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Pharmacodynamic and Clinical Outcome Study of Mongersen in Patients With Crohn's Disease

This study is currently recruiting participants.
See Contacts and Locations
Verified August 2017 by Celgene
Sponsor:
Information provided by (Responsible Party):
Celgene
ClinicalTrials.gov Identifier:
NCT02685683
First received: February 15, 2016
Last updated: August 31, 2017
Last verified: August 2017
  Purpose
This study is designed to explore mechanism of action of mongersen (GED-0301) 160 mg once daily in patients with active Crohn's Disease

Condition Intervention Phase
Crohn's Disease Drug: GED-0301 Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2, Open-label Study to Explore the Pharmacodynamic and Clinical Effects of Mongersen (GED-0301) in Subjects With Active Crohn's Disease

Resource links provided by NLM:


Further study details as provided by Celgene:

Primary Outcome Measures:
  • Change from baseline of Smad7 (Mothers Against Decapentaplegic homolog 7) expression in the intestinal mucosa [ Time Frame: Baseline and Week 12 ]
    Smad7 is a protein important in cell interactions and stands for "Mothers against decapentaplegic homolog 7" protein


Secondary Outcome Measures:
  • Change from baseline in messenger ribonucleic acid (mRNA) expression of inflammatory cytokines [ Time Frame: Baseline and Week 12 ]
    Change from baseline in the mRNA expression of inflammatory cytokines such as, but not limited to, interleukin (IL)-10, IL-25, chemokine (C-C motif) ligand 20 (CCL20) and tumor necrosis factor alpha (TNF-α) in the intestinal mucosa at Week 12, from biopsy samples during ileocolonoscopy

  • The proportion of subjects achieving clinical remission, defined as a Crohn's Disease Activity Index (CDAI) score < 150, at Weeks 4, 8, and 12 [ Time Frame: Up to week 12 ]
    The CDAI is a measure of disease activity in patients with Crohn's Disease.

  • Change from baseline in the Simple Endoscopic Score for Crohn's Disease (SES-CD) at Week 12 and 52 [ Time Frame: Week 12 and week 52 ]
    The SES-CD is a measure that indicates mucosal inflammation in the end part of the ileum (or small intestine) and colon (or large intestine) in patients with Crohn's Disease

  • Number of Adverse Events [ Time Frame: Up to Week 56 ]
    The evaluation of safety and tolerability of GED-0301, assessed by the type, frequency and severity of adverse events, and its relationship to investigational product (IP), discontinuation due to adverse events, and clinically significant changes in electrocardiograms (ECGs), vital signs, and/or laboratory findings


Estimated Enrollment: 20
Actual Study Start Date: April 4, 2016
Estimated Study Completion Date: October 28, 2019
Estimated Primary Completion Date: January 26, 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: GED-0301 Induction (160mg) followed by intermittent 160 mg
GED-0301 160 mg "by mouth" (PO) daily (QD) for 12 weeks, followed by alternating GED 0301 160 mg QD for 4 weeks and no IP for 4 week, up to Week 100
Drug: GED-0301
During Induction period patient will receive mongersen 160 mg daily for 12 weeks. This study also offers subjects the option to continue with maintenance treatment at the discretion of the Investigator, beginning after the Week 12 Visit with alternating no IP for 4 weeks, followed by mongersen (GED-0301) 160 mg QD for 4 weeks, up through the Week 52 Visit during the Maintenance Period. At Week 52, subjects who have achieved an endoscopic improvement of >50% from baseline based on the SES-CD, as assessed by the central reader, and clinical improvement (HBI <7), will have the option to continue receiving treatment for an additional year, up through Year 2 (ie, the Week 100 Visit).
Other Name: Mongersen

Detailed Description:

Subjects will be screened to provide 20 enrolled subjects who complete 12 weeks of mongersen (GED-0301) 160 mg QD treatment as open-label therapy.

The study will consist of 4 periods:

  • Screening Period - up to 4 weeks
  • Induction Period - 12 weeks (Week 0 Visit through Week 12 Visit)
  • Maintenance Period - 88 weeks (after Week 12 Visit through Week 100 Visit)
  • Follow-up Period - 4 weeks (ie, no IP taken)

Subjects who prematurely discontinue treatment from this study prior to Week 100 will have an Early Termination Visit and also enter the 4-week Follow-up Period.

At the Screening Visit, all subjects who meet the entrance criteria will be eligible to enter the study. The number of subjects with previous exposure to Tumor Necrosis Factor-Alpha (TNF-α) blockers is targeted to be approximately 40% (ie, approximately 8 subjects).

  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female ≥ 18 years of age.
  • 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)
  • 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.
  • Subject must use protocol approved contraception

Exclusion Criteria:

  • Diagnosis of ulcerative colitis (UC), indeterminate colitis, ischemic colitis, microscopic colitis, radiation colitis or diverticular disease-associated colitis
  • Crohn's Disease (CD) manifestations such as abscesses, short bowel syndrome; or intestinal strictures with prestenotic dilatation, requiring procedural intervention or not passable with an adult colonoscope.
  • Intestinal resection within 6 months or any intra-abdominal surgery within 3 months prior to the Screening Visit
  • Ileostomy or a colostomy
  • Prior treatment with more than 2 TNF-α blockers (eg, infliximab or adalimumab).
  • Prior treatment with any integrin antagonists (eg, natalizumab or vedolizumab).
  • Subject is pregnant or breastfeeding.
  • Subject has received prior treatment with mongersen (GED-0301), or participation in a clinical study involving mongersen (GED-0301).
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02685683

Contacts
Contact: Associate Director Clinical Trial Disclosure 1-888-260-1599 clinicaltrialdisclosure@celgene.com

Locations
Italy
Fondazione IRCCS Policlinico San Matteo Recruiting
Pavia, Italy, 27100
Policlinico Tor Vergata Recruiting
Rome, Italy, 0133
Sponsors and Collaborators
Celgene
Investigators
Study Director: Keith Usiskin, MD Celgene
  More Information

Responsible Party: Celgene
ClinicalTrials.gov Identifier: NCT02685683     History of Changes
Other Study ID Numbers: GED-0301-CD-005
Study First Received: February 15, 2016
Last Updated: August 31, 2017

Keywords provided by Celgene:
Crohn's Disease
GED-0301
Open-label
Pharmacodynamic
Mongersen

Additional relevant MeSH terms:
Crohn Disease
Inflammatory Bowel Diseases
Gastroenteritis
Gastrointestinal Diseases
Digestive System Diseases
Intestinal Diseases

ClinicalTrials.gov processed this record on September 21, 2017