E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Anemia in children with Inflammatory Bowel Disease (IBD) |
Anemie bij kinderen met inflammatoire darmziekten |
|
E.1.1.1 | Medical condition in easily understood language |
Anemia in children with chronic inflamed intestines |
Bloedarmoede bij kinderen met chronisch ontstoken darmen |
|
E.1.1.2 | Therapeutic area | Body processes [G] - Digestive System and Oral Physiological Phenomena [G10] |
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 |
The goal of this multi center, randomized controlled trial is to determine effectiveness of IV administration of ferric carboxymaltose to patients with CU/CD with regard to improvement of laboratory markers of the iron metabolism and improvement of quality of life compared to oral iron supplementation. Primary objective: the elevation of hemoglobin with 1.25 mmol/l after administration of IV ferric carboxymaltose therapy. Secondary objectives: QoL measurements, measurement of clinical and biochemical disease activity according to PCDAI and PUCAI scoring systems (see 1.5.4), laboratory markers to measure replenishment of iron stores, side effects on liver functioning and electrolyte homeostasis. |
|
E.2.2 | Secondary objectives of the trial |
Secondary objectives: QoL measurements, measurement of clinical and biochemical disease activity according to PCDAI and PUCAI scoring systems (see 1.5.4), laboratory markers to measure replenishment of iron stores, side effects on liver functioning and electrolyte homeostasis. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Children attending a pediatrician/ pediatric gastro- enterologist 2. Suffering from CD/CU (diagnosed according to Porto criteria) 3. Anemia according to WHO criteria 4. Written informed consent of parent with authority. 5. Children aged 0 – 18 years 6. 6.Ability to understand and speak Dutch language |
|
E.4 | Principal exclusion criteria |
1. Allergic reactions to intravenous iron therapy 2. Previous administration of IV iron therapy 3. Known hemochromatosis |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Primary outcome variables in the study are: • Elevation of Hb with 1.25 mmol/L three months after administration of IV ferric carboxymaltose therapy in comparison to the Hb level at inclusion in the study |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
After 4 weeks, after 3 and after 6 months |
|
E.5.2 | Secondary end point(s) |
Secondary outcome variables in the study are: • QoL will be measured at baseline (T=0), 4 weeks (T=1), 3 months (T=2) and 6 months (T=3) after the start of the intervention. • Clinical disease activity according to PCDAI and PUCAI scoring system at T=0, T=2, T=3 • Laboratory markers for: o Effectiveness of IV iron therapy in replenishment of iron stores: Ht, cell indices, thrombocytes, MCV, ferritin, TIBC, serum iron level, transferrin saturation, reticulocyte count, sTfR, CRP, transferrin receptors to log ferritin(TfR-F ratio), transferine/log ferritine ratio, hepcidin o Side effects of IV iron therapy on liver: AST, ALT, AF, total protein, albumin o Side effects on electrolyte homeostasis: phosphate |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
After 4 weeks, after 3 and 6 months |
|
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 | No |
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) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
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 | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Yes |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
|
E.8.2.4 | Number of treatment arms in the trial | 2 |
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 | 4 |
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
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |