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
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 3 N=61 An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Factor XIII Deficiency
Primary: Adverse Events — 42; 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 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 3 N=41 A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Factor XIII Deficiency
Primary: The Incidence of Spontaneous Bleeding Events Requiring Treatment (Treatment is Defined as Administration of a FXIII-Containing Product to Treat the Bleeding Event) — 0…
Phase 2 N=15 A Study of the Use of Factor XIII Concentrate in Patients With Inherited FXIII Deficiency
Factor XIII Deficiency
Primary: Peak FXIII Concentration at Steady State — 0.9 Units/mL
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
N/A N=72 Clinical Trial of Factor XIII (FXIII) Concentrate
Hemophilia · Factor XIII Deficiency
Primary: Response to Treatment of Bleeding Events Requiring Additional Factor XIII Infusions — 30; 1; 0; 3 Number of bleeds
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=246 An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A
Hemophilia A · Severe Hemophilia A
Primary: Incidence of Inhibitor Formation to FVIII in Previously Treated Patients (PTPs) With 100 Exposure Days (EDs) to CSL627 — 0 percentage of paticipants
Phase 3 N=25 A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa
Hemophilia A With Inhibitors · Hemophilia B With Inhibitors
Primary: Proportion of Successfully Treated Mild/Moderate Bleeding Episodes Per FDA Requirement. — 0.654; 0.603 Proportion of successfully treated BEs
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=84 Pharmacokinetic, Efficacy, and Safety Study of Recombinant Factor VIII Single Chain (rVIII-SingleChain) in Children With Severe Hemophilia A
Congenital Hemophilia A
Primary: Treatment Success — 96.3 Percentage of treated bleeding events
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 2 N=4 Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
Adeno-Associated Virus (AAV) · Blood Coagulation Disorder · Blood Coagulation Disorders, Inherited
Primary: Number of Participants With Adverse Events (AEs) — 4 Participants
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 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 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 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=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
N/A N=103 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 Treatment
Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor · Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor · Haemophilia A
Primary: Mean Number of Daily Active Minutes of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study…
Phase 3 N=108 An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Previously Untreated Males With Severe Hemophilia A
Hemophilia A
Primary: Percentage of Participants With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay — 31.11 percentage of participants
Phase 3 N=15 Recombinant Von Willebrand Factor in Subjects With Severe Von Willebrand Disease Undergoing Surgery
Von Willebrand Disease
Primary: Overall Hemostatic Efficacy as Assessed by the Investigator (Hemophilia Physician) — 11; 4; 7; 0 Participants
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=6 Safety of a Single Intravenous Dose of Recombinant Factor XIII in Children With Congenital FXIII A-subunit Deficiency
Congenital Bleeding Disorder · Congenital FXIII Deficiency
Primary: Area Under the Concentration vs. Time Curve (AUC) — 250.25 IU*h/mL
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 3 N=165 Study to Evaluate the Safety, Pharmacokinetics and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Subjects With Severe Hemophilia A
Severe Hemophilia A
Primary: Incidence Rate of FVIII Inhibitor Development — 0; 0; 0; 0 percentage of 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 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 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