Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Patients With Severe Haemophilia A

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Pharmacokinetics Evaluation of Recombinant Coagulation Factor VIII Injection in Subjects With Hemophilia A. Safety and Efficacy of Turoctocog Alfa in Haemophilia A Subjects Study Evaluating ReFacto AF in Severe Hemophilia A A Multi-centre, Comparative, Double Blind, Randomised Cross-over Trial Investigating Single Dose Pharmacokinetics and Safety of Turoctocog Alfa Pegol From the Pivotal Process and Turoctocog Alfa Pegol From the Commercial Process in Patients With Severe Haemophilia A An Open Study to Investigate the Safety and Efficacy of Optivate® in Severe Haemophilia A Patients. 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A Study of the Impact of Hemophilia and Its Treatment on Brain Development, Thinking and Behaviour in Children With Hemophilia The Effect of Patient Counseling on Adolescent Hemophilia Patient Compliance With Bleeding Logs Early Prophylaxis Immunologic Challenge (EPIC) Study Exit Interviews to Assess Impact of Infusion Frequency in Hemophilia A Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Patients With Severe Haemophilia A Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5 PK Driven Prophylaxis for Hemophilia A Personalized Medicine for Canadians With Hemophilia Lentiviral FVIII Gene Therapy Retrospective Study in Chinese Pediatric Hemophilia A Patients With rFⅧ Contained Regular Prophylaxis Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A Efficacy and Safety of Recombinant Factor VIII (GreenGene) in Patients With Hemophilia A Study to Compare How the Body Distributes and Excretes the Drugs Jivi (BAY 94-9027) and Adynovi in Patients With Severe Hemophilia A (Bleeding Disorder Resulting From a Lack of Blood Clotting Factor VIII) Individualized Prophylaxis for Severe Hemophilia A Children A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors Safety and Efficacy of Long-term Treatment With SCT800 in Previously Treated Hemophilia A Patients. Study of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Subjects With Severe Hemophilia A Safety,Efficacy and Pharmacokinetics Evaluation of SCT800 in Previously Treated Paediatric Patients With Severe Haemophilia A. An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A Study of FVIIa Variant BAY86-6150 (B0189) in Subjects With Moderate or Severe Hemophilia Types A or B With or Without Inhibitors Prophylaxis Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A Evaluation of SCT800 in Prophylaxis Therapy on Previous Treated Patients With Severe Hemophilia A A Study of Emicizumab Administered Subcutaneously (SC) in Pediatric Participants With Hemophilia A and Factor VIII (FVIII) Inhibitors Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Paediatric Patients With Severe Haemophilia A Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Pediatric Subjects With Hemophilia A Clinical Study to Investigate the PK, Efficacy, and Safety of Wilate in Patients With Severe Hemophilia A Long-Term Safety and Efficacy of rFVIIIFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia A 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) Study to Evaluate the Efficacy and Safety of PF-07055480 in Moderately Severe to Severe Hemophilia A Adults Pharmacokinetic Study of ADVATE 3000 IU in Previously Treated Patients With Severe Hemophilia A Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients Exercise Versus DDAVP in Patients With Mild Hemophilia A A Trial to Compare Prophylaxis Therapy to On-demand Therapy With a New Full Length Recombinant FVIII in Patients With Severe Hemophilia A Evaluate Efficacy and Safety of Recombinant Factor VIII （rFVIII）Treatment of Severe or Moderately Severe Hemophilia A Study of rFVIIIFc for ITI in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI Therapies Study Investigating a PEGylated Recombinant Factor VIII (BAX 855) for Hemophilia A (PROLONG-ATE Study) Safety, Efficacy Evaluation of Recombinant Coagulation Factor VIII Injection in Subjects With Hemophilia A. Efficacy and Safety Study of Human-cl rhFVIII in PTPs With Severe Hemophilia A Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety and Immunogenicity of a Recombinant FVIII in Patients With Severe Hemophilia A Efficacy and Safety Study of SCT800 for On-demand Treatment With in Patients With Hemophilia A Safety and Efficacy Study of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients Undergoing Surgery Safety, Efficacy and Pharmacokinetics of GreenGene™ F to Previously Treated Patients With Severe Hemophilia A Phase 3 Efficacy and Safety Study of BAX 855 in Severe Hemophilia A Patients Undergoing Surgical Procedures The Hemophilia Ultrasound Project An Expanded Access Program of Emicizumab in Participants With Hemophilia A With Inhibitors PF-05208756, Moroctocog Alfa (AF-CC), Xyntha For Hemophilia A Safety and Efficacy Extension Study of GreenGene™ F in Previously Treated Patients Diagnosed With Severe Hemophilia A Pharmacokinetic Comparison of Advate rAHF-PFM With Recombinate rAHF in Patients With Severe Hemophilia A A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Given Every 4 Weeks in Participants With Hemophilia A A Gene Transfer Study for Hemophilia A PHYSICAL ACTIVITY AND FVIII CLEARANCE: RELEVANCE FOR PERSONALIZED THERAPY IN SEVERE HAEMOPHILIA A (PHYSEMO) Prophylaxis Versus on Demand Treatment for Children With Hemophilia A Study of PF-05208756, Moroctocog Alfa (AF-CC), Xyntha For Male Chinese Subjects With Hemophilia A Long-Term Safety and Efficacy of SPK-8011 in Males With Hemophilia A An Open Study to Investigate the Safety and Efficacy of Optivate® in Haemophilia A Patients Undergoing Surgery. Observational Study on Safety of Room Temperature Stable NovoSeven® in Patients With Haemophilia A or B ADVATE/ ADYNOVI Hemophilia A Outcome Database (AHEAD) Pharmacokinetics of Single Bolus Dose of NovoSeven® in Paediatric and Adult Patients With Haemophilia A or B in a Non- Bleeding State Investigating Safety and Pharmacokinetics of 2 Different Single Doses of NNC128-0000-2011 in Haemophilia A or B Patients Safety and Mode of Action of a Single Dose and Multiple Doses of Long Acting Activated Recombinant Human Factor VII in Patients With Haemophilia A and B Gene Therapy for Haemophilia A. Health Related Quality of Life of Youth and Young Adults With Hemophilia A Trial to Evaluate the Effect of Secondary Prophylaxis With rFVIII Therapy in Severe Hemophilia A Adult and/or Adolescent Subjects Compared to That of Episodic Treatment Web-based Application for the Population Pharmacokinetic Service – Phase 1 Assessing Physician and Hemophilia A Patient Reasons and Expectations for Switching Treatment to Kovaltry & Jivi: A Nested Study Within an Existing Registry Study Comparing Blood Levels of ReFacto and Advante in Hemophilia A Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Adult Patients With Severe Hemophilia A Subclinical Joint Bleeding in Irish Adults With Severe Haemophilia A on Personalised Prophylaxis Regimens Safety/Efficacy Study to Assess Whether FVIII/VWF Concentrate Can Induce Immune Tolerance in Haemophilia A Patients Multicentre, Non-controlled, Prospective, Post-Marketing Safety Study Following Long-Term Prophylactic OptivateTreatment in Subjects With Severe Haemophilia A Validation Study of a cOmputer Pharmacokinetic Tool to assIst in the Follow up Care of haeMophilia A Patients Joint Status in Subjects With Severe Hemophilia A in Relation to Different Treatment Regimens Safety Study of Alphanate in Previously Treated Patients With Severe Hemophilia A Treatment of Hemophilia A Patients With FVIII Inhibitors Comparison of Prophylaxis and On-demand Treatment in Children With Moderate to Severe Hemophilia A An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Untreated Males With Severe Hemophilia A Assessment of the Risk of Inhibitor Formation in Previously Treated Patients With Severe Hemophilia A Efficacy and Safety of ADVATE Standard Prophylaxis to Hemophilia A Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A National Study of a Pharmacokinetic-Focused Educational Package for Patients With Severe Haemophilia A Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A Drug Use Investigation of Kovaltry in Hemophilia A Patients Safety, Immunogenicity and Hemostatic Efficacy of PEGylated Recombinant FVIII (BAX 855) in Previously Untreated Patients (PUPs) < 6 Years of Age With Severe Hemophilia A 2-cohort Study of Adult Patients With Severe Hemophilia A in Greece Study of Emicizumab Prophylaxis in Participants With Hemophilia A With or Without Inhibitors Undergoing Minor Surgical Procedures Efficacy, Safety, and Pharmacokinetic Study of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Participants Individually Tailored Prophylaxis in Patients With Severe Hemophilia A Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients Retrospective Chart Review to Evaluate Safety and Tolerability of ADVATE Among Previously Untreated Patients in China With Moderate to Severe Hemophilia A Comparison of Different Prophylaxis Regimens for Moderate to Severe Hemophilia A Pediatric Patients BAY79-4980 Compared to rFVIII-FS in Previously Treated Patients With Severe Hemophilia A Clot Formation and Clot Stability in Patients With Severe Haemophilia A Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A RIXUBIS PMS India (RIXUBIS PMS) Trial of Rituximab Versus Oral Cyclophosphamide to Eradicate or Suppress Autoimmune Anti-Factor VIII Antibodies in Acquired Hemophilia A Gene Therapy Study in Severe Haemophilia A Patients Moroctocog Alfa (AF-CC) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Hemophilia A Patients Study Evaluating Inhibitor Specificity in Hemophilia A A Prospective Study to Collect High-Quality Documentation of Bleeds, Health-Related Quality of Life (HRQoL), and Safety Outcomes in Patients With Hemophilia A Treated With Standard-of-Care Treatment Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg Study Evaluating B-Domain Deleted Recombinant Factor VIII (BDDrFVIII, ReFacto AF) in Patients With Hemophilia A Undergoing Elective Major Surgery Efficacy and Cost Effectiveness of Pharmacokinetic Dosing in Haemophilia A 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 Evaluate Efficacy and Safety of ADVATE in the Standard Prophylaxis Treatment of Severe or Moderately Severe Hemophilia A A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors Study of Biostate® in Children With Hemophilia A Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Congenital Hemophilia A Extension at 10 Years of the: “Observational Study Evaluating Efficacy and Costs of 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of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients – A Continuation of Clinical Study 069901 Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a “Shuttle”. A Trial Investigating Safety and Efficacy of Treatment With BAY94-9027 in Severe Hemophilia A Joint Health Study Study Evaluating Safety And Efficacy Of Moroctocog Alfa (AF-CC) In Previously Treated Hemophilia A Patients A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors UK – EHL Outcomes Registry Study to Establish Bioequivalence of ReFacto AF (BDDrFVIII) With Advate (FLrFVIII) in Hemophilia A Evaluation of Long-term Safety of ADYNOVI/ADYNOVATE (Antihaemophilic Factor [Recombinant] PEGylated, Rurioctocog Alfa Pegol) in Patients With Haemophilia A – An ADYNOVI/ADYNOVATE Post-Authorisation Safety Study (PASS) Platelet Function in Patients With Hemophilia A Study to Learn More About the Physical Activity Level of Patients Suffering From Hemophilia A Treated With Damoctocog Alfa Pegol (LIFE ACTIVE Study) Pharmacokinetic Characterization of the Hemophilia A Population in Spain Using myPKFiT® Impact of Conservative 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Patients Without Inhibitors Phase I/II Study of Monoclonal Factor IX Concentrate for Factor IX Deficiency The Effectiveness of Recombinant Fusion Protein Linking Coagulation Factor IX With Recombinant Albumin (rIX-FP) in Severe Hemophilia B Patients Switching From Previous Factor IX Treatment The Safety and Tolerability of SerpinPC in Healthy Men and in Men With Severe Blood Disorders (Haemophilia A and B) IMMUNINE Pre-Treatment Study Efficacy and Safety of NNC-0156-0000-0009 During Surgical Procedures in Subjects With Haemophilia B Study of Ataluren (PTC124®) in Hemophilia A and B Safety of 40K Pegylated Recombinant Factor IX in Non-Bleeding Patients With Haemophilia B Fc factOrs and Real-World hemophiliA Patient-ReporteD Outcomes Bridging Hemophilia B Experiences, Results and Opportunities Into Solutions (B-HERO-S) Study Evaluating Approach to Treatment of Haemophilia A and B in Spain Global Epidemiologic Study of Preexisting Immunity to AAV in Adults With Severe Hemophilia Safety 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Mutation p.Ile112Thr : Discrepancy Between Factor IX Level and Bleeding Phenotype LTFU for Gene Transfer Subjects With Hemophilia B A Phase 1/2 Study of SHP648, an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects Adverse Event Data Collection From External Registries on Nonacog Beta Pegol Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B Patients Lentiviral FIX Gene Therapy An Open Study to Investigate the Safety and Efficacy of Replenine®-VF in Severe Haemophilia B Patients Study to Describe the Allergic Reactions to Factor IX in Patients With Hemophilia B A Study to Evaluate the Real-world Usage and Effectiveness of Elocta and Alprolix in Patients With Haemophilia A or B A Long-Term Follow-Up Study of Haemophilia B Patients Who Have Undergone Gene Therapy A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B Long-Term Safety, Tolerability, and Efficacy of 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Brief Title

Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Patients With Severe Haemophilia A

Official Title

Prospective, Open-label, Multicenter Phase 3b Study to Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Previously Treated Adult Patients With Severe Haemophilia A

Brief Summary

      To compare the number of breakthrough bleeds under tailored prophylaxis with Human cell line
      recombinant factor FVIII (Human-cl rhFVIII) with the historical bleeding rate from patients
      who received Human-cl rhFVIII as on demand treatment.

Detailed Description

      There were 3 phases in this study: (1) An initial pharmacokinetic (PK) assessment in which
      participants received a single infusion of 60±5 IU/kg of Human-cl rhFVIII; blood samples were
      collected for 72 hours following the infusion. (2) Prophylactic Treatment-Phase I during
      which participants received infusions of 30-40 IU/kg of human-cl rhFVIII every other day or
      3x/week for 1-3 months. (3) Prophylactic Treatment-Phase II during which the dose and dosing
      interval were determined individually from data gathered in the initial PK assessment. The
      maximum dosing interval with a dose of ≤ 60-80 IU/kg that maintains a trough level of ≥ 0.01
      IU/mL was determined. Participants were treated for 6 months.

Study Phase

Phase 3

Study Type

Interventional

Primary Outcome

Annualized Number of Bleeding Episodes (BE) in Phase II

Secondary Outcome

 Annualized Number of Spontaneous Bleeding Episodes (BE) in Phase II

Condition

Severe Haemophilia A

Intervention

Human-cl rhFVIII

Study Arms / Comparison Groups

 Human-cl rhFVIII
Description:  Up to 60-80 IU/kg of intravenous Human-cl rhFVIII was administered at an individually determined dose and dose interval.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Biological

Estimated Enrollment

66

Start Date

August 2013

Completion Date

January 2015

Primary Completion Date

January 2015

Eligibility Criteria

        Inclusion Criteria:

          -  Severe haemophilia A (FVIII:C < 1%) according to medical history.

          -  Male patients ≥ 18 years old.

          -  Previous treatment with a FVIII concentrate (regular prophylaxis with good compliance
             or on-demand treatment) for at least 150 exposure days (EDs).

          -  Good documentation regarding dosing and bleeding frequency in the 6 months preceding
             study start.

          -  Immunocompetence (CD4+ count > 200/microliter).

          -  HIV-negative, if positive, viral load < 200 particles/microliter or < 400,000
             copies/mL.

          -  Freely given written informed consent

        Exclusion Criteria:

          -  Any coagulation disorder other than haemophilia A.

          -  Present or past FVIII inhibitor activity (> 0.6 Bethesda Unit [BU])

          -  Severe liver or kidney disease.

Gender

Male

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, ,

Location Countries

Austria

Location Countries

Austria

Administrative Informations

NCT ID

NCT01863758

Organization ID

GENA-21

Responsible Party

Sponsor

Study Sponsor

Octapharma

Study Sponsor

, ,

Verification Date

July 2017