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An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A

Phase 1
Conditions
Hemophilia ASevere hemophilia A
MedDRA version: 16.1Level: LLTClassification code 10060613Term: Hemophilia A (Factor VIII)System Organ Class: 100000004850
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Registration Number
EUCTR2013-003262-13-IT
Lead Sponsor
CSL Behring GmbH
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
ot Recruiting
Sex
Male
Target Recruitment
200
Inclusion Criteria

• Males of any age who have been diagnosed with severe hemophilia A (FVIII activity levels < 1%)
• Participated in a previous CSL-sponsored clinical study with rVIII-SingleChain.

Are the trial subjects under 18? yes
Number of subjects for this age range: 75
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 124
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

• Known or suspected hypersensitivity to rVIII-SingleChain or to any excipients of rVIII-SingleChain or Chinese hamster ovary (CHO) proteins.
• Currently receiving a therapy not permitted during the study.

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Main Objective: The primary objective of this study is to evaluate the safety of long term use of rVIII-SingleChain;Secondary Objective: The secondary objectives of the study are:<br>• To measure the incidence rate of inhibitor formation to FVIII after 10 EDs and after 50 EDs<br>• To collect and evaluate additional efficacy information on the prophylaxis and treatment of bleeding events<br>• To assess the hemostatic efficacy of rVIII-SingleChain for subjects who undergo surgery<br>• To characterize the safety profile of rVIII-SingleChain;Primary end point(s): The primary outcome measure is the incidence rate of inhibitor formation to FVIII over 100 EDs.;Timepoint(s) of evaluation of this end point: At the closest visit after 100 EDs (up to three years). <br>
Secondary Outcome Measures
NameTimeMethod
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