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Study of Recombinant Coagulation Factor VIII Fc Fusion Protein, BIIB031, in Previously Treated Pediatric Subjects With Hemophilia A (Kids ALONG)

This study has been completed.
Sponsor:
Collaborator:
Swedish Orphan Biovitrum
Information provided by (Responsible Party):
Biogen Idec
ClinicalTrials.gov Identifier:
NCT01458106
First received: October 20, 2011
Last updated: November 4, 2014
Last verified: November 2014
  Purpose

The primary objective of the study is to evaluate the safety of Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) in previously treated pediatric subjects with hemophilia A. Secondary objectives of this study in this study population are as follows: To evaluate the efficacy of Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) for prevention and treatment of bleeding episodes; To evaluate and assess the PK of Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc); and To evaluate Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) consumption for prevention and treatment of bleeding episodes.


Condition Intervention Phase
Hemophilia A
Biological: BIIB031 (rFVIIIFc)
Biological: FVIII (PK subgroup only)
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-Label, Multicenter Evaluation of Safety, Pharmacokinetics, and Efficacy of Recombinant Coagulation Factor VIII Fc Fusion Protein, BIIB031, in the Prevention and Treatment of Bleeding Episodes in Pediatric Subjects With Hemophilia A

Resource links provided by NLM:


Further study details as provided by Biogen Idec:

Primary Outcome Measures:
  • Frequency of inhibitor development [ Time Frame: Up to Week 26 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Annualized Number of Bleeding Episodes [ Time Frame: Up to Week 26 +/- 7 days ] [ Designated as safety issue: No ]
  • Annualized Number of Spontaneous Joint Bleeding Episodes [ Time Frame: Up to Week 26 +/- 7 days ] [ Designated as safety issue: No ]
  • Response to Treatment With Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) for Bleeding Episodes [ Time Frame: Up to Week 26 +/- 7 days ] [ Designated as safety issue: No ]
  • Total Annualized Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) Consumption per Participant [ Time Frame: Up to Week 26 +/- 7 days ] [ Designated as safety issue: No ]
  • Time From Last Injection of Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) to Bleeding Episode [ Time Frame: Up to Week 26 +/- 7 days ] [ Designated as safety issue: No ]
  • Number of Injections of Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) Required to Resolve a Bleeding Episode [ Time Frame: Up to Week 26 +/- 7 days ] [ Designated as safety issue: No ]
  • Dose per Injection of Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) Required to Resolve a Bleeding Episode [ Time Frame: Up to Week 26 +/- 7 days ] [ Designated as safety issue: No ]
  • Pharmacokinetics (PK) Parameter: Maximum Concentration [ Time Frame: Up to 72 (+/-7) hours after start of injection ] [ Designated as safety issue: No ]
  • Pharmacokinetics (PK) Parameter: Half-life (t1/2) [ Time Frame: Up to 72 (+/-7) hours after start of injection ] [ Designated as safety issue: No ]
  • Pharmacokinetics (PK) Parameter: clearance (CL) [ Time Frame: Up to 72 (+/-7) hours after start of injection ] [ Designated as safety issue: No ]
  • Pharmacokinetics (PK) Parameter: volume of distribution at steady state (Vss) [ Time Frame: Up to 72 (+/-7) hours after start of injection ] [ Designated as safety issue: No ]
  • Pharmacokinetics (PK) Parameter: dose normalized area under the curve (DNAUC) [ Time Frame: Up to 72 (+/-7) hours after start of injection ] [ Designated as safety issue: No ]
  • Pharmacokinetics (PK) Parameter: Mean Residence Time (MRT) [ Time Frame: Up to 72 (+/-7) hours after start of injection ] [ Designated as safety issue: No ]
  • Pharmacokinetics (PK) Parameter: Incremental Recovery [ Time Frame: Up to 72 (+/-7) hours after start of injection ] [ Designated as safety issue: No ]

Enrollment: 50
Study Start Date: November 2012
Study Completion Date: December 2013
Primary Completion Date: December 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: All participants
Participants can choose to enter the PK subgroup or can directly enter the prophylactic treatment (non-PK subgroup)
Biological: BIIB031 (rFVIIIFc)
IV administration in all participants
Other Names:
  • BIIB031
  • Recombinant Human Coagulation Factor VIII Fusion Protein
  • ELOCTATE
Biological: FVIII (PK subgroup only)
Baseline prestudy FVIII dosing in participants who enter the PK subgroup

Detailed Description:

Previously treated pediatric participants will be treated with a prophylactic regimen of recombinant coagulation factor VIII Fc protein (rFVIIIFc). Pharmacokinetic (PK) analysis of pre-study factor VIII (FVIII) and Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) will be performed in a sub-group of the study participants prior to commencement of prophylactic treatment for all subjects.

  Eligibility

Ages Eligible for Study:   up to 11 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Severe hemophilia A defined as <1 IU/dL (<1%).
  • <12 years of age and weight ≥13 kg.
  • History of at least 50 documented prior exposure days to FVIII
  • No current, or history of, inhibitor development to FVIII

Key Exclusion Criteria:

  • Other coagulation disorders in addition to Hemophilia A
  • History of anaphylaxis associated with any FVIII or IV immunoglobulin administration

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: NCT01458106

  Show 28 Study Locations
Sponsors and Collaborators
Biogen Idec
Swedish Orphan Biovitrum
Investigators
Study Director: Medical Director Biogen Idec
  More Information

No publications provided

Responsible Party: Biogen Idec
ClinicalTrials.gov Identifier: NCT01458106     History of Changes
Other Study ID Numbers: 8HA02PED, 2011-003073-28
Study First Received: October 20, 2011
Last Updated: November 4, 2014
Health Authority: Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
Australia: Department of Health and Ageing Therapeutic Goods Administration
Hong Kong: Department of Health
South Africa: Medicines Control Council
Ireland: Medical Ethics Research Committee
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
United States: Food and Drug Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency

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

ClinicalTrials.gov processed this record on November 19, 2014