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Tests treatment safety and results for PIDs Linked to PI3K
Official title Long-term Safety and Efficacy of Leniolisib in PIDs With Immune Dysregulation
ClinicalTrials.gov ID: NCT06990529
What this study is testing
What is Leniolisib?
Leniolisib is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for pids linked to pi3k.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 to 75
You may be able to join 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...
You likely can't join 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...
- Corticosteroids above 25 mg prednisone or equivalent per day within 2 weeks prior to first dosing of study medication.
See the full eligibility criteria
Who can join
- 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...
What rules you out
- 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...
- Uncontrolled hypertension
- Congestive heart failure (New York Heart Association status of class III or IV)
- Diagnosis of electrocardiogram (ECG) abnormalities indicating a significant risk of safety
- Chronic obstructive pulmonary disease (Global Initiative for Chronic Obstructive Lung Disease [GOLD] stage 3-4)
- Chronic need for supplemental oxygen or invasive or non-invasive respiratory support
- Major GI tract surgery that may affect drug absorption (such as gastric bypass surgery, gastroenterostomy)
- Acute pancreatitis
- Liver failure or clinically significant liver disease or dysfunction as indicated by ALT or AST greater than 2.5 times the upper limit of normal, bilirubin greater than 2 times the upper limit of normal, INR greater...
- History of significant renal injury/renal disease severely affecting renal function or presence of impaired renal function as indicated by estimated glomerular filtration rate (eGFR) of less than 30 mL/min/1.73 m2.
- 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.
- Administration of live vaccines (this includes any attenuated live vaccines) starting from 6 weeks prior to first dosing of study medication, during the study, and up to 7 days after the last dose of leniolisib.
- Subject has a previous diagnosis of lymphoma that has been treated with chemotherapy, radiotherapy, or transplant within 1 year prior to first dosing of study medication or is anticipated to require lymphoma treatment...
- Subject has a history of malignancy (except lymphoma) within 3 years prior to first dosing of study medication or has evidence of residual disease from a previously diagnosed malignancy, except for adequately treated...
- Subject has uncontrolled post-transplant lymphoproliferative disease-like Epstein-Barr-virus-related lymphoproliferative disease.
- Subject has had major surgery requiring hospitalization or radiotherapy within 4 weeks prior to first dosing of study medication or has a planned or expected major surgical procedure during the study period.
- Pregnant or nursing (lactating) women.
- An individual of child-bearing potential who is physiologically capable of becoming pregnant, unless using highly effective methods of contraception.
The study team makes the final eligibility decision.
Where it's taking place
- Bethesda, Maryland, United States
- Burlington, Massachusetts, United States
- New York, New York, United States
- Valencia, Spain
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years to 75 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bethesda, Maryland, United States; Burlington, Massachusetts, United States; New York, New York, United States; Valencia, Spain. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.