E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Anaemic subjects with lymphoid malignancies and functional iron deficiency receiving chemotherapy |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10025631 |
E.1.2 | Term | Malignant lymphoid neoplasm NOS |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10022970 |
E.1.2 | Term | Iron deficiency |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10002034 |
E.1.2 | Term | Anaemia |
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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 |
Evaluation of efficacy of Ferric carboxymaltose (FCM) given without erythropoiesis stimulating agents (ESA) in the correction of haemoglobin (Hb) levels in anaemic subjects with lymphoproliferative disorder (LPD), undergoing chemotherapy. |
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E.2.2 | Secondary objectives of the trial |
• Describe safety and tolerability of FCM. • Describe the effect of treatment with FCM on iron status variables in LPD subjects. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Subjects (male or female) aged ≥18, suffering from indolent non-Hodgkin’s lymphoma, multiple myeloma or chronic lymphocytic leukaemia on any chemotherapy excluding anthracycline containing.
2. Life expectancy at least 6 months.
3. Received at least 12 weeks (or 3 cycles) of treatment in the current course of chemotherapy before start of iron therapy.
4. 8.5 g/dL ≤Hb ≤10.5 g/dL at time of randomisation.
5. Iron-restricted erythropoiesis as defined: • Stainable iron in bone marrow combined with transferrin saturation (TSAT) ≤20% OR • where the evaluation of stainable iron in bone marrow is not possible or available: - ferritin >30 ng/mL (women) or >40 ng/mL (men) and - TSAT ≤20%
6. Signed informed consent (before any study procedure).
7. Females of child-bearing potential must have a negative urine pregnancy test. |
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E.4 | Principal exclusion criteria |
1. Any anaemia treatment within 4 weeks before inclusion (including red blood cell transfusion, ESA treatment and any oral/parenteral iron supplementation).
2. Subjects weighing <35 kg.
3. Subjects with increase in Hb during the chemotherapy (>1 g/dL rise between initiation of CT and screening laboratory value).
4. Folate deficiency (serum folate <4.5 nmol/L) and/or vitamin B12 deficiency (serum cobalamin <145 pmol/L).
5. Ongoing haemolysis defined as serum haptoglobin <0.2 g/L.
6. Recent significant bleeding/surgery.
7. Monotherapy with immunotherapy agents.
8. Known chronic renal failure, creatinine >125 μmol/L.
9. Anthracycline containing chemotherapy regimens.
10. Clinically relevant active inflammatory disease other than the malignant disease (according to the judgement of the Investigator).
11. Clinically relevant ongoing infectious disease including known human immunodeficiency virus.
12. Serum-ferritin >800 ng/mL.
13. Ongoing significant neurological or psychiatric disorders including psychotic disorders or dementia.
14. Significant cardiovascular disease prior to study inclusion including myocardial infarction within 12 months prior to study inclusion, congestive heart failure New York Heart Association (NYHA) Grade III or IV, or poorly controlled hypertension according to the judgment of the Investigator.
15. Elevation of liver enzymes (aspartate aminotransferase, alanine aminotransferase) over 3 times above the normal range or known acute hepatic disorder.
16. Subject currently is enrolled in or has not yet completed at least 30 days since ending other investigational device or drug study(ies), or subject is receiving other investigational agent(s).
17. Females who are evidently pregnant (e.g., positive HCG test) or are breast feeding.
18. Subject is not using adequate contraceptive precautions. Adequate contraceptive precautions are defined as those which result in a low failure rate (i.e., less than 1% per year) when used consistently and correctly such as implants, injectables, combined oral contraceptives, some intra-uterine devices, sexual abstinence or vasectomised partner. Non-childbearing potential includes being surgically sterilised at least 6 months prior to the study or post-menopausal, defined as amenorrhea for at least 12 months.
19. Subject has known sensitivity to any of the products to be administered during dosing.
20. Subject will not be available for follow-up assessment.
21. Subject has any kind of disorder that compromises the ability of the subject to give written informed consent and/or to comply with study procedures. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Mean change in Hb from baseline to Weeks 4, 6 and 8 (end of treatment) in the absence of any red cell transfusion or ESA treatment. |
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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 | 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 | 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 | 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 |
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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 | 5 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 15 |
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 | Information not present in EudraCT |
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
|
Last visit of the last patient |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
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 | 1 |
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 |