{"disease":{"id":"hypophosphatasia","name":"Hypophosphatasia","therapeutic_area":"","data":{},"enrichment_level":0,"last_enriched_at":null,"visit_count":0,"created_at":"2026-04-19T18:31:21.150Z","updated_at":"2026-04-19T18:39:44.960Z","meddra_pt":null,"meddra_code":null,"icd10_codes":null,"synonyms":[],"prevalence_global":null,"prevalence_us":null,"marketed_drug_count":1,"pipeline_drug_count":0,"trial_count":2},"drugs":{"marketed":[{"drug_id":"asfotase-alfa","indication_name":"Hypophosphatasia","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":"Strensiq","generic_name":"ASFOTASE ALFA","company_name":"AstraZeneca","drug_phase":"marketed","molecular_target":"","drug_class":"Tissue-nonspecific Alkaline Phosphatase [EPC]","quality_score":10,"revenue":null,"mechanism":"Strensiq works by replacing the deficient tissue-nonspecific alkaline phosphatase enzyme in patients with hypophosphatasia."}],"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":"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":"NCT02306720","title":"Registry of Patients With Hypophosphatasia","phase":"","overall_status":"ENROLLING_BY_INVITATION","enrollment_count":1571,"lead_sponsor_name":"Alexion Pharmaceuticals, Inc.","has_results":false},{"nct_id":"NCT06079281","title":"Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa","phase":"PHASE3","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":124,"lead_sponsor_name":"Alexion Pharmaceuticals, Inc.","has_results":false},{"nct_id":"NCT05062629","title":"United States Hypophosphatasia Molecular Research Center","phase":"","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":66,"lead_sponsor_name":"Children's Mercy Hospital Kansas City","has_results":false},{"nct_id":"NCT04018287","title":"Circulating miRNAs and Bone Microstructure in Adults With Hypophosphatasia","phase":"","overall_status":"UNKNOWN","enrollment_count":60,"lead_sponsor_name":"Medical University of Vienna","has_results":false},{"nct_id":"NCT06161142","title":"Characteristics of Hypophosphatasia in Adult Patients in Rheumatology","phase":"","overall_status":"UNKNOWN","enrollment_count":60,"lead_sponsor_name":"University of Bonn","has_results":false},{"nct_id":"NCT07390240","title":"The Effect of Monoallelic Variants in the ALPL Gene on the Natural Course of Hypophosphatasia in Russia","phase":"","overall_status":"RECRUITING","enrollment_count":55,"lead_sponsor_name":"AstraZeneca","has_results":false},{"nct_id":"NCT04195763","title":"Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa)","phase":"","overall_status":"COMPLETED","enrollment_count":50,"lead_sponsor_name":"Alexion Pharmaceuticals, Inc.","has_results":false},{"nct_id":"NCT06079372","title":"Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa","phase":"PHASE3","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":43,"lead_sponsor_name":"Alexion Pharmaceuticals, Inc.","has_results":false},{"nct_id":"NCT04181164","title":"Evaluation of Bone Architecture and Bone Strength in Adults With Hypophosphatasia (HPP)","phase":"","overall_status":"UNKNOWN","enrollment_count":30,"lead_sponsor_name":"Hvidovre University Hospital","has_results":false},{"nct_id":"NCT06079359","title":"Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP","phase":"PHASE3","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":30,"lead_sponsor_name":"Alexion Pharmaceuticals, Inc.","has_results":false},{"nct_id":"NCT05234567","title":"A Prospective Sub-Study of the Global Hypophosphatasia Registry","phase":"","overall_status":"RECRUITING","enrollment_count":30,"lead_sponsor_name":"Alexion Pharmaceuticals, Inc.","has_results":false},{"nct_id":"NCT02797821","title":"Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP)","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":27,"lead_sponsor_name":"Alexion Pharmaceuticals, Inc.","has_results":true},{"nct_id":"NCT03418389","title":"Evaluate and Monitor Physical Performance of Adults Treated With Asfotase Alfa for Hypophosphatasia","phase":"","overall_status":"COMPLETED","enrollment_count":23,"lead_sponsor_name":"Dr. Lothar Seefried","has_results":false},{"nct_id":"NCT04925804","title":"Unraveling Genetics of HypoPhosPhatasia (HPP Genetics)","phase":"","overall_status":"COMPLETED","enrollment_count":16,"lead_sponsor_name":"CENTOGENE GmbH Rostock","has_results":false},{"nct_id":"NCT04980248","title":"Study of ALXN1850 in Participants With Hypophosphatasia (HPP)","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":15,"lead_sponsor_name":"Alexion Pharmaceuticals, Inc.","has_results":true},{"nct_id":"NCT04222452","title":"The PORTRAIT Study","phase":"","overall_status":"UNKNOWN","enrollment_count":14,"lead_sponsor_name":"Sheffield Teaching Hospitals NHS Foundation Trust","has_results":false},{"nct_id":"NCT05890794","title":"Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia","phase":"PHASE1, PHASE2","overall_status":"COMPLETED","enrollment_count":12,"lead_sponsor_name":"AM-Pharma","has_results":true},{"nct_id":"NCT06015750","title":"Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ®) for Hypophosphatasia","phase":"PHASE4","overall_status":"WITHDRAWN","enrollment_count":0,"lead_sponsor_name":"Alexion Pharmaceuticals, Inc.","has_results":false},{"nct_id":"NCT02496689","title":"Expanded Access Program for Asfotase Alfa Treatment for Patients With Infantile- or Juvenile-onset Hypophosphatasia (HPP)","phase":"","overall_status":"APPROVED_FOR_MARKETING","enrollment_count":null,"lead_sponsor_name":"Alexion Pharmaceuticals, Inc.","has_results":false}],"total":21},"guidelines":[],"source":"Drug Landscape verified database"}