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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02289417




Registration number
NCT02289417
Ethics application status
Date submitted
10/11/2014
Date registered
13/11/2014

Titles & IDs
Public title
Efficacy and Safety Study of Apremilast to Treat Active Ulcerative Colitis
Scientific title
A Phase 2, Randomized, Placebo-controlled, Multicenter Study to Investigate the Efficacy and Safety of Apremilast (CC-10004) for Treatment of Subjects With Active Ulcerative Colitis
Secondary ID [1] 0 0
2014-002981-64
Secondary ID [2] 0 0
CC-10004-UC-001
Universal Trial Number (UTN)
Trial acronym
UC
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Ulcerative Colitis 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders
Oral and Gastrointestinal 0 0 0 0
Inflammatory bowel disease

Intervention/exposure
Study type
Interventional(has expanded access)
Description of intervention(s) / exposure
Experimental: Apremilast 30 mg PO BID - Apremilast 30 mg by mouth (PO) twice a day (BID) for 12 weeks

After 12 weeks:

* Participants who achieve at least a 20% decrease from baseline in the total Mayo score (TMS) will continue to receive apremilast 30 mg BID for an additional 40 weeks. (Wk 52)
* Participants who do not achieve at least a 20% decrease from baseline in the TMS will receive apremilast 40 mg BID for an additional 40 weeks (Wk 52)

After 52 weeks, participants who are eligible for the Extension Phase will continue to receive the same dose of apremilast assigned at Wk 12 (30 mg BID or 40 mg BID) for an additional 52 weeks (Wk 104)

Experimental: Apremilast 40 mg PO BID - Apremilast 40 mg by mouth (PO) twice a day (BID) for 12 weeks

After 12 weeks, participants assigned to the 40 mg BID dose of apremilast at baseline will continue to receive apremilast 40 mg BID for an additional 40 weeks (Wk 52)

After 52 weeks, participants who are eligible for the extension Phase will continue to receive apremilast 40 mg BID for an additional 52 weeks (Wk 104)

Placebo comparator: Placebo BID - Identically matching placebo by mouth (PO) twice a day (BID) for 12 weeks. After 12 weeks all participants randomized to placebo at baseline will be re-randomized to receive apremilast 30 mg or 40 mg BID for an additional 40 weeks (Wk 52)

After Wk 52, participants who are eligible for the extension phase will continue to receive the same dose of apremilast assigned at Wk 12 (30 mg BID or 40 mg BID) for an additional 52 weeks (Wk 104)
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Participants Who Achieved a Clinical Remission by Total Mayo Score (TMS) at Week 12
Timepoint [1] 0 0
Week 12
Secondary outcome [1] 0 0
Percentage of Participants Who Achieved a Clinical Response by Total Mayo Score and the Reduction in the Rectal Bleeding Subscore at Week 12
Timepoint [1] 0 0
Week 12
Secondary outcome [2] 0 0
Percentage of Participants Who Achieved an Endoscopic Remission at Week 12
Timepoint [2] 0 0
Week 12
Secondary outcome [3] 0 0
Percentage of Participants Who Achieved an Endoscopic Response at Week 12
Timepoint [3] 0 0
Week 12
Secondary outcome [4] 0 0
Percentage of Participants Who Achieved a Rectal Bleeding Subscore (RBS) of = 1 at Week 12
Timepoint [4] 0 0
Week 12
Secondary outcome [5] 0 0
Percentage of Participants Who Achieved Clinical Remission in the Modified Mayo Subscore (MMS) at Week 12
Timepoint [5] 0 0
Week 12
Secondary outcome [6] 0 0
Percentage of Participants Who Achieved Clinical Response in the Modified Mayo Subscore (MMS) at Week 12
Timepoint [6] 0 0
Week 12
Secondary outcome [7] 0 0
Percentage of Participants Who Achieved Clinical Remission in the Partial Mayo Subscore (PMS) With no Individual Subscore >1 at Week 8
Timepoint [7] 0 0
Week 8
Secondary outcome [8] 0 0
Percentage of Participants Who Achieved Clinical Response in the Partial Mayo Subscore at Week 8
Timepoint [8] 0 0
Week 8
Secondary outcome [9] 0 0
The Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAEs) During the Placebo-Controlled Phase
Timepoint [9] 0 0
From the first dose of investigational product (IP) and no later than 28 days after the last dose of IP for those who had completed the study or discontinued (D/C) early; maximum duration of exposure to treatment was 12.00 weeks
Secondary outcome [10] 0 0
The Number of Participants Who Discontinued Apremilast Due to Treatment Emergent Adverse Events During the Placebo-Controlled Period
Timepoint [10] 0 0
From the first dose of IP and no later than 28 days after the last dose of IP for those who had completed the study or discontinued early; median duration of exposure to treatment was 12.00 weeks
Secondary outcome [11] 0 0
The Number of Participants Who Experienced TEAEs During the Apremilast (APR) Exposure Period (Active Treatment Phase) Through Week 52
Timepoint [11] 0 0
From first dose of IP and no later than 28 days after last dose of IP for those who completed the active treatment phase or D/C early; median duration of exposure = 41.00, 44.15 and 40.00 weeks respectively for 30 mg, 40 mg and 30 mg/40 mg APR arms
Secondary outcome [12] 0 0
The Number of Participants Who Experienced TEAEs During Week 52 to Week 104 (Extension Phase)
Timepoint [12] 0 0
From the first dose of IP at Week 52 and no later than 28 days after the last dose of IP for those who completed the study or had discontinued early; median exposure of apremilast for the total apremilast group was 52 weeks.

Eligibility
Key inclusion criteria
Subjects must satisfy the following criteria to be enrolled in the study:

* Male or female aged 18 and over at the time of signing the informed consent.
* Must understand and voluntarily sign an informed consent form prior to any study related assessments/procedures being conducted.
* Diagnosis of ulcerative colitis (UC) with a duration of at least 3 months prior to the Screening Visit..
* Total Mayo Score (TMS) = 6 to = 11 (range: 0-12) at baseline, prior to randomization in the study.
* Endoscopic subscore = 2 (range: 0-3) on the Mayo score prior to randomization in the study.
* Subjects must have had a therapeutic failure, been intolerant to, or have a contraindication to, at least one of the following: oral aminosalicylates (ie, 5-aminosalicylic acid [5-ASA] compounds or sulfasalazine [SSZ]), budesonide, systemic corticosteroids, or immunosuppressants (eg, 6-mercaptopurine [6-MP], azathioprine [AZA], or methotrexate [MTX]).
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
The presence of any of the following will exclude a subject from enrollment:

* Diagnosis of Crohn's disease, indeterminate colitis, ischemic colitis, microscopic colitis, radiation colitis or diverticular disease-associated colitis.
* Ulcerative colitis restricted to the distal 15 cm or less (eg, ulcerative proctitis).
* Subjects who have had surgery as a treatment for UC or who, in the opinion of the Investigator, are likely to require surgery for UC during the study.
* Clinical signs suggestive of fulminant colitis or toxic megacolon.
* Prior use of any tumor necrosing factor (TNF) inhibitor (or any biologic agent).
* Prior use of mycophenolic acid, tacrolimus, sirolimus, cyclosporine or thalidomide.
* Use of intravenous (IV) corticosteroids within 2 weeks of the Screening Visit.
* Use of immunosuppressants (AZA, 6-MP or MTX) within 8 weeks of the Screening Visit.
* Use of topical treatment with 5-ASA or corticosteroid enemas or suppositories within 2 weeks of the Screening Visit.
* History of any clinically significant neurological, renal, hepatic, gastrointestinal, pulmonary, metabolic, cardiovascular, psychiatric, endocrine, hematological disorder or disease, or any other medical condition that, in the investigator's opinion, would preclude participation in the study.

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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
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
8/01/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
3/06/2019
Sample size
Target
Accrual to date
Final
170
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Concord Repatriation General Hospital - Concord
Recruitment hospital [2] 0 0
Liverpool Hospital - Liverpool
Recruitment hospital [3] 0 0
Mater Adult Hospital - South Brisbane
Recruitment hospital [4] 0 0
Footscray Hospital - Footscray
Recruitment hospital [5] 0 0
Royal Melbourne Hospital - Parkville
Recruitment postcode(s) [1] 0 0
2139 - Concord
Recruitment postcode(s) [2] 0 0
2170 - Liverpool
Recruitment postcode(s) [3] 0 0
4101 - South Brisbane
Recruitment postcode(s) [4] 0 0
3011 - Footscray
Recruitment postcode(s) [5] 0 0
3050 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Connecticut
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Iowa
Country [7] 0 0
United States of America
State/province [7] 0 0
Kentucky
Country [8] 0 0
United States of America
State/province [8] 0 0
Massachusetts
Country [9] 0 0
United States of America
State/province [9] 0 0
Michigan
Country [10] 0 0
United States of America
State/province [10] 0 0
Mississippi
Country [11] 0 0
United States of America
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New York
Country [12] 0 0
United States of America
State/province [12] 0 0
Ohio
Country [13] 0 0
United States of America
State/province [13] 0 0
Tennessee
Country [14] 0 0
United States of America
State/province [14] 0 0
Texas
Country [15] 0 0
United States of America
State/province [15] 0 0
Utah
Country [16] 0 0
United States of America
State/province [16] 0 0
Washington
Country [17] 0 0
Bulgaria
State/province [17] 0 0
Plovdiv
Country [18] 0 0
Bulgaria
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Sofia
Country [19] 0 0
Bulgaria
State/province [19] 0 0
Varna
Country [20] 0 0
Canada
State/province [20] 0 0
Manitoba
Country [21] 0 0
Canada
State/province [21] 0 0
Ontario
Country [22] 0 0
Czechia
State/province [22] 0 0
Brno
Country [23] 0 0
Czechia
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Hradec Kralove
Country [24] 0 0
Czechia
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Hradec Králové
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Czechia
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Slany
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France
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Amiens
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France
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Clichy
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France
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Nantes
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France
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Nice
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France
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Pierre Bénite
Country [31] 0 0
France
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Saint Priest en Jarez
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France
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Vandoeuvre les Nancy
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Germany
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Berlin
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Germany
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Frankfurt
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Germany
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Keil
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Germany
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Minden
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Hungary
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Budapest
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Hungary
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Debrecen
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Hungary
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Mosonmagyaróvár
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Hungary
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Szeged
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Hungary
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Szekszárd
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Hungary
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Vác
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Italy
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Bologna
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Italy
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Milan
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Italy
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Palermo
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Italy
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Roma
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Netherlands
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Groningen
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Netherlands
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Rotterdam
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New Zealand
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Auckland
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New Zealand
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Christchurch
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New Zealand
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Dunedin
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New Zealand
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Hamilton
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Poland
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Bialystok
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Bydgoszcz
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Czestochowa
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Katowice
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Sopot
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Szczecin
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Torun
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Warsaw
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Warszawa
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Poland
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Wroclaw
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Russian Federation
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Kazan
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Russian Federation
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Moscow
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Russian Federation
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Rostov on Don
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Russian Federation
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Saratov
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Russian Federation
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St Petersburg
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Ukraine
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Ivano-Frankivsk
Country [69] 0 0
Ukraine
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Kharkiv
Country [70] 0 0
Ukraine
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Kirovograd
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Ukraine
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Kremenchuk
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Ukraine
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Lviv
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Ukraine
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Odesa
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Ukraine
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Uzhgorod
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Ukraine
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Vinnytsia
Country [76] 0 0
Ukraine
State/province [76] 0 0
Zaporizhzhia

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

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
MD
Address 0 0
Amgen
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?
De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF), Clinical study report (CSR)
When will data be available (start and end dates)?
Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study.
Available to whom?
Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors. If not approved, a Data Sharing Independent Review Panel will arbitrate and make the final decision. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the URL below.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: http://www.amgen.com/datasharing


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT02289417