NCT06549114

Leniolisib for Immune Dysregulation in PIDs

A Study to Assess Safety and Tolerability, and Explore Efficacy of Leniolisib for Immune Dysregulation in Primary Immunodeficiency Disorders

• Status: Active Not 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 study is an exploratory, non-randomized, open-label, within-patient dose escalation study. The primary objective is to assess safety and tolerability of leniolisib. Secondary objectives include assessments of PK/PD, and to explore clinical efficacy measures with administration of three different dose levels of leniolisib.

Detailed description

Patients ages 12-75 diagnosed with genetically defined PID disorders linked to PI3K signaling. This includes disorders caused by pathogenic variants in SOCS1, PTEN, CTLA4, NFKB1 (variants leading to NFKB pathway activation), FAS (germline or somatic), or RAS-associated leukoproliferative disorder caused by somatic variants in NRAS or KRAS (not juvenile myelomonocytic leukemia \[JMML\]). 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 30 mg BID for 4 weeks, and then 70 mg BID for 12 weeks. Leniolisib dose increase at the individual subject level will occur if no safety or tolerability issues have been identified by the Investigator that precludes the planned dose escalation. Subjects not continuing leniolisib treatment outside of the current protocol will be followed up, with the EOS visit planned to occur approximately 28 days after last dose of leniolisib.

Conditions

• Primary Immunodeficiency Disorders (PIDs)

Interventions

Type	Name	Description
DRUG	Leniolisib	The doses selected will range from 10 to 70 mg twice daily (BID) (resulting in total daily doses ranging from 20 to 140 mg per day).

Timeline

• Start date: 2024-10-21
• Primary completion: 2026-11-01
• Completion: 2026-11-01
• First posted: 2024-08-12
• Last updated: 2026-02-13

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study