NCT06990529

Long-term Safety and Efficacy of Leniolisib in PIDs With Immune Dysregulation

An Open-label, Single-arm Extension Study to Evaluate the Long-term Safety, Tolerability, and Efficacy of Leniolisib for Immune Dysregulation in Primary Immunodeficiency Disorders

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Pharming Technologies B.V. · Industry
• Sex: All
• Age: 12 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

This is an open-label extension (OLE) study to extend treatment to patients with primary immunodeficiency (PID) disorders linked to phosphoinositide 3-kinase delta signaling who participated in a prior study of leniolisib, LE 7201. The primary objective is to assess long-term safety and tolerability of leniolisib. Secondary and exploratory objectives include various efficacy and immunophenotyping measures for leniolisib.

Conditions

• PIDs Linked to PI3K

Interventions

Type	Name	Description
DRUG	Leniolisib	All subjects will receive leniolisib film-coated tablets (FCTs) at the same dose they were receiving when they completed the preceding study (10, 30, or 70 mg twice daily \[BID\]).

Timeline

• Start date: 2025-04-29
• Primary completion: 2028-09-18
• Completion: 2029-04-13
• First posted: 2025-05-25
• Last updated: 2026-02-13

Locations

4 sites across 2 countries: United States, Spain

Regulatory

• FDA-regulated drug study