Trial record 3 of 8 for:    rFVIIIFc

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):
Biogen
ClinicalTrials.gov Identifier:
NCT01454739
First received: September 29, 2011
Last updated: June 18, 2015
Last verified: June 2015
  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
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
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 Biogen:

Primary Outcome Measures:
  • Frequency of inhibitor development [ Time Frame: Up to 4 years ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: No ]
  • The annualized number of spontaneous joint bleeding episodes per participant [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]
  • The total number of days of exposure per participant per year [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]
  • The consumption of rFVIIIFc as total dose per kg per participant per year [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]
  • Physician's global assessment of response to treatment using a 4-point scale [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
  • Investigator/Surgeon assessment of hemostatic response to surgery using the 4-point bleeding response scale [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]
    Using the eDiary, each participant or the participant's caregiver will rate the treatment response to any bleeding episode using the following 4-point scale (excellent, good, moderate, or none).

  • Number of injections and dose per injection to maintain hemostasis during the surgical period [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]
  • Estimated blood loss (mL) during surgery and post-operative period [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]
  • Number of blood product units transfused during surgery [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]

Estimated Enrollment: 194
Study Start Date: December 2011
Estimated Study Completion Date: December 2018
Estimated Primary Completion Date: December 2018 (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

Genders 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

Key Exclusion Criteria:

  • Confirmed positive high-titer inhibitor test

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 74 Study Locations
Sponsors and Collaborators
Biogen
Investigators
Study Director: Medical Director Biogen
  More Information

No publications provided

Responsible Party: Biogen
ClinicalTrials.gov Identifier: NCT01454739     History of Changes
Other Study ID Numbers: 8HA01EXT, 2011-003072-37
Study First Received: September 29, 2011
Last Updated: June 18, 2015
Health Authority: Israel: Ministry of Health
Belgium: Federal Agency for Medicinal Products and Health Products
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Switzerland: Swissmedic
Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
Ireland: Irish Medicines Board
Spain: Agencia Española de Medicamentos y Productos Sanitarios
Australia: Department of Health and Ageing Therapeutic Goods Administration
India: Ministry of Health
Hong Kong: Department of Health
Italy: Ministry of Health
South Africa: Department of Health
Austria: Agency for Health and Food Safety
New Zealand: Medsafe
Japan: Pharmaceuticals and Medical Devices Agency (PMDA)
Brazil: Ministry of Health
Sweden: Medical Products Agency
Germany: Federal Institute for Drugs and Medical Devices
Canada: Health Canada
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
United States: Food and Drug Administration

Keywords provided by Biogen:
rFVIIIFc
A-LONG Extension

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

ClinicalTrials.gov processed this record on August 27, 2015