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



Status:Recruiting
Conditions:Anemia, Hematology
Therapuetic Areas:Hematology
Healthy:No
Age Range:Any
Updated:4/3/2019
Start Date:October 13, 2014
End Date:August 30, 2023
Contact:Trial Registration Coordinator
Email:clinicaltrials@cslbehring.com

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A Phase III 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

This multicenter, open-label, phase 3 extension study will investigate the safety and
efficacy of rVIII‑SingleChain for prophylaxis and on‑demand treatment of bleeding episodes in
at least 200 previously treated patients (PTPs) with severe congenital hemophilia A and
previous exposure to FVIII products who achieve at least 100 exposure days (EDs) to
rVIII-SingleChain in this study, as well as in at least 50 previously untreated patients
(PUPs) with no previous exposure to any FVIII product who achieve at least 50 EDs to
rVIII-SingleChain in this study. A substudy (open to both PTPs and PUPs) will investigate the
use of rVIII-SingleChain in surgery. A substudy (open to PUPs who develop an inhibitor to
rVIII-SingleChain) will investigate the use of rVIII-SingleChain in immune tolerance
induction (ITI) therapy.


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
We found this trial at
12
sites
Milwaukee, Wisconsin 53233
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Aurora, Colorado 80045
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Aurora, CO
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Chicago, Illinois 60612
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Chicago, IL
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Dallas, Texas 75216
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Dallas, TX
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Hartford, Connecticut 06106
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Hartford, CT
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Houston, Texas 77004
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Houston, TX
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Las Vegas, NV
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Melbourne,
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Melbourne,
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Miami, Florida 33136
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Miami, FL
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New Orleans, Louisiana 70112
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New Orleans, LA
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Sacramento, California 95819
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Sacramento, CA
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San Diego, California 92103
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San Diego, CA
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