A Study of Efficacy and Safety of Mongersen (GED-0301) for the Treatment of Adult and Adolescent Subjects With Active Crohn's Disease

• ClinicalTrials.gov Identifier: NCT02974322
• Recruitment Status: Withdrawn
• First Posted: November 28, 2016
• Last Update Posted: November 14, 2017
• Sponsor: Celgene
• Information provided by (Responsible Party): Celgene

Tracking Information

• First Submitted Date: November 23, 2016
• First Posted Date: November 28, 2016
• Last Update Posted Date: November 14, 2017
• Estimated Study Start Date: December 1, 2017
• Estimated Primary Completion Date: November 17, 2018 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: November 23, 2016)

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) [ Time Frame: week 12 ]

The proportion of subjects achieving clinical remission at Week 12.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: November 28, 2016)

• Clinical remission defined by an average daily liquid or soft stool frequency and abdominal pain score (US) and CDAI [ Time Frame: Week 4, week 12 ]
  The proportion of subjects achieving clinical remission at Week 4, Week 12.
• Endoscopic improvement by the Simple Endoscopic Score for Crohn's Disease (SES-CD) [ Time Frame: Week 12 ]
  Endoscopic improvement of the mucosa at Week 12.
• Clinical response defined by CDAI [ Time Frame: Week 4, week 12 ]
  The proportion of subjects achieving clinical response at Week 4, Week 12.
• Clinical remission defined by PCDAI [ Time Frame: Week 12 ]
  The proportion of subjects achieving clinical remission at Week 12 (adolescent subjects only).
• Adverse Events (AEs) [ Time Frame: Up to 20 weeks ]
  Incidence and severity of adverse events
• Pharmacokinetics (PK)- plasma concentration of GED-0301 [ Time Frame: Week 4, week 8 ]
  The plasma concentration of GED-0301 at Week 4, Week 8

Original Secondary Outcome Measures (submitted: November 23, 2016)

• Clinical remission defined by an average daily liquid or soft stool frequency and abdominal pain score (US) and CDAI [ Time Frame: Week 4, week 12 ]
  The proportion of subjects achieving clinical remission at Week 4, Week 12.
• Endoscopic improvement by the Simple Endoscopic Score for Crohn's Disease (SES-CD) [ Time Frame: Week 12 ]
  Endoscopic improvement of the mucosa at Week 12.
• Clinical response defined by CDAI [ Time Frame: Week 4, week 12 ]
  The proportion of subjects achieving clinical response at Week 4, Week 12.
• Clinical remission defined by PCDAI [ Time Frame: Week 12 ]
  The proportion of subjects achieving clinical remission at Week 12 (adolescent subjects only).
• Adverse Events (AEs) [ Time Frame: Up to 20 weeks ]
  Incidence and severity of adverse events
• Pharmacokinetics (PK)- GED-0301 [ Time Frame: Week 4, week 8 ]
  The plasma concentration of GED-0301 at Week 4, Week 8

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Study of Efficacy and Safety of Mongersen (GED-0301) for the Treatment of Adult and Adolescent Subjects With Active Crohn's Disease
• Official 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

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.

• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Crohn Disease

Intervention

• Drug: GED-0301
  GED-0301
  Other Name: Mongersen
• Drug: Placebo
  Placebo

Study Arms

• Experimental: GED-0301 1 x 160 mg once daily
  GED-0301 1 x 160 mg tablet once daily (QD)
  Intervention: Drug: GED-0301
• Experimental: GED-0301 - 4 x 40 mg once daily
  GED-0301 4 x 40 mg tablets once daily (QD)
  Intervention: Drug: GED-0301
• Placebo Comparator: Placebo once daily
  Placebo once daily (QD)
  Intervention: Drug: Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Withdrawn
• Actual Enrollment (submitted: November 9, 2017): 0
• Original Estimated Enrollment (submitted: November 23, 2016): 798
• Estimated Study Completion Date: December 24, 2018
• Estimated Primary Completion Date: November 17, 2018 (Final data collection date for primary outcome measure)

Eligibility Criteria

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).

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 12 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Australia, Austria, Belgium, Canada, Croatia, Czechia, Denmark, Finland, France, Germany, Greece, Hungary, Israel, Italy, Korea, Republic of, Latvia, Netherlands, Poland, Portugal, Romania, Russian Federation, Serbia, Slovakia, Spain, Sweden, Switzerland, Turkey, Ukraine, United Kingdom, United States
• Removed Location Countries: Bosnia and Herzegovina, Czech Republic

Administrative Information

• NCT Number: NCT02974322
• Other Study ID Numbers: GED-0301-CD-003
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Current Responsible Party: Celgene
• Original Responsible Party: Celgene Corporation
• Current Study Sponsor: Celgene
• Original Study Sponsor: Celgene Corporation
• Collaborators: Not Provided

Investigators

• Study Director: Denesh Chitkara, MD; Celgene Corporation

• PRS Account: Celgene
• Verification Date: November 2017