Hoffmann-La Roche: Hemophilia A trials

The registry lists 25 Hemophilia A studies with Hoffmann-La Roche as lead sponsor. 5 are open to participants now and 13 have results posted.

Phase 1 6Phase 2 4Phase 3 12Phase 4 2No phase 5(a study in two phases counts in both)

Studies

Open to participants first, then studies with results posted, then the rest; newest registration first within each.

• Clinical Trial of NXT007 for the Prophylactic Treatment of Hemophilia A in Infants and ChildrenRecruitingNCT07838194Phase 3
• A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia ARecruitingNCT07416604Phase 3
• A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia ARecruitingNCT07416526Phase 3
• A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia ARecruitingNCT07226206Phase 1/2
• A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia ARecruitingNCT05987449Phase 1/2
• A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without InhibitorsActive, not recruitingNCT04431726Phase 3Results posted
• An Observational Study to Evaluate Physical Activity, Bleeding Incidence and Health Related Quality of Life, in Participants With Haemophilia A Without Inhibitors Receiving Standard of Care TreatmentCompletedNCT04165135Results posted
• A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII InhibitorsCompletedNCT04158648Phase 3Results posted
• Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII InhibitorsCompletedNCT03734588Phase 1/2Results posted
• A Study to Investigate the Pharmacokinetics, Safety, and Tolerability of Emicizumab in Healthy Chinese VolunteersCompletedNCT03380780Phase 1Results posted
• Study of Emicizumab Prophylaxis in Participants With Hemophilia A With or Without Inhibitors Undergoing Minor Surgical ProceduresTerminatedNCT03361137Phase 4Results posted
• Efficacy, Safety, and Pharmacokinetic Study of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A ParticipantsCompletedNCT03315455Phase 3Results posted
• A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With InhibitorsCompletedNCT03191799Phase 3Results posted
• A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Given Every 4 Weeks in Participants With Hemophilia ACompletedNCT03020160Phase 3Results posted
• A Gene Transfer Study for Hemophilia ACompletedNCT03003533Phase 1/2Results posted
• A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without InhibitorsCompletedNCT02847637Phase 3Results posted
• A Study of Emicizumab Administered Subcutaneously (SC) in Pediatric Participants With Hemophilia A and Factor VIII (FVIII) InhibitorsCompletedNCT02795767Phase 3Results posted
• A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants With InhibitorsCompletedNCT02622321Phase 3Results posted
• Study of a Gene Therapy Treatment for Hemophilia AWithdrawnNCT06297486Phase 3
• 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 ProphylaxisActive, not recruitingNCT05181618Phase 4
• Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A ParticipantsCompletedNCT03876301
• Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia AActive, not recruitingNCT03432520
• An Expanded Access Program of Emicizumab in Participants With Hemophilia A With InhibitorsApproved for marketingNCT03154437
• A Prospective Study to Collect High-Quality Documentation of Bleeds, Health-Related Quality of Life (HRQoL), and Safety Outcomes in Patients With Hemophilia A Treated With Standard-of-Care TreatmentCompletedNCT02476942
• Hemophilia B Gene Therapy With AAV8 VectorTerminatedNCT01620801Phase 1