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

NCT06990529 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: LE7X01
• Study Type: interventional
• Target: 12
• Locations: 2 countries
• Sites: 4

Brief 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

Outcome Measures

Primary Outcomes (1)

• To assess the long-term safety and tolerability of leniolisib

  Adverse events (AEs)

  From Baseline to approximately 3 years of Treatment

Secondary Outcomes (21)

• Impact of leniolisib on hemoglobin

  From Baseline to approximately 3 years of Treatment

• Impact of leniolisib on platelets

  From Baseline to approximately 3 years of Treatment

• Impact of leniolisib on neutrophils

  From Baseline to approximately 3 years of Treatment

• Impact of leniolisib on GLILD or other PID-related ILD

  From Baseline to approximately 3 years of Treatment

• Impact of leniolisib on pulmonary function

  From Baseline to approximately 3 years of Treatment

Study Arms (1)

Treatment Arm

EXPERIMENTAL

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

Drug: Leniolisib

Interventions

Leniolisib DRUG

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 \[BID\]).

Treatment Arm

Eligibility Criteria

• Age: 12 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Subject must have participated in LE 7201.
• Subject is deemed by the Investigator to benefit from continued leniolisib therapy.
• Subject or their legal representatives (for a patient under the age of 18 years) must be able to communicate with the Investigator and understand and comply with the requirements of the study, including an ability to provide written informed consent before any assessment is performed.

You may not qualify if:

• Subject has had a successful allogeneic hematopoietic stem cell transplant.
• Previous or concurrent use of immunosuppressive medication, such as:
• Use of an mTOR inhibitor or a PI3K delta inhibitor, besides leniolisib, within 3 weeks prior to first dosing of study medication.
• Rituximab or other B-cell depleting antibodies, belimumab, cyclophosphamide, or alemtuzumab within 6 months prior to first dosing of study medication.
• Cyclosporine A, mycophenolate mofetil, 6-mercaptopurine, azathioprine, methotrexate, tacrolimus, ruxolitinib or other Janus kinase (JAK) inhibitors within 3 weeks prior to first dosing of study medication.
• Corticosteroids above 25 mg prednisone or equivalent per day within 2 weeks prior to first dosing of study medication.
• Other immunosuppressive agents expected to have a significant impact on immune cell number or function.
• Subject is receiving concurrent treatment with another investigational therapy or use of another investigational therapy less than 4 weeks or 5 half lives (whichever is longer) prior to first dosing of study medication.
• History of hypersensitivity to the study drug or to drugs of similar chemical classes.
• Current use of medication known to be a strong inhibitor or moderate or strong inducer, of isoenzyme cytochrome P450 (CYP)3A.
• Current use of medications that to a larger extent are breast cancer resistant protein (BCRP), organic anion transporting polypeptide (OATP)1B1, and/or OATP1B3 substrates.
• History of acquired immunodeficiency diseases, including a positive human immunodeficiency virus (HIV) test result at screening.
• Uncontrolled chronic or recurrent infectious disease (except those considered to be characteristic of PID), or evidence of tuberculosis (TB) infection as defined by a positive QuantiFERON TB-Gold test at Screening.
• Any surgical or medical condition which may jeopardize the subject in case of participation in the study, or might significantly alter the absorption, distribution, metabolism, or excretion of drugs (conditions due to underlying clinical PID phenotype may be permitted):
• Uncontrolled hypertension

Sponsors & Collaborators

• Pharming Technologies B.V.: lead
• Aixial Group: collaborator

Study Sites (4)

National Institute of Health

Bethesda, Maryland, 20892, United States

RECRUITING

Lahey Hospital & Medical Center

Burlington, Massachusetts, 01805, United States

RECRUITING

Mount Sinai Hospital

New York, New York, 10029, United States

RECRUITING

IIS La Fe

Valencia, Spain

RECRUITING

MeSH Terms

Interventions

leniolisib

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 8, 2025
• First Posted: May 25, 2025
• Study Start: April 29, 2025
• Primary Completion (Estimated): September 18, 2028
• Study Completion (Estimated): April 13, 2029
• Last Updated: February 13, 2026

Record last verified: 2025-05

Locations

ES (1); US (3)