Brief Title
An Open-label Safety, Efficacy and Pharmacokinetic Study of a Recombinant FVIII Compared to Recombinant Human Antihemophilic FVIII in Patients With Severe Hemophilia A
Official Title
A Phase I/III Open-label, Multicenter, Crossover Safety, Efficacy and Pharmacokinetic Study of Recombinant Coagulation Factor VIII (rFVIII) Compared to Recombinant Human Antihaemophilic Factor VIII (rFVIII; INN: Octocog Alfa) in Subjects With Hemophilia A, and a Repeat PK, Safety and Efficacy Study
Brief Summary
This is an open-label, non-randomized, efficacy, safety and pharmacokinetic (PK) study
comparing octocog alfa and rVIII-SingleChain. The study consists of three parts, a PK period
(Part 1), a continuation of dosing safety and efficacy period (Part 2) and a safety,
efficacy, and repeat PK period (Part 3) and also includes a surgical sub-study for subjects
enrolled in Parts 2 and 3.
Study Phase
Phase 2/Phase 3
Study Type
Interventional
Primary Outcome
Treatment Success
Secondary Outcome
AUC0-∞ (Part 1)
Condition
Hemophilia A
Intervention
rVIII-SingleChain
Study Arms / Comparison Groups
Recombinant Factor VIII (rFVIII)
Description:
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
175
Start Date
February 2012
Completion Date
December 2014
Primary Completion Date
December 2014
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of severe hemophilia A defined as <1% FVIII:C documented in medical records.
- Males between 18 and 65 years of age (Parts 1 and 2).
- Males between 12 and 65 years of age (Part 3).
- Subjects who have received or are currently receiving FVIII products (plasma-derived
and/or recombinant FVIII) and have had >150 exposure days (EDs) with a FVIII product
- Written informed consent for study participation obtained before undergoing any study
specific procedures.
Exclusion Criteria:
- Any history of or current FVIII inhibitors
- Any first order family history of FVIII inhibitors
- Use of an Investigational Medicinal Product within 30 days prior to the first
rVIII-SingleChain administration.
- Administration of any cryoprecipitate, whole blood or plasma within 30 days prior to
administration of rVIII-SingleChain or reference product.
- Known hypersensitivity (allergic reaction or anaphylaxis) to any FVIII product or
hamster protein.
- Any known congenital or acquired coagulation disorder other than congenital FVIII
deficiency.
- Platelet count < 100,000/µL at screening.
- Human immunodeficiency virus (HIV) positive subjects with a CD4 count < 200/mm3, in
their medical history or at screening if available results are older than one year.
(HIV positive subjects may participate in the study and antiviral therapy are
permitted, at the discretion of the Investigator).
- Subject currently receiving IV immunomodulating agents such as immunoglobulin or
chronic systemic corticosteroid treatment.
- Subject with serum aspartate aminotransferase (AST) or serum alanine aminotransferase
(ALT) values > 5 times (x) the upper limit of normal (ULN) at Screening.
- Subjects with serum creatinine values > 2 x ULN at Screening.
- Evidence of thrombosis, including deep vein thrombosis, stroke, pulmonary embolism,
myocardial infarction and arterial embolus within 3 months prior to Day 1.
- Experienced life-threatening bleeding episode or had major surgery or an orthopedic
surgical procedure during the 3 months prior to Day 1.
Gender
Male
Ages
12 Years - 65 Years
Accepts Healthy Volunteers
No
Contacts
Program Director, ,
Location Countries
Australia
Location Countries
Australia
Administrative Informations
NCT ID
NCT01486927
Organization ID
CSL627_1001
Secondary IDs
2011-002393-23
Responsible Party
Sponsor
Study Sponsor
CSL Behring
Study Sponsor
Program Director, Study Director, CSL Behring
Verification Date
June 2016