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

Bioverativ

Status and phase: Completed; Phase 3

Conditions: Hemophilia A

Treatments: Drug: rFVIIIFc

Study type: Interventional

Funder types: Industry

Identifiers

NCT01454739

8HA01EXT

2011-003072-37

Details and patient eligibility

About

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.

Full 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), 997HA307 (NCT02083965) and 997HA309 (NCT02502149).

Enrollment: 240 patients

Sex: Male

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Subjects who have completed previous rFVIIIFc studies (NCT01181128, NCT02083965, NCT01458106 and NCT02502149)
• 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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240 participants in 2 patient groups

On-Demand

Experimental group

Description:

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.

Treatment:

Drug: rFVIIIFc

Prophylaxis

Experimental group

Description:

Tailored prophylaxis, Weekly prophylaxis or Personalized prophylaxis available.

Treatment:

Drug: rFVIIIFc