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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02974322




Registration number
NCT02974322
Ethics application status
Date submitted
23/11/2016
Date registered
28/11/2016
Date last updated
14/11/2017

Titles & IDs
Public title
A Study of Efficacy and Safety of Mongersen (GED-0301) for the Treatment of Adult and Adolescent Subjects With Active Crohn's Disease
Scientific title
A Phase 3, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of Mongersen (GED-0301) for the Treatment of Adult and Adolescent Subjects With Active Crohn's Disease
Secondary ID [1] 0 0
GED-0301-CD-003
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Crohn Disease 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Inflammatory bowel disease
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders
Oral and Gastrointestinal 0 0 0 0
Crohn's disease

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - GED-0301
Treatment: Drugs - Placebo

Experimental: GED-0301 1 x 160 mg once daily - GED-0301 1 x 160 mg tablet once daily (QD)

Experimental: GED-0301 - 4 x 40 mg once daily - GED-0301 4 x 40 mg tablets once daily (QD)

Placebo Comparator: Placebo once daily - Placebo once daily (QD)


Treatment: Drugs: GED-0301
GED-0301

Treatment: Drugs: Placebo
Placebo

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

Outcomes
Primary outcome [1] 0 0
Clinical remission defined by an average daily liquid or soft stool frequency and abdominal pain score (US) and Crohn's Disease Activity Index (CDAI) (Rest of World) - The proportion of subjects achieving clinical remission at Week 12.
Timepoint [1] 0 0
week 12
Secondary outcome [1] 0 0
Clinical remission defined by an average daily liquid or soft stool frequency and abdominal pain score (US) and CDAI - The proportion of subjects achieving clinical remission at Week 4, Week 12.
Timepoint [1] 0 0
Week 4, week 12
Secondary outcome [2] 0 0
Endoscopic improvement by the Simple Endoscopic Score for Crohn's Disease (SES-CD) - Endoscopic improvement of the mucosa at Week 12.
Timepoint [2] 0 0
Week 12
Secondary outcome [3] 0 0
Clinical response defined by CDAI - The proportion of subjects achieving clinical response at Week 4, Week 12.
Timepoint [3] 0 0
Week 4, week 12
Secondary outcome [4] 0 0
Clinical remission defined by PCDAI - The proportion of subjects achieving clinical remission at Week 12 (adolescent subjects only).
Timepoint [4] 0 0
Week 12
Secondary outcome [5] 0 0
Adverse Events (AEs) - Incidence and severity of adverse events
Timepoint [5] 0 0
Up to 20 weeks
Secondary outcome [6] 0 0
Pharmacokinetics (PK)- plasma concentration of GED-0301 - The plasma concentration of GED-0301 at Week 4, Week 8
Timepoint [6] 0 0
Week 4, week 8

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

- Male or female = 12 years

- Subject is able to swallow the IP tablets

- Active Crohn's disease (CD) disease as determined by the Crohn's Disease Activity
Index (CDAI) score and the Simple Endoscopic Score for Crohn's Disease (SES-CD)

- Must meet a determined average minimum number of daily stools or rating of abdominal
pain over a 7day period

- Subject must have failed or experienced intolerance to at least one of the following:
budesonide; systemic corticosteroids; immunosuppressants (eg, azathiopurine,
6-mercaptopurine, or methotrexate); or biologics for the treatment of CD (ie,
infliximab, adalimumab, certolizumab, or vedolizumab).
Minimum age
12 Years
Maximum age
No limit
Gender
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
ulcerative colitis (UC), indeterminate colitis, ischemic colitis, microscopic colitis,
radiation colitis or diverticular disease-associated colitis, or colitis due to
immunodeficiency

- Local manifestations of Crohn's Disease (CD) such as abscesses, short bowel syndrome;
or other disease complications for which surgery might be indicated or could confound
the evaluation of efficacy

- Subject has strictures with prestenotic dilatation, requiring procedural intervention,
or with obstructive symptoms. In addition, subjects with colonic strictures that are
not passable with an age-appropriate colonoscope, or strictures in the ileum or
ileocecal valve that are fibrotic in nature, will be excluded

- Intestinal resection within 6 months or any intra-abdominal surgery within 3 months
prior to the Screening Visit

- Ileostomy or a colostomy

- Subject has a history of any clinically significant medical condition that, in the
investigator's opinion, would prevent the subject from participating in the study

- Adolescents with delayed growth or pubertal development who are on corticosteroids at
baseline and who should not continue treatment with the same dose of corticosteroids
until the Week 12 visit.

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 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Withdrawn
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,QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
Concord Repatriation General Hospital - Concord
Recruitment hospital [3] 0 0
Centre For Digestive Diseases - Five Dock
Recruitment hospital [4] 0 0
Liverpool Hospital - Liverpool
Recruitment hospital [5] 0 0
Royal Brisbane and Women's Hospital - Herston
Recruitment hospital [6] 0 0
Mater Adult Hospital - South Brisbane
Recruitment hospital [7] 0 0
Princess Alexandra Hospital - Woolloongabba
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Royal Adelaide Hospital Institute of Medical and Veterinary Science - Adelaide
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Box Hill Hospital - Box Hill
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The Alfred Hospital - Melbourne
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Fiona Stanley Hospital - Murdoch
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Ballarat Base Hospital - Ballarat
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Monash Medical Centre Clayton - Bentleigh East
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St Vincents Hospital Melbourne - Fitzroy
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2050 - Camperdown
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2139 - Concord
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2170 - Liverpool
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4029 - Herston
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4102 - Woolloongabba
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5000 - Adelaide
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3128 - Box Hill
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3004 - Melbourne
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6150 - Murdoch
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3350 - Ballarat
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3165 - Bentleigh East
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3065 - Fitzroy
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Celgene
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of study is to test the effect of an experimental medication GED-0301(mongersen)
and evaluate its safety in patients (= 12 years of age) with active Crohn's disease. The
study will test GED-0301 compare to placebo for 12 weeks. The study treatment is blinded
which means that patients and the study doctor will not know which treatment has been
assigned. Patients in this study will be allowed treatment with stable doses of oral
aminosalicylates, oral corticosteroids, immunosupressants and antibiotics for the treatment
of Crohn's disease. Adolescent patients will also be allowed treatment with stable doses of
exclusive enteral nutrition and growth hormone.

All patients who complete the study will have the option to enter a long term active
treatment study.
Trial website
https://clinicaltrials.gov/show/NCT02974322
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Denesh Chitkara, MD
Address 0 0
Celgene 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

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT02974322