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Phase 3b Continuation Study of the Safety and Efficacy of Prophylactic BAX 855 in PTPs With Severe Hemophilia A

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Baxalta US Inc.
ClinicalTrials.gov Identifier:
NCT01945593
First received: September 16, 2013
Last updated: March 6, 2017
Last verified: March 2017
  Purpose
To continue the evaluation of the safety and efficacy of prophylaxis with BAX 855 for the prevention and treatment of bleeding episodes in previously treated patients (PTPs) (children and adults from 0 to 75 years of age) with severe hemophilia A.

Condition Intervention Phase
Hemophilia A
Biological: PEGylated Recombinant Factor VIII
Phase 3

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Prevention
Official Title: A Phase 3b Continuation Study of the Safety and Efficacy of PEGylated Recombinant Factor VIII (PEG-rFVIII; BAX 855) in Prophylaxis of Bleeding in Previously Treated Patients With Severe Hemophilia A

Resource links provided by NLM:


Further study details as provided by Baxalta US Inc.:

Primary Outcome Measures:
  • Development of inhibitory antibodies to Factor VIII (FVIII) [ Time Frame: 36 months ]
  • Annualized bleed rate (ABR) - Spontaneous bleeds [ Time Frame: 36 months ]
    Determined by the development of spontaneous bleeds (bleeds not associated with trauma)


Secondary Outcome Measures:
  • Total annualized bleed rate (ABR) - Spontaneous and traumatic bleeding episodes [ Time Frame: 36 months ]
  • Rate of success of BAX 855 for treatment of breakthrough bleeding episodes [ Time Frame: 36 months ]
    A breakthrough bleeding episode (BE) is a BE during prophylaxis

  • Number of BAX 855 infusions needed to treat bleeding episodes [ Time Frame: 36 months ]
  • Time intervals between bleeding episodes [ Time Frame: 36 months ]
  • Weight-adjusted consumption of BAX 855 [ Time Frame: 36 months ]
  • Occurrence of Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: 36 months ]
  • Changes in vital signs and clinical laboratory parameters [ Time Frame: 36 months ]
    Clinical laboratory parameters include hematology, clinical chemistry, and lipids

  • Immunogenicity: Binding antibodies [ Time Frame: 36 months ]
    • Binding antibodies (IgG and IgM) to Factor VIII, BAX855, and polyethylene glycol (PEG)
    • A 72-hour washout period is required

  • Immunogenicity: Anti-Chinese hamster ovary (CHO) antibodies [ Time Frame: 36 months ]
    A 72-hour washout period is required

  • Change from baseline in patient reported outcomes: Bleed and pain severity [ Time Frame: 36 months ]
    Bleed and pain severity measured using the Haemo-SYM questionnaire

  • Change from baseline in patient reported outcomes: health-related quality of life (HRQoL) [ Time Frame: 36 months ]
    HRQoL assessed using the Short Form-36 Questionnaire (SF-36)


Estimated Enrollment: 250
Study Start Date: October 2013
Estimated Study Completion Date: October 2017
Estimated Primary Completion Date: October 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: BAX855
30-80 ±5 IU/kg twice weekly to once per week. After each consecutive 6 months of being treated in this study, the dose and/or frequency may be adjusted, depending on the participant's spontaneous annualized bleed rate (ABR) estimated at those intervals.
Biological: PEGylated Recombinant Factor VIII
Other Name: BAX 855

  Eligibility

Ages Eligible for Study:   up to 75 Years   (Child, Adult, Senior)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Main Inclusion Criteria:

  • Participants from other BAX 855 studies can be provided with the continuation study informed consent form (ICF) prior to the end of study visit to review and consider participation in this continuation study. These participants will complete any additional screening assessments within 2 weeks of the previous study's end of study visit and will return to the study site within 6 weeks of the previous study end of study visit to confirm eligibility for this continuation study.
  • BAX 855 naïve participants who are ≥ 12 years of age can only be enrolled in this continuation study after enrollment in the phase 2/3 pivotal study is closed. BAX 855 naïve participants who are < 12 years of age and in countries where the pediatric PTP study is being conducted can only be enrolled in this continuation study after enrollment in the pediatric PTP study is closed.
  • Participants coming from other BAX 855 studies into the continuation study, will be considered enrolled only after they have completed all procedures and assessments at the End of Study Visit in the previous BAX 855 study
  • Participant and/or legal representative has/have voluntarily provided signed informed consent
  • Participant is from 0 to 75 years of age at screening
  • Participant is male with severe hemophilia A (Factor VIII (FVIII) clotting activity < 1%) as confirmed by central laboratory at screening (after at least a 72-hour washout period) or a documented FVIII clotting activity <1% (confirmation is only required for BAX 855 naïve participants)
  • 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 has a Karnofsky or Lansky performance score of ≥ 60.
  • Participant is human immunodeficiency virus negative (HIV-); or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm^3, as confirmed by central laboratory at screening.
  • Participant is hepatitis C virus negative (HCV-) by antibody or PCR testing (if positive, antibody titer will be confirmed by PCR), as confirmed by central laboratory at screening; or HCV+ with chronic stable hepatitis.
  • Participant is willing and able to comply with the requirements of the protocol

Main Exclusion Criteria:

  • Participant has detectable FVIII inhibitory antibodies (≥ 0.4 Bethesda unit (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).
  • Participant has known hypersensitivity towards mouse or hamster proteins, polyethylene glycol (PEG), or Tween 80.
  • Participant has severe chronic hepatic dysfunction.
  • Participant has severe renal impairment.
  • Participant has current or recent (<30 days) use of other PEGylated drugs prior to study participation or scheduled use of such drugs during study participation.
  • Participant has participated in another clinical study involving an investigational product (IP) other than BAX 855 or device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
  • Participant has medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance.
  • Participant is a family member or employee of the investigator.
  Contacts and Locations
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Please refer to this study by its ClinicalTrials.gov identifier: NCT01945593

  Show 89 Study Locations
Sponsors and Collaborators
Baxalta US Inc.
Investigators
Study Director: William Savage, MD, PhD Baxalta, now part of Shire
  More Information

Responsible Party: Baxalta US Inc.
ClinicalTrials.gov Identifier: NCT01945593     History of Changes
Other Study ID Numbers: 261302
2013-002236-24 ( EudraCT Number )
Study First Received: September 16, 2013
Last Updated: March 6, 2017

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

ClinicalTrials.gov processed this record on May 23, 2017