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…
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
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=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 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 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=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 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=159 A Phase 3 Open-label Interventional Study of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein, Efanesoctocog Alfa (BIVV001), in Patients With Severe Hemophilia A
Factor VIII Deficiency
Primary: Estimated Annualized Bleeding Rate (ABR) in Arm A: Prophylaxis — 0.71 episodes per participant per year
Phase 3 N=120 A Study of PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A
Hemophilia A
Primary: Number of Participants With FVIII Inhibitor Development — 11 Participants
Phase 3 N=28 Trial to Evaluate the Efficacy and Safety of a New Full Length Recombinant Human FVIII for Hemophilia A
Blood Coagulation Disorders · Hemophilia A
Primary: Part A - Area Under the Drug Concentration-time Curve (AUC) — 1889.23; 1583.91 Int.units x hours/deciliters (IU*h/dL)
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=6 Study of Next-Generation Recombinant Factor IX Variant in Adult Subjects With Hemophilia B
Hemophilia B
Primary: Number of Subjects Who Achieved FIX Level ≥12% — 3; 4; 4; 4 Participants
Phase 2 N=11 Study of Coagulation Factor VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia A and B
Hemophilia A With Inhibitor · Hemophilia B With Inhibitor
Primary: Bleeding Episode Prevention Success — 1.4640 score on a scale
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
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 1 N=11 Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia
Hemophilia A · Hemophilia B · Hemophilia A With Inhibitor
Primary: Comparative MarzAA Activity by Dose Level/Stage - AUC0-∞ and AUC0-last — 1390.0; 516.4; 849.4; 1060.0 h*ng/mL
Phase 3 N=22 Phase 3 Efficacy and Safety Study of BAX 855 in Severe Hemophilia A Patients Undergoing Surgical Procedures
Hemophilia A
Primary: Global Hemostatic Efficacy Assessment Score (GHEA) - Composed of 3 Individual Ratings — 100.0; 100.0; 100.0; 100.0 Percentage of surgeries
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 3 N=30 BAX 326 Surgery Study in Hemophilia B Patients
Hemophilia B
Primary: Intraoperative Hemostatic Efficacy — 37; 20; 17; 1 surgeries
Phase 3 N=138 Study Investigating a PEGylated Recombinant Factor VIII (BAX 855) for Hemophilia A (PROLONG-ATE Study)
Hemophilia A
Primary: Annualized Bleeding Rate (ABR) — 4.3; 43.4 Bleeds per year — p=<0.0001
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 3 N=27 Phase III Study of Coagulation FVIIa (Recombinant) in Congenital Hemophilia A or B Patients With Inhibitors
Hemophilia A With Inhibitors · Hemophilia B With Inhibitors
Primary: Proportion of Successfully Treated Mild/Moderate Bleeding Episodes — .849; .932 Proportion of Success of BEs
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 2 N=16 A Safety, Tolerability, and Pharmacokinetics Study of a Single Intravenous Injection of Recombinant Coagulation Factor VIII Fc - Von Willebrand Factor - XTEN Fusion Protein (rFVIIIFc-VWF-XTEN) (BIVV001) in Previously Treated Adults With Severe Hemophilia A (EXTEN-A)
Hemophilia A
Primary: Number of Participants With Treatment Emergent Adverse Events (TEAE) and Treatment Emergent Serious Adverse Event (TESAE) During Advate Treatment Period — 2; 1; 1; 0…
Phase 4 N=26 Dose-Response Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A
Hemophilia A
Primary: Initial Recovery — 1.7; 1.6; 1.8 IU/dL per IU/kg — p=0.1662
Phase 3 N=134 Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)
Hemophilia A
Primary: Change From Baseline in Annualized Number of Bleeding Episodes Irrespective of Exogenous FVIII Replacement Treatment [Annualized Bleeding Rate (ABR) for All Bleeds] in…
Phase 3 N=1 Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
Hemophilia A
Primary: Change of the Median Factor VIII (FVIII) Activity — 4.1 IU/dL
Phase 3 N=110 Human Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated Patients
Severe Hemophilia A
Primary: Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors — 17; 11; 28 Participants
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 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