Ignite Creation Date:
2025-12-25 @ 12:07 AM
Ignite Modification Date:
2026-01-01 @ 12:24 PM
Study NCT ID:
NCT06897358
Status:
RECRUITING
Last Update Posted:
2025-10-15
First Post:
2025-03-12
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Leniolisib for Immune Dysregulation in CVID
Sponsor:
Pharming Technologies B.V.
Organization:
Study Overview
Official Title:
A Study to Assess Safety and Tolerability, and Explore Efficacy of Leniolisib for Immune Dysregulation in Common Variable Immunodeficiency (CVID)
Status:
RECRUITING
Status Verified Date:
2025-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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.
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.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: