{"disease":{"id":"activated-pi3k-delta-syndrome","name":"activated pi3k delta syndrome"},"drugs":{"marketed":[{"drug_id":"leniolisib","indication_name":"Activated PI3K delta syndrome (APDS)","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":"Joenja","generic_name":"LENIOLISIB","company_name":"Pharming","drug_phase":"marketed","molecular_target":"Phosphatidylinositol 4,5-bisphosphate 3-kinase catalytic subunit delta isoform","drug_class":"","quality_score":57,"revenue":null,"mechanism":"Joenja works by blocking the activity of a specific enzyme called PI3K delta, which is involved in the immune system's response."}],"pipeline":[],"offLabel":[],"totalMarketed":1,"totalPipeline":0},"trials":{"data":[{"nct_id":"NCT02859727","title":"Extension to the Study of Efficacy of CDZ173 in Patients With APDS/PASLI","phase":"PHASE2, PHASE3","overall_status":"TERMINATED","enrollment_count":37,"lead_sponsor_name":"Pharming Technologies B.V.","has_results":true},{"nct_id":"NCT03383380","title":"Rapamycin Treatment for Activated Phosphoinositide 3-Kinase δ Syndrome","phase":"PHASE1, PHASE2","overall_status":"COMPLETED","enrollment_count":30,"lead_sponsor_name":"Children's Hospital of Fudan University","has_results":false},{"nct_id":"NCT06694363","title":"New Biomarker-based Strategy to Screen and Monitor for Activated Phosphoinositide 3-kinase δ Syndrome","phase":"","overall_status":"RECRUITING","enrollment_count":14,"lead_sponsor_name":"Assistance Publique - Hôpitaux de Paris","has_results":false},{"nct_id":"NCT02593539","title":"Safety, Pharmacokinetic (PK) and Pharmacodynamic (PD) Study of Repeat Doses of Inhaled Nemiralisib in Patients With APDS/PASLI","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":5,"lead_sponsor_name":"GlaxoSmithKline","has_results":true}],"total":4},"guidelines":[],"source":"Drug Landscape verified database"}