{"disease":{"id":"gm2-gangliosidosis","name":"gm2 gangliosidosis"},"drugs":{"marketed":[],"pipeline":[],"offLabel":[],"totalMarketed":0,"totalPipeline":0},"trials":{"data":[{"nct_id":"NCT03047369","title":"The Myelin Disorders Biorepository Project","phase":"","overall_status":"RECRUITING","enrollment_count":12000,"lead_sponsor_name":"Children's Hospital of Philadelphia","has_results":false},{"nct_id":"NCT02699190","title":"LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies","phase":"","overall_status":"COMPLETED","enrollment_count":236,"lead_sponsor_name":"Children's Hospital of Philadelphia","has_results":true},{"nct_id":"NCT04470713","title":"Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2","phase":"","overall_status":"COMPLETED","enrollment_count":226,"lead_sponsor_name":"Idorsia Pharmaceuticals Ltd.","has_results":false},{"nct_id":"NCT07054515","title":"A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis","phase":"PHASE3","overall_status":"RECRUITING","enrollment_count":147,"lead_sponsor_name":"Azafaros A.G.","has_results":false},{"nct_id":"NCT07082543","title":"A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis","phase":"PHASE3","overall_status":"RECRUITING","enrollment_count":75,"lead_sponsor_name":"Azafaros A.G.","has_results":false},{"nct_id":"NCT04624789","title":"Registry Gangliosidoses","phase":"","overall_status":"UNKNOWN","enrollment_count":40,"lead_sponsor_name":"SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt)","has_results":false},{"nct_id":"NCT03759665","title":"N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":30,"lead_sponsor_name":"IntraBio Inc","has_results":true},{"nct_id":"NCT03822013","title":"Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD)","phase":"PHASE3","overall_status":"TERMINATED","enrollment_count":30,"lead_sponsor_name":"Tehran University of Medical Sciences","has_results":false},{"nct_id":"NCT07399704","title":"A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease","phase":"PHASE2","overall_status":"RECRUITING","enrollment_count":21,"lead_sponsor_name":"Azafaros A.G.","has_results":false},{"nct_id":"NCT04798235","title":"First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis","phase":"PHASE1, PHASE2","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":3,"lead_sponsor_name":"Dr. Anupam Sehgal","has_results":false}],"total":10},"guidelines":[],"source":"Drug Landscape verified database"}