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Study to Evaluate the Safety, Pharmacokinetics and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Subjects With Severe Hemophilia A

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Biogen Idec
ClinicalTrials.gov Identifier:
NCT01181128
First received: August 12, 2010
Last updated: December 6, 2012
Last verified: December 2012
History of Changes
  Purpose

The study is to investigate safety, pharmacokinetics (the determination of the concentration of drug in blood over time) and efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in previously treated subjects with severe hemophilia A


Condition Intervention Phase
Severe Hemophilia A
 Drug: rFVIIIFc
 Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: ALONG: An Open-Label, Multicenter Evaluation of the Safety, Pharmacokinetics, and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in the Prevention and Treatment of Bleeding in Previously Treated Subjects With Severe Hemophilia A

Resource links provided by NLM:

MedlinePlus related topics: Hemophilia
U.S. FDA Resources

Further study details as provided by Biogen Idec:

Primary Outcome Measures:
  • Clinically notable changes from baseline in physical examinations, vital signs, lab values, and incidence of AEs and inhibitor development [ Time Frame: 156 weeks ] [ Designated as safety issue: Yes ]
  • Annual number of bleeding episodes (spontaneous and traumatic) [ Time Frame: 156 weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Total annualized rFVIIIFc consumption per subject [ Time Frame: 156 weeks ] [ Designated as safety issue: Yes ]
  • Evaluation of PK parameter estimates of rFVIIIFc and rFVIII [ Time Frame: 156 weeks ] [ Designated as safety issue: Yes ]
  • Evaluation of subject's response to treatment [ Time Frame: 156 weeks ] [ Designated as safety issue: Yes ]
  • To evaluate the efficacy of rFVIIIFc used in a surgical subgroup [ Time Frame: 156 weeks ] [ Designated as safety issue: Yes ]

Enrollment: 150
Study Start Date: November 2010
Study Completion Date: October 2012
Primary Completion Date: August 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: A
Individualized Prophylaxis
 Drug: rFVIIIFc
IV administration
Experimental: B
Weekly Prophylaxis
 Drug: rFVIIIFc
IV administration
Experimental: C
Episodic (on demand) treatment
 Drug: rFVIIIFc
IV administration

Detailed Description:

The current hemophilia standard of care for the prevention of bleeds and arthropathy is to maintain FVIII activity level above 1%. Due to the short half-life of the current FVIII products, prophylaxis therapy will require injection of 2-3 times per week or every other day. Treatment usually involves intravenous access, an invasive procedure, especially difficult in children. Episodic treatment will involve 1-3 injections to treat bleeding episodes, depending on the severity of the hemorrhage. Severe hemophilia patients will treat prophylactically with the long-lasting recombinant factor VIII Fc fusion protein (rFVIIIFc) in an interval to maintain FVIII activity level above 1%. Any bleeding episodes will be reported. The response to treatment will also be recorded to access the effectiveness of the rFVIIIFc.

  Eligibility

Ages Eligible for Study:   12 Years and older
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male, ≥12 years of age and weigh at least 40 kg
  • Diagnosed with severe hemophilia A defined as <1 IU/dL (<1%) endogenous Factor VIII)
  • History of at least 150 documented prior exposure days to any Factor VIII product
  • Platelet count ≥100,000 cells/μL

Exclusion Criteria:

  • History of Factor VIII inhibitors
  • Kidney and liver dysfunction
  • Diagnosed with other coagulation disorder(s) other than hemophilia A
  • Prior history of hypersensitivity or anaphylaxis associated with any FVIII or IV immunoglobulin administration
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01181128

  Show 71 Study Locations
Sponsors and Collaborators
Biogen Idec
  More Information

No publications provided

Responsible Party: Biogen Idec
ClinicalTrials.gov Identifier: NCT01181128     History of Changes
Other Study ID Numbers: 997HA301
Study First Received: August 12, 2010
Last Updated: December 6, 2012
Health Authority: 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 May 19, 2013