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Long-Term Safety and Efficacy of rFVIIIFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia A (ASPIRE)

This study is enrolling participants by invitation only.
Sponsor:
Information provided by (Responsible Party):
Bioverativ Therapeutics Inc.
ClinicalTrials.gov Identifier:
NCT01454739
First received: September 29, 2011
Last updated: June 5, 2017
Last verified: June 2017
  Purpose
The primary objective of the study is to evaluate the long-term safety of recombinant human Factor VIII Fc fusion protein (rFVIIIFc) in participants with hemophilia A. The secondary objective of the study is to evaluate the efficacy of rFVIIIFc in the prevention and treatment of bleeding episodes in participants with hemophilia A.

Condition Intervention Phase
Hemophilia A Drug: rFVIIIFc Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: No masking
Primary Purpose: Treatment
Official Title: An Open-Label, Multicenter Evaluation of the Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) in the Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects With Hemophilia A

Resource links provided by NLM:


Further study details as provided by Bioverativ Therapeutics Inc.:

Primary Outcome Measures:
  • Frequency of inhibitor development [ Time Frame: Up to 4 years ]
    Participants will be tested for development of inhibitors at time points throughout the study based on exposure days (ED). One ED is equivalent to a 24 hour period in which rFVIIIFc is dosed.


Secondary Outcome Measures:
  • The annualized number of bleeding episodes (spontaneous and traumatic) per participant [ Time Frame: Up to 4 years ]
  • The annualized number of spontaneous joint bleeding episodes per participant [ Time Frame: Up to 4 years ]
  • The total number of days of exposure per participant per year [ Time Frame: Up to 4 years ]
  • The consumption of rFVIIIFc as total dose per kg per participant per year [ Time Frame: Up to 4 years ]
  • Physician's global assessment of response to treatment using a 4-point scale [ Time Frame: Up to 4 years ]
    Investigators will record assessments of each participant's response to his rFVIIIFc regimen grading on the following 4-point scale: excellent, effective, partially effective, ineffective.

  • Participant's assessment of response to treatment using a 4-point scale [ Time Frame: Up to 4 years ]

Estimated Enrollment: 194
Study Start Date: December 2011
Estimated Study Completion Date: August 2017
Estimated Primary Completion Date: July 14, 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: On-Demand
The individual dose of rFVIIIFc to treat bleeding episodes will be based on participant's clinical condition, type and severity of the bleeding event, and if indicated, Factor VIII (FVIII) levels.
Drug: rFVIIIFc
Administered as specified in the treatment arm.
Other Names:
  • Eloctate
  • recombinant coagulation factor VIII Fc fusion protein
  • BIIB031
  • antihemophilic factor (recombinant) Fc fusion protein
  • efmoroctocog alfa
Experimental: Prophylaxis
Tailored prophylaxis, Weekly prophylaxis or Personalized prophylaxis available.
Drug: rFVIIIFc
Administered as specified in the treatment arm.
Other Names:
  • Eloctate
  • recombinant coagulation factor VIII Fc fusion protein
  • BIIB031
  • antihemophilic factor (recombinant) Fc fusion protein
  • efmoroctocog alfa

Detailed Description:
Participant will follow either a prophylaxis or on-demand regimen. The starting dose in this study will be determined by the clinical profile of the participant in the preceding studies A-LONG - 997HA301 (NCT01181128), pediatric study 8HA02PED (NCT01458106) and 997HA307 (NCT02083965).
  Eligibility

Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Subjects who have completed previous rFVIIIFc studies (NCT01181128, NCT02083965 and NCT01458106)
  • Ability to understand purposes and risks of the study and to provide signed and dated informed consent (or assent, as applicable).

Key Exclusion Criteria:

  • Confirmed positive high-titer inhibitor (≥5.00 BU/mL).

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01454739

  Show 77 Study Locations
Sponsors and Collaborators
Bioverativ Therapeutics Inc.
Investigators
Study Director: Medical Director Bioverativ Therapeutics Inc.
  More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Bioverativ Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT01454739     History of Changes
Other Study ID Numbers: 8HA01EXT
2011-003072-37
Study First Received: September 29, 2011
Last Updated: June 5, 2017

Keywords provided by Bioverativ Therapeutics Inc.:
rFVIIIFc
A-LONG Extension

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 June 28, 2017