An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A
Hemophilia A, Severe Hemophilia A
About this trial
This is an interventional treatment trial for Hemophilia A
Eligibility Criteria
Inclusion Criteria:
PTPs:
- Males of any age who have been diagnosed with severe congenital hemophilia A (FVIII activity levels < 1%) and who participated in a previous CSL-sponsored clinical study with rVIII-SingleChain.
- Males 0 to <65 years age who have been diagnosed with severe congenital hemophilia A (FVIII activity levels < 1%), who have at least 50 EDs to any FVIII product, and who are not currently enrolled in a CSL-sponsored clinical study with rVIII-SingleChain.
PUPs:
- Males 0 to <18 years of who have been diagnosed with severe congenital hemophilia A (FVIII activity levels < 1%)
- No prior exposure to any Factor VIII product (with the exception of short-term use of blood products).
ITI substudy:
- PUPs who have developed a confirmed inhibitor to rVIII-SingleChain in the main study.
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.
- Serum creatinine > 2 x upper limit of normal, alanine aminotransferase or aspartate aminotransferase > 5 x upper limit of normal at Screening (if specified)
- Any first-order family (eg, siblings) history of FVIII inhibitors
- For PTPs not rolling over directly from a CSL-sponsored clinical study with rVIII-SingleChain: any history of or current FVIII inhibitors
Sites / Locations
- Study Site 8400213
- Study Site 8400241
- Study Site 8400118
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- Study Site 8400240
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- Study Site 0360014
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- Study Site 2680001
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- Study Site 8040007
- Study Site 8040005
- Study Site 8260008
Arms of the Study
Arm 1
Arm 2
Experimental
Experimental
Previously treated patients (PTPs)
Previously untreated patients (PUPs)
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.