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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/NCT02316353
Registration number
NCT02316353
Ethics application status
Date submitted
10/12/2014
Date registered
12/12/2014
Date last updated
14/11/2018
Titles & IDs
Public title
A Study to Evaluate the Long-term Clinical Safety and Efficacy of Subcutaneously Administered C1-esterase Inhibitor in the Prevention of Hereditary Angioedema
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Scientific title
An Open-label, Randomized Study to Evaluate the Long-term Clinical Safety and Efficacy of Subcutaneous Administration of Human Plasma-derived C1-esterase Inhibitor in the Prophylactic Treatment of Hereditary Angioedema
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Secondary ID [1]
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2014-001054-42
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Secondary ID [2]
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CSL830_3002
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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:
Hereditary Angioedema Types I and II
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Condition category
Condition code
Cardiovascular
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Diseases of the vasculature and circulation including the lymphatic system
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Blood
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Other blood disorders
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Cardiovascular
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Other cardiovascular diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Other interventions - C1-esterase inhibitor
Experimental: C1-INH - low-volume dose - A low-volume dose of C1-INH will be administered subcutaneously twice a week for up to 52 weeks (up to 146 weeks extension period).
Experimental: C1-INH - medium-volume dose - A medium-volume dose of C1-INH will be administered subcutaneously twice a week for up to 52 weeks (up to 146 weeks extension period).
Other interventions: C1-esterase inhibitor
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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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Person-time Incidence Rates (Subject Based)
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Assessment method [1]
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Subject-based Analysis for Person-Time Incidence Rate = (the total number of subjects who experienced the event during the respective treatment) / (the sum of the date each subject experienced the event - the subject's start date + 1 day) / (365.25 days). The analysis population for this endpoint was the Safety Population: the Safety Population comprised all subjects who provided informed consent / assent, who were randomized, and who received at least 1 dose or a partial dose of CSL830.
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Timepoint [1]
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Up to 146 weeks.
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Primary outcome [2]
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The Person-time Incidence Rates (Event Based)
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Assessment method [2]
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Event-based Analysis for Person-Time Incidence Rate = (the total number of events documented during the respective treatment) / (the sum of each subject's end date - the subject's start date + 1 day) / (365.25 days). The analysis population for this endpoint was the Safety Population: The Safety Population comprised all subjects who provided informed consent / assent, who were randomized, and who received at least 1 dose or a partial dose of CSL830.
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Timepoint [2]
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Up to 146 weeks.
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Secondary outcome [1]
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Percentage of Subjects Who Have Solicited Adverse Events (AEs)
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Assessment method [1]
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The number of subjects having at least 1 solicited local AE during a treatment were divided by the number of subjects in the corresponding treatment. The analysis population for this endpoint was the Safety Population: The Safety Population comprised all subjects who provided informed consent / assent, who were randomized, and who received at least 1 dose or a partial dose of CSL830.
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Timepoint [1]
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Up to 146 weeks
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Secondary outcome [2]
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Percentage of Injections Followed by At Least One Solicited Adverse Event
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Assessment method [2]
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The percent of injections followed by at least one solicited adverse event. The analysis population for this endpoint was the Safety Population: The Safety Population comprised all subjects who provided informed consent / assent, who were randomized, and who received at least 1 dose or a partial dose of CSL830. This was assessed across all participants, calculated as total number of events following injections / total number of injections across all participants, in each Arm.
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Timepoint [2]
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Up to 146 weeks
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Secondary outcome [3]
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Percentage of Subjects Who Become Seropositive for Human Immunodeficiency Virus (HIV-1/-2), Hepatitis B Virus, or Hepatitis C Virus.
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Assessment method [3]
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Blood samples to be tested for HIV-1/-2, HBV, and HCV. The analysis population for this endpoint was the Safety Population: The Safety Population comprised all subjects who provided informed consent / assent, who were randomized, and who received at least 1 dose or a partial dose of CSL830.
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Timepoint [3]
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Up to 146 weeks
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Secondary outcome [4]
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Percentage of Subjects Who Experience a Time-normalized HAE Attack Frequency of <1 HAE Attack Per 4-Week Period
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Assessment method [4]
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The percentage of subjects with a time-normalized merged HAE attack frequency of <1 HAE attack per 4-week period. The analysis population for this endpoint was the Intent-to-Treat (ITT) Population: The ITT Population comprised all subjects who provided informed consent / assent and were randomized, regardless of whether or not they received CSL 830.
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Timepoint [4]
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Up to 146 weeks
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Secondary outcome [5]
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Percentage of Subjects Who Are Responders
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Assessment method [5]
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A responder was defined as a subject with a = 50% reduction in the time-normalized number of HAE attacks on CSL830 relative to the time-normalized number of HAE attacks used to qualify for participation in the current study. The analysis population for this endpoint was the Intent-to-Treat (ITT) Population: The ITT Population comprised all subjects who provided informed consent / assent and were randomized, regardless of whether or not they received CSL830. Not all subjects in the ITT were available for this outcome measure.
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Timepoint [5]
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Up to 146 weeks
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Eligibility
Key inclusion criteria
- Males or females aged 6 years or older.
- A confirmed diagnosis of HAE type I or II.
- HAE attacks over a consecutive 2-month period that required acute treatment, medical
attention, or caused significant functional impairment.
- For subjects who have used oral therapy for prophylaxis against HAE attacks within 3
months of first study visit: use of a stable regimen within 3 months of the first
study visit.
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Minimum age
6
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Incurable malignancies.
- Any clinical condition that will interfere with the evaluation of C1-INH therapy.
- Clinically significant history of poor response to C1-esterase therapy for the
management of HAE.
- Suspected or confirmed diagnosis of acquired HAE or HAE with normal C1-INH.
- Inability to have HAE managed pharmacologically with on-demand treatment.
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Study design
Purpose of the study
Prevention
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Allocation to intervention
Randomised controlled 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
31/12/2014
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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
21/09/2017
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Sample size
Target
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Accrual to date
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Final
126
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Recruitment in Australia
Recruitment state(s)
NSW
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Recruitment hospital [1]
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Study Site - Campbelltown
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Recruitment postcode(s) [1]
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2560 - Campbelltown
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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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Alabama
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United States of America
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Arizona
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United States of America
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California
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United States of America
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Maryland
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United States of America
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Ohio
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United States of America
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Oklahoma
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United States of America
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Oregon
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United States of America
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Pennsylvania
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Texas
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United States of America
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Virginia
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Canada
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Ontario
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Canada
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State/province [12]
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Quebec
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Czechia
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State/province [13]
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Plzen
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Germany
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State/province [14]
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Hesse
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Germany
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Berlin
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Germany
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Frankfurt
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Germany
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Mainz
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Hungary
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Budapest
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Israel
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Tel Aviv
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Israel
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Tel Hashomer
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Italy
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Catania
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Italy
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Milano
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Romania
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Cluj Napoca
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Spain
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Madrid
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Spain
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Valencia
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United Kingdom
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State/province [26]
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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
The aim of this study is to assess the long-term safety of C1-esterase inhibitor (C1-INH) in
preventing hereditary angioedema (HAE) attacks when it is administered under the skin of
subjects with HAE. The safety of participating subjects will be assessed for up to 54 weeks.
The long-term efficacy of C1-INH will also be assessed. Each eligible subject will enter the
treatment phase, wherein subjects will be randomized to treatment with either low- or
medium-volume C1-INH. Subjects who have an insufficient treatment response during the study
will be given an opportunity to undergo a dose increase. The study aims to enroll eligible
subjects who completed study CSL830_3001 (NCT01912456). Subjects who did not participate in
study CSL830_3001 may also participate, if eligible and if space permits. Subjects from the
United States (US) who complete Treatment Period 2 will be allowed to participate in an
Extension Period. During the Extension Period participating US subjects will continue to
receive treatment with open-label CSL830 for up to an additional 88 weeks.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02316353
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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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Global Clinical Program Director
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Address
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CSL Behring
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Fax
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Email
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Contact person for public queries
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Address
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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/NCT02316353
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