Dose-escalation Trial of the Safety, Pharmacokinetics, and Pharmacodynamics of Iron Isomaltoside (Monofer®)

Study Overview
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Brief Summary
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The trial is an open-label, 4 cohorts, sequential, dose-escalating, single dose trial.

Detailed Description
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IDA is highly prevalent in subjects with cancer and gastrointestinal diseases such as inflammatory bowel diseases, 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 treatment of these subjects includes controlling the bleeding and replenishing lost iron.

Official Title
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Phase I Dose-escalation Trial of the Safety, Pharmacokinetics, and Pharmacodynamics of Iron Isomaltoside (Monofer®) Administered as Single Bolus Injections or Infusions in Japanese Subjects With Iron Deficiency Anemia

Conditions
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Anemia, Deficiency Diseases, Anemia, Iron Deficiency, Hematologic Disease, Iron Metabolism Disorders

Intervention / Treatment
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* Drug: Iron Isomaltoside


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Japanese man or woman ≥ 20 years, < 65 years of age Hb of ≥ 9.0 g/dL, < 12.0 g/dL for women and < 13.0 g/dL for men Serum ferritin < 25 ng/mL TIBC ≥ 360 μg/dL Body weight ≥ 50 kg Willingness to participate and signing the informed consent form Exclusion criteria include: Anemia caused by conditions other than iron deficiency Cancer IV or oral iron treatment, or blood transfusion 4 weeks prior to screening Erythropoiesis stimulating agent (ESA) treatment prior to screening Imminent expectation of blood transfusion on part of treating physician Iron overload or disturbances (e.g. hemochromatosis and hemo-siderosis) Known hypersensitivity reaction to iv iron preparations Decompensated liver cirrhosis or active hepatitis Active acute or chronic infections Pregnant or nursing women. Planned elective surgery during the trial

Ages Eligible for Study
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Minimum Age: 20 Years
Maximum Age: 64 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: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: cohort 1 iron isomaltoside<br>treated with first dose level of iron isomaltoside | Drug: Iron Isomaltoside<br>* The trial is a dose escalating trial.<br>* Other names: Monofer;|
| Experimental: cohort 2 iron isomaltoside<br>treated with second dose level of iron isomaltoside | Drug: Iron Isomaltoside<br>* The trial is a dose escalating trial.<br>* Other names: Monofer;|
| Experimental: cohort 3 iron isomaltoside<br>treated with third dose level of iron isomaltoside | Drug: Iron Isomaltoside<br>* The trial is a dose escalating trial.<br>* Other names: Monofer;|
| Experimental: cohort 4 iron isomaltoside<br>treated with fourth dose level of iron isomaltoside | Drug: Iron Isomaltoside<br>* The trial is a dose escalating trial.<br>* Other names: Monofer;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of adverse events | Proportion of adverse events will be collected and evaluated for relatedness, severity, seriousness, and expectedness. | 1 week | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Maximum plasma drug concentration [Cmax] |  | 1 week | 
| Area Under the Curve [AUC] |  | 1 week | 
| Time to reach one-half of the maximum drug concentration [T1/2] |  | 1 week | 
| Time to reach maximum drug concentration [Tmax] |  | 1week | 
| Change in concentration of hemoglobin (g/dL) |  | 1 week | 
| Change in concentration of serum ferritin (ng/mL) |  | 1 week | 
| Change in concentration of total iron binding capacity (μg/dL ) |  | 1 week | 
| Change in concentrations of transferrin saturation (%) |  | 1 week |