NCT06897358

Leniolisib for Immune Dysregulation in CVID

A Study to Assess Safety and Tolerability, and Explore Efficacy of Leniolisib for Immune Dysregulation in Common Variable Immunodeficiency (CVID)

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Pharming Technologies B.V. · Industry
• Sex: All
• Age: 12 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

In this study, common variable immunodeficiency (CVID) patients will all receive the study drug, leniolisib, for a treatment period of 6 months. Participants will start on a lower dose of leniolisib, followed by a mid and then a higher dose level. The primary goal is to assess the safety and tolerability of leniolisib, and secondary goal is to assess the potential for leniolisib to provide benefits for patients.

Detailed description

The study includes administration of increasing dose levels of leniolisib in approximately 20 CVID patients presenting with clinical manifestations of immune dysregulation at study entry. Enrollment will include both patients without and those with genetic drivers identified for their CVID. All subjects participating will receive leniolisib film-coated tablets (FCTs) with a planned dose regimen consisting of a starting dose of 10 mg twice daily (BID) for 4 weeks, followed by a dose escalation to 30 mg BID for 4 weeks, and then 70 mg BID for an additional 16 weeks. Dose adjustments are allowed during treatment if deemed clinically necessary. Subjects not continuing leniolisib treatment outside the current protocol will be followed up, with the EoS visit planned to occur approximately 28 days after last dose of leniolisib.

Conditions

• Common Variable Immunodeficiency (CVID)

Interventions

Type	Name	Description
DRUG	Leniolisib	Planned dose will range from 10 mg twice daily to 70 mg twice daily

Timeline

• Start date: 2025-02-12
• Primary completion: 2026-10-01
• Completion: 2026-10-01
• First posted: 2025-03-26
• Last updated: 2026-02-12

Locations

7 sites across 3 countries: United States, Spain, United Kingdom

Regulatory

• FDA-regulated drug study