Years) with APDS (Activated Phosphoinositide 3-Kinase Delta Syndrome) Followed by an Open-label Long-term Extension
SEX AT BIRTH: All
HEALTHY PARTICIPANTS: No
CONDITION: Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS)
Learn More About This Research Study
For more information, please contact study coordinator, Erica Denallo at 216-286-7453 or complete the form below.
Study Purpose
The purpose of this research study is to find out how well the investigational study drug leniolisib is tolerated and the efficacy of leniolisib for the treatment of APDS (Activated Phosphoinositide 3-Kinase Delta Syndrome). This is a 2-part study and includes a screening period (up to 7 weeks), a treatment period of Part I (up to 12 weeks), a treatment period of Part II (up to 52 weeks), and a follow-up period (up to 4 weeks). If your child completes the study, he or she will participate for a total of 75 weeks and visit the study site around 11 times. The duration of the appointment will vary according on the procedures conducted.
Who Can Participate
Participants between the ages of 1 and 6 who are diagnosed with Activated Phosphoinositide 3-Kinase Delta Syndrome may be eligible to participate.