E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
Crohn’s disease (an Inflammatory Bowel Disease) |
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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 | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10021315 |
E.1.2 | Term | Ileitis terminal |
E.1.2 | System Organ Class | 100000004856 |
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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 |
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E.2.2 | Secondary objectives of the trial |
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 |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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. |
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E.4 | Principal 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-α 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. |
|
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 |
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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 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. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | Yes |
E.8.1.6 | Cross over | No |
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 | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
Different dose or regimen of the study treatment. |
|
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
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 18 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 371 |
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 |
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 |
Romania |
Russian Federation |
Serbia |
Slovakia |
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 |