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Long Term Safety and Efficacy Study of Teriflunomide 7 mg or 14 mg in Patients With Relapsing-Remitting Multiple Sclerosis

Phase 3
Completed
Conditions
Multiple Sclerosis
Interventions
Registration Number
NCT00803049
Lead Sponsor
Sanofi
Brief Summary

The primary objective of this study was to document the long-term safety and tolerability of teriflunomide in Multiple Sclerosis (MS) participants with relapse.

The secondary objective was to document the long-term efficacy on disability progression, relapse rate and Magnetic Resonance Imaging (MRI) parameters.

Detailed Description

Participants completing the EFC6049 (HMR1726D/3001) study were given the opportunity to continue in the extension study;

* participants receiving teriflunomide 7 mg or 14 mg were blindly maintained on the same dose of teriflunomide.

* participants receiving placebo were randomized at a 1:1 ratio to teriflunomide 7 mg or 14 mg.

The study period per participant was broken down as follows:

* Double-blind treatment: up to a maximum of 288 weeks or until teriflunomide was commercially available in the country where participant lived,

* Post-washout follow-up: 4 weeks after last treatment intake. No post-washout follow up if participant continued on teriflunomide treatment by obtaining its commercial form after end of the study.

The total duration of the extension was 292 weeks (about 6 years) from the first participant enrolled or until teriflunomide is commercially available in the country where participant lived.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
742
Inclusion Criteria
  • Participant who completed the previous double-blind placebo-controlled study EFC6049 and who did not meet criteria for treatment withdrawal.
  • Willingness to participate in a long-term safety/efficacy trial.
Exclusion Criteria
  • Any known condition or circumstance that would prevent in the investigator's opinion, compliance or completion of the study.

The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Teriflunomide 7 mg/7 mgTeriflunomide (HMR1726)Participants who completed treatment of teriflunomide 7 mg tablet QD for 108 weeks in EFC6049 study, continued their treatment with teriflunomide 7 mg tablet QD for 288 weeks in this extension study.
Placebo/Teriflunomide 14 mgTeriflunomide (HMR1726)Participants who completed treatment of placebo (for teriflunomide) tablet QD for 108 weeks in EFC6049, study received teriflunomide 14 mg tablet QD for 288 weeks in this extension study.
Teriflunomide 14 mg/14 mgTeriflunomide (HMR1726)Participants who completed treatment of teriflunomide 14 mg tablet QD for 108 weeks in EFC6049 study, continued their treatment with teriflunomide 14 mg tablet QD for 288 weeks in this extension study.
Placebo/Teriflunomide 7 mgTeriflunomide (HMR1726)Participants who completed treatment of placebo (for teriflunomide) tablet once daily (QD) for 108 weeks in EFC6049 study, received teriflunomide tablet 7 mg QD for 288 weeks in this extension study.
Primary Outcome Measures
NameTimeMethod
Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs)Baseline (LTS6050) up to 28 days after last dose of study drug up to 450 weeks

Adverse event (AE) was defined as any untoward medical occurrence in a participant who received investigational medicinal product (IMP) without regard to possibility of causal relationship with this treatment. TEAEs: AEs that developed or worsened or became serious during on-treatment period which was defined as the period from the time of first dose of study drug (in LTS6050) up to 4 weeks (28 days) after last dose of study drug. Serious adverse event (SAE) was defined as any untoward medical occurrence that resulted in any of the following outcomes: death, life-threatening, required initial or prolonged in-patient hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect, or considered as medically important event. Any TEAE included both serious and non-serious AEs.

Secondary Outcome Measures
NameTimeMethod
Time to 12 Week Sustained Disability Progression (DP): Kaplan-Meier Estimates of the Rate of DPUp to 10.8 years (EFC6049: 108 weeks + LTS6050: 450 weeks)

Sustained DP defined as sustained increase of at least 1 point from baseline (EFC6049) expanded disability status scale (EDSS) score (0.5 point for participants with baseline EDSS\>5.5) persisting for at least 12 weeks. EDSS: an ordinal scale qualifies disability in participants with MS. EDSS total score range: 0 (normal neurological examination) to 10 (death due to Multiple Sclerosis \[MS\]). Probability of DP at 12 weeks was estimated using Kaplan-Meier method on time to DP defined as date of first DP minus (-) date of randomization in EFC6049 study +1 day. Participants free of DP (no DP observed on treatment) were censored at the date of last on-treatment EDSS evaluation in LTS6050. 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.

Time to 24 Week Sustained Disability Progression (DP): Kaplan-Meier Estimates of the Rate of DPUp to 10.8 years (EFC6049: 108 weeks + LTS6050: 450 weeks)

Sustained DP was defined as sustained increase of at least 1 point from baseline (EFC6049) EDSS score (0.5 point for participants with baseline EDSS\>5.5) persisting for at least 24 weeks. EDSS: an ordinal scale qualifies disability in participants with MS. EDSS total score range: 0 (normal neurological examination) to 10 (death due to MS). Probability of DP at 24 weeks was estimated using Kaplan-Meier method on time to DP defined as date of first DP minus (-) date of randomization in EFC6049 study +1 day. Participants free of DP (no DP observed on treatment) were censored at the date of last on-treatment EDSS evaluation in LTS6050. 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.

Percentage of Participants Free of Sustained Disability Progression (DP)Up to 10.8 years since EFC6049 randomization (EFC6049: 108 weeks + LTS6050: 450 weeks)

Sustained DP was defined as sustained increase of at least 1 point from baseline (EFC6049) EDSS score (0.5 point for participants with baseline EDSS\>5.5) persisting for at least 12 weeks and 24 weeks. EDSS is an ordinal scale in half-point increments that qualifies disability in participants with MS. It consists of 8 ordinal rating scales assessing seven functional systems (visual, brainstem, pyramidal, cerebellar, sensory, bowel/bladder, cerebral) as well as ambulation. EDSS total score ranges from 0 (normal neurological examination) to 10 (death due to MS), where higher scores indicates worse neurological function. Percentage of participants who were considered as free of disability progression confirmed after 12 week sustained progression and 24 week sustained progression were reported. Analysis for this outcome measure was performed on combined data of EFC6049 and LTS6050 study, as pre-specified in protocol.

Annualized MS Relapse Rate (ARR): Poisson Regression EstimatesUp to 8 years since LTS6050 randomization

ARR was obtained from total number of confirmed relapses that occurred during treatment period divided by sum of treatment durations in LTS6050 study only. 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 EDSS score or Functional System (FS) scores. EDSS: an ordinal scale qualifies disability. EDSS total score range: 0 (normal neurological examination) to 10 (death due to MS). FSS: to assess the neurological function. Total score range: 0 (normal) - 6(worse), higher scores = worse neurological function. To account for the different treatment duration 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 enrolment and baseline EDSS stratum as covariates).

Magnetic Resonance Imaging (MRI) Assessment: Change From Baseline in Total Volume of Abnormal Lesions (Burden of Disease [BOD]) at Week 192 Since LTS6050 RandomizationBaseline, Week 192

BOD was assessed by cerebral MRI and defined as the total volume of all abnormal brain tissue (calculated as the sum of the total volume of T2-lesion component and T1-hypointense lesion component).

Trial Locations

Locations (116)

Investigational Site Number 4604

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Sittard-Geleen, Netherlands

Investigational Site Number 3505

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Ivano-Frankovsk, Ukraine

Investigational Site Number 3510

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Kharkiv, Ukraine

Investigational Site Number 3508

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Lviv, Ukraine

Investigational Site Number 1601

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Wien, Austria

Investigational Site Number 3803

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Santiago, Chile

Investigational Site Number 1209

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Toronto, Canada

Investigational Site Number 1604

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Innsbruck, Austria

Investigational Site Number 1211

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St. John'S, Canada

Investigational Site Number 1212

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Gatineau, Canada

Investigational Site Number 1038

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Ft. Wayne, Indiana, United States

Investigational Site Number 3805

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Viña Del Mar, Chile

Investigational Site Number 1205

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Greenfield Park, Canada

Investigational Site Number 2414

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Nantes Cedex 01, France

Investigational Site Number 2003

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Wiesbaden, Germany

Investigational Site Number 1032

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Maitland, Florida, United States

Investigational Site Number 1207

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Winnipeg, Canada

Investigational Site Number 2410

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Paris Cedex 12, France

Investigational Site Number 4605

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Breda, Netherlands

Investigational Site Number 3604

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Tønsberg, Norway

Investigational Site Number 4201

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Coimbra, Portugal

Investigational Site Number 3008

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Bialystok, Poland

Investigational Site Number 3005

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Lodz, Poland

Investigational Site Number 4601

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Nijmegen, Netherlands

Investigational Site Number 5003

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Izmir, Turkey

Investigational Site Number 5006

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Izmir, Turkey

Investigational Site Number 2007

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Hannover, Germany

Investigational Site Number 2008

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Münster, Germany

Investigational Site Number 3203

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Moscow, Russian Federation

Investigational Site Number 3207

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Nizhny Novgorod, Russian Federation

Investigational Site Number 2404

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Limoges Cedex, France

Investigational Site Number 2405

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Nancy Cedex, France

Investigational Site Number 2009

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Rostock, Germany

Investigational Site Number 3401

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Stockholm, Sweden

Investigational Site Number 2201

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Tampere, Finland

Investigational Site Number 1802

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Basel, Switzerland

Investigational Site Number 3009

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Bialystok, Poland

Investigational Site Number 3002

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Warszawa, Poland

Investigational Site Number 2001

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Berlin, Germany

Investigational Site Number 2803

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Firenze, Italy

Investigational Site Number 2012

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Erbach, Germany

Investigational Site Number 2005

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Gießen, Germany

Investigational Site Number 2010

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Offenbach, Germany

Investigational Site Number 2813

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Roma, Italy

Investigational Site Number 2403

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Clermont Ferrand Cedex 1, France

Investigational Site Number 2406

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Rennes Cedex, France

Investigational Site Number 2203

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Helsinki, Finland

Investigational Site Number 2206

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Pori, Finland

Investigational Site Number 3003

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Warszawa, Poland

Investigational Site Number 3504

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Dnipropetrovsk, Ukraine

Investigational Site Number 2819

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Bari, Italy

Investigational Site Number 2415

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Besancon, France

Investigational Site Number 2011

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Berlin, Germany

Investigational Site Number 3205

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Moscow, Russian Federation

Investigational Site Number 3202

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St-Petersburg, Russian Federation

Investigational Site Number 3208

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Novosibirsk, Russian Federation

Investigational Site Number 2402

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Montpellier Cedex 5, France

Investigational Site Number 2000

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Bochum, Germany

Investigational Site Number 2004

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Essen, Germany

Investigational Site Number 2411

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Toulouse, France

Investigational Site Number 3201

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St-Petersburg, Russian Federation

Investigational Site Number 2809

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Pavia, Italy

Investigational Site Number 3006

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Lublin, Poland

Investigational Site Number 5005

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Kocaeli, Turkey

Investigational Site Number 2609

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Plymouth, United Kingdom

Investigational Site Number 3501

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Zaporizhzhya, Ukraine

Investigational Site Number 2600

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Newcastle Upon Tyne, United Kingdom

Investigational Site Number 2608

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London, United Kingdom

Investigational Site Number 3802

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Santiago, Chile

Investigational Site Number 4203

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Lisboa, Portugal

Investigational Site Number 4801

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Glostrup, Denmark

Investigational Site Number 4804

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Sønderborg, Denmark

Investigational Site Number 4802

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Vejle, Denmark

Investigational Site Number 2808

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Milano, Italy

Investigational Site Number 2824

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Roma, Italy

Investigational Site Number 5001

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Sihhiye / Ankara, Turkey

Investigational Site Number 1208

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Calgary, Canada

Investigational Site Number 1201

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Halifax, Canada

Investigational Site Number 1203

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Montreal, Canada

Investigational Site Number 1206

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London, Canada

Investigational Site Number 1202

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Quebec, Canada

Investigational Site Number 1204

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Ottawa, Canada

Investigational Site Number 2604

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Dundee, United Kingdom

Investigational Site Number 2607

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London, United Kingdom

Investigational Site Number 2601

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Nottingham, United Kingdom

Investigational Site Number 2606

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Sheffield, United Kingdom

Investigational Site Number 2602

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Stoke On Trent, United Kingdom

Investigational Site Number 2407

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Nice Cedex, France

Investigational Site Number 3206

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St-Petersburg, Russian Federation

Investigational Site Number 1602

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Wien, Austria

Investigational Site Number 1210

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Vancouver, Canada

Investigational Site Number 3801

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Santiago, Chile

Investigational Site Number 4101

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Olomouc, Czech Republic

Investigational Site Number 1501

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Tartu, Estonia

Investigational Site Number 2408

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Dijon, France

Investigational Site Number 2413

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Lille Cedex, France

Investigational Site Number 2401

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Lyon Cedex 03, France

Investigational Site Number 2409

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Marseille, France

Investigational Site Number 2827

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Fidenza, Italy

Investigational Site Number 2814

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Gallarate, Italy

Investigational Site Number 2812

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Padova, Italy

Investigational Site Number 4602

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'S Hertogenbosch, Netherlands

Investigational Site Number 3601

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Oslo, Norway

Investigational Site Number 3509

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Uzhgorod, Ukraine

Investigational Site Number 2202

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Turku, Finland

Investigational Site Number 1502

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Tallinn, Estonia

Investigational Site Number 1603

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Graz, Austria

Investigational Site Number 3804

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Santiago, Chile

Investigational Site Number 3001

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Poznan, Poland

Investigational Site Number 3506

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Kharkiv, Ukraine

Investigational Site Number 3004

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Lublin, Poland

Investigational Site Number 3007

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Gdansk, Poland

Investigational Site Number 3502

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Odessa, Ukraine

Investigational Site Number 3507

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Vinnitsa, Ukraine

Investigational Site Number 1037

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Allentown, Pennsylvania, United States

Investigational Site Number 1033

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Detroit, Michigan, United States

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