Actively Recruiting
Long-term Safety and Efficacy of Leniolisib in PIDs With Immune Dysregulation
Led by Pharming Technologies B.V. · Updated on 2026-02-13
12
Participants Needed
4
Research Sites
206 weeks
Total Duration
On this page
Sponsors
P
Pharming Technologies B.V.
Lead Sponsor
A
Aixial Group
Collaborating Sponsor
AI-Summary
What this Trial Is About
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
Official Title
Long-term Safety and Efficacy of Leniolisib in PIDs With Immune Dysregulation
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Must have participated in the LE 7201 study
- Investigator believes the participant will benefit from continued leniolisib treatment
- Participant or legal representative can communicate with the Investigator and comply with study requirements, including providing written informed consent
You will not qualify if you...
- Successful allogeneic hematopoietic stem cell transplant
- Use of immunosuppressive medications such as mTOR inhibitors, PI3K delta inhibitors other than leniolisib within 3 weeks prior to starting study drug
- Use of rituximab, belimumab, cyclophosphamide, alemtuzumab within 6 months prior to starting study drug
- Use of cyclosporine A, mycophenolate mofetil, 6-mercaptopurine, azathioprine, methotrexate, tacrolimus, ruxolitinib, or other Janus kinase inhibitors within 3 weeks prior to starting study drug
- Use of corticosteroids above 25 mg prednisone or equivalent per day within 2 weeks prior to starting study drug
- Use of other immunosuppressive agents significantly affecting immune cells
- Concurrent or recent use of other investigational therapies within 4 weeks or 5 half-lives before starting study drug
- History of allergy to leniolisib or similar drugs
- Current use of strong inhibitors or inducers of cytochrome P450 3A enzyme
- Use of medications that are substrates of breast cancer resistant protein or organic anion transporting polypeptides
- History of acquired immunodeficiency diseases including positive HIV test at screening
- Uncontrolled chronic or recurrent infections (except typical PID infections) or positive tuberculosis test
- Medical conditions that increase risk during study or affect drug metabolism, including uncontrolled hypertension, severe heart failure, ECG abnormalities posing safety risk, severe COPD, need for respiratory support, major gastrointestinal surgery affecting absorption, acute pancreatitis, significant liver or kidney disease
- Positive hepatitis B or C tests at screening
- Administration of live vaccines within 6 weeks before starting study drug, during study, or within 7 days after last dose
- Recent or planned treatment for lymphoma or other malignancies within specified time frames
- Uncontrolled lymphoproliferative disease related to Epstein-Barr virus
- Major surgery or radiotherapy within 4 weeks prior to starting study drug or planned during study
- Pregnancy or breastfeeding
- Individuals capable of becoming pregnant not using effective contraception
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 4 locations
1
National Institute of Health
Bethesda, Maryland, United States, 20892
Actively Recruiting
2
Lahey Hospital & Medical Center
Burlington, Massachusetts, United States, 01805
Actively Recruiting
3
Mount Sinai Hospital
New York, New York, United States, 10029
Actively Recruiting
4
IIS La Fe
Valencia, Spain
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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