MedPath

A Study to Evaluate the Efficacy, Safety, and Tolerability of Mirabegron in Men With OAB Symptoms While Taking Tamsulosin Hydrochloride for Lower Urinary Tract Symptoms (LUTS) Due to Benign Prostatic Hyperplasia (BPH)

Phase 4
Completed
Conditions
Benign Prostatic Hyperplasia
Overactive Bladder
Interventions
Registration Number
NCT02757768
Lead Sponsor
Astellas Pharma Global Development, Inc.
Brief Summary

The purpose of the study was to assess the efficacy, safety, and tolerability of mirabegron versus placebo in men with overactive bladder (OAB) symptoms while taking tamsulosin hydrochloride for lower urinary tract symptoms (LUTS) due to Benign Prostatic Hyperplasia (BPH).

Detailed Description

At Screening (Visit 1), participants entered into a 4-week open label tamsulosin hydrochloride 0.4 mg QD run-in period prior to being randomized into the 12-week double-blind treatment period (Visit 2). At conclusion of the 4-week tamsulosin hydrochloride run-in period, participants completed a 3-day diary just prior to Baseline (Visit 2). Approximately 7 days prior to Visit 2 participants received a phone call reminding them about the diary and to answer any questions.

If participants met all entry criteria at the end of the tamsulosin hydrochloride run-in period, participants were randomized to 1 of 2 treatment groups (mirabegron or placebo) for 12 weeks of treatment in addition to the continuation of tamsulosin hydrochloride 0.4 mg QD. Those participants randomized to Mirabegron started at 25 mg and increased to 50 mg after 4 weeks. Those participants randomized to placebo started blinded product matched to the mirabegron 25 mg tablet and increased to blinded product matched to 50 mirabegron after 4 weeks. Once a participant increased dose, the participant remained on that dose for the remainder of the study unless for safety reasons was required to discontinue study drug.

A training diary was completed in the first 2 weeks of the tamsulosin hydrochloride run-in period. During this evaluation period at least one telephone contact took place with the participant. Diaries were completed at home, using the electronic patient-reported outcome (ePRO) device, for 3 consecutive days prior to each visit: Baseline (Visit 2), Week 4 (Visit 3), Week 8 (Visit 4), and Week 12/End of Treatment (Visit 5). Site staff contacted the participant approximately 7 days prior to the scheduled visit to remind the participant to complete the electronic diary, review completion instruction and review changes to concomitant medications and adverse events (if applicable).

Three days before Visits 2 (Baseline), 3 (Week 4), 4 (Week 8), and 5 (Week 12), participants completed a 3-day diary, using the ePRO device in which the participant recorded micturition frequency, urgency (PPIUS), incontinence and volume voided. In addition, the diary captured morning and evening blood pressure and pulse rate measurements via Home Blood Pressure Monitoring (HBPM). At Visit 1, International Prostate Symptom Score (IPSS) was completed. At Visits 2, 3, 4, and 5, participants completed the IPSS, EQ-5D-5L, OAB-q, PPBC, and TS-VAS. Maximum urinary flow (Qmax) was measured at Visit 1 (Screening/tamsulosin hydrochloride run-in) and Visit 5 (Week 12/End of Treatment). Post-void residual volume (PVR) was assessed at Screening/tamsulosin hydrochloride run-in (Visit 1), Baseline (Visit 2) and at Week 4 (Visit 3), Week 8 (Visit 4), and Week 12/End of Treatment (Visit 5). A follow-up phone call (Visit 6) was conducted 4-weeks after End of Treatment (Visit 5). Total study participation was approximately 20 weeks.

Recruitment & Eligibility

Status
COMPLETED
Sex
Male
Target Recruitment
715
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboPlaceboParticipants received matching placebo in addition to oral tamsulosin hydrochloride daily throughout the study.
PlaceboTamsulosin HydrochlorideParticipants received matching placebo in addition to oral tamsulosin hydrochloride daily throughout the study.
MirabegronMirabegronParticipants received initial dose of 25 mg of mirabegron which was increased to 50 mg after 4 weeks. In addition to mirabegron participants received 0.4 mg of oral tamsulosin hydrochloride daily throughout the study.
MirabegronTamsulosin HydrochlorideParticipants received initial dose of 25 mg of mirabegron which was increased to 50 mg after 4 weeks. In addition to mirabegron participants received 0.4 mg of oral tamsulosin hydrochloride daily throughout the study.
Primary Outcome Measures
NameTimeMethod
Change From Baseline to End of Treatment (EoT) in Mean Number of Micturitions Per DayBaseline and Week 12

Participants recorded micturitions in the e-diary during three days. The mean number of micturitions was calculated as the average number of times a participant recorded a micturition per day during the 3-day period. Only voluntary micturitions were counted and the episodes of incontinence were not included.

Secondary Outcome Measures
NameTimeMethod
Change From Baseline to Week 4, Week 8, Week 12 and EoT in Mean Volume Voided Per MicturitionBaseline and Weeks 4, 8, and 12

The mean volume voided per micturition during 3 days of the 3-day micturition diary period.

Change From Baseline to Week 4, Week 8, Week 12 and EoT in Mean Number of Urgency Incontinence Episodes Per DayBaseline and Weeks 4, 8 and 12

Urgency Incontinence was defined as the complaint of involuntary leakage accompanied by or immediately proceeded by urgency. The mean number of urgency incontinence episodes was calculated as the average number of times a participant recorded an urgency incontinence episode per day during the 3-day micturition diary period.

Change From Baseline to Week 4, Week 8, Week 12 and EoT in HRQL Subscale Coping ScoreBaseline and Weeks 4, 8, and 12

The OAB-q is a self-reported questionnaire with 33 questions relating to symptom bother and health-related quality of life (HRQL). The HRQL portion consists of 25 HRQL items comprising 4 HRQoL subscales (Coping, Concern, Sleep, and Social Interaction), each item was scored 1-6. A higher score on OAB-q HRQL indicated a better response. The lowest and highest possible scores for the Coping subscale ranges from 8 to 48. The Coping score was calculated by adding the 8 response scores and transforming to a scale from 0 to 100, with higher scores indicating better quality of life. A positive change from baseline indicated an improvement.

Change From Baseline to Week 4, Week 8, Week 12 and EoT in HRQL Subscale Social Interaction ScoreBaseline and Weeks 4, 8, and 12

The OAB-q is a self-reported questionnaire with 33 questions relating to symptom bother and health-related quality of life (HRQL). The HRQL portion consists of 25 HRQL items comprising 4 HRQoL subscales (Coping, Concern, Sleep, and Social Interaction), each item was scored 1-6. A higher score on OAB-q HRQL indicated a better response. The lowest and highest possible scores for the Social Interaction subscale ranges from 5 to 30. The Social Interaction score was calculated by adding the 5 response scores and transforming to a scale from 0 to 100, with higher scores indicating better quality of life. A positive change from baseline indicated an improvement.

Change From Baseline to Week 4, Week 8, Week 12 and EoT in Mean Number of Nocturia Episodes Per 24 HoursBaseline and Weeks 4, 8, and 12

A nocturia episode was defined as waking at night one or more time to void (i.e., any voiding associated with sleep disturbance between the date/time the participant goes to bed with the intention to sleep until the date/time the participant gets up in the morning with the intention to stay awake). A night time episode of incontinence is not considered a nocturia episode. The mean number of nocturia episodes per day (24 hours) was calculated as the average number of times a participant recorded a nocturia episode per day during the 3-day micturition diary period.

Change From Baseline to Week 4, Week 8, Week 12 and EoT in Treatment Satisfaction Visual Analog Scale (TS-VAS)Baseline and Weeks 4, 8, and 12

The TS-VAS is a visual analog scale that asks participants to rate their satisfaction with the treatment by placing a vertical mark on a line that runs from 0 (No, not at all) to 100 (Yes, completely).

Change From Baseline to Week 4, Week 8, and Week 12 in Mean Number of Micturitions Per DayBaseline and Weeks 4, 8, and 12

Participants recorded micturitions in the e-diary during three days. The mean number of micturitions was calculated as the average number of times a participant recorded a micturition per day during the 3-day period. Only voluntary micturitions were counted and the episodes of incontinence were not included.

Change From Baseline to Week 4, Week 8, Week 12 and EoT in Mean Number of Incontinence Episodes Per DayBaseline and Weeks 4, 8, and 12

An incontinence episode was defined as the complaint of any involuntary leakage of urine. The mean number of incontinence episodes per 24 hours was calculated as the average number of times a participant recorded an incontinence episode per day during the 3-day micturition diary period.

Change From Baseline to Week 4, Week 8, Week 12 and EoT in International Prostate Symptom Score (IPSS) Total ScoreBaseline and Weeks 4, 8, and 12

The International Prostate Symptom Score (IPSS) consists of 7 questions concerning urinary symptoms and 1 question concerning quality of life (QoL) with total score and subscores (voiding, storage and QoL). The IPSS total score classification ranges from mild (0 to 7) to moderate (8 to 19) or severe (20 to 35). Higher IPSS scored indicated more severe symptoms.

Change From Baseline to Week 4, Week 8, Week 12 and EoT in Symptom Bother ScoreBaseline and Weeks 4, 8, and 12

Overactive bladder symptoms were assessed using the Symptom Bother Scale of the Overactive Bladder questionnaire (OAB-q). The OAB-q is a self-reported questionnaire with 33 questions relating to symptom bother and health-related quality of life (HRQoL). The symptom bother portion consists of 8 questions, rated on a 6-point Likert scale (1 through 6). The total symptom bother score was calculated from the 8 answers and then transformed to range from 0 (least severity) to 100 (worst severity). Lower scores on OAB-q symptom bother indicate a better response.

Change From Baseline to Week 4, Week 8, Week 12 and EoT in Mean Number of Urgency Episodes (Grade 3 or 4) Per DayBaseline and Weeks 4, 8, and 12

Urgency was defined as a complaint of a sudden, compelling desire to pass urine, which is difficult to defer. An urgency episode was defined as any micturition or incontinence episode with a severity of grade 3 or 4, assessed by participants based on the Patient Perception of Intensity of Urgency Scale (PPIUS), where 0 = No urgency; 1 = Mild urgency; 2 = Moderate urgency, could delay voiding a short while; 3 = Severe urgency, could not delay voiding; 4 = Urge incontinence, leaked before arriving to the toilet. The mean number of urgency episodes (grade 3 and/or 4) per day was calculated as the average number of times a participant recorded an urgency episode (grade 3 and/or 4) with or without incontinence per day during the 3-day micturition diary period.

Change From Baseline to Week 4, Week 8, Week 12 and EoT in Total Health Related Quality of Life (HRQL) ScoreBaseline and Weeks 4, 8, and 12

The OAB-q is a self-reported questionnaire with 33 questions relating to symptom bother and health-related quality of life (HRQoL). The HRQoL portion consists of 25 HRQoL items comprising 4 HRQoL subscales (Coping, Concern, Sleep, and Social Interaction), each item was scored 1-6. The total score was calculated by adding the 4 HRQoL subscale scores and transforming to a scale from 0 to 100, with higher scores indicating better quality of life. A higher score on OAB-q HRQL indicated a better response.

Change From Baseline to Week 4, Week 8, Week 12 and EoT in HRQL Subscale Concern ScoreBaseline and Weeks 4, 8, and 12

The OAB-q is a self-reported questionnaire with 33 questions relating to symptom bother and health-related quality of life (HRQL). The HRQL portion consists of 25 HRQL items comprising 4 HRQoL subscales (Coping, Concern, Sleep, and Social Interaction), each item was scored 1-6. A higher score on OAB-q HRQL indicated a better response. The lowest and highest possible scores for the Concern subscale ranges from 7 to 42. The Concern score was calculated by adding the 7 response scores and transforming to a scale from 0 to 100, with higher scores indicating better quality of life. A positive change from baseline indicated an improvement.

Change From Baseline to Week 4, Week 8, Week 12 and EoT in IPSS Subscale Voiding ScoreBaseline and Weeks 4, 8, and 12

The International Prostate Symptom Score (IPSS) consists of 7 questions concerning urinary symptoms and 1 question concerning quality of life (QoL) with total score and subscores (voiding, storage and QoL). Each question is assigned points from 0 to 5 indicating increasing severity of the particular symptom. The subscale voiding score is the sum of the responses to 4 voiding symptoms questions (incomplete emptying, intermittency, weak stream, and straining). The lowest and highest possible scores range from 0 to 20 (mildly symptomatic to severely symptomatic). A higher score is indicative of worse condition and a negative change from baseline indicates an improvement.

Change From Baseline to Week 4, Week 8, Week 12, and EoT in IPSS Subscale Storage ScoreBaseline and Weeks 4, 8, and 12

The International Prostate Symptom Score (IPSS) consists of 7 questions concerning urinary symptoms and 1 question concerning quality of life (QoL) with total score and subscores (voiding, storage and QoL). Each question is assigned points from 0 to 5 indicating increasing severity of the particular symptom. The subscale storage score is the sum of the responses to 3 storage symptoms questions (frequency, urgency, and nocturia). The lowest and highest possible scores range from 0 to 15 (mildly symptomatic to severely symptomatic). A higher score is indicative of worse condition and a negative change from baseline indicates an improvement.

Change From Baseline to Week 4, Week 8, Week 12 and EoT in IPSS Subscale Quality of Life (QoL) ScoreBaseline and Weeks 4, 8, and 12

The International Prostate Symptom Score (IPSS) consists of 7 questions concerning urinary symptoms and 1 question concerning quality of life (QoL) with total score and subscores (voiding, storage and QoL). The QoL assessment was a single question asking the participant how he would feel about tolerating his current level of symptoms for the rest of his life. The lowest and highest possible score ranges from 0 to 6 (very pleased to terrible). A higher score is indicative of worse condition and a negative change from baseline indicates an improvement.

Change From Baseline to Week 4, Week 8, Week 12 and EoT in HRQL Subscale Sleep ScoreBaseline and Weeks 4, 8 and 12

The OAB-q is a self-reported questionnaire with 33 questions relating to symptom bother and health-related quality of life (HRQL). The HRQL portion consists of 25 HRQL items comprising 4 HRQoL subscales (Coping, Concern, Sleep, and Social Interaction), each item was scored 1-6. A higher score on OAB-q HRQL indicated a better response. The lowest and highest possible scores for the Sleep subscale ranges from 5 to 30. The Sleep score was calculated by adding the 5 response scores and transforming to a scale from 0 to 100, with higher scores indicating better quality of life. A positive change from baseline indicated an improvement.

Change From Baseline to Week 4, Week 8, Week 12 and EoT in European Quality of Life in 5 Dimensions and 5 Levels (EQ-5D-5L Questionnaire) UtilitiesBaseline and Weeks 4, 8, and 12

The EQ-5D-5L is an international standardized non-disease specific generic instrument for describing and valuing health status. It has a multidimensional measure of health-related QoL, capable of being expressed as a single index value and specifically designed to complement other health status measures. The EQ-5D-5L has five dimensions: Mobility, Self-Care, Usual Activities, Pain/Discomfort, and Anxiety/Depression. Each dimension has 5 response levels (e.g., 1=no problems, 2=slight problems, 3=moderate problems, 4=severe problems, and 5=extreme problems/unable to perform the activity). Health-state utility (HSU) data are estimates of the preference for a given state of health on a cardinal numeric scale, where a value of 1.0 represents full health, 0.0 represents dead, and negative values represent states worse than death. Missing EoT values were imputed using LOCF method.

Change From Baseline to Week 4, Week 8, Week 12 and EoT in Patient Perception of Bladder Condition (PPBC)Baseline and Weeks 4, 8, and 12

The PPBC is a validated, global assessment tool using a 6-point Likert scale that asks participants to rate their subjective impression of their current bladder condition. Participants assessed their bladder condition using this scale: 1. Does not cause me any problems at all; 2. Causes me some very minor problems; 3. Causes me some minor problems; 4. Causes me (some) moderate problems; 5. Causes me severe problems; 6. Causes me many severe problems. A higher score indicated a worse perception of bladder condition.

Change From Baseline to Week 4, Week 8, Week 12 and EoT in Total Urgency and Frequency Score (TUFS)Baseline and Weeks 4, 8 and 12

The TUFS was calculated by adding the PPIUS scores of every void in a participant's 3-day diary, and dividing this by the number of days recorded in the diary. Due to a programming failure in the e-diary data for the number of pads used was not collected.

Trial Locations

Locations (79)

Site US01009

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Homewood, Alabama, United States

Site US01005

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Huntsville, Alabama, United States

Site US01070

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Hawaiian Gardens, California, United States

Site US01081

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Los Angeles, California, United States

Site US01021

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Murrieta, California, United States

Site US01068

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San Diego, California, United States

Site US01064

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Valley Village, California, United States

Site US01097

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Englewood, Colorado, United States

Site US01026

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Pompano Beach, Florida, United States

Site US01034

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West Des Moines, Iowa, United States

Site US01025

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Saint Petersburg, Florida, United States

Site US01027

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Wellington, Florida, United States

Site US01041

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Baltimore, Maryland, United States

Site US01060

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Kansas City, Missouri, United States

Site US01003

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Watertown, Massachusetts, United States

Site US01016

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Boston, Massachusetts, United States

Site US01031

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Albuquerque, New Mexico, United States

Site US01015

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Garden City, New York, United States

Site US01019

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Plainview, New York, United States

Site US01069

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Cary, North Carolina, United States

Site US01079

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Maiden, North Carolina, United States

Site US01042

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Clarksville, Tennessee, United States

Site US01056

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Knoxville, Tennessee, United States

Site US01063

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Newport News, Virginia, United States

Site CA15001

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Brampton, Ontario, Canada

Site US01078

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Knoxville, Tennessee, United States

Site CA15003

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Barrie, Ontario, Canada

Site CA15002

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Sherbrooke, Quebec, Canada

Site CA15005

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Toronto, Ontario, Canada

Site CA15011

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Quebec City, Quebec, Canada

Site CZ42004

🇨🇿

Benesov, Czechia

Site CZ42005

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Prague 10, Czechia

Site FR33004

🇫🇷

Colmar Cedex, France

Site CZ42006

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Domazlice, Czechia

Site CZ42001

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Sternberk, Czechia

Site CZ42003

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Praha 4, Czechia

Site FR33002

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Pierre Benite Cedex, France

Site DE49002

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Duisburg, Germany

Site DE49004

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Hamburg, Germany

Site DE49001

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Hagenow, Germany

Site DE49006

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Halle (Saale), Germany

Site DE49003

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Lutherstadt Eisleben, Germany

Site DE49005

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Hettstedt, Germany

Site DE49007

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Sangerhausen, Germany

Site IT39003

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Catanzaro, Italy

Site IT39002

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Avellino, Italy

Site IT39006

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Pisa, Italy

Site IT39004

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Florence, Italy

Site IT39005

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Latina, Italy

Site IT39010

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Chieti, Italy

Site IT39008

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Vasto, Italy

Site IT39001

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Treviglio, Italy

Site PL48004

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Warszawa, Poland

Site PL48005

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Wrocław, Poland

Site ES34007

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Barcelona, Spain

Site ES34001

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Madrid, Spain

Site ES34009

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Lugo, Spain

Site ES34004

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Vigo, Spain

Site ES34002

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Sevilla, Spain

Site ES34003

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Miranda de Ebro, Spain

Site ES34006

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Valencia, Spain

Site GB44005

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Bristol, United Kingdom

Site GB44011

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Bristol, United Kingdom

Site GB44006

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York, United Kingdom

Site GB44002

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Reading, United Kingdom

Site GB44004

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Newcastle upon Tyne, United Kingdom

Site PL48007

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Wroclaw, Poland

Site PL48006

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Katowice, Poland

Site CA15014

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Victoriaville, Quebec, Canada

Site US01012

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Anchorage, Alaska, United States

Site CA15009

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Sherbrooke, Quebec, Canada

Site CA15008

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Calgary, Alberta, Canada

Site US01067

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Billings, Montana, United States

Site US01075

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Knoxville, Tennessee, United States

Site US01072

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Bristol, Tennessee, United States

Site US01094

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Shreveport, Louisiana, United States

Site US01061

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Omaha, Nebraska, United States

Site US01029

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Edison, New Jersey, United States

Site PL48001

🇵🇱

Piaseczno, Poland

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