Phase 4 N=21 Prophylaxis Regimen for Hemophilia A Patients
Hemophilia A · Prophylaxis of Bleeding
Primary: Overall Number of Participants With Favorable Outcome on the Score-assigned Prophylaxis Regimen — 12 participants
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Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 3 N=212 A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)
Hemophilia B · Hemophilia A
Primary: Annualized Bleeding Rate (ABR) for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia B Participants: Efficacy Analysis Set — 3.61…
Phase 3 N=36 A Study to Learn How Safe the Study Treatment BAY94-9027 is and How it Affects the Body in Previously Treated Children Aged 7 to Less Than 12 Years With Severe Hemophilia A, a Genetic Bleeding Disorder That is Caused by the Lack of a Protein Called Clotting Factor 8 (FVIII) in the Blood
Treatment of Bleeding · Prophylaxis of Bleeding · Hemophilia A
Primary: Adverse Events of Special Interest (AESI) Hypersensitivity and Loss of Efficacy Associated With the First 4 Exposure Days (EDs) Leading to Discontinuation — 1 Participants
Phase 4 N=50 Moroctocog Alfa (AF-CC) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Hemophilia A Patients
Hemophilia A
Primary: Percentage of Participants Who Developed Factor VIII (FVIII) Inhibitors — 0 percentage of participants
N/A N=104 Multidisciplinary Evaluation of Patients With Hemophilia
Haemophilia
Primary: Assess the Joint Damage — 0.36; 0 points
Phase 2 N=23 Rituximab to Treat Severe Hemophilia A
Hemophilia A
Primary: Proportion of Subjects With Major Response, i.e. Inhibitor Level Falls to Less Than 5 BU/mL Between Weeks 6 to 22 and Remains Below 5 BU/mL at 5-7 Days Following…
Phase 3 N=5 Study of TAK-672 in Participants With Acquired Hemophilia A
Acquired Hemophilia A
Primary: Percentage of Participants With Severe Bleeding Episodes Who Demonstrated Response to TAK-672 Therapy at 24 Hours After the Initiation of Treatment — 100 percentage of…
N/A N=66 An Institutional Pilot Study to Investigate Physical Activity Patterns in Boys With Hemophilia
Hemophilia A · Hemophilia B · Factor VIII Deficiency
Primary: MVPA — 48; 55 minutes per day — p=0.32
Phase 4 N=23 Study of Safety And Efficacy Of ReFacto AF In Previously Untreated Hemophilia A Patients In The Usual Care Setting
Hemophilia A
Primary: Percentage of Participants Who Developed Clinically Significant Factor VIII (FVIII) Inhibitors During the Course of the Study — 21.74 Percentage of participants
Phase 3 N=97 A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B
Hemophilia B
Primary: Total Number of Participants Who Developed Inhibitors Against Factor IX (FIX) — 0; 1 Participants
Phase 3 N=16 Prospective Biomarkers of Bone Metabolism in Hemophilia A
Hemophilia · Bone Disease
Primary: Bone Biomarker Density (BMD) — -0.74; -0.16; -0.16 Z-Score
Phase 3 N=134 A Trial Investigating Safety and Efficacy of Treatment With BAY94-9027 in Severe Hemophilia A
Hemophilia A
Primary: Annualized Number of Total Bleeds in On-demand Treatment Arm (Weeks 0 -36) and in Each Prophylaxis Arm (Weeks 10 - 36, Excluding Rescue Bleeds) - Part A, Main Trial…
Phase 3 N=57 Clinical Study to Investigate the PK, Efficacy, and Safety of Wilate in Patients With Severe Hemophilia A
Severe Hemophilia A
Primary: Total Annualized Bleeding Rate (TABR) — 2.10 No. of BEs / year (ABR) — p=<0.0001
Phase 3 N=85 Efficacy, Safety, and Pharmacokinetic Study of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Participants
Hemophilia A
Primary: Model-Based Annualized Bleeding Rate for Treated Bleeds — 27.0; 1.0; 1.0; 1.2 Treated bleeds per year — p=<0.0001
Phase 3 N=240 Long-Term Safety and Efficacy of rFVIIIFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia A
Hemophilia A
Primary: Number of Participants With Any Positive Inhibitor Development — 0; 0; 0 Participants