A long-term extension study to evaluate the drug mongersen (GED-0301) for the treatment of Crohn’s disease

• Register: EUCTR
• Last refreshed on: 29 January 2018
• Main ID: EUCTR2015-001963-37-HU
• Date of registration: 17/12/2015
• Prospective Registration: Yes
• Primary sponsor: Celgene Corporation
• Public title: A long-term extension study to evaluate the drug mongersen (GED-0301) for the treatment of Crohn’s disease
• Scientific title: A Phase 3, long-term active treatment extension study of mongersen (GED-0301) in subjects with Crohn’s disease - A long-term study to explore the safety of GED-0301 in Crohn's disease
• Date of first enrolment: 15/02/2016
• Target sample size: 1414
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-001963-37
• 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: no; Other: yes; Other specify the comparator: Different dose or regimen of the study treatment.; Number of treatment arms in the trial: 5
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

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

Contacts

• Name: ClinicalTrialDisclosure
• Affiliation: Celgene Corporation

• Name: ClinicalTrialDisclosure
• Affiliation: Celgene Corporation

Key inclusion & exclusion criteria

Inclusion criteria:
Inclusion Criteria for Adult Subjects:
1. Subject is a male or female = 18 years of age at the time of signing the informed consent form (ICF).
2. Subject must understand and voluntarily sign an ICF prior to any study-related assessments/procedures being conducted.
3. Subject is willing and able to adhere to the study visit schedule and other protocol requirements.
4. Subject must have completed through Week 12 in the previous GED-0301 study AND either:
- Completed participation through the last study treatment visit
at Week 52 in Study GED-0301-CD-002 or at Week 12 in Study GED-0301-CD-003
- Met the “early escape criteria” and were discontinued after Week 12 in Study GED-0301-CD-002.
5. Females of childbearing potential (FCBP) must have a negative pregnancy test at screening and enrollment (Visits 1 and 2). FCBP must either practice true abstinence from heterosexual contact or use none of the options while on IP and for at least 28 days after taking the last dose of IP.
Inclusion Criteria for Adolescent Subjects:
1. male or female, 12 to 17 years of age at the time of assent/informed consent in core GED-0301-CD-003 study and must affirmatively agree to participate in this study by signing an assent with a parent/legal guardian who can understand and voluntarily sign an ICF. Adolescent subjects who turn 18 by the screening visit for GED-0301-CD004 study must also understand and voluntarily sign an ICF prior to any study-related assessments/procedures being conducted.
2. able to swallow the IP tablets.
3. willing and able to adhere to study visit schedule and protocol requirements, and a parent or legal guardian willing to supervise adherence to protocol requirements.
4. must have completed through the Week 12 Visit in Study GED-0301CD-003.
5. Females of childbearing potential (FCBP)5 must have a negative pregnancy test at screening and enrollment (Visits 1 and 2). FCBP must either practice true abstinence from heterosexual contact or use one of the approved contraceptive options while on IP and for at least 28 days after taking the last dose of IP.
Are the trial subjects under 18? yes
Number of subjects for this age range: 100
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1243
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 71

Exclusion criteria:
1. Subject had experienced a serious adverse event related to the IP while participating in the core Phase 3 GED-0301 study.
2. Subject has any continuing serious medical condition, laboratory abnormality, or psychiatric illness that occurred while participating in the core Phase 3 GED-0301 study.
3. Subject has or had a flare or worsening of CD that, in the opinion of the Investigator, would not be in the best interest for the subject to participate in this long-term active treatment study.
4. Subject has initiated biologic agents, such as TNF-a blockers or integrin antagonists while, or after participating in the core Phase 3 GED-0301 study.
5. Subject diagnosed with colorectal cancer or confirmed diagnosis of colorectal dysplasia (with the exception of adenomatous colonic polyps that have been completely resected) while participating in the core Phase 3 GED-0301 study.
6. Newly diagnosed malignancy while participating in the previous Phase 3 GED-0301 study.
7. Subject is pregnant or breastfeeding.
8. Subject has been newly diagnosed with substance abuse.
9. New condition that may put subject at risk or confound the ability to interpret data from the study.
10. Known hypersensitivity to oligonucleotides, GED-0301 or any ingredient in the IP.


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

Health Condition(s) or Problem(s) studied

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

Active Crohn's disease
MedDRA version: 20.0 Level: LLT Classification code 10021315 Term: Ileitis terminal System Organ Class: 100000004856

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

Primary Outcome(s)

Primary end point(s): Safety of GED-0301, assessed by type, frequency and severity of adverse events, and its relationship to investigational product, discontinuation due to adverse events, and clinically significant changes in electrocardiograms (ECGs), vital signs, and/or laboratory findings

Secondary Objective: Secondary Objective (Adolescent Subjects):
- evaluate the efficacy of GED-0301 on clinical activity
- evaluate long-term endoscopic outcomes of GED-0301
- evaluate the long-term changes in linear growth in response to GED-0301

Main Objective: To evaluate the long-term safety of oral GED-0301 in subjects with Crohn’s disease

Timepoint(s) of evaluation of this end point: Through Week 208 and 4 weeks postdose

Secondary Outcome(s)

Secondary end point(s): Secondary endpoints are not included for adult subjects in this study.
Secondary endpoints for Adolescent subjects from GED-0301-CD-003:
- The proportion of subjects with clinical remission at Week 40.
- The proportion of subjects with endoscopic remission defined as SES-CD = 2 at Week 40.
- The proportion of subjects who have clinical remission, defined as a PCDAI = 10 points at Week 40.
- The change from baseline (GED-0301-CD-003) in weight, height, body mass index (BMI), and height velocity z-scores (adjusted for chronological age) at Week 40.

Timepoint(s) of evaluation of this end point: not applicable

Secondary ID(s)

GED-0301-CD-004

2015-001963-37-LV

Source(s) of Monetary Support

Celgene Corporation