A Phase 3 Open Label, Multicenter, Extension Study to Assess the Safety and Efficacy of Recombinant Coagulation Factor VIII (rVIII-SingleChain, CSL627) in Subjects with Severe Hemophilia A
- Conditions
- Hemophilia A10005330
- Registration Number
- NL-OMON47544
- Lead Sponsor
- CSL Behring GmbH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 14
Eligible subjects will be males of any age who have been diagnosed with severe
hemophilia A (FVIII activity levels < 1%) and have participated in a previous
CSL-sponsored clinical study with rVIIISingleChain., Arm 1 (PTPs): subjects of
any age who have participated in a previous CSL-sponsored clinical study with
rVIII-SingleChain. , Arm 2 (PUPs): subjects 0 to < 18 years of age who have not
been exposed to any prior FVIII product (except for short-term use of blood
products)., Arm 3 (PTPs): subjects 0 to < 65 years of age who have had at least
50 EDs to a prior FVIII product and who are not currently enrolled in a
rVIII-SingleChain study, ITI substudy:
• PUPs who have developed a confirmed inhibitor to rVIII-SingleChain in the
main study.
Subjects meeting any of the following exclusion criteria must not be enrolled
into the study:
• Currently receiving a therapy not permitted during the study, as defined in
Section 7.2 of the study protocol
• Previous participation in the current study
• Mental condition rendering the subject (or the subject's legally acceptable
representative[s]) unable to understand the nature, scope and possible
consequences of the study
• Known or suspected hypersensitivity to rVIII-SingleChain or to any
excipients of rVIII SingleChain or Chinese hamster ovary (CHO) proteins
• Any issue that, in the opinion of the investigator, would render the subject
unsuitable for participation in the study., • Known or suspected
hypersensitivity to rVIII SingleChain, to any excipients of rVIII SingleChain,
or to CHO proteins
• Currently receiving a therapy not permitted during the study
• Serum creatinine > 2 times the upper limit (x ULN), alnine aminotransferase
(ALT) or aspartate aminotransferase (AST) > 5 x ULN at Screening (if specified
for a particular arm)
• Any first-order family (eg, siblings) history of FVIII inhibitors
• Arm 3: Any history of or current FVIII inhibitors
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The primary outcome measure is the incidence rate of inhibitor formation to<br /><br>FVIII over 100 EDs of rVIII-SingleChain in PTPs (Arm 1 and 3) and the incidence<br /><br>of high-titer inhibitor formation over 50 EDs of rVIII-SingleChain in PUPs (Arm<br /><br>2).<br /><br><br /><br>Treatment success for major bleeding episodes in PUPs.<br /><br>Annualized spontaneous bleeding rate in PUPs.</p><br>
- Secondary Outcome Measures
Name Time Method <p>• The rate of treatment success for bleeding episodes defined as a rating of<br /><br>excellent or good on the investigator's clinical assessment of hemostatic<br /><br>efficacy 4-point scale.<br /><br>• The annualized bleeding rate (traumatic and non-traumatic) during prophylaxis<br /><br>and on-demand treatment.<br /><br>• The proportion of bleeding episodes requiring 1, 2, 3, or > 3 infusions of<br /><br>rVIII-SingleChain to achieve hemostasis.<br /><br>• The mean actual dose per kg per subject per year; the consumption of factor<br /><br>VIII, expressed as number of infusions and IU/kg per month and per year, as<br /><br>well as IU/kg per event (prophylaxis and ondemand).<br /><br>• Investigator*s assessment of hemostatic efficacy of rVIII-SingleChain for<br /><br>subjects who undergo surgery</p><br>