Common variable immunodeficiency
Conditions
Brief summary
Adverse events (AEs; via assessment of vital signs, safety labs, physical exams and overall trial safety monitoring)
Detailed description
Percent change from baseline in splenomegaly measured by 3D volume and 2D size of spleen, Percent change from baseline in lymphoproliferation measured as the SPD in the index lesions selected per the Cheson methodology, Percent change from baseline in lymphoproliferation measured as SPD of measurable non-index lesions selected as per the Cheson methodology, Hemoglobin over time, Platelet count over time, Absolute neutrophil count over time, Change in CT evidence of ILD evaluated using Hartmann scoring methodology, Change in pulmonary function testing, including forced expiratory volume 1 (FEV1), forced vital capacity (FVC), total lung capacity (TLC), residual volume (RV), diffusion capacity for carbon monoxide (DLCO), Absolute numbers and percentages of leukocyte populations (including lymphocyte subsets) over time, Percentages of naïve B cells and CD21low B cells over time, Percentage of T regulatory cells over time, Levels of CXCL13 over time, Levels of soluble IL-2Rα over time, Define the PK parameters for leniolisib in patients with CVID
Interventions
Sponsors
Pharming Technologies B.V.
Eligibility
Sex/Gender
All
Age
0 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Adverse events (AEs; via assessment of vital signs, safety labs, physical exams and overall trial safety monitoring) | — |
Secondary
| Measure | Time frame |
|---|---|
| Percent change from baseline in splenomegaly measured by 3D volume and 2D size of spleen, Percent change from baseline in lymphoproliferation measured as the SPD in the index lesions selected per the Cheson methodology, Percent change from baseline in lymphoproliferation measured as SPD of measurable non-index lesions selected as per the Cheson methodology, Hemoglobin over time, Platelet count over time, Absolute neutrophil count over time, Change in CT evidence of ILD evaluated using Hartmann scoring methodology, Change in pulmonary function testing, including forced expiratory volume 1 (FEV1), forced vital capacity (FVC), total lung capacity (TLC), residual volume (RV), diffusion capacity for carbon monoxide (DLCO), Absolute numbers and percentages of leukocyte populations (including lymphocyte subsets) over time, Percentages of naïve B cells and CD21low B cells over time, Percentage of T regulatory cells over time, Levels of CXCL13 over time, Levels of soluble IL-2Rα over time, Defi | — |
Countries
Spain
Outcome results
None listed