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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03112603




Registration number
NCT03112603
Ethics application status
Date submitted
4/04/2017
Date registered
13/04/2017

Titles & IDs
Public title
A Study of Ruxolitinib vs Best Available Therapy (BAT) in Patients With Steroid-refractory Chronic Graft vs. Host Disease (GvHD) After Bone Marrow Transplantation (REACH3)
Scientific title
A Phase III Randomized Open-label Multi-center Study of Ruxolitinib vs. Best Available Therapy in Patients With Corticosteroid-refractory Chronic Graft vs Host Disease After Allogeneic Stem Cell Transplantation (REACH3)
Secondary ID [1] 0 0
CINC424D2301
Secondary ID [2] 0 0
INCB 18424-365 (REACH3)
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Graft-versus-host Disease (GVHD) 0 0
Condition category
Condition code
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional(has expanded access)
Description of intervention(s) / exposure
Treatment: Drugs - Ruxolitinib
Treatment: Drugs - Extracorporeal photopheresis (ECP)
Treatment: Drugs - Low-dose methotrexate (MTX)
Treatment: Drugs - Mycophenolate mofetil (MMF)
Treatment: Drugs - mechanistic Target of Rapamycin (mTOR) inhibitors (everolimus or sirolimus)
Treatment: Drugs - Infliximab
Treatment: Drugs - Rituximab
Treatment: Drugs - Pentostatin
Treatment: Drugs - Imatinib
Treatment: Drugs - Ibrutinib

Experimental: Ruxolitinib - Ruxolitinib for the treatment period and extension period.

Active comparator: Best Available Therapy - Best available therapy for the treatment period and extension period, with optional crossover to ruxolitinib after Cycle 6.


Treatment: Drugs: Ruxolitinib
Ruxolitinib twice daily at the protocol-defined starting dose.

Treatment: Drugs: Extracorporeal photopheresis (ECP)
Best available therapy (BAT) will be selected by the investigator for each participant. BAT may not include experimental agents (ie, those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements. The BAT in this study will be among the following treatments currently used in this setting (no other types or combinations of BATs are permitted in this study).

Treatment: Drugs: Low-dose methotrexate (MTX)
Patients will receive BAT based on the Investigator's opinion, taking into account the manufacturer's instructions, labeling, subject's medical condition, and institutional guidelines for any dose adjustment.

Treatment: Drugs: Mycophenolate mofetil (MMF)
Patients will receive BAT based on the Investigator's opinion, taking into account the manufacturer's instructions, labeling, subject's medical condition, and institutional guidelines for any dose adjustment.

Treatment: Drugs: mechanistic Target of Rapamycin (mTOR) inhibitors (everolimus or sirolimus)
Patients will receive BAT based on the Investigator's opinion, taking into account the manufacturer's instructions, labeling, subject's medical condition, and institutional guidelines for any dose adjustment.

Treatment: Drugs: Infliximab
Patients will receive BAT based on the Investigator's opinion, taking into account the manufacturer's instructions, labeling, subject's medical condition, and institutional guidelines for any dose adjustment.

Treatment: Drugs: Rituximab
Patients will receive BAT based on the Investigator's opinion, taking into account the manufacturer's instructions, labeling, subject's medical condition, and institutional guidelines for any dose adjustment.

Treatment: Drugs: Pentostatin
Patients will receive BAT based on the Investigator's opinion, taking into account the manufacturer's instructions, labeling, subject's medical condition, and institutional guidelines for any dose adjustment.

Treatment: Drugs: Imatinib
Patients will receive BAT based on the Investigator's opinion, taking into account the manufacturer's instructions, labeling, subject's medical condition, and institutional guidelines for any dose adjustment.

Treatment: Drugs: Ibrutinib
Patients will receive BAT based on the Investigator's opinion, taking into account the manufacturer's instructions, labeling, subject's medical condition, and institutional guidelines for any dose adjustment.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Efficacy of Ruxolitinib Versus Investigator's Choice Best Available Therapy (BAT) in Participants With Moderate or Severe Steroid Refractory Chronic Graft Versus Host Disease (SR-cGvHD) Assessed by Overall Response Rate (ORR) at the Cycle 7 Day 1 Visit
Timepoint [1] 0 0
Cycle 7 Day 1 (each cycle was comprised of 4 weeks)
Secondary outcome [1] 0 0
Rate of Participants With Clinically Relevant Improvement of the Modified Lee cGvHD Symptom Scale Score
Timepoint [1] 0 0
Baseline; Cycle 7 Day 1 (each cycle was comprised of 4 weeks)
Secondary outcome [2] 0 0
Rate of Failure-free Survival (FFS)
Timepoint [2] 0 0
Baseline to when the last participant reached Cycle 7 Day 1 (each cycle was comprised of 4 weeks)
Secondary outcome [3] 0 0
Rate of FFS at Study Completion
Timepoint [3] 0 0
From Baseline to Last Participant Last Visit (LPLV) (approximately 5 years)
Secondary outcome [4] 0 0
Best Overall Response (BOR) at Cycle 7 Day 1
Timepoint [4] 0 0
up to Cycle 7 Day 1 (each cycle was comprised of 4 weeks)
Secondary outcome [5] 0 0
BOR During Cross-over Treatment With Ruxolitinib
Timepoint [5] 0 0
from Crossover Cycle 1 Day 1 to any time point up to and including Crossover Cycle 7 Day 1 (each cycle was comprised of 4 weeks)
Secondary outcome [6] 0 0
ORR at the End of Cycle 3
Timepoint [6] 0 0
Cycle 4 Day 1 (each cycle was comprised of 4 weeks)
Secondary outcome [7] 0 0
Duration of Response Through Study Completion
Timepoint [7] 0 0
from first response to LPLV (approximately 5 years)
Secondary outcome [8] 0 0
Overall Survival (OS)
Timepoint [8] 0 0
from the date of randomization to the date of death due to any cause up to LPLV (approximately 5 years)
Secondary outcome [9] 0 0
Cumulative Incidence of Non-relapse Mortality (NRM)
Timepoint [9] 0 0
Months 3, 6, 12, 18, 24, 30, and 36
Secondary outcome [10] 0 0
Percentage of Participants With a = 50% Reduction in Daily Corticosteroid Dose
Timepoint [10] 0 0
from Day 15 up to Day 182
Secondary outcome [11] 0 0
Percentage of Participants Successfully Tapered Off of All Corticosteroids
Timepoint [11] 0 0
up to Day 179
Secondary outcome [12] 0 0
Cumulative Incidence of Malignancy Relapse/Recurrence (MR)
Timepoint [12] 0 0
Months 3, 6, 12, 18, 24, 30, and 36
Secondary outcome [13] 0 0
Change From Baseline in Functional Assessment of Cancer Therapy - Bone Marrow Transplantation (FACT-BMT)
Timepoint [13] 0 0
Baseline; up to Cycle 39 Day 1 (each cycle was comprised of 4 weeks)
Secondary outcome [14] 0 0
Change From Baseline in EQ-5D-5L
Timepoint [14] 0 0
Baseline; up to Cycle 39 Day 1 (each cycle was comprised of 4 weeks)
Secondary outcome [15] 0 0
Number of Participants With Any Treatment-emergent Adverse Event (TEAE)
Timepoint [15] 0 0
from Baseline to LPLV (approximately 5 years)
Secondary outcome [16] 0 0
Cmax of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses
Timepoint [16] 0 0
Extensive Sampling Schedule: Cycle 1 Days 1 and 15: predose; 0.5, 1, 1.5, 4, 6, and 9 hours post-dose. Sparse Sampling Schedule: Cycle 1 Days 1 and 15: predose; 1.5 hours post-dose
Secondary outcome [17] 0 0
AUClast of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses
Timepoint [17] 0 0
Extensive Sampling Schedule: Cycle 1 Days 1 and 15: predose; 0.5, 1, 1.5, 4, 6, and 9 hours post-dose. Sparse Sampling Schedule: Cycle 1 Days 1 and 15: predose; 1.5 hours post-dose
Secondary outcome [18] 0 0
AUCinf of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses
Timepoint [18] 0 0
Extensive Sampling Schedule: Cycle 1 Days 1 and 15: predose; 0.5, 1, 1.5, 4, 6, and 9 hours post-dose. Sparse Sampling Schedule: Cycle 1 Days 1 and 15: predose; 1.5 hours post-dose
Secondary outcome [19] 0 0
CL/F of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses
Timepoint [19] 0 0
Extensive Sampling Schedule: Cycle 1 Days 1 and 15: predose; 0.5, 1, 1.5, 4, 6, and 9 hours post-dose. Sparse Sampling Schedule: Cycle 1 Days 1 and 15: predose; 1.5 hours post-dose
Secondary outcome [20] 0 0
Vz/F of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses
Timepoint [20] 0 0
Extensive Sampling Schedule: Cycle 1 Days 1 and 15: predose; 0.5, 1, 1.5, 4, 6, and 9 hours post-dose. Sparse Sampling Schedule: Cycle 1 Days 1 and 15: predose; 1.5 hours post-dose
Secondary outcome [21] 0 0
Tmax of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses
Timepoint [21] 0 0
Extensive Sampling Schedule: Cycle 1 Days 1 and 15: predose; 0.5, 1, 1.5, 4, 6, and 9 hours post-dose. Sparse Sampling Schedule: Cycle 1 Days 1 and 15: predose; 1.5 hours post-dose
Secondary outcome [22] 0 0
t1/2 of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses
Timepoint [22] 0 0
Extensive Sampling Schedule: Cycle 1 Days 1 and 15: predose; 0.5, 1, 1.5, 4, 6, and 9 hours post-dose. Sparse Sampling Schedule: Cycle 1 Days 1 and 15: predose; 1.5 hours post-dose
Secondary outcome [23] 0 0
Utilization of Medical Resources
Timepoint [23] 0 0
from Baseline to LPLV (approximately 5 years)

Eligibility
Key inclusion criteria
* Have undergone allogeneic stem cell transplantation (alloSCT) from any donor source (matched unrelated donor, sibling, haplo-identical) using bone marrow, peripheral blood stem cells, or cord blood. Recipients of non-myeloablative, myeloablative, and reduced intensity conditioning are eligible
* Evident myeloid and platelet engraftment: Absolute neutrophil count (ANC) > 1000/mm^3 and platelet count > 25,000/ mm^3
* Participants with clinically diagnosed moderate to severe cGvHD according to NIH Consensus Criteria prior to randomization:

* Moderate cGvHD: At least one organ (not lung) with a score of 2, 3 or more organs involved with a score of 1 in each organ, or lung score of 1
* Severe cGvHD: at least 1 organ with a score of 3, or lung score of 2 or 3
* Participants currently receiving systemic or topical corticosteroids for the treatment of cGvHD for a duration of < 12 months prior to Cycle 1 Day 1 (if applicable), and have a confirmed diagnosis of steroid-refractory cGvHD defined per 2014 NIH consensus criteria irrespective of the concomitant use of a calcineurin inhibitor (CNI), as follows:

* A lack of response or disease progression after administration of minimum prednisone 1 mg/kg/day for at least 1 week, OR
* Disease persistence without improvement despite continued treatment with prednisone at > 0.5 mg/kg/day or 1 mg/kg/every other day for at least 4 weeks, OR
* Increase to prednisolone dose to > 0.25 mg/kg/day after 2 unsuccessful attempts to taper the dose
* Participant must accept to be treated with only one of the following BAT options on Cycle 1 Day 1 (additions and changes are allowed during the course of the study, but only with BAT from the following BAT options): extracorporeal photopheresis (ECP), low-dose methotrexate (MTX), mycophenolate mofetil (MMF), mTOR inhibitors (everolimus or sirolimus), infliximab, rituximab, pentostatin, imatinib, ibrutinib
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Participants who have received 2 or more systemic treatment for cGvHD in addition to corticosteroids ± CNI for cGvHD
* Patients that transition from active aGvHD to cGvHD without tapering off corticosteroids ± CNI and any systemic treatment

* Patients receiving up to 30 mg by mouth once a day of hydrocortisone (i.e., physiologic replacement dose) of corticosteroids are allowed.
* Participants who were treated with prior JAK inhibitors for aGvHD; except when the participant achieved complete or partial response and has been off JAK inhibitor treatment for at least 8 weeks prior to Cycle 1 Day 1
* Failed prior alloSCT within the past 6 months from Cycle 1 Day 1
* Participants with relapsed primary malignancy, or who have been treated for relapse after the alloSCT was performed
* Steroid refractory cGvHD occurring after a non-scheduled donor lymphocyte infusion (DLI) administered for preemptive treatment of malignancy recurrence. Participants who have received a scheduled DLI as part of their transplant procedure and not for management of malignancy relapse are eligible
* Any corticosteroid therapy for indications other than cGvHD at doses > 1 mg/kg/day methylprednisolone or equivalent within 7 days of Cycle 1 Day 1

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
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
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - Darlinghurst
Recruitment hospital [2] 0 0
Novartis Investigative Site - Parkville
Recruitment postcode(s) [1] 0 0
2010 - Darlinghurst
Recruitment postcode(s) [2] 0 0
3002 - Parkville
Recruitment outside Australia
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Arizona
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California
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Connecticut
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Delaware
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Florida
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Illinois
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Indiana
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Kansas
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Kentucky
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Massachusetts
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Nebraska
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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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Tennessee
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Texas
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Washington
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Wisconsin
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Austria
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Tyrol
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Graz
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Linz
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Lyon Cedex 08
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Montpellier cedex 5
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Saint Priest en Jarez Cedex
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Vandoeuvre les Nancy cedex
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Koeln
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Leipzig
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Muenster
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Ulm
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Würzburg
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Greece
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Athens
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Hungary
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Budapest
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India
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Maharashtra
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India
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Delhi
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India
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Vellore
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Israel
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Haifa
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Israel
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Jerusalem
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Israel
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Petach Tikva
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Israel
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Tel Aviv
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Italy
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AN
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Italy
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BG
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Italy
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BO
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Italy
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BS
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Italy
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GE
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Italy
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MI
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Italy
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PA
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Italy
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PE
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Italy
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PR
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Italy
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PV
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Italy
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RM
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Italy
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TO
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Italy
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UD
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Italy
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Perugia
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Japan
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Aichi
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Japan
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Fukuoka
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Japan
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Hokkaido
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Japan
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Hyogo
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Japan
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Kanagawa
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Japan
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Miyagi
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Japan
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Osaka
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Japan
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Tochigi
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Japan
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Tokyo
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Japan
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Kyoto
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Japan
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Okayama
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Jordan
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Amman
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Korea, Republic of
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Seoul
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Netherlands
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The Netherlands
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Netherlands
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Leiden
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Netherlands
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Nijmegen
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Rotterdam
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Norway
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Oslo
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Poland
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Dolnoslaskie
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Poland
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Slaskie
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Poland
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Katowice
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Poland
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Kraków
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Poland
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Warszawa
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Portugal
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Lisboa
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Portugal
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Porto
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Puerto Rico
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Ponce
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Romania
State/province [126] 0 0
Bucharest
Country [127] 0 0
Russian Federation
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Moscow
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Russian Federation
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Saint Petersburg
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Saudi Arabia
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Riyadh
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Spain
State/province [130] 0 0
Andalucia
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Spain
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Andalucía
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Spain
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Castilla Y Leon
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Spain
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Catalunya
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Spain
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Cataluña
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Spain
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Comunidad Valenciana
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Spain
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Galicia
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Spain
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Murica
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Spain
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Pais Vasco
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Spain
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Barcelona
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Spain
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Las Palmas de Gran Canaria
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Spain
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Madrid
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Sweden
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Göteborg
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Sweden
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Uppsala
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Switzerland
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Basel
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Switzerland
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Zürich
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Turkey
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Ankara
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Turkey
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Antalya
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United Kingdom
State/province [148] 0 0
Glasgow
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United Kingdom
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London
Country [150] 0 0
United Kingdom
State/province [150] 0 0
Manchester

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

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
Rodica Morariu-Zamfir
Address 0 0
Incyte Corporation
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)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

Results publications and other study-related documents

No documents have been uploaded by study researchers.