A study to evaluate the drug Mongersen (GED-0301) for the treatment of Crohn's disease in adults and adolescents.

• Register: EUCTR
• Last refreshed on: 6 November 2017
• Main ID: EUCTR2015-001924-40-AT
• Date of registration: 18/01/2017
• Prospective Registration: Yes
• Primary sponsor: Celgene Corporation
• Public title: A study to evaluate the drug Mongersen (GED-0301) for the treatment of Crohn's disease in adults and adolescents.
• Scientific title: A Phase 3, randomized, double-blind, placebo-controlled, multicenter study to investigate the efficacy and safety of mongersen (GED-0301) for the treatment of adult and adolescent subjects with active Crohn’s disease - A Study of Efficacy and Safety of GED-0301 in Adults and Adolescents with Crohn's Disease
• Date of first enrolment: 21/02/2017
• Target sample size: 798
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-001924-40
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: no Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 3
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Argentina, Australia, Austria, Belgium, Bosnia and Herzegovina, Brazil, Canada, Chile, Colombia, Croatia, Czech Republic, Denmark, Finland, France, Germany, Greece, Hungary, Israel, Italy, Korea, Republic of, Latvia, Malaysia, Mexico, Netherlands, Norway, Poland, Portugal, Romania, Russian Federation, Serbia, Slovakia, Spain, Sweden, Switzerland, Taiwan, Turkey, Ukraine, United Kingdom, United States

Contacts

• Name: ClinicalTrialDisclosure
• Affiliation: Celgene Corporation

• Name: ClinicalTrialDisclosure
• Affiliation: Celgene Corporation

Key inclusion & exclusion criteria

Inclusion criteria:
• Diagnosis of CD with a duration of at least 3 months prior to the Screening Visit
• Presence of ileitis, ileocolitis, or colitis, as determined by ileocolonoscopy at screening
• Active disease, defined as a CDAI score = 220 and = 450 at screening
• Must have a 7-day average daily liquid or soft stool frequency = 3.5 or abdominal pain = 1.5 at screening
• Must have a total SES-CD = 6 at screening, or the ileum segmental SES-CD = 4 at screening
• Must have failed or experienced intolerance to at least one of the following: budesonide; systemic corticosteroids; immunosuppressants (ie, AZA, 6-MP, or MTX); or biologics for the treatment of CD (ie, infliximab, adalimumab, certolizumab, or vedolizumab)

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 658
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 40

Exclusion criteria:
1. diagnosis of UC, indeterminate colitis, ischemic colitis, microscopic colitis, radiation colitis, diverticular disease-associated colitis, or colitis due to immunodeficiency.
2. local manifestations of CD such as abscesses, short bowel syndrome, or other disease complications for which surgery might be indicated or which could confound the evaluation of efficacy.
3. 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.
4. intestinal resection within 6 months or any intra-abdominal surgery within 3 months prior to the Screening Visit.
5. ileostomy or a colostomy.
6. prior treatment with mycophenolic acid, tacrolimus, sirolimus, cyclosporine, thalidomide or apheresis (eg, Adacolumn®) within 8 weeks prior to the Screening Visit.
7. intravenous (IV) corticosteroids within 2 weeks prior to the Screening Visit.
8. changed or discontinued the dose of oral aminosalicylates within 2 weeks prior to the Screening Visit.
9. changed or discontinued the dose of oral corticosteroids (prednisone = 20 mg/day or equivalent, budesonide = 9 mg/day) within 3 weeks prior to the Screening Visit.
10. 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 Week 12 visit.
11. immunosuppressants (eg, AZA, 6-MP, or MTX) within 12 weeks prior to the Screening Visit and has changed or discontinued the dose of immunosuppressants within 8 weeks prior to the Screening Visit.
12. topical GI treatments, such as, 5-aminosalicylic acid (5-ASA) or corticosteroid enemas or suppositories within 2 weeks prior to the Screening Visit.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Active Crohn's disease
MedDRA version: 20.0 Level: LLT Classification code 10021315 Term: Ileitis terminal System Organ Class: 100000016693

Therapeutic area: Diseases [C] - Digestive System Diseases [C06]

Intervention(s)

Product Name: Mongersen
Product Code: GED-0301
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: mongersen
CAS Number: 1443994-98-6
Current Sponsor code: GED-0301
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 40-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use

Product Name: Mongersen
Product Code: GED-0301
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: mongersen
CAS Number: 1443994-98-6
Current Sponsor code: GED-0301
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 160-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use

Primary Outcome(s)

Main Objective: Evaluate efficacy of GED-0301 at Week 12, administered as either a single 160 mg tablet or as four 40 mg tablets, compared with placebo on clinical activity in subjects with active CD

Primary end point(s): Proportion of subjects achieving clinical remission, defined as a CDAI score < 150

Secondary Objective: • evaluate efficacy of GED-0301 compared with placebo on endoscopic outcomes
• evaluate efficacy of GED-0301 at Week 4 and Week 12 compared with placebo on clinical activity
• evaluate efficacy of GED-0301 compared with placebo on clinical activity in adolescent subjects (aged 12 to 17 years, inclusive)
• evaluate systemic exposure of GED-0301 using PK sampling
• evaluate safety and tolerability of GED-0301

Timepoint(s) of evaluation of this end point: Week 12

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: As given above

Secondary end point(s): Proportion of subjects who have a clinical response, defined as a decrease from baseline in CDAI score = 100 points, at Week 12
Proportion of subjects with endoscopic response-25 (ER-25), defined as a reduction of at least 25% from baseline in SES-CD, at Week 12
Proportion of subjects who have a clinical response, defined as a decrease from baseline in CDAI = 100 points, at Week 4
Proportion of subjects achieving clinical remission, defined as a CDAI score < 150, at Week 4
Proportion of subjects with endoscopic response–50 (ER-50), defined as a reduction of at least 50% in SESCD
compared with baseline, at Week 12
Proportion of subjects achieving clinical remission, defined as a PCDAI score = 10 at Week 12 (adolescent subjects only)
plasma concentration of GED-0301 at Week 4 and Week 8
Type, frequency, severity, seriousness, and relationship of AEs to IP
Number of subjects who discontinue IP due to any AE through Week 12
Clinically significant changes in vital signs, ECG, and/or laboratory findings through Week 12 and 4-week Follow-up

Secondary ID(s)

GED-0301-CD-003

Source(s) of Monetary Support

Celgene Corporation