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

Tracking Information
First Received Date ^ICMJE	August 12, 2010
Last Updated Date	December 6, 2012
Start Date ^ICMJE	November 2010
Primary Completion Date	August 2012 (final data collection date for primary outcome measure)
Current Primary Outcome Measures ^ICMJE (submitted: August 13, 2010)	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 ]
Original Primary Outcome Measures ^ICMJE (submitted: August 12, 2010)	• 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 ] • Annualized number of bleeding episodes (spontaneous and traumatic) [ Time Frame: 156 weeks ] [ Designated as safety issue: Yes ]
Change History	Complete list of historical versions of study NCT01181128 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ^ICMJE (submitted: November 18, 2010)	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 ]
Original Secondary Outcome Measures ^ICMJE (submitted: August 12, 2010)	• Total annualized rFVIIIFc consumption per subject [ Time Frame: 156 weeks ] [ Designated as safety issue: Yes ] • Evaluation of PK parameter estimates of rFVIIIFc and rFVIII at baseline and at Week 12-28 [ 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 ]
Current Other Outcome Measures ^ICMJE	Not Provided
Original Other Outcome Measures ^ICMJE	Not Provided

Descriptive Information
Brief Title ^ICMJE	Study to Evaluate the Safety, Pharmacokinetics and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Subjects With Severe Hemophilia A
Official Title ^ICMJE	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
Brief Summary	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
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.
Study Type ^ICMJE	Interventional
Study Phase	Phase 2 Phase 3
Study Design ^ICMJE	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Crossover Assignment Masking: Open Label Primary Purpose: Treatment
Condition ^ICMJE	Severe Hemophilia A
Intervention ^ICMJE	Drug: rFVIIIFc IV administration
Study Arm (s)	Experimental: A Individualized Prophylaxis Intervention: Drug: rFVIIIFc Experimental: B Weekly Prophylaxis Intervention: Drug: rFVIIIFc Experimental: C Episodic (on demand) treatment Intervention: Drug: rFVIIIFc
Publications *	Not Provided
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information
Recruitment Status ^ICMJE	Completed
Enrollment ^ICMJE	150
Completion Date	October 2012
Primary Completion Date	August 2012 (final data collection date for primary outcome measure)
Eligibility Criteria ^ICMJE	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
Gender	Male
Ages	12 Years and older
Accepts Healthy Volunteers	No
Contacts ^ICMJE	Contact information is only displayed when the study is recruiting subjects
Location Countries ^ICMJE	United States, Australia, Austria, Belgium, Brazil, Canada, France, Germany, Hong Kong, India, Israel, Italy, Japan, New Zealand, Poland, South Africa, Spain, Sweden, Switzerland, United Kingdom

Administrative Information
NCT Number ^ICMJE	NCT01181128
Other Study ID Numbers ^ICMJE	997HA301
Has Data Monitoring Committee	Yes
Responsible Party	Biogen Idec
Study Sponsor ^ICMJE	Biogen Idec
Collaborators ^ICMJE	Not Provided
Investigators ^ICMJE	Not Provided
Information Provided By	Biogen Idec
Verification Date	December 2012
^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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