Pediatric Patients Aged 4 to 11 Years With APDS

Trial ID or NCT#

NCT05438407

Status

not recruiting iconNOT RECRUITING

Purpose

This is a 2-part, prospective, open-label, single arm, multicenter study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PDx), and efficacy of leniolisib in at least 15 pediatric patients (aged 4 to 11 years) with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS).

Official Title

An Open-label, Single Arm Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Leniolisib in Pediatric Patients Aged 4 to 11 Years With Activated Phosphoinositide 3-Kinase Delta Syndrome Followed by an Open-label Long-term Extension

Eligibility Criteria

Ages Eligible for Study: 4 Years to 11 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers: No
Exclusion Criteria:
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Investigator(s)

Yael Gernez
Clinical Associate Professor, Pediatrics - Immunology and Allergy

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Contact

Yael Gernez, MD

View on ClinicalTrials.gov