Leniolisib for Primary Immunodeficiency

Overseen By Gulbu Uzel, MD

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Pharming Technologies B.V.

Must be taking: Leniolisib

Must not be taking: Immunosuppressants, Corticosteroids, CYP3A inhibitors, others

Disqualifiers: HIV, Uncontrolled infections, Hepatitis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: Approximately 3 years

12 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Eligibility Criteria

This trial is for patients with primary immunodeficiency disorders related to PI3K delta signaling who were previously in the leniolisib study LE 7201. It aims to continue treatment and further evaluate safety and effectiveness.

Inclusion Criteria

Subject is deemed by the Investigator to benefit from continued leniolisib therapy

Subject must have participated in LE 7201

I can communicate with the study team and follow the study requirements.

Exclusion Criteria

History of acquired immunodeficiency diseases, including a positive human immunodeficiency virus (HIV) test result at screening

A positive hepatitis B surface antigen (HBsAg), positive hepatitis B polymerase chain reaction (PCR), positive hepatitis C PCR, or positive hepatitis C antibody result at screening

I haven't had major surgery or radiotherapy in the last 4 weeks and don't plan to during the study.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive leniolisib film-coated tablets to assess long-term safety, tolerability, and efficacy

Approximately 3 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants continue to receive leniolisib to evaluate long-term safety and efficacy

Long-term

Treatment Details

Interventions

• Leniolisib

Trial Overview The trial tests the long-term safety, tolerability, and efficacy of a drug called Leniolisib for individuals with immune dysregulation due to primary immunodeficiency disorders.

Participant Groups

Group I: Treatment Arm Experimental Treatment 1 Intervention

All subjects will receive leniolisib film-coated tablets (FCTs)

Who Is Running the Clinical Trial?

Pharming Technologies B.V.

Lead Sponsor

Trials

23

Recruited

1,400+

Aixial Group

Industry Sponsor

Trials

4

Recruited

90+