{"disease":{"id":"severe-hemophilia-b","name":"severe hemophilia b"},"drugs":{"marketed":[{"drug_id":"gbt021601","indication_name":"Treatment of adult patients with severe hemophilia A with inhibitors","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":"Gbt021601","generic_name":"gbt021601","company_name":"Pfizer","drug_phase":"marketed","molecular_target":"unknown","drug_class":"unknown","quality_score":22,"revenue":null,"mechanism":""}],"pipeline":[],"offLabel":[],"totalMarketed":1,"totalPipeline":0},"trials":{"data":[{"nct_id":"NCT03655223","title":"Early Check: Expanded Screening in Newborns","phase":"","overall_status":"ENROLLING_BY_INVITATION","enrollment_count":30000,"lead_sponsor_name":"RTI International","has_results":false},{"nct_id":"NCT06938542","title":"Palliative Care Needs of Children With Rare Diseases and Their Families","phase":"NA","overall_status":"ENROLLING_BY_INVITATION","enrollment_count":480,"lead_sponsor_name":"Children's National Research Institute","has_results":false},{"nct_id":"NCT05889754","title":"Reliability and Validity of the Turkish Version of the PedHAL","phase":"","overall_status":"UNKNOWN","enrollment_count":50,"lead_sponsor_name":"Hasan Kalyoncu University","has_results":false},{"nct_id":"NCT05981274","title":"A Study on the Bone-health Effectiveness of Applying Recombinant Factor VIII Fc (rFVIIIFc) to Patients With Hemophilia A (Prototype A)","phase":"","overall_status":"RECRUITING","enrollment_count":45,"lead_sponsor_name":"Tri-Service General Hospital","has_results":false},{"nct_id":"NCT06155955","title":"Comparison of Outcomes Between Low Dose Emicizumab and Factor VIII in Clinically Severe Hemophilia A","phase":"PHASE2, PHASE3","overall_status":"UNKNOWN","enrollment_count":15,"lead_sponsor_name":"Chulalongkorn University","has_results":false},{"nct_id":"NCT05281185","title":"Clinical Outcomes of Low Dose PK-guided EHL FVIII Concentrates Versus Standard Prophylaxis in Severe Haemophilia A","phase":"PHASE4","overall_status":"COMPLETED","enrollment_count":15,"lead_sponsor_name":"Chulalongkorn University","has_results":false}],"total":6},"guidelines":[],"source":"Drug Landscape verified database"}