Brief Title
An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A
Official Title
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
Brief Summary
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 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.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Incidence of inhibitor formation to FVIII in PTPs with 100 EDs to rVIII-SingleChain
Secondary Outcome
Treatment success in PTPs
Condition
Hemophilia A
Intervention
rVIII‑SingleChain
Study Arms / Comparison Groups
Previously treated patients (PTPs)
Description: 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.
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
245
Start Date
October 13, 2014
Completion Date
July 1, 2025
Primary Completion Date
July 1, 2025
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
Gender
Male
Ages
N/A - N/A
Accepts Healthy Volunteers
No
Contacts
Program Director, ,
Location Countries
Australia
Location Countries
Australia
Administrative Informations
NCT ID
NCT02172950
Organization ID
CSL627_3001
Secondary IDs
2013-003262-13
Responsible Party
Sponsor
Study Sponsor
CSL Behring
Study Sponsor
Program Director, Study Director, CSL Behring
Verification Date
June 2020