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
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Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
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 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 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 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=33 Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B
Hemophilia B
Primary: Percentage of Participants With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay — 3.03 percentage of 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 2 N=17 A Safety and Efficacy Study of a Recombinant Factor IX in Patients With Severe Hemophilia B
Hemophilia B
Primary: Number of Subjects With Treatment-related Adverse Events — 0 participants
Phase 3 N=32 Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B
Hemophilia B
Primary: Degree of Hemorrhage Control by Treatment Regimen — 53; 116; 105; 74 Bleeding Episodes
Phase 3 N=50 Study Comparing On-Demand Treatment With Two Prophylaxis Regimens Of BeneFIX In Patients With Severe Hemophilia B
Hemophilia B
Primary: Annualized Number of Bleeding Episodes — 35.1; 4.6; 2.6 episodes — p=<0.0001
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 3 N=63 A Safety and Efficacy Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B
Hemophilia B
Primary: Change in Frequency of Spontaneous Bleeding Events Between On-demand and Prophylaxis Treatments (Annualized) — 15.43; 0.00 bleeds/year/subject — p=<0.0001
Phase 3 N=30 Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B
Hemophilia B
Primary: Occurence of Factor IX (FIX) Inhibitor Development — 0; 0; 0; 0 percentage of participants
Phase 1 N=9 Dose-escalation Study to Investigate the Safety, PK, and PD of ISU304/CB2679d in Hemophilia B Patients
Hemophilia B
Primary: Number of Adverse Events (AEs) After the Administration of Investigational Products (IP) — 0; 10; 10; 69 adverse events
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 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=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=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=123 Study of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Participants With Hemophilia B
Severe Hemophilia B
Primary: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities — 2; 0; 2; 0 participants
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=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=28 Pivotal Study (Pharmacokinetics, Efficacy, Safety) of BAX 326 (rFIX) in Hemophilia B Patients
Hemophilia B
Primary: Study Part 1- Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours Per Dose — 14.30; 13.42 (IU·hr/dL) / (IU/kg)
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 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 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 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 4 N=20 BAY14-2222 Prophylaxis and Joint Function Improvement (Adults)
Hematologic Disease · Hemophilia A
Primary: Number of Joint Bleeds — 18.5; 1.5 Number of joint bleeds — p=<0.001
Phase 3 N=218 BAX 855 Continuation
Hemophilia A
Primary: Number of Participants With Inhibitory Antibodies to Factor VIII (FVIII) — 0; 0; 0; 0 Participants
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 3 N=21 Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B
Hemophilia B
Primary: Annualized Bleeding Rate (ABR) — 2.93 bleeding episodes per year