Long-term Safety and Efficacy of Leniolisib in PIDs With Immune Dysregulation
ConditionPIDs Linked to PI3K
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age12-75
SponsorPharming Technologies B.V.
About 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 does not accept healthy volunteersQualifiers
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.
Disqualifiers
Subject has had a successful allogeneic hematopoietic stem cell transplant.
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.
Trial design
Design model
Single group
Treatments tested in this trial
Leniolisib
DrugAll 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 groups
12 Participants
are divided into 1 treatment groupGroup A: Treatment ArmExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
To assess the long-term safety and tolerability of leniolisib
Adverse events (AEs)
Time frame
From Baseline to approximately 3 years of TreatmentSecondary outcomes
1
Impact of leniolisib on hemoglobin
Hemoglobin over time
Time frame
From Baseline to approximately 3 years of Treatment2
Impact of leniolisib on platelets
Platelet count over time
Time frame
From Baseline to approximately 3 years of Treatment3
Impact of leniolisib on neutrophils
Absolute neutrophil count (ANC) over time
Time frame
From Baseline to approximately 3 years of Treatment4
Impact of leniolisib on GLILD or other PID-related ILD
Computed tomography (CT) evidence of granulomatous lymphocytic interstitial lung disease (ILD) or other PID-related ILD evaluated using Hartmann scoring methodology over time
Time frame
From Baseline to approximately 3 years of TreatmentSponsors and contacts
Click on the lead sponsor to view all of their trials.
Pharming Technologies B.V.
Lead sponsor
Aixial Group
Collaborator
Source ClinicalTrials.gov