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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01500551




Registration number
NCT01500551
Ethics application status
Date submitted
22/12/2011
Date registered
28/12/2011

Titles & IDs
Public title
Long-Term Safety Study Of Tofacitinib In Patients With Juvenile Idiopathic Arthritis
Scientific title
A LONG-TERM, OPEN-LABEL FOLLOW-UP STUDY OF TOFACITINIB FOR TREATMENT OF JUVENILE IDIOPATHIC ARTHRITIS (JIA)
Secondary ID [1] 0 0
2011-004915-22
Secondary ID [2] 0 0
A3921145
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Juvenile Idiopathic Arthritis 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Osteoarthritis
Inflammatory and Immune System 0 0 0 0
Rheumatoid arthritis

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Tofacitinib
Treatment: Drugs - Tofacitinib

Experimental: Tofacitinib - All patients will be in tofacitinib treatment group.


Treatment: Drugs: Tofacitinib
Tofacitinib will be administered orally BID (twice daily) approximately 12 hours (±2 hours) apart, once in the morning and once in the evening, based on body weight for all subjects for all three index studies (A3921103, A3921104, and A3921165)

5 mg BID Dose Level:

Body Weight (Dose in tablet \[mg BID\] or solution \[ml BID\]) 5 - \< 7 kg (2 mg or 2 ml) 7 - \< 10 kg (2.5 mg or 2.5 ml) 10 - \<15 kg (3 mg or 3 ml) 15 - \<25 kg (3.5 mg or 3.5 ml) 25 - \<40 kg (4 mg or 4 ml) \>=40 kg (5 mg or 5 ml)

Oral solution (1 mg/mL concentration) will be used for subjects weighing \<40 kg. Oral tablets (5 mg) will be used for subjects weighing \>=40 kg; subjects who are unable to swallow tablets will have the option of taking oral solution.

Subjects will swallow study tablets whole and will not manipulate or chew tablets prior to swallowing.

Treatment: Drugs: Tofacitinib
For subjects rolling over from study A3921103 and actively participating in this study at the time of Protocol Amendment 6 and receiving a dosage of tofacitinib in accordance with the dosing scheme specified in Protocol Amendment 5, investigators will have the option of maintaining the subject's current dosage regimen from index study A3921103 (if the desired clinical response has been attained with no safety concern) or adjusting the dosage regimen in accordance with the dosing scheme specified in this section.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Standard laboratory safety data and adverse event (AE) reports. Body weight, height and Tanner Stages will collected to assess growth and physical development.
Timepoint [1] 0 0
up to 8 years
Secondary outcome [1] 0 0
Physician global evaluation of disease activity at each visit.
Timepoint [1] 0 0
up to 8 years
Secondary outcome [2] 0 0
Number of joints with active arthritis at each visit.
Timepoint [2] 0 0
up to 8 years
Secondary outcome [3] 0 0
Number of joints with limitation of motion at each visit.
Timepoint [3] 0 0
up to 8 years
Secondary outcome [4] 0 0
Index of inflammation (C-reactive protein [CRP] and Erythrocyte Sedimentation Rate [ESR]) at each visit.
Timepoint [4] 0 0
up to 8 years
Secondary outcome [5] 0 0
Parent's Assessment of Physical Function (Childhood Health Assessment Questionnaire [CHAQ]Disability Index)at each visit.
Timepoint [5] 0 0
up to 8 years
Secondary outcome [6] 0 0
Parent's Assessment of Child's Arthritis Pain (Childhood Health Assessment Questionnaire [CHAQ] Discomfort Index, Visual Analog Scale [VAS])at each visit.
Timepoint [6] 0 0
up to 8 years
Secondary outcome [7] 0 0
Parent's Global Assessment of Overall Wellbeing (Childhood Health Assessment Questionnaire [CHAQ] subsection, Visual Analog Scale [VAS])at each visit.
Timepoint [7] 0 0
up to 8 years
Secondary outcome [8] 0 0
JIA American College of Rheumatology (ACR) response and occurrence of JIA ACR disease flare at each visit.
Timepoint [8] 0 0
up to 8 years
Secondary outcome [9] 0 0
JIA ACR Clinical Inactive Disease status and Clinical Remission on Medication at each visit.
Timepoint [9] 0 0
up to 8 years
Secondary outcome [10] 0 0
Change from baseline in Juvenile Arthritis Disease Activity Score (JADAS) 27- CRP and JADAS 27-ESR, and occurrence of JADAS minimum disease activity and inactive disease at each visit.
Timepoint [10] 0 0
up to 8 years
Secondary outcome [11] 0 0
In subjects with Enthesitis Related Arthritis (ERA): Change from baseline in the Tender Entheseal Assessment, Modified Schober's Test, Overall Back Pain and Nocturnal Back Pain responses at various visits.
Timepoint [11] 0 0
up to 8 years
Secondary outcome [12] 0 0
In subjects with psoriatic arthritis (PsA): Change from baseline in body surface area (BSA) affected by psoriasis and Physician's Global Assessment (PGA) of psoriasis) at various visits.
Timepoint [12] 0 0
up to 8 years
Secondary outcome [13] 0 0
In subjects with sJIA: "Absence of Fever", defined as absence of fever due to sJIA in the week preceding the assessment at each visit.
Timepoint [13] 0 0
up to 8 years
Secondary outcome [14] 0 0
Eligibility of tapering defined per protocol for corticosteroids
Timepoint [14] 0 0
up to 8 years
Secondary outcome [15] 0 0
Eligibility of tapering defined per protocol for methotrexate
Timepoint [15] 0 0
up to 8 years
Secondary outcome [16] 0 0
Eligibility of tapering defined per protocol for leflunomide
Timepoint [16] 0 0
Up to 8 years
Secondary outcome [17] 0 0
Eligibility of tapering defined per protocol for tofacitinib
Timepoint [17] 0 0
Up to 8 years

Eligibility
Key inclusion criteria
* Pediatric subjects with JIA aged from 2 to less than 18 years who met entry criteria for the qualifying/index study and in the opinion of the investigator have sufficient evidence of JIA disease activity to warrant use of tofacitinib as a DMARD. Subjects turning 18 years of age during participation in the qualifying/index study or subsequently will be eligible for participation in this study.
* The subject has discontinued disallowed concomitant medications for the required time prior to the first dose of study drug, as defined in Appendix 1, and is taking only those concomitant medications in doses and frequency allowed by the protocol.
* Fertile male subjects and female subjects of childbearing potential who are, in the opinion of the investigator, sexually active and at risk for pregnancy with their partner(s) must be using a highly effective method of contraception as outlined in this protocol throughout the study and for at least 28 days after the last dose of study medication.
* Subjects must have previously completed participation in a qualifying study of tofacitinib for the treatment of JIA. Subjects who have required earlier discontinuation of treatment in a qualifying study for reasons other than tofacitinib related serious adverse events may be eligible.
Minimum age
2 Years
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* persistent oligoarthritis, and undifferentiated JIA.
* Infections:

1. Chronic infections.
2. Any infection requiring hospitalization, parenteral antimicrobial therapy or judged to be opportunistic by the investigator within the 3 months prior to the first dose of study drug.
3. Any treated infections within 2 weeks of baseline visit.
4. A subject known to be infected with human immunodeficiency virus (HIV), hepatitis B or hepatitis C virus.
5. History of infected joint prosthesis with prosthesis still in situ.
* History of recurrent (more than one episode) herpes zoster or disseminated (a single episode) herpes zoster or disseminated (a single episode) herpes simplex.

Study design
Purpose of the study
Treatment
Allocation to intervention
NA
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
18/03/2013
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
22/03/2025
Actual
Sample size
Target
Accrual to date
Final
276
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
The Children's Hospital at Westmead - Westmead
Recruitment hospital [2] 0 0
The Royal Children's Hospital - Parkville
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
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Arkansas
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California
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Connecticut
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District of Columbia
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Florida
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Georgia
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United States of America
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Illinois
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Indiana
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Massachusetts
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Minnesota
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New Jersey
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New York
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North Carolina
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Ohio
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Oklahoma
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Oregon
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Pennsylvania
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Texas
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Utah
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Washington
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Argentina
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Santa FE
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Argentina
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Tucuman
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Argentina
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Caba
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Belgium
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Brussels
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Belgium
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Gent
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Belgium
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Leuven
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Brazil
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Bahia
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Brazil
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Minas Gerais
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Brazil
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Parana
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Brazil
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RIO Grande DO SUL
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SAO Paulo
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Brazil
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Rio de Janeiro
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Brazil
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Sao Paulo
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Canada
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Alberta
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Canada
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British Columbia
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Canada
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Ontario
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Canada
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Quebec
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China
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Chongqing
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China
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Guangdong
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China
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Hubei
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China
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Hunan
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China
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Jiangsu
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China
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Shaanxi
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China
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Sichuan
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China
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Zhejiang
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China
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Beijing
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China
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Shanghai
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Costa Rica
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San Jose
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Germany
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Bayern
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Germany
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Bad Bramstedt
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Germany
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Berlin
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Germany
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Bremen
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Germany
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Dresden
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Germany
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Garmisch-partenkirchen
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Germany
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Hamburg
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Germany
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Sankt Augustin
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Germany
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Sendenhorst
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Hungary
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Budapest
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India
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Gujarat
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India
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WEST Bengal
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India
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New Delhi
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Israel
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Haifa
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Israel
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Kfar Saba
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Israel
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Ramat Gan
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Italy
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Milan
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Italy
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Genova
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Mexico
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Jalisco
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Mexico
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Nuevo LEON
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Mexico
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San Luis De Potosí
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Mexico
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San Luis Potosi
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Poland
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Bydgoszcz
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Poland
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Krakow
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Lodz
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Sosnowiec
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Poland
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Warszawa
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Russian Federation
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Republic OF Bashkortostan
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Russian Federation
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Moscow
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Russian Federation
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Saint-Petersburg
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Russian Federation
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Tolyatti
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Slovakia
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Bratislava
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Slovakia
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Kosice
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Slovakia
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Piestany
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South Africa
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Cape Town
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South Africa
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Kwazulu-natal
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South Africa
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Durban
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Spain
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Madrid
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Spain
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Valencia
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Turkey
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Ankara
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Turkey
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Istanbul
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Turkey
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Kadikoy / Istanbul
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Turkey
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Kayseri
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Ukraine
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Dnipro
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Ukraine
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Ivano-Frankivsk
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Ukraine
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Vinnytsia
Country [96] 0 0
United Kingdom
State/province [96] 0 0
WEST Midlands
Country [97] 0 0
United Kingdom
State/province [97] 0 0
Glasgow

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Pfizer
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Pfizer CT.gov Call Center
Address 0 0
Pfizer
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT01500551