NCT05438407

Pediatric Patients Aged 4 to 11 Years With APDS

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

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 15 (estimated)
• Sponsor: Pharming Technologies B.V. · Industry
• Sex: All
• Age: 4 Years – 11 Years
• Healthy volunteers: Not accepted

Summary

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).

Detailed description

Part I will consist of a 12-week period to assess the safety and efficacy of treatment with leniolisib. Part II will consist of a 1-year, long-term, safety follow-up extension with a possible interim analysis. The leniolisib doses to be used in study were selected based on safety, tolerability, PK, and PDx data from the adult Phase 2/3 study, as well as PK modeling data. In both parts of the study, leniolisib will be administered orally based on weight.

Conditions

• APDS

Interventions

Type	Name	Description
DRUG	Leniolisib	The doses selected range from 20 to 70 mg BID (resulting in total daily doses ranging from 40 to 140 mg per day) based on weight. The doses will be administered as (a combination of) 10 mg and 30 mg tablets

Timeline

• Start date: 2023-02-01
• Primary completion: 2025-10-31
• Completion: 2025-12-30
• First posted: 2022-06-30
• Last updated: 2025-10-28

Locations

7 sites across 3 countries: United States, France, Japan

Regulatory

• FDA-regulated drug study