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…
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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…
N/A N=22 Platelet-Rich Plasma Intra-Articular Injection in Treating Hemophilic Arthropathy
Hemophilia · Hemophilic Arthropathy
Primary: Pain (Visual Analogue Scale) and Change From Baseline at 1, 2, 3 &6 Months — 5.0; 4.1; 2.5; 1.8 scores on a scale — p=<0.05
Phase 3 N=29 Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Acquired Hemophilia A
Acquired Hemophilia A
Primary: Percentage of Serious Bleeding Episodes Responsive to OBI-1 — 100 percentage of serious bleeding episodes — p=<0.001
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=104 Multidisciplinary Evaluation of Patients With Hemophilia
Haemophilia
Primary: Assess the Joint Damage — 0.36; 0 points
Phase 2 N=10 Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B
Hemophilia B
Primary: Number of Participants With Adverse Events — 5; 5 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 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
Phase 2 N=15 A Gene Therapy Study for Hemophilia B
Hemophilia B
Primary: Number of Participants With Clinically Significant Change From Baseline in Physical Examination Findings — 0 Participants
Phase 1 N=29 Phase 1 Safety, Pharmacokinetics And Pharmacodynamics Study Of Recombinant Factor VIIa Variant (813d) In Adult Subjects With Hemophilia
Hemophilia A
Primary: Change From Baseline in Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP) — 122.0; 130.2; 120.2; 124.2 mmHg
N/A N=17 Bilateral Deficit Phenomenon in Patients With Haemophilic Arthropathy
Hemophilia
Primary: Assessment of Bilateral Index of Maximal Peak Force — -40.86 percentage
Phase 2 N=26 PF-06741086 Multiple Dose Study in Severe Hemophilia
Hemophilia A or B
Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) — 7; 4; 6; 4 Participants
Phase 4 N=30 Prophylaxis Versus on Demand Treatment for Children With Hemophilia A
Hemophilia A
Primary: Difference of Annualized Number of All Bleeds Between On-demand and Prophylaxis Period — 56.00 Bleeds
Phase 4 N=73 Prophylaxis Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A
Hemophilia A
Primary: Mean Transformed Annualized Bleed Rate Estimates From Each of the 1-year Prophylaxis Regimens — 1.61; 1.46 (bleeds/year)^(1/2) — p=0.6016
Phase 4 N=56 Canadian Hemophilia Prophylaxis Study
Severe Hemophilia A
Primary: Number of Participants Who Developed Target Joint Bleeding — 17 Participants
Phase 1 N=13 Study of PF-05208756, Moroctocog Alfa (AF-CC), Xyntha For Male Chinese Subjects With Hemophilia A
Hemophilia A
Primary: Maximum Plasma FVIII Activity (Cmax) — 1.147 IU/milliliter (mL)
Phase 2 N=4 A Study of BAX 888 in Male Adults With Severe Hemophilia A
Hemophilia A
Primary: Number of Participants With BAX 888-Related Adverse Events (AEs) — 2; 2 Participants
Phase 2 N=11 A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A
Hemophilia A
Primary: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) — 2; 2; 2; 5 Participants
Phase 2 N=20 PF-06741086 Long-term Treatment in Severe Hemophilia
Hemophilia A or B
Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), TEAEs by Severity, and Serious Adverse Events (SAEs) (All Causality and Treatment-Related) — 5; 2…
Phase 2 N=25 A Gene Transfer Study for Hemophilia A
Hemophilia A
Primary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) — 2; 3; 9; 11 Participants
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=63 Phase 3/4 Study of a Recombinant Protein-Free Factor VIII (rAHF-PFM): Comparison of Continuous Infusion Versus Intermittent Bolus Infusion in Hemophilia A Subjects Undergoing Major Orthopedic Surgery
Hemophilia A
Primary: Cumulative Packed Red Blood Cell (PRBC) Volume in the Drainage Fluid During the First 24 Hours Following Surgery in Subjects Receiving ADVATE (rAHF-PFM) by Bolus (BI) or…
Phase 3 N=67 HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
Hemophilia B
Primary: Annualized Bleeding Rate (ABR) for All Bleeding Episodes — 4.19; 1.51 bleeds/year/participants
Phase 4 N=87 PF-05208756, Moroctocog Alfa (AF-CC), Xyntha For Hemophilia A
Hemophilia A
Primary: Percentage of Participants With Factor VIII (FVIII) Inhibitors — 8.22; 7.14 percentage of participants
Phase 1 N=6 Study to Evaluate Safety and Tolerability of a Single Dose of PF-06741086 in Chinese Adult Participants With Severe Hemophilia
Severe Hemophilia
Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) — 1; 0; 0 Participants
Phase 3 N=152 A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors
Hemophilia A
Primary: Annualized Bleeding Rate (ABR) for Treated Bleeds — 38.2; 1.5; 1.3; 1.6 treated bleed rate per year — p=<0.0001
Phase 3 N=55 Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients
Hemophilia A
Primary: Factor VIII Inhibitor Development — 29.1 percentage
Phase 2 N=15 Gene Therapy Study in Severe Haemophilia A Patients (270-201)
Severe Haemophilia A
Primary: Number of Participants With Treatment Emergent Adverse Events — 1; 1; 6; 7 Participants
Phase 3 N=36 Efficacy and Safety Study of Human-cl rhFVIII in PTPs With Severe Hemophilia A
Severe Hemophilia A
Primary: Efficacy Assessment After a Total of at Least 50 EDs Per Subject at the End of the Study at 6 Months — 0.188 Bleeds per month