E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
E.1.1.1 | Medical condition in easily understood language |
Crohn’s disease (an Inflammatory Bowel Disease) |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Digestive System Diseases [C06] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10021315 |
E.1.2 | Term | Ileitis terminal |
E.1.2 | System Organ Class | 100000004856 |
|
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the long-term safety of oral GED-0301 in subjects with Crohn’s disease |
|
E.2.2 | Secondary objectives of the trial |
Secondary objectives are not included in this study. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
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 OR 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). While on IP and for at least 28 days after taking the last dose of IP, FCBP who engage in activity in which conception is possible must use one of the approved contraceptive options
6. Male subjects when engaging in sexual activity with females who are able to become pregnant must use barrier contraception while on IP and for at least 28 days after the last dose. |
|
E.4 | Principal exclusion criteria |
1. Subject had experienced a serious adverse event related to the IP while participating in the previous Phase 3 GED-0301 study.
2. Subject has any continuing serious medical condition, laboratory abnormality, or psychiatric illness that occurred while participating in the previous 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-α blockers or integrin antagonists.
5. Subject has been diagnosed with colorectal cancer or colorectal dysplasia (with the exception of adenomatous colonic polyps that have been completely resected) while participating in the previous Phase 3 GED-0301 study.
6. Subject has a 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. Subject has developed a known hypersensitivity to oligonucleotides, GED-0301 or any ingredient in the IP. |
|
E.5 End points |
E.5.1 | 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 |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Through Week 208 and 4 weeks postdose |
|
E.5.2 | Secondary end point(s) |
Secondary endpoints are not included in this study. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | Yes |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 5 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 311 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Australia |
Austria |
Belgium |
Bulgaria |
Canada |
Croatia |
Czech Republic |
Denmark |
Estonia |
France |
Germany |
Greece |
Hungary |
Israel |
Italy |
Korea, Republic of |
Latvia |
Netherlands |
New Zealand |
Norway |
Poland |
Romania |
Russian Federation |
Serbia |
Slovakia |
South Africa |
Spain |
Sweden |
Switzerland |
Turkey |
Ukraine |
United Kingdom |
United States |
|
E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
End of Study is defined as either the date of the LVLS to complete the post-treatment follow-up, or the date of receipt of the last data point from the LS that is required for primary, secondary and/or exploratory analysis, as prespecified in the protocol, whichever is the later date. |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 4 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 3 |
E.8.9.2 | In all countries concerned by the trial days | 0 |