Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Leniolisib · 5 trials · 4 indications
To assess number of Participants with TEAEs, SAEs, and AEs leading to discontinuation of study drug
Number of Participants with change in clinical laboratory test results (hematology, blood
Number of Participants with change in vital signs
Number of Participants with change in physical examination findings
Number of Participants with change in electrocardiograms (ECGs)
Number of Participants with change in growth and physical development
To assess the impact of leniolisib on lymphadenopathy, patients will be scanned in an MRI or a CT scanner as based on clinical practice and local regulation. Index lesions will be selected from measurable nodal and extra nodal lesions as per the Cheson methodology. The same imaging modality will be used throughout the study for the same patient. Patients will be assessed by MRI, or in sites where local practice and local authorities/IECs/IRBs approve CT scans for research purposes using a low-dose CT scan.
To assess change in the PDx effect of leniolisib will be assessed using ex vivo stimulated and unstimulated
For the assessment of the impact of leniolisib on lymphoproliferation, patients will be scanned in an MRI or a CT scanner as based on clinical practice and local regulation. Index lesions will be selected from measurable nodal and extra nodal lesions as per the Cheson methodology. The same imaging modality will be used throughout the study for the same patient. Patients will be assessed by MRI, or in sites where local practice and local authorities/IECs/IRBs approve CT scans for research purposes using a low-dose CT scan.
Adverse events (AEs)
To assess the number of AEs/SAEs and number of participants with AEs/SAEs
• To assess the number of AEs/SAEs and number of participants with AEs/SAEs
| Arm | Type | Description |
|---|---|---|
| Leniolisib | EXPERIMENTAL | Leniolisib film-coated granules in 10, 15 and 20 mg strengths administered orally BID by body weight for 12 weeks for Part I and for 1 year for Part II. |
| Treatment Arm | EXPERIMENTAL | All subjects will receive leniolisib film-coated tablets (FCTs) |
| Name | Type | Description |
|---|---|---|
| Leniolisib | DRUG | The doses selected will range from 10 to 50 mg twice daily (BID) (resulting in total daily doses ranging from 20 to 100 mg per day). |
Inclusion Criteria: 1. Patient is male or female and between the age of 1 to 6 years old at time of the first study procedure. 2. Patient weighs ≥8 and ≤37 kg at baseline. 3. Patient has a confirmed PI3Kδ genetic mutation of either the PIK3CD (APDS1) or PIK3R1 (APDS2) gene. 4. Patient has at least ...
Leniolisib is an investigational small molecule being developed for primary immunodeficiency disorders (PIDs) linked to PI3K, including activated PI3K delta syndrome (APDS), and for common variable immunodeficiency (CVID). It is currently in clinical development and has not been approved by the FDA.
Leniolisib targets PI3K, specifically the PI3K delta isoform, as indicated by its '-lisib' suffix. It is a small molecule designed to modulate the PI3K pathway, which is implicated in immune dysregulation associated with primary immunodeficiency disorders.
Leniolisib is being developed by Pharming Group N.V., a biopharmaceutical company listed on the stock exchange under the ticker PHAR. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with APDS and related conditions.
Leniolisib is in Phase 3 clinical development for pediatric patients with APDS. Additionally, it is being studied in Phase 2 trials for CVID and other PIDs linked to PI3K. It is an investigational drug and has not received FDA approval.
Leniolisib is being evaluated in several trials, including NCT05438407 (Phase 3, pediatric APDS patients aged 4-11), NCT05693129 (Phase 3, pediatric APDS patients aged 1-6), NCT06897358 (Phase 2, CVID), and NCT06990529 (Phase 2, PIDs linked to PI3K). All trials are active.
Leniolisib is not known to be the same as any other drug. It is a distinct small molecule targeting PI3K delta, developed by Pharming Group N.V. for primary immunodeficiency disorders. No alternative names have been reported.