{"disease":{"id":"neuronal-ceroid-lipofuscinosis-type-2","name":"neuronal ceroid lipofuscinosis type 2"},"drugs":{"marketed":[],"pipeline":[],"offLabel":[],"totalMarketed":0,"totalPipeline":0},"trials":{"data":[{"nct_id":"NCT05687474","title":"Baby Detect : Genomic Newborn Screening","phase":"","overall_status":"COMPLETED","enrollment_count":6824,"lead_sponsor_name":"Centre Hospitalier Universitaire de Liege","has_results":false},{"nct_id":"NCT04476862","title":"Cerliponase Alfa Observational Study in the US","phase":"","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":35,"lead_sponsor_name":"BioMarin Pharmaceutical","has_results":false},{"nct_id":"NCT05791864","title":"A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With TTX-381 for the Ocular Manifestations Associated With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease","phase":"PHASE1, PHASE2","overall_status":"RECRUITING","enrollment_count":16,"lead_sponsor_name":"Tern Therapeutics, LLC","has_results":false},{"nct_id":"NCT02678689","title":"A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":14,"lead_sponsor_name":"BioMarin Pharmaceutical","has_results":true},{"nct_id":"NCT05152914","title":"Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2","phase":"PHASE1, PHASE2","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":5,"lead_sponsor_name":"David L Rogers, MD","has_results":false}],"total":5},"guidelines":[],"source":"Drug Landscape verified database"}