IDA-01 A Randomised, Open-Label, Comparative Study of Intravenous Iron Isomaltoside 1000 (Monofer®) and Iron Sucrose

Study Overview
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Brief Summary
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The purpose of the trial is to evaluate and compare the effect of iron isomaltoside 1000 to iron sucrose in its ability to increase haemoglobin (Hb) in subjects with IDA when oral iron preparations are ineffective or cannot be used or where there is a clinical need to deliver iron rapidly.

Detailed Description
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IDA is highly prevalent in subjects with gastrointestinal diseases and cancer, menstruating or pregnant women, and subjects who have undergone bariatric procedure. IDA can have a substantial medical and quality of life (QoL) burden on the subjects and the treatment of these subjects includes controlling the bleeding and replenishing lost iron. Oral iron administration is often used in the clinical practice at many clinics; however, oral iron may not be tolerated by all subjects. Hence, there is a need for an alternative iron treatment in subjects, who do not tolerate oral iron. This study is planned to compare the efficacy and safety of iron isomaltoside 1000 with another parenteral iron preparation (iron sucrose) in subjects with IDA and who are intolerant or unresponsive to oral iron therapy or who need iron rapidly.

Official Title
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A Phase III, Randomised, Open-Label, Comparative Study of Intravenous Iron Isomaltoside 1000 (Monofer®) and Iron Sucrose in Subjects With Iron Deficiency Anaemia and Who Are Intolerant or Unresponsive to Oral Iron Therapy or Who Need Iron Rapidly (PROVIDE)

Conditions
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Iron Deficiency Anaemia

Intervention / Treatment
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* Drug: iron isomaltoside 1000 (Monofer®)
* Drug: iron sucrose (Venofer®)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Men or women > 18 years having IDA caused by different aetiologies such as abnormal uterine bleeding, gastrointestinal diseases, cancer, bariatric procedures (gastric bypass operations), and other conditions leading to significant blood loss and with a documented history of intolerance or unresponsiveness to oral iron therapy for at least one month prior to study enrollment or where there at investigators judgment is a clinical need to deliver iron rapidly Hb < 11 g/dL TSAT < 20 % S-ferritin < 100 ng/mL Willingness to participate and signing the informed consent form Exclusion Criteria: Anaemia predominantly caused by factors other than IDA (e.g. anaemia with untreated vitamin B12 or folate deficiency, haemolytic anaemia) Iron overload or disturbances in utilisation of iron (e.g. haemochromatosis and haemosiderosis) Decompensated liver cirrhosis or active hepatitis (ALAT > 3 times upper limit of normal) Active acute or chronic infections (assessed by clinical judgement supplied with white blood cells (WBC) and C-reactive protein (CRP)) Body weight < 50 kg Rheumatoid arthritis with symptoms or signs of active inflammation Pregnant or nursing women. In order to avoid pregnancy, women have to be surgically sterile or use adequate contraception (e.g. intrauterine devices, hormonal contraceptives, or double barrier method) during the whole study period and after the study has ended for at least 5 times plasma biological half-life of the investigational medicinal product History of multiple allergies Known hypersensitivity to parenteral iron or any excipients in the investigational drug products Erythropoietin treatment within 8 weeks prior to the screening visit Other iron treatment or blood transfusion within 4 weeks prior to the screening visit Planned elective surgery during the study Participation in any other clinical study within 3 months prior to the screening Any other medical condition that, in the opinion of Investigator, may cause the subject to be unsuitable for the completion of the study or place the subject at potential risk from being in the study, e.g. uncontrolled hypertension, unstable ischaemic heart disease, or uncontrolled diabetes mellitus

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: iron isomaltoside 1000 (Monofer®)<br>iron isomaltoside 1000 (Monofer®) | Drug: iron isomaltoside 1000 (Monofer®)<br> <br> |
| Active Comparator: iron sucrose (Venofer®)<br>iron sucrose (Venofer®) | Drug: iron sucrose (Venofer®)<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Subjects With an Haemoglobin (Hb) Increase of ≥ 2 g/dL From Baseline at Any Time From Week 1 to Week 5 | The primary efficacy endpoint of the trial was the count of subjects with an Hb increase of ≥ 2 g/dL from baseline at any time from week 1 to week 5. 'Any time' implied that if the endpoint was met at a time-point prior to or at week 5, the effect (increase of ≥ 2 g/dL) did not have to be maintained throughout the trial in order for a subject to be a responder. Number of responders (i.e. a subject with increase in Hb ≥ 2 g/dL from baseline at any time from week 1 to week 5) and percentages according to number of subjects in the analysis set were summarised. | From baseline to week 5 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Hb Concentration |  | From baseline to week 2, 4 and 5 | 
| Change in Serum (s)-Ferritin Concentration |  | From baseline to week 1, 2, 4, and 5 | 
| Change in Transferrin Saturation (TSAT) |  | From baseline to week 1, 2, 4, and 5 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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IDA, Iron deficiency, iron deficiency anaemia and who are intolerant or unresponsive to oral iron therapy