An Efficacy and Safety Study of Mongersen (GED-0301) in Subjects With Active Ulcerative Colitis

NCT02601300 | Completed Phase 2 Results

• 1 other identifier: GED-0301-UC-002
• Study Type: interventional
• Target: 41
• Locations: 6 countries
• Sites: 36

Brief Summary

This is a phase 2, open-label, multicenter study to explore the efficacy and safety of oral GED- 0301 in subjects with active UC, defined as a modified Mayo score (MMS) ≥ 4 and ≤ 9 and a Mayo endoscopic subscore≥ 2. Approximately 40 subjects will be enrolled using an Interactive Voice Response System (IVRS) or an Interactive Web Response System (IWRS) to receive open-label, oral GED-0301 160 mg for duration of 52 week treatment. Enrollment of subjects with previous exposure to TNF-α blockers will be limited to approximately 15 subjects. The number of subjects with extensive colitis is targeted to comprise approximately 50% of the entire study population.

Conditions

Colitis, Ulcerative

Keywords

Ulcerative Colitis; GED-0301; Mongersen; Efficacy; Safety

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants Who Achieved Clinical Remission in the Modified Mayo Score (MMS) at Week 8

  Clinical remission was defined as a modified Mayo score of ≤ 2, with no individual subscore \> 1, at Week 8. The MMS was based on a modification of the total Mayo score (TMS) which included the stool frequency, rectal bleeding, and endoscopic subscores of the TMS and excluded the Physician's Global Assessment (PGA) subscore, since this was a global measure that is subjective in nature. The MMS ranges from 0 to 9 points with higher scores indicating greater disease severity. The endoscopy subscores was centrally reviewed. Two-sided confidence intervals for the within-group percentage were based on the Wilson score method.

  Baseline to Week 8

Secondary Outcomes (8)

• Percentage of Participants Who Achieved a Modified Mayo Score of ≤ 2, With Rectal Bleeding Subscore (RBS) of 0 and Stool Frequency Subscore (SFS) and Mayo Endoscopic Subscore ≤ 1 at Week 8

  Baseline to Week 8

• Percentage of Participants Who Achieved a Mayo Endoscopic Subscore of ≤ 1 at Week 8

  Baseline to Week 8

• Percentage of Participants Who Achieved a Mayo Endoscopic Subscore of ≤ 1 by Individual Segment at Week 8

  Baseline to Week 8

• Percentage of Participants Who Achieved a Clinical Response in the Modified Mayo Score at Week 8

  Baseline to Week 8

• Percentage of Participants Who Achieved a Mayo Endoscopic Response at Week 8

  Baseline and Week 8

Study Arms (1)

GED-0301 160 mg once daily (QD)

EXPERIMENTAL

Patients will receive oral GED-0301 160 mg once daily (QD)for duration of 52 week treatment.

Drug: GED-0301

Interventions

GED-0301 DRUG

Also known as: Mongersen

GED-0301 160 mg once daily (QD)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects must satisfy the following criteria to be enrolled in the study:
• Subject is ≥ 18 years of age at the time of signing the informed consent form (ICF).
• Subject is able to understand and voluntarily sign an informed consent form (ICF)prior to conducting any study related assessments/procedures.
• Subject is willing and able to adhere to the study visit schedule and other protocol requirements.
• Subject must have diagnosis of Ulcerative Colitis (UC) with a duration of at least 3 months prior to screening.
• Subject must have moderate to severe Ulcerative Colitis (UC), defined as Modified Mayo score (MMS) ≥ 4 to ≤ 9 with rectal bleeding subscore (RBS) ≥ 1 at screening.
• Subject must have a Mayo endoscopic subscore ≥ 2 at screening.
• Subject must have failed or experienced intolerance to at least one of the following:
• aminosalicylates; budesonide; systemic corticosteroids; immunosuppressants (eg,6-mercaptopurine (6-MP), or azathioprine (AZA)) or Tumor necrosis factor (TNF)-α blockers (eg, infliximab, adalimumab, or golimumab)
• Subject must meet the following laboratory criteria:
• White blood cell count ≥ 3000/mm3 (≥ 3.0 X 109/L)
• Platelet count ≥ 100,000/mm3 (≥ 100 X 109/L)
• Serum creatinine ≤ 1.5 mg/dL (≤ 132.6 μmol/L)
• Aspartate transaminase (AST/serum glutamic oxaloacetic transaminase (SGOT)) and alanine transaminase (ALT/serum pyruvic transaminase (SGPT)2.5 X upper limit of normal (ULN)
• Total bilirubin ≤ 2 mg/dL (≤ 34 μmol/L) unless there is a confirmed diagnosis of Gilbert's disease

You may not qualify if:

• The presence of any of the following will exclude a subject from enrollment:
• Subject has a diagnosis of Crohn's Disease (CD), indeterminate colitis, ischemic colitis, microscopic colitis, radiation colitis or diverticular disease-associated colitis.
• Subject has ulcerative colitis restricted to distal 15 cm or less (eg, ulcerative proctitis).
• Subject had surgery as a treatment for ulcerative colitis (UC)or who, in the opinion of the Investigator, is likely to require surgery for ulcerative colitis (UC) during the study.
• Subject has clinical signs suggestive of fulminant colitis or toxic megacolon.
• Subject is stool positive for any enteric pathogen or Clostridium difficile (C. difficile) toxin at screening.
• Subject has history of colorectal cancer or colorectal dysplasia.
• Prior treatment with more than 2 Tumor necrosis factor (TNF)-α blockers (eg, infliximab, adalimumab, or golimumab).
• Prior treatment with any integrin antagonists (eg, natalizumab or vedolizumab).
• Use of Tumor necrosis factor (TNF)-α blockers within 8 weeks of the screening.
• Subject had prior treatment with mycophenolic acid, tacrolimus, sirolimus, cyclosporine, thalidomide or apheresis (eg, Adacolumn®) for the treatment of ulcerative colitis (UC). In addition, prior use of any of these treatment modalities for an indication other than ulcerative colitis (UC) within 8 weeks of screening is also excluded.
• Subject has received intravenous (IV) corticosteroids within 2 weeks of screening.
• Subject has received topical treatment with 5 aminosalicylic acid (5-ASA) or corticosteroid enemas or suppositories within 2 weeks of screening.
• Subject has received total parenteral nutrition (TPN) within 4 weeks of screening.
• Subject has a 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 prevent the subject from participation in the study.

Sponsors & Collaborators

• Celgene: lead

Study Sites (36)

Macks Research Group

Newport Beach, California, 92660, United States

Location

Medical Associates Research Associates

San Diego, California, 92123, United States

Location

Florida Research Network, LLC

Gainesville, Florida, 32605, United States

Location

University of Miami School of Medicine

Miami, Florida, 33136, United States

Location

Gastroenterology Group of Naples

Naples, Florida, 34102, United States

Location

Shafran Gastroenterology Center

Winter Park, Florida, 32789, United States

Location

University of Kentucky

Lexington, Kentucky, 40536, United States

Location

Chevy Chase Clinical Research

Chevy Chase, Maryland, 20815, United States

Location

Dartmouth Hitchcock Medical Center

Lebanon, New Hampshire, 03756, United States

Location

Concorde Medical Group

New York, New York, 10016, United States

Location

Rochester General Hospital

Rochester, New York, 14621, United States

Location

Case Western Reserve University

Cleveland, Ohio, 44106, United States

Location

Gastroenterology Center of The Midsouth PC

Germantown, Tennessee, 38138, United States

Location

Nashville Gastrointestinal Specialists

Nashville, Tennessee, 37211, United States

Location

Vanderbilt University

Nashville, Tennessee, 37212, United States

Location

Texas Digestive Disease Consultants - Dallas

Dallas, Texas, 75231, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Texas Digestive Disease Consultants - Southlake

Southlake, Texas, 76092, United States

Location

McGuire Veterans Affairs Medical Center

Richmond, Virginia, 23249, United States

Location

Dean Medical Center

Madison, Wisconsin, 53715, United States

Location

Multiprofile Hospotal for Active Treatment- Sveti Nikolay Chudotvoretz - LOM EOOD

Lom, 3600, Bulgaria

Location

Multiprofile Hospital for Active Treatment Doverie AD

Sofia, 1632, Bulgaria

Location

Multiprofile Hospital for Active Treatment Sveti Panteleimon - Sofia AD

Sofia, 1712, Bulgaria

Location

GI Research Institute

Vancouver, British Columbia, V6Z 2K5, Canada

Location

London Health Sciences Centre, University Hospital

London, Ontario, N6A 5A5, Canada

Location

Toronto Digestive Disease Associates Inc

Vaughan, Ontario, L4L 4Y7, Canada

Location

Pandy Kalman Megyei Korhaz

Siófok, 8600, Hungary

Location

Centrum Medyczne sw. Lukasza

Częstochowa, 42-200, Poland

Location

Endoskopia Sp. z o.o.

Sopot, 81-756, Poland

Location

Centrum Zdrowia Matki, Dziecka i Mlodziezy

Warsaw, 00-632, Poland

Location

LexMedica Osrodek Badan Klinicznych

Wroclaw, 53-025, Poland

Location

Fakultna nemocnica s poliklinikou F. D. Roosevelta

Banská Bystrica, 975 17, Slovakia

Location

Univerzitna nemocnica Bratislava

Bratislava, 82606, Slovakia

Location

IBD Centrum s.r.o.

Bratislava, 83104, Slovakia

Location

KM Management, spol. s r.o.

Nitra, 949 01, Slovakia

Location

GASTRO I., s.r.o.

Prešov, 080 01, Slovakia

Location

MeSH Terms

Conditions

Colitis, Ulcerative

Interventions

GED0301

Condition Hierarchy (Ancestors)

Colitis; Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Inflammatory Bowel Diseases; Colonic Diseases; Intestinal Diseases

Results Point of Contact

• Title: Anne McClain, Senior Manager, Clinical Trial Disclosure
• Organization: Celgene Corporation

ClinicalTrialDisclosure@Celgene.com

888-260-1599

Study Officials

• Keith Usiskin, M.D

  Celgene Corporation

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 6, 2015
• First Posted: November 10, 2015
• Study Start: December 14, 2015
• Primary Completion: September 6, 2016
• Study Completion: August 8, 2017
• Last Updated: October 17, 2018
• Results First Posted: September 12, 2018

Record last verified: 2018-10

Locations

US (20); SL (5); BU (3); HU (1); PL (4); CA (3)