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 |
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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 | 10045365 |
E.1.2 | Term | Ulcerative colitis |
E.1.2 | System Organ Class | 100000016670 |
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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 |
The objective of the study is to evaluate the long-term safety, tolerability, and maintenance of clinical response, of repeated administration of adalimumab in pediatric subjects with ulcerative colitis who participated in, and successfully completed, Protocol M11-290 through Week 52. |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
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E.3 | Principal inclusion criteria |
1. Subject must have successfully enrolled in and completed Protocol M11-290 through Week 52.
2. Subject of legal age, and/or parent or legal guardian, as required, has voluntarily signed and dated an Institutional Review Board (IRB)/Independent Ethics Committee (IEC) approved informed consent form, after the nature of the study has been explained and the subject of legal age, and/or Subject's parent, or legal guardian, as required, has had the opportunity to ask questions. The informed consent must be signed before any study-specific procedures are performed. Subjects will be included in all discussions, and if required, their signature on an assent form will be obtained.
Diagnosis and Main Criteria for Inclusion/Exclusion (Continued):
Main Inclusion (Continued):
3. Parent or legal guardian of subject who is not of legal age, as required, must be willing to actively supervise storage and administration of study drug and to ensure that the time of each dose is accurately recorded in the subject's electronic diary.
If a subject is of a legal age, subjects must be able and willing to self-administer SC injections or have a qualified person available to administer SC injections and to ensure that the time of each dose is accurately recorded in the subject's electronic diary.
4. If female, subject who is either not of childbearing potential, defined as pre-menstrual, or is of childbearing potential and is practicing an approved method of birth control throughout the study and for 150 days after last dose of the study drug.
Examples of approved methods of birth control include the following (see local informed consent for more detail):
• Total abstinence from sexual intercourse;
• Condoms, sponge, foam, jellies, diaphragm or intrauterine device (IUD);
• Hormonal contraceptives for 90 days prior to study drug administration;
• A vasectomized partner.
5. Subject is judged to be in good medical condition, as determined by the Principal Investigator based upon results of clinical and laboratory evaluations done throughout the preceding ulcerative colitis Study M11-290.
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E.4 | Principal exclusion criteria |
1. Subject is considered by the Investigator, for any reason, to be an unsuitable candidate for continuing therapy in the Study M10-870.
2. Female subjects who are pregnant or currently breastfeeding or considering becoming pregnant during the study.
3. Subject with Crohn's disease (CD) or indeterminate colitis (IC).
4. History of clinically significant drug or alcohol abuse in the last 12 months.
5. History of demyelinating disease (including myelitis) or neurologic symptoms suggestive of demyelinating disease.
6. History of chronic or active hepatitis B infection, human immunodeficiency virus (HIV) infection, any immunodeficiency syndrome, or active TB (regardless of receiving treatment).), or active viral infection that, based on the investigator’s clinical assessment, makes the subject an unsuitable candidate for the study.
7. History of moderate to severe congestive heart failure (NYHA class III or IV), recent cerebrovascular accident and any other condition which would put the subject at risk by participation in the study.
8. History of malignancy (including lymphoma and leukemia) other than a successfully treated non metastatic cutaneous squamous cell or basal cell carcinoma or localized carcinoma in situ of the cervix.
9. Subject who is planning surgical bowel resection at any time point while enrolled in the study.
10. Known hypersensitivity to adalimumab or its excipients.
11. Current diagnosis of fulminant colitis and /or toxic megacolon.
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E.5 End points |
E.5.1 | Primary end point(s) |
This study will utilize the PMS (and Mayo score if available) and PUCAI to determine efficacy of the study drug |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84 and 96, 108, 120, 144, 168, 192, 216, 240, 264 and 288/Premature Discontinuation |
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E.5.2 | Secondary end point(s) |
Safety analyses will be performed on all subjects who receive at least one dose of study drug. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84 and 96, 108, 120, 132, 144, 156, 168, 180, 192, 204, 216, 228, 240, 252, 264, 276 and 288/Premature Discontinuation |
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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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
Open label extension study |
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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 | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 24 |
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 |
Canada |
European Union |
Israel |
Japan |
New Zealand |
United States |
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E.8.7 | Trial has a data monitoring committee | No |
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
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The end of study is defined as the date of the Last Subject Last Visit, or the last follow-up contact, whichever is longer. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 5 |
E.8.9.1 | In the Member State concerned months | 8 |
E.8.9.1 | In the Member State concerned days | 21 |
E.8.9.2 | In all countries concerned by the trial years | 5 |
E.8.9.2 | In all countries concerned by the trial months | 8 |
E.8.9.2 | In all countries concerned by the trial days | 21 |