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 |
|
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10025631 |
E.1.2 | Term | Malignant lymphoid neoplasm NOS |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10002034 |
E.1.2 | Term | Anaemia |
E.1.2 | System Organ Class | 10005329 - Blood and lymphatic system disorders |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10022970 |
E.1.2 | Term | Iron deficiency |
E.1.2 | System Organ Class | 10027433 - Metabolism and nutrition disorders |
|
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. |
|
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. |
|
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. |
|
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. |
|
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. |
|
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) | 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 | 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 |
|
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 | 1 |
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 |