Severe Hemophilia A Clinical Trial
Official title:
R34 Pilot Feasibility Randomized, Noninferiority, Cross-Over Trial of Once-Weekly vs. Thrice-Weekly Prophylaxis With Recombinant Factor VIII in Adults With Severe Hemophilia A
The purpose of this pilot R34 trial is to determine the feasibility of a large single dose Phase III study of hemophilia adult prophylaxis comparing once weekly with thrice-weekly recombinant factor VIII. Efficacy will measured by bleeding frequency, factor usage, joint range of motion, cost, quality-of-life, F.VIII level, and inter-dose hypocoagulability by thrombin generation. Safety will be measured by inhibitor formation and bleeding events unresponsive to up to two rescue doses.
The purpose of this 52-week pilot R34 randomized, open-label, non-inferiority, cross-over study is to determine the feasibility of a large single dose Phase III study of hemophilia adult prophylaxis. The primary efficacy endpoint will be bleeding frequency. Secondary endpoints will include factor usage, joint range of motion, cost, quality-of-life, and inter-dose hypocoagulability by thrombin generation time and F.VIII activity will also be determined. Safety will be measured by the frequency of bleeding unresponsive to up to two rescue treatments. Inhibitor formation by anti-F.VIII Bethesda assay, and clinical frequency of thrombosis and allergic reactions will also be assessed. Subject acceptance and adherence to the treatment interventions will be determined; and web-based data entry of case report forms, digital range-of-motion images, and quality-of-life instrument will be implemented. The relation of bleeding frequency to relative inter-dose hypocoagulability, will be assessed by inter-dose thrombin generation time (TGT), endogenous thrombin potential (ETP), and factor VIII levels. Optimal blood sample collection and shipping methods will be determined. For all tests, we will estimate and determine completeness and congruency, in order to determine adjustments or revisions required before initiating a large phase III Randomized clinical trial. All testing will be exploratory, so that we may determine if the test, approach are realistic, and to estimate standard deviations for future power analyses. ;
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Open Label, Primary Purpose: Treatment
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Withdrawn |
NCT01051544 -
Study of First TIME Immunotolerance Induction in Severe Hemophilia A Patients With Inhibitor at High Risk of Failure: Comparison With FVIII Concentrates With or Without Von Willebrand Factor - RES.I.S.T. Naive
|
N/A | |
| Completed |
NCT02172950 -
An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A
|
Phase 3 | |
| Completed |
NCT01051076 -
Rescue Immunotolerance Study in Induction of Immune Tolerance (ITI)-Experienced Patients (RES.I.S.T. Experienced)
|
N/A | |
| Completed |
NCT03376516 -
Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Paediatric Patients With Severe Haemophilia A
|
Phase 3 | |
| Completed |
NCT01181128 -
Study to Evaluate the Safety, Pharmacokinetics and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Subjects With Severe Hemophilia A
|
Phase 3 | |
| Not yet recruiting |
NCT06136507 -
Study of Efficacy and Safety of FRSW107 in Pediatric Patients With Severe Hemophilia A
|
Phase 3 | |
| Completed |
NCT02083965 -
Pharmacokinetics of rFVIIIFc at Two Vial Strengths
|
Phase 1 | |
| Completed |
NCT01085344 -
Canadian Hemophilia Prophylaxis Study
|
Phase 4 | |
| Completed |
NCT04864743 -
A Study to Evaluate the Pharmacokinetics，Safety and Tolerability of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection
|
Phase 1 | |
| Completed |
NCT01341912 -
Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)
|
Phase 3 | |
| Completed |
NCT01125813 -
Efficacy and Safety Study of Human-cl rhFVIII in PTPs With Severe Hemophilia A
|
Phase 3 | |
| Active, not recruiting |
NCT05181618 -
A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis
|
Phase 4 | |
| Terminated |
NCT04046848 -
Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Adult Patients With Severe Hemophilia A
|
Phase 1/Phase 2 | |
| Completed |
NCT06137092 -
rFVIII-Fc (Produced by AryoGen Pharmed Co.) Pharmacokinetic Study
|
Phase 3 | |
| Active, not recruiting |
NCT04431726 -
A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors
|
Phase 3 | |
| Recruiting |
NCT05935358 -
Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study
|
Phase 4 | |
| Completed |
NCT01712438 -
Human Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated Patients
|
Phase 3 | |
| Recruiting |
NCT06142552 -
Phase 3 Clinical Project of Pegylated Recombinant Human Coagulation Factor VIII-Fc Fusion Protein
|
Phase 3 | |
| Completed |
NCT02954575 -
Clinical Study to Investigate the PK, Efficacy, and Safety of Wilate in Patients With Severe Hemophilia A
|
Phase 3 | |
| Completed |
NCT01027377 -
Study of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Subjects With Severe Hemophilia A
|
Phase 1 |