MedPath

Efficacy and Safety of Teriflunomide in Patients With Relapsing Multiple Sclerosis and Treated With Interferon-beta

Phase 3
Terminated
Conditions
Multiple Sclerosis Relapse
Interventions
Registration Number
NCT01252355
Lead Sponsor
Sanofi
Brief Summary

The primary objective was to demonstrate the effect of teriflunomide, in comparison to placebo, on frequency of Multiple Sclerosis (MS) relapses in patients with relapsing forms of MS who are treated with Interferon-beta (IFN-beta).

The secondary objectives were:

* Assess the effect of teriflunomide, in comparison to placebo, when added to IFN-beta on:

* Disease activity as measured by brain Magnetic Resonance Imaging (MRI)

* Disability progression

* Burden of disease and disease progression as measured by brain MRI

* Evaluate the safety and tolerability of teriflunomide when added to IFN-beta therapy

* Assess the pharmacokinetics of teriflunomide in use in addition to baseline IFN-beta therapy

* Assess associations between variations in genes and clinical outcomes (safety and efficacy)

* Assess other measures of efficacy of teriflunomide such as fatigue and health-related quality of life

* Assess measures of health economics (hospitalization due to relapse, including the length of stay and any admission to intensive care unit)

Detailed Description

The study period per patient was expected to be between 56 and 160 weeks depending on when the patient was randomized and this included the following:

* a screening period up to 4 weeks,

* a treatment period expected to be between 48 and 152 weeks,

* 4-week post rapid elimination follow-up period.

Patients were to continue on treatment until a fixed common end date which was approximately 48 weeks after randomization of the last patient.

For those patients who completed the treatment period, a long term extension study of approximately 1 year (including teriflunomide alone) was initially planned to be proposed.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
534
Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Teriflunomide 7 mg + IFN-betaTeriflunomideTeriflunomide 7 milligram (mg) once a day concomitantly with IFN-beta therapy.
Teriflunomide 14 mg + IFN-betaTeriflunomideTeriflunomide 14 mg once a day concomitantly with IFN-beta therapy.
Placebo + IFN-betaPlacebo (for teriflunomide)Placebo (for teriflunomide) once a day concomitantly with IFN-beta therapy.
Teriflunomide 7 mg + IFN-betaInterferon-beta (IFN-beta)Teriflunomide 7 milligram (mg) once a day concomitantly with IFN-beta therapy.
Placebo + IFN-betaInterferon-beta (IFN-beta)Placebo (for teriflunomide) once a day concomitantly with IFN-beta therapy.
Teriflunomide 14 mg + IFN-betaInterferon-beta (IFN-beta)Teriflunomide 14 mg once a day concomitantly with IFN-beta therapy.
Primary Outcome Measures
NameTimeMethod
Annualized Relapse Rate (ARR) (Poisson Regression Estimates)Up to a maximum of 108 weeks depending on time of enrollment

ARR is the total number of confirmed relapses that occurred during the treatment period divided by the total number of patient-years treated. Each episode of relapse (appearance, or worsening of a clinical symptom that was stable for at least 30 days, that persisted for a minimum of 24 hours in the absence of fever) was to be confirmed by an increase in Expanded Disability Status Scale (EDSS) score or Functional System scores. To account for the different treatment durations among participants, a Poisson regression model with robust error variance was used (total number of confirmed relapses as response variable; log-transformed treatment duration as "offset" variable; treatment group, region of enrollment and IFN-beta dose stratum, and number of relapses in the year prior to randomization as covariates).

Secondary Outcome Measures
NameTimeMethod
Brain Magnetic Resonance Imaging (MRI) Assessment: Number of Gadolinium Enhancing (Gd-enhancing) T1-lesions Per Scan (Poisson Regression Estimates)Up to a maximum of 108 weeks depending on time of enrollment

Number of Gd-enhancing T1-lesions per scan is the total number of Gd-enhancing T1-lesions that occurred during the treatment period divided by the total number of scans performed during the treatment period. To account for the different number of scans among participants, a Poisson regression model with robust error variance was used (total number of Gd-enhancing T1-lesions as response variable; log-transformed number of scans as offset variable; treatment group, region of enrollment, IFN-beta dose stratum and baseline number of Gd-enhancing T1-lesions as covariates).

Time to 12-Week Sustained Disability ProgressionUp to a maximum of 108 weeks depending on time of enrollment

The 12-week sustained disability progression was defined as an increase from baseline of at least 1-point in EDSS score (at least 0.5-point for participants with baseline EDSS score \>5.5) that persisted for at least 12 weeks. Probability of disability progression was to be estimated using Kaplan-Meier method.

Brain MRI Assessment: Volume of Gd-enhancing T1-lesions Per MRI ScanUp to a maximum of 108 weeks depending on time of enrollment

Total volume of Gd-enhancing T1-lesions per scan is the sum of the volumes of Gd-enhancing T1-lesions observed during the treatment period divided by the total number of scans performed during the treatment period.

Brain MRI Assessment: Change From Baseline in Total Lesion Volume (Burden of Disease) at Week 24Baseline, Week 24

The total lesion volume (burden of disease) is the total volumes of hyperintense on T2 plus hypointense on T1 as measured by MRI scan. Least-square means were estimated using a Mixed-effect model with repeated measures (MMRM) on cubic root transformed volume data with factors for treatment, region, IFN-beta dose stratum, visit, treatment-by-visit interaction, cubic root transformed baseline burden of disease, and baseline-by-visit interaction.

Time to Relapse: Kaplan-Meier Estimates of the Probability of no Relapse at Week 24, 48, and 72Up to a maximum of 108 weeks depending on time of enrollment

Probability of no relapse at 24, 48 and 72 weeks was estimated using Kaplan-Meier method on the time to relapse defined as the time from randomization to first EDSS confirmed relapse. Participants free of confirmed relapse (no EDSS confirmed relapse observed on treatment) were censored at the date of the last study drug intake. Kaplan-Meier method consists in computing probabilities of non-occurrence of event at any observed time of event and multiplying successive probabilities for time \<=t by any earlier computed probabilities to estimate the probability of being event-free for the amount of time t.

Change From Baseline in Fatigue Impact Scale (FIS) Total Score at Week 24Baseline, Week 24

FIS is a participant-reported scale that qualifies the impact of fatigue on daily life in participants with MS.

Change From Baseline in Short Form Generic Health Survey - 36 Items, Version 2 (SF-36v2) Summary Scores at Week 24Baseline, Week 24

SF-36 scale is a generic, self-administered, health-related quality-of-life (QOL) instrument.

Resource Utilization When RelapseUp to a maximum of 108 weeks depending on time of enrollment

Resource utilization each time a participant experiences an MS relapse, specifically the number of hospitalizations, the number of over night spent in the hospital and number of intensive care admissions if hospitalized were to be reported.

Overview of Adverse Events (AEs)First study drug intake up to 28 days after last study drug intake, for up to 112 weeks

AEs are any unfavorable and unintended sign, symptom, syndrome, or illness observed by the investigator or reported by the participant during the study.

Trial Locations

Locations (185)

Investigational Site Number 170001

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Barranquilla, Colombia

Investigational Site Number 170007

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Bogota, Colombia

Investigational Site Number 233002

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Tallinn, Estonia

Investigational Site Number 170009

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Medellin, Colombia

Investigational Site Number 233001

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Tartu, Estonia

Investigational Site Number 246006

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Hyvinkää, Finland

Investigational Site Number 246002

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Pori, Finland

Investigational Site Number 840021

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St. Petersburg, Florida, United States

Investigational Site Number 840047

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Tampa, Florida, United States

Investigational Site Number 840037

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Elk Grove Village, Illinois, United States

Investigational Site Number 840005

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Cordova, Alaska, United States

Investigational Site Number 840011

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Oceanside, California, United States

Investigational Site Number 840004

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Tampa, Florida, United States

Investigational Site Number 840007

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Fargo, North Dakota, United States

Investigational Site Number 840009

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Missoula, Montana, United States

Investigational Site Number 840040

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Round Rock, Texas, United States

Investigational Site Number 840002

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Nashville, Tennessee, United States

Investigational Site Number 840030

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St Louis, Missouri, United States

Investigational Site Number 840015

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New York, New York, United States

Investigational Site Number 036005

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Chatswood, Australia

Investigational Site Number 840049

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Cullman, Alabama, United States

Investigational Site Number 840013

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Ormond Beach, Florida, United States

Investigational Site Number 840020

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San Antonio, Texas, United States

Investigational Site Number 840032

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Vienna, Virginia, United States

Investigational Site Number 840003

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Phoenix, Arizona, United States

Investigational Site Number 124003

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Gatineau, Canada

Investigational Site Number 840043

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Tulsa, Oklahoma, United States

Investigational Site Number 246001

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Turku, Finland

Investigational Site Number 276015

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Berlin, Germany

Investigational Site Number 348006

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Budapest, Hungary

Investigational Site Number 276010

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Hamburg, Germany

Investigational Site Number 032002

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Argentina, Argentina

Investigational Site Number 250004

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Montpellier Cedex 5, France

Investigational Site Number 246003

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Helsinki, Finland

Investigational Site Number 276012

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Bonn, Germany

Investigational Site Number 276020

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Bamberg, Germany

Investigational Site Number 250001

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Nancy Cedex, France

Investigational Site Number 276003

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Bayreuth, Germany

Investigational Site Number 276005

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Dresden, Germany

Investigational Site Number 276032

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Düsseldorf, Germany

Investigational Site Number 250010

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Clermont Ferrand Cedex 1, France

Investigational Site Number 056005

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Charleroi, Belgium

Investigational Site Number 208002

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Aarhus C, Denmark

Investigational Site Number 250002

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Lyon Cedex 03, France

Investigational Site Number 276018

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Erbach, Germany

Investigational Site Number 276024

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Kassel, Germany

Investigational Site Number 276013

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Mainz, Germany

Investigational Site Number 276026

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Wuppertal, Germany

Investigational Site Number 170005

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Bogota, Colombia

Investigational Site Number 300001

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Athens, Greece

Investigational Site Number 380009

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Catania, Italy

Investigational Site Number 380011

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Napoli, Italy

Investigational Site Number 250003

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Besancon, France

Investigational Site Number 276004

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Erlangen, Germany

Investigational Site Number 250006

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Nantes Cedex 01, France

Investigational Site Number 276031

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Rostock, Germany

Investigational Site Number 440004

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Klaipeda, Lithuania

Investigational Site Number 620004

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Coimbra, Portugal

Investigational Site Number 620003

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Setubal, Portugal

Investigational Site Number 300006

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Thessaloniki, Greece

Investigational Site Number 276023

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Minden, Germany

Investigational Site Number 276008

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Wiesbaden, Germany

Investigational Site Number 348005

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Szekesfehervar, Hungary

Investigational Site Number 410002

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Goyang-Si, Korea, Republic of

Investigational Site Number 440003

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Siauliai, Lithuania

Investigational Site Number 578002

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Tønsberg, Norway

Investigational Site Number 703002

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Martin, Slovakia

Investigational Site Number 380002

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Cefalù, Italy

Investigational Site Number 380006

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Padova, Italy

Investigational Site Number 348002

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Budapest, Hungary

Investigational Site Number 643011

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St-Petersburg, Russian Federation

Investigational Site Number 724005

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Madrid, Spain

Investigational Site Number 643003

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St-Petersburg, Russian Federation

Investigational Site Number 380005

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Roma, Italy

Investigational Site Number 620001

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Amadora, Portugal

Investigational Site Number 643001

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Kemerovo, Russian Federation

Investigational Site Number 380003

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Fidenza, Italy

Investigational Site Number 380004

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Gallarate, Italy

Investigational Site Number 643015

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Novosibirsk, Russian Federation

Investigational Site Number 348007

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Zalaegerszeg, Hungary

Investigational Site Number 643006

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Nizhny Novgorod, Russian Federation

Investigational Site Number 380008

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Roma, Italy

Investigational Site Number 643009

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Rostov-On-Don, Russian Federation

Investigational Site Number 643017

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St-Petersburg, Russian Federation

Investigational Site Number 724003

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Girona, Spain

Investigational Site Number 440002

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Kaunas, Lithuania

Investigational Site Number 620002

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Coimbra, Portugal

Investigational Site Number 788002

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Manouba, Tunisia

Investigational Site Number 752003

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Stockholm, Sweden

Investigational Site Number 380010

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Napoli, Italy

Investigational Site Number 380014

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Verona, Italy

Investigational Site Number 703001

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Trnava, Slovakia

Investigational Site Number 643014

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Yaroslavl, Russian Federation

Investigational Site Number 643004

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Nizhny Novgorod, Russian Federation

Investigational Site Number 724009

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Córdoba, Spain

Investigational Site Number 826004

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Plymouth, United Kingdom

Investigational Site Number 643005

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Smolensk, Russian Federation

Investigational Site Number 788004

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Sfax, Tunisia

Investigational Site Number 752001

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Stockholm, Sweden

Investigational Site Number 724002

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Barcelona, Spain

Investigational Site Number 826001

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Salford, United Kingdom

Investigational Site Number 752004

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Göteborg, Sweden

Investigational Site Number 826008

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Birmingham, United Kingdom

Investigational Site Number 826005

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Leeds, United Kingdom

Investigational Site Number 724004

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Madrid, Spain

Investigational Site Number 788006

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Tunis, Tunisia

Investigational Site Number 840034

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Chicago, Illinois, United States

Investigational Site Number 840028

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Baltimore, Maryland, United States

Investigational Site Number 840041

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Baltimore, Maryland, United States

Investigational Site Number 300003

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Heraklion, Greece

Investigational Site Number 300002

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Athens, Greece

Investigational Site Number 528005

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Nieuwegein, Netherlands

Investigational Site Number 528002

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Sittard-Geleen, Netherlands

Investigational Site Number 276022

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Hennigsdorf, Germany

Investigational Site Number 276001

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Leipzig, Germany

Investigational Site Number 840023

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Albuquerque, New Mexico, United States

Investigational Site Number 276016

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Berlin, Germany

Investigational Site Number 276021

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Berlin, Germany

Investigational Site Number 840016

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Clinton Township, Michigan, United States

Investigational Site Number 840031

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St Louis, Missouri, United States

Investigational Site Number 840027

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Charlotte, North Carolina, United States

Investigational Site Number 032004

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Caba, Argentina

Investigational Site Number 032003

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Buenos Aires, Argentina

Investigational Site Number 036010

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New Lambton, Australia

Investigational Site Number 036004

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Kogarah, Australia

Investigational Site Number 036008

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Bedford Park, Australia

Investigational Site Number 040001

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Graz, Austria

Investigational Site Number 036001

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Heidelberg West, Australia

Investigational Site Number 056002

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Leuven, Belgium

Investigational Site Number 040004

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Linz, Austria

Investigational Site Number 056003

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Hasselt, Belgium

Investigational Site Number 056004

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Gent, Belgium

Investigational Site Number 056006

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La Louvière, Belgium

Investigational Site Number 056001

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Sijsele-Damme, Belgium

Investigational Site Number 056007

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Wilrijk, Belgium

Investigational Site Number 076009

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Joinville, Brazil

Investigational Site Number 076012

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Passo Fundo, Brazil

Investigational Site Number 076003

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Porto Alegre, Brazil

Investigational Site Number 076007

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Sao Paulo, Brazil

Investigational Site Number 076013

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Sao Paulo, Brazil

Investigational Site Number 124005

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Calgary, Canada

Investigational Site Number 124004

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Edmonton, Canada

Investigational Site Number 124006

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Kingston, Canada

Investigational Site Number 124007

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Montreal, Canada

Investigational Site Number 124008

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Ottawa, Canada

Investigational Site Number 124002

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Regina, Canada

Investigational Site Number 124009

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Winnipeg, Canada

Investigational Site Number 124001

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Sherbrooke, Canada

Investigational Site Number 152004

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Santiago, Chile

Investigational Site Number 152005

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Viña Del Mar, Chile

Investigational Site Number 152003

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Santiago, Chile

Investigational Site Number 246004

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Oulu, Finland

Investigational Site Number 276009

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Bad Mergentheim, Germany

Investigational Site Number 276002

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Münster, Germany

Investigational Site Number 276028

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Freiburg, Germany

Investigational Site Number 276006

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Gießen, Germany

Investigational Site Number 348009

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Eger, Hungary

Investigational Site Number 348010

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Budapest, Hungary

Investigational Site Number 348003

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Esztergom, Hungary

Investigational Site Number 348001

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Szeged, Hungary

Investigational Site Number 380012

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Montichiari, Italy

Investigational Site Number 410004

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Seoul, Korea, Republic of

Investigational Site Number 410001

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Seoul, Korea, Republic of

Investigational Site Number 528001

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Breda, Netherlands

Investigational Site Number 528006

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Venray, Netherlands

Investigational Site Number 643012

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Kaluga, Russian Federation

Investigational Site Number 643007

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Kazan, Russian Federation

Investigational Site Number 643013

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Moscow, Russian Federation

Investigational Site Number 643010

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Rostov-On-Don, Russian Federation

Investigational Site Number 643016

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Samara, Russian Federation

Investigational Site Number 643018

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St-Petersburg, Russian Federation

Investigational Site Number 643002

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St-Petersburg, Russian Federation

Investigational Site Number 724001

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Barcelona, Spain

Investigational Site Number 724007

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Murcia, Spain

Investigational Site Number 788005

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Monastir, Tunisia

Investigational Site Number 724008

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Sevilla, Spain

Investigational Site Number 826006

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Liverpool, United Kingdom

Investigational Site Number 826003

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London, United Kingdom

Investigational Site Number 840036

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Fort Collins, Colorado, United States

Investigational Site Number 840012

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Maitland, Florida, United States

Investigational Site Number 840055

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Pompano Beach, Florida, United States

Investigational Site Number 840033

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Louisville, Kentucky, United States

Investigational Site Number 840006

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Bismark, North Dakota, United States

Investigational Site Number 840046

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Dayton, Ohio, United States

Investigational Site Number 840017

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Toledo, Ohio, United States

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