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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02172950
Registration number
NCT02172950
Ethics application status
Date submitted
23/06/2014
Date registered
24/06/2014
Date last updated
27/10/2021
Titles & IDs
Public title
An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A
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Scientific 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
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Secondary ID [1]
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2013-003262-13
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Secondary ID [2]
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CSL627_3001
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Hemophilia A
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Severe Hemophilia A
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Condition category
Condition code
Blood
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Clotting disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Other interventions - rVIII-SingleChain
Experimental: Previously treated patients (PTPs) - 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.
Experimental: 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 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.
Other interventions: rVIII-SingleChain
Recombinant single-chain coagulation factor VIII
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Intervention code [1]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Incidence of Inhibitor Formation to FVIII in Previously Treated Patients (PTPs) With 100 Exposure Days (EDs) to CSL627
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Assessment method [1]
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Timepoint [1]
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At the closest visit after 100 EDs (up to 5 years).
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Primary outcome [2]
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Number of Previously Untreated Patients (PUPs) With High-titer Inhibitor Formation to FVIII With at Least 50 EDs to CSL627
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Assessment method [2]
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High-titer inhibitor is defined as an inhibitor titer of = 5 Bethesda units/mL.
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Timepoint [2]
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At the closest visit after 50 EDs (up to 5 years).
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Primary outcome [3]
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Percent Treatment Success for Major Bleeding Episodes in PUPs
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Assessment method [3]
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Percentage of major bleeding episodes treated successfully where treatment success for a bleeding episode is defined as a rating of "excellent" or "good" on the investigator's clinical assessment of hemostatic efficacy 4-point scale "excellent, good, moderate or poor/no response". Major bleeding episodes are defined as bleeding episodes for which a subject is required to seek treatment at the hemophilia center or that threatens the subject's life or loss of limb.
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Timepoint [3]
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Up to 5 years
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Primary outcome [4]
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Annualized Spontaneous Bleeding Rate in PUPs
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Assessment method [4]
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The annualized spontaneous bleeding rate for PUPs taking prophylaxis and on-demand treatment regimens.
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Timepoint [4]
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Up to 5 years
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Secondary outcome [1]
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Percentage of Bleeding Episodes Treated Successfully in PTPs
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Assessment method [1]
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Percentage of bleeding episodes treated successfully where treatment success for a bleeding episode is defined as a rating of "excellent" or "good" on the investigator's clinical assessment of hemostatic efficacy 4-point scale "excellent, good, moderate or poor/no response".
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Timepoint [1]
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Up to 5 years
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Secondary outcome [2]
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Annualized Bleeding Rate in PTPs and PUPs
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Assessment method [2]
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The annualized bleeding rate for PTPs and PUPs taking prophylaxis and on-demand treatment regimens
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Timepoint [2]
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Up to 5 years
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Secondary outcome [3]
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Percentage of Bleeding Episodes Requiring 1, 2, 3, or > 3 Injections of CSL627 to Achieve Hemostasis in PTPs and PUPs
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Assessment method [3]
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Timepoint [3]
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Up to 5 years
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Secondary outcome [4]
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Mean Number of On-demand Infusions of CSL627
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Assessment method [4]
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Timepoint [4]
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Up to 5 years
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Secondary outcome [5]
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Mean On-demand Dose Administered of CSL627
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Assessment method [5]
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Timepoint [5]
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Up to 5 years
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Secondary outcome [6]
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Mean Prophylaxis Dose Administered of CSL627
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Assessment method [6]
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Timepoint [6]
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Up to 5 years
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Secondary outcome [7]
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Mean Total Amount of CSL627 Administered During Surgery Period in PTPs
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Assessment method [7]
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Timepoint [7]
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Day of surgery up to 336 hours post-surgery
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Secondary outcome [8]
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Total Amount of CSL627 Administered During Surgery Period in PUPs
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Assessment method [8]
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Timepoint [8]
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Day of surgery up to 336 hours post-surgery
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Secondary outcome [9]
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Hemostatic Efficacy of rVIII-SingleChain for PTPs and PUPs Who Undergo Surgery
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Assessment method [9]
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The investigator will rate the efficacy of the rVIII-SingleChain treatment during surgery based on a hemostatic efficacy four point rating scale of "excellent, good, moderate or poor/no response".
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Timepoint [9]
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From the start of surgery through the post-operative recovery (generally up to 14 days after surgery)
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Secondary outcome [10]
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Incidence of Inhibitor Formation to FVIII in PTPs After 10 EDs and After 50 EDs
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Assessment method [10]
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Timepoint [10]
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Up to 5 years
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Secondary outcome [11]
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Percentage of PTPs and PUPs Developing Antibodies Against CSL627
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Assessment method [11]
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Timepoint [11]
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PTPs: At the closest visit after 100 EDs (up to 5 years). PUPs: At the closest visit after 50 EDs (up to 5 years).
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Secondary outcome [12]
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Percentage of PTPs and PUPs Developing Antibodies to Chinese Hamster Ovary (CHO) Proteins
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Assessment method [12]
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Timepoint [12]
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PTPs: At the closest visit after 100 EDs (up to 5 years). PUPs: At the closest visit after 50 EDs (up to 5 years).
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Secondary outcome [13]
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Number of PUPs With High-titer Inhibitor Formation to FVIII After 10 EDs With CSL627
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Assessment method [13]
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High-titer inhibitor is defined as an inhibitor titer of = 5 Bethesda units/mL.
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Timepoint [13]
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At the closest visit after 10 EDs (up to 5 years)
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Secondary outcome [14]
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Number of PUPs With Low-titer Inhibitor Formation to FVIII After 10 EDs and After 50 EDs With CSL627
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Assessment method [14]
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Low-titer inhibitor is defined as an inhibitor titer of less than 5 Bethesda units/mL.
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Timepoint [14]
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At the closest visit after 10 and after 50 EDs (up to 5 years)
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Secondary outcome [15]
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Incidence of Total Inhibitor Formation to FVIII in PUPs
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Assessment method [15]
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Timepoint [15]
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Up to 5 years
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Secondary outcome [16]
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Percent Treatment Success for Non-major Bleeding Episodes in PUPs
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Assessment method [16]
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Percentage of bleeding episodes treated successfully where treatment success for a bleeding episode is defined as a rating of "excellent" or "good" on the investigator's clinical assessment of hemostatic efficacy 4-point scale "excellent, good, moderate or poor/no response". Non-major bleeding episodes are those not requiring treatment at the hemophilia center or not threatening subject's life or loss of limb.
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Timepoint [16]
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Up to 5 years
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Secondary outcome [17]
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Percentage of PUPs With Clinically Significant Abnormal Vital Signs Values After First Infusion of CSL627
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Assessment method [17]
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Vital signs assessments include heart rate, blood pressure, and body temperature. Clinical significance of an abnormality will be assessed by the investigator.
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Timepoint [17]
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Up to 6 hours after first infusion
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Secondary outcome [18]
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Percentage of PUPs With Treatment-emergent Clinically Significant Abnormal Vital Signs Values
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Assessment method [18]
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Vital signs assessments include heart rate, blood pressure, and body temperature. Clinical significance of an abnormality will be assessed by the investigator.
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Timepoint [18]
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Up to 5 years
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Eligibility
Key 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.
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Minimum age
No limit
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Maximum age
No limit
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Sex
Males
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Can healthy volunteers participate?
No
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Key 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
13/10/2014
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
19/01/2021
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Sample size
Target
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Accrual to date
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Final
246
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Study Site 0360014 - Melbourne
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Recruitment hospital [2]
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Study Site 0360028 - Nedlands
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Recruitment hospital [3]
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Study Site 0360031 - Perth
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Recruitment postcode(s) [1]
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3052 - Melbourne
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Recruitment postcode(s) [2]
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WA 6009 - Nedlands
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Recruitment postcode(s) [3]
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WA 6000 - Perth
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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United States of America
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Colorado
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United States of America
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Connecticut
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United States of America
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Florida
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United States of America
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Illinois
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United States of America
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State/province [6]
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Louisiana
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Country [7]
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United States of America
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State/province [7]
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Texas
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Country [8]
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United States of America
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State/province [8]
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Wisconsin
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Country [9]
0
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Austria
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State/province [9]
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Graz
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Country [10]
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Austria
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State/province [10]
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Linz
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Austria
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Wien
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Canada
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Saint John
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Czechia
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Hradec Kralove
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France
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State/province [14]
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Brest
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Country [15]
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France
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State/province [15]
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Le Kremlin Bicetre
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France
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State/province [16]
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Lille Cedex
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Country [17]
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France
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Nantes
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France
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Paris
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Georgia
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Tbilisi
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Germany
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Bonn
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Germany
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Frankfurt
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Germany
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Giessen
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Germany
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Hannover
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Hungary
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Debrecen
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Ireland
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Dublin
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Italy
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Milano
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Japan
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Hyogo
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Japan
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Nagoya
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Japan
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Okayama
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Japan
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Saitama
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Japan
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Tokyo
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Lebanon
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Beirut
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Malaysia
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Kuala Lumpur
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Netherlands
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Amsterdam Zuidoost
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Netherlands
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Nijmegen
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Netherlands
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Utrecht
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Philippines
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Cebu City
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Philippines
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Davao City
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Poland
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Gdansk
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Poland
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Krakow
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Poland
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Rzeszow
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Poland
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Wroclaw
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Portugal
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Porto
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Romania
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Bucharest
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Romania
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Timisoara
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South Africa
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Parktown
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Spain
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A Coruna
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Spain
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Barcelona
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Spain
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Madrid
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Spain
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Valencia
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Switzerland
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Luzern
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Thailand
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Bangkok
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Thailand
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Chiang Mai
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Thailand
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Khon Kaen
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Thailand
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Songkhla
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Ukraine
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Dnipropetrovsk
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Ukraine
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Lviv
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United Kingdom
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State/province [58]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
CSL Behring
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02172950
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Program Director
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Address
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CSL Behring
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02172950
Download to PDF