E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
iron deficiency in chronic inflammatory bowel disease |
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E.1.1.1 | Medical condition in easily understood language |
iron deficiency in chronic inflammatory bowel disease |
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E.1.1.2 | Therapeutic area | Diseases [C] - Digestive System Diseases [C06] |
MedDRA Classification |
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 efficacy of iron in comparison to placebo in reducing platelet activity as measured by platelet aggregation |
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E.2.2 | Secondary objectives of the trial |
• To evaluate the effect of iron in comparison to placebo on markers of platelet activation and platelets counts
• To evaluate the efficacy of iron in comparison to placebo in normalizing iron deficiency
• The evaluate the effect of iron in comparison to placebo on megakaryocytic growth factors
• To evaluate the change of disease activity
• To evaluate the safety of this treatment
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Male or female, inpatient or outpatient, aged at least 18 years and not more than 60 years.
2. Transferrin saturation (TfS) <16% or ferritin < 100g/l
3. Previously diagnosed inflammatory bowel disease (Crohn’s disease or ulcerative colitis)
4. Females of child-bearing potential must have a negative urine pregnancy test at screening and be practicing an acceptable method of birth control during the study and for up to 1month after the last dose of the study medication. Acceptable methods of birth control include barrier methods (including male and female condoms), diaphragms (cervical caps) with intravaginal spermicide (including jellies, foams and suppositories), intra-uterine devices or hormonal contraceptives. Non-childbearing potential includes being surgically sterilised at least 6 months prior to the study or postmenopausal with no menstrual bleeding
5. Demonstrate the ability to understand the requirements of the study, provide written informed consent, abide by the study restrictions, and agree to undergo the required assessments.
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E.4 | Principal exclusion criteria |
1. Harvey Bradshaw Index >7 (10) , Mayo Score >5 (11)
2. Significant anemia (hemoglobin <9 g/dl)
3. Bodyweight < 50kg
4. Blood transfusions or iron therapy during the previous 4 weeks, or erythropoietin treatment within the 8 weeks prior to enrolment.
5. Concomitant therapy with prednisolon above 20mg/d, 6-mercaptopurine, infliximab or azathioprine must have been initiated at least 4 months prior to study and the dose must be stable for at least 8 weeks. Other drugs with known effects on megakaryopoiesis (e.g. interferon-alpha).
6. Severe concomitant disease or need for surgery within 8 weeks
7. Hemochromatosis or other iron-storage disorders
8. Treatment with an investigational drug within the 30 days prior to enrolment
9. Active severe infection or malignancy other than carcinoma in situ of the cervix and non-melanoma skin cancer.
10. Bone Marrow Disease (MDS, thalassemia, etc)
11. Active or chronic liver or kidney disease. Serum albumin <25 g/L or serum creatinine >20 mg/L
12. Significant cardiovascular disease, including myocardial infarction within 12 months prior to study inclusion, congestive heart failure NYHA (New York Heart Association) grade III or IV, or poorly controlled hypertension according to the judgment of the investigator. Known hypersensitivity to FERINJECT®
13. Positive for HIV 1/HIV 2 antibodies (anti HIV) (HIV: human immunodeficiency virus).
14. Positive for hepatitis B surface-antigen (HBsAg), hepatitis C virus antibody (anti HCV) and evidence for active hepatitis, i.e., abnormal liver function test (LFT) results.
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary efficacy endpoint is the change in platelet aggregation as measured by platelet aggregometry. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
• Markes of platelet activity (p-selectin expression on platelets, p-selectin in serum, sCD40L) and platelet counts.
• Change in megakaryocytic growth factors such as thrombopoietin and Interleukin-3
• Change in iron parameters (ferritin, hemoglobin, transferrin, transferrin saturation, soluble transferrin-receptor, hepcidin)
• Change in disease activity (Harvey-Bradshaw index and Mayoscore without endoscopy)
• Change in inflammation parameters such as C-reactive protein, ESR, IL-6 and IL-11 and calprotectin.
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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 | Yes |
E.6.4 | Safety | No |
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | Yes |
E.8.1.4 | Double blind | No |
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 | Yes |
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
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |