Pharming LE3302 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: Any
- Age: Child (Birth - 17)
- Accepting Healthy People: No
- Type: Interventional / Therapeutic
- Trial Phase: Phase III
- Conditions Being Studied: Protein Kinase C
Study Purpose
APDS is a rare disease caused by genetic variations in either of two genes known as PIK3CD or PIK3R1. Both of these genes are vital to the development and function of immune cells in the body. Currently there are no approved therapies for APDS. The purpose of this study is to find out how well study drug leniolisib is tolerated and the efficacy of leniolisib for the treatment of APDS (Activated Phosphoinositide 3-Kinase Delta Syndrome).
Locations
UH Cleveland Medical Center
11100 Euclid Avenue
Cleveland OH, 44106
- UH IRB: SITE00001982
- StudyID: 2022-0864
- ClinicalTrials.gov: NCT05693129
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