Study Investigating a PEGylated Recombinant Factor VIII (BAX 855) for Hemophilia A (PROLONG-ATE Study)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Baxter Healthcare Corporation
ClinicalTrials.gov Identifier:
NCT01736475
First received: November 21, 2012
Last updated: November 21, 2013
Last verified: November 2013
  Purpose

To assess efficacy and safety, including immunogenicity of BAX 855 administered as prophylaxis and as on-demand therapy in adult and adolescent (12-65 years) previously treated patients (PTPs) with severe hemophilia A To determine the pharmacokinetic (PK) parameters of BAX 855.


Condition Intervention Phase
Hemophilia A
Biological: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method
Biological: PEGylated Recombinant Factor VIII
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Factorial Assignment
Masking: Open Label
Primary Purpose: Prevention
Official Title: A Phase 2/3, Multi-Center, Open Label Study of Efficacy, Safety, and Pharmacokinetics of PEGylated Recombinant Factor VIII (BAX 855) Administered for Prophylaxis and Treatment of Bleeding in Previously Treated Patients With Severe Hemophilia A

Resource links provided by NLM:


Further study details as provided by Baxter Healthcare Corporation:

Primary Outcome Measures:
  • Annualized Bleeding Rate (ABR) [ Time Frame: 9 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Rate of success of BAX 855 for treatment of bleeding episodes [ Time Frame: 9 months ] [ Designated as safety issue: No ]
    Hemostatic effect of BAX855 on treatment of bleeding episodes assessed on an objective four point efficacy scale: Excellent, Good, Fair, and None

  • Number of BAX 855 infusions needed for the treatment of bleeding episodes [ Time Frame: 9 months ] [ Designated as safety issue: No ]
  • Time intervals between bleeding episodes [ Time Frame: 9 months ] [ Designated as safety issue: No ]
  • Weight-adjusted consumption of BAX 855 [ Time Frame: 9 months ] [ Designated as safety issue: No ]
  • Occurrence of Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: 9 months ] [ Designated as safety issue: Yes ]
  • Changes in vital signs and clinical laboratory parameters [ Time Frame: 9 months ] [ Designated as safety issue: Yes ]
  • Immunogenicity [ Time Frame: 9 months ] [ Designated as safety issue: Yes ]
  • Patient Reported Outcomes - Bleed and pain severity [ Time Frame: 9 months ] [ Designated as safety issue: No ]
    Bleed and pain severity- change from baseline

  • Patient Reported Outcomes - Health Related Quality of Life (HQRoL) [ Time Frame: 9 months ] [ Designated as safety issue: No ]
    HQRoL- change from baseline

  • Pharmacokinetics (Pk) [ Time Frame: 9 months ] [ Designated as safety issue: No ]

    BAX 855 PK parameters based on FVIII levels following an initial single dose of

    BAX 855 and after ≥50 exposure days (EDs) to BAX 855. Pk parameters include:

    Plasma half-life (T1/2), Mean residence time, Total body clearance (CL), Incremental recovery over time (IR), Area under the concentration versus time curve from 0 to infinity (AUC0-∞), Apparent volume of distribution at steady state (Vss), Maximum plasma concentration Cmax, Time to maximum concentration in plasma (Tmax)



Enrollment: 159
Study Start Date: January 2013
Estimated Study Completion Date: August 2014
Estimated Primary Completion Date: June 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Prophylaxis Biological: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method
Pharmacokinetic (PK) evaluation of ADVATE
Other Name: ADVATE
Biological: PEGylated Recombinant Factor VIII
Pharmacokinetic (PK) evaluation of BAX 855
Other Name: BAX 855
Biological: PEGylated Recombinant Factor VIII
Prophylaxis treatment
Other Name: BAX 855
Experimental: On-demand Biological: PEGylated Recombinant Factor VIII
On-demand treatment
Other Name: BAX 855

  Eligibility

Ages Eligible for Study:   12 Years to 65 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Main Inclusion Criteria:

  • Participant and/or legal representative has/have voluntarily provided signed informed consent
  • Participant is 12 to 65 years old at the time of screening
  • Participant is male with severe hemophilia A (Factor VIII (FVIII) clotting activity < 1%) as confirmed by central laboratory at screening after the appropriate washout period or a documented FVIII clotting activity <1%
  • Participant has been previously treated with plasma-derived FVIII concentrates or recombinant FVIII for ≥150 documented exposure days (EDs)
  • Participant is currently receiving prophylaxis or on-demand therapy with FVIII
  • Participant is willing and able to comply with the requirements of the protocol

Main Exclusion Criteria:

  • Participant has detectable FVIII inhibitory antibodies (≥ 0.6 Bethesda Units (BU) using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening
  • Participant has history of FVIII inhibitory antibodies (≥ 0.4 BU using the Nijmegen modification of the Bethesda assay or ≥ 0.6 BU using the Bethesda assay) at any time prior to screening
  • Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease).
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01736475

  Show 72 Study Locations
Sponsors and Collaborators
Baxter Healthcare Corporation
Investigators
Study Director: Brigitt Abbuehl, MD Baxter Innovations GmbH
  More Information

No publications provided

Responsible Party: Baxter Healthcare Corporation
ClinicalTrials.gov Identifier: NCT01736475     History of Changes
Other Study ID Numbers: 261201, 2012-003599-38
Study First Received: November 21, 2012
Last Updated: November 21, 2013
Health Authority: Australia: Department of Health and Ageing Therapeutic Goods Administration
Austria: Agency for Health and Food Safety
Bulgaria: Bulgarian Drug Agency
Czech Republic: State Institute for Drug Control
Germany: Paul-Ehrlich-Institut
Israel: Israeli Health Ministry Pharmaceutical Administration
Japan: Pharmaceuticals and Medical Devices Agency
Korea: Food and Drug Administration
Lithuania: State Medicine Control Agency - Ministry of Health
Malaysia: Ministry of Health
Netherlands: Medicines Evaluation Board (MEB)
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
Spain: Agencia Española de Medicamentos y Productos Sanitarios
Sweden: Medical Products Agency
Switzerland: Federal Office of Public Health
Taiwan: Department of Health
Ukraine: State Pharmacological Center - Ministry of Health
United Kingdom: Medicines and Healthcare Products Regulatory Agency
United States: Food and Drug Administration

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on April 21, 2014