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Disease · MONDO_0010604

Trials54

Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs. 13 more are not shown, since the checks do not confirm them.

TrialTitlePhaseParticipantsResults first postedConditionsDrugsLinked bySource
NCT01154231BeneFIX Drug Use Results Survey [All-Case Surveillance]3142017-04-11Hemophilia BCOAGULATION FACTOR IX RECOMBINANT HUMANIts record names the diseaseClinicalTrials.gov
NCT00484185Post Marketing Surveillance To Observe Safety and Efficacy Of BeneFIX In Patients With Hemophilia B1832013-08-12Hemophilia BCOAGULATION FACTOR IX RECOMBINANT HUMANIts record names the diseaseClinicalTrials.gov
NCT01027364Study of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Participants With Hemophilia BPhase 31232014-09-04Severe Hemophilia BCOAGULATION FACTOR IX RECOMBINANT HUMAN, EFTRENONACOG ALFAIts record names the diseaseClinicalTrials.gov
NCT03417245A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without InhibitorsPhase 31202022-02-04Hemophilia A, Hemophilia BFITUSIRANIts record names the diseaseClinicalTrials.gov
NCT01174446Pivotal Study (Pharmacokinetics, Efficacy, Safety) of BAX 326 (rFIX) in Hemophilia B PatientsPhase 3862013-11-25Hemophilia BCOAGULATION FACTOR IX RECOMBINANT HUMANIts record names the diseaseClinicalTrials.gov
NCT03549871A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent ProphylaxisPhase 3802023-02-06HemophiliaFITUSIRANIts record names the diseaseClinicalTrials.gov
NCT00768287Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia BPhase 2/3772018-09-10Hemophilia BCOAGULATION FACTOR IX RECOMBINANT HUMANIts record names the diseaseClinicalTrials.gov
NCT01333111Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B PatientsPhase 3742017-07-28Congenital Bleeding Disorder, Haemophilia BNONACOG BETA PEGOLOpen Targets' reading, confirmed by NLM's indexing of its papersClinicalTrials.gov
NCT01395810Safety and Efficacy of NNC-0156-0000-0009 After Long-Term Exposure in Patients With Haemophilia B: An Extension to Trials NN7999-3747 and NN7999-3773Phase 3712018-05-09Congenital Bleeding Disorder, Haemophilia BNONACOG BETA PEGOLOpen Targets' reading, confirmed by NLM's indexing of its papersClinicalTrials.gov
NCT02336178Safety and Efficacy of Benefix in Patients With Hemophilia B in Usual Care Settings in ChinaPhase 4702017-05-04HEMOPHILIA BCOAGULATION FACTOR IX RECOMBINANT HUMANIts record names the diseaseClinicalTrials.gov
NCT03569891HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B PatientsPhase 3672022-10-10Hemophilia BETRANACOGENE DEZAPARVOVECIts record names the diseaseClinicalTrials.gov
NCT03417102A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With InhibitorsPhase 3602021-12-21Hemophilia A, Hemophilia BFITUSIRANIts record names the diseaseClinicalTrials.gov
NCT02141074Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia BPhase 3542023-12-29Congenital Bleeding Disorder, Haemophilia BNONACOG BETA PEGOLOpen Targets' reading of its recordClinicalTrials.gov
NCT03861273A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia BPhase 3512024-03-27Hemophilia BFIDANACOGENE ELAPARVOVECIts record names the diseaseClinicalTrials.gov
NCT01757405Recombinant Factor VIIa BI (rFVIIa BI) Treatment of Acute Bleeding Episodes Per an On-demand RegimenPhase 3402016-10-25Hemophilia A, Hemophilia BEPTACOG ALFA (ACTIVATED)Its record names the diseaseClinicalTrials.gov
NCT00866606Study Evaluating On-Demand Treatment With BeneFIX In Chinese SubjectsPhase 3352011-01-04Hemophilia BCOAGULATION FACTOR IX RECOMBINANT HUMANIts record names the diseaseClinicalTrials.gov
NCT02554773An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or BPhase 1/2342024-06-05Hemophilia A, Hemophilia BFITUSIRANIts record names the diseaseClinicalTrials.gov
NCT02234310Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia BPhase 3332020-07-31Hemophilia BEFTRENONACOG ALFAIts record names the diseaseClinicalTrials.gov
NCT01440946Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia BPhase 3302015-06-18Hemophilia BEFTRENONACOG ALFAIts record names the diseaseClinicalTrials.gov
NCT01507896BAX 326 Surgery Study in Hemophilia B PatientsPhase 3302017-02-15Hemophilia BCOAGULATION FACTOR IX RECOMBINANT HUMANIts record names the diseaseClinicalTrials.gov

diseases-2026-09-18-0.1.0

Sources 6

  • ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
  • ChEMBL, through Open TargetsCC BY-SA 3.0
  • ClinicalTrials.govNo license stated
  • NLM's MeSHNLM's terms
  • Open TargetsCC0 1.0
  • PubMedNLM's terms
Each source's terms
  • ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
  • ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
  • ClinicalTrials.govnone stated
  • NLM's MeSHNLM's
  • Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
  • PubMedNLM's terms

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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