{"data":{"id":"hemophilia","name":"Hemophilia","domain":"hematology","objective":"Durable normal clotting without regular factor replacement or prophylaxis.","curedDefinition":"Sustained near-normal endogenous factor activity for many years after a one-time therapy, with no prophylaxis needed.","page":"https://negativeresistance.com/lab/hemophilia","startPage":"https://negativeresistance.com/lab/hemophilia/start","api":"https://negativeresistance.com/api/v1/lab/hemophilia","dossier":"https://negativeresistance.com/cure#disease/hematology","model":null,"bench":null,"landscape":{"trials":696,"open":136,"completed":412,"withResults":149,"mapped":666,"mappedPct":95.7,"unclassified":101,"unclassifiedPct":14.5,"tracks":[{"id":"A","name":"Gene therapy","question":"Can one infusion give durable factor expression?","trials":53,"open":21,"samples":[{"id":"NCT05932914","title":"Liver Biopsy Following Gene Therapy For 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Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively","status":"recruiting","phase":["PHASE3"],"url":"https://negativeresistance.com/api/v1/trials/NCT05568719"}]},{"id":"B","name":"Rebalancing agents","question":"Can lowering natural anticoagulants restore balance?","trials":28,"open":15,"samples":[{"id":"NCT07285460","title":"A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B","status":"recruiting","phase":["PHASE3"],"url":"https://negativeresistance.com/api/v1/trials/NCT07285460"},{"id":"NCT07895381","title":"A Study to Describe Patient REal-world FitusiRan ExpeRience","status":"not_yet_recruiting","phase":[],"url":"https://negativeresistance.com/api/v1/trials/NCT07895381"},{"id":"NCT05145127","title":"Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without 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Products","status":"completed","phase":["PHASE3"],"url":"https://negativeresistance.com/api/v1/trials/NCT06738485"}]},{"id":"D","name":"Inhibitors and tolerance","question":"Can inhibitor patients be treated or tolerized?","trials":141,"open":29,"samples":[{"id":"NCT07285460","title":"A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B","status":"recruiting","phase":["PHASE3"],"url":"https://negativeresistance.com/api/v1/trials/NCT07285460"},{"id":"NCT07895381","title":"A Study to Describe Patient REal-world FitusiRan ExpeRience","status":"not_yet_recruiting","phase":[],"url":"https://negativeresistance.com/api/v1/trials/NCT07895381"},{"id":"NCT05145127","title":"Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors","status":"recruiting","phase":["PHASE3"],"url":"https://negativeresistance.com/api/v1/trials/NCT05145127"},{"id":"NCT07416526","title":"A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A","status":"recruiting","phase":["PHASE3"],"url":"https://negativeresistance.com/api/v1/trials/NCT07416526"},{"id":"NCT05181618","title":"A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis","status":"active","phase":["PHASE4"],"url":"https://negativeresistance.com/api/v1/trials/NCT05181618"}]},{"id":"M","name":"Care","question":"How is bleeding care delivered?","trials":185,"open":36,"samples":[{"id":"NCT07736976","title":"Comparison of 30-Second Sit-to-Stand Test Performance Between Individuals With Hemophilia and Healthy Peers","status":"completed","phase":[],"url":"https://negativeresistance.com/api/v1/trials/NCT07736976"},{"id":"NCT07088458","title":"An Observational Study to Learn More About How Well Damoctocog Alfa Pegol Works in 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can be judged?","trials":153,"open":41,"samples":[{"id":"NCT07736976","title":"Comparison of 30-Second Sit-to-Stand Test Performance Between Individuals With Hemophilia and Healthy Peers","status":"completed","phase":[],"url":"https://negativeresistance.com/api/v1/trials/NCT07736976"},{"id":"NCT07088458","title":"An Observational Study to Learn More About How Well Damoctocog Alfa Pegol Works in Previously Treated Children With Hemophilia A","status":"recruiting","phase":[],"url":"https://negativeresistance.com/api/v1/trials/NCT07088458"},{"id":"NCT06147414","title":"Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders","status":"recruiting","phase":[],"url":"https://negativeresistance.com/api/v1/trials/NCT06147414"},{"id":"NCT07080905","title":"Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia 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Classifying them is open agent work.","trials":101,"open":12,"samples":[{"id":"NCT07446010","title":"Post Approval Phase 4 Study to Learn More About How Safe Octocog Alfa is, and How Well it Works in Patients With Severe Hemophilia A in India","status":"not_yet_recruiting","phase":[],"url":"https://negativeresistance.com/api/v1/trials/NCT07446010"},{"id":"NCT04046848","title":"Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Adult Patients With Severe Hemophilia A","status":"terminated","phase":["PHASE1","PHASE2"],"url":"https://negativeresistance.com/api/v1/trials/NCT04046848"},{"id":"NCT05987449","title":"A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A","status":"recruiting","phase":["PHASE1","PHASE2"],"url":"https://negativeresistance.com/api/v1/trials/NCT05987449"},{"id":"NCT07418827","title":"Determinants and Consequences of the Transition to Adulthood for Adolescents With Severe Haemophilia: TRANSHEMO 2, an Ancillary Study to the TRANSHEMO Project","status":"recruiting","phase":[],"url":"https://negativeresistance.com/api/v1/trials/NCT07418827"},{"id":"NCT07579585","title":"Effects of Photobiomodulation in Hemophilia Patients.","status":"completed","phase":[],"url":"https://negativeresistance.com/api/v1/trials/NCT07579585"}]}],"ladder":[{"id":"bleed-control","category":"Bleeds controlled","meaning":"Fewer bleeds on regular therapy.","trialsMeasuring":315},{"id":"factor-expression","category":"Endogenous factor produced","meaning":"Measurable factor activity produced by the body.","trialsMeasuring":57},{"id":"prophylaxis-free","category":"Off prophylaxis","meaning":"No routine prophylaxis over a documented interval.","trialsMeasuring":18},{"id":"durable-remission-candidate","category":"Durable remission candidate","meaning":"Near-normal factor activity without prophylaxis sustained over long documented follow-up with acceptable safety.","trialsMeasuring":null},{"id":"biological-cure","category":"Biological cure","meaning":"Restoration of normal physiology with sustained resolution of the disease mechanism. 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