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

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

The trial is an open-label, 4 cohorts, sequential, dose-escalating, single dose trial.

Study Overview

• Status: Completed
• Conditions: Anemia; Iron Metabolism Disorders; Deficiency Diseases; Anemia, Iron Deficiency; Hematologic Disease
• Intervention / Treatment: Drug: Iron Isomaltoside

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Actual): 24
• Phase: Phase 1

Contacts and Locations

Study Locations

• Japan
  • Tokyo, Japan, 171-0014
    • Ikebukuro

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 20 years to 64 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Japanese man or woman ≥ 20 years, < 65 years of age
2. Hb of ≥ 9.0 g/dL, < 12.0 g/dL for women and < 13.0 g/dL for men
3. Serum ferritin < 25 ng/mL
4. TIBC ≥ 360 μg/dL
5. Body weight ≥ 50 kg
6. Willingness to participate and signing the informed consent form

Exclusion criteria include:

1. Anemia caused by conditions other than iron deficiency
2. Cancer
3. IV or oral iron treatment, or blood transfusion 4 weeks prior to screening
4. Erythropoiesis stimulating agent (ESA) treatment prior to screening
5. Imminent expectation of blood transfusion on part of treating physician
6. Iron overload or disturbances (e.g. hemochromatosis and hemo-siderosis)
7. Known hypersensitivity reaction to iv iron preparations
8. Decompensated liver cirrhosis or active hepatitis
9. Active acute or chronic infections
10. Pregnant or nursing women.
11. Planned elective surgery during the trial

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

4

Arms and Interventions

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

What is the study measuring?

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	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

Collaborators and Investigators

• Sponsor: Pharmacosmos A/S

Study record dates

Study Major Dates

• Study Start (Actual): January 6, 2017
• Primary Completion (Actual): June 4, 2017
• Study Completion (Actual): June 4, 2017

Study Registration Dates

• First Submitted: December 22, 2016
• First Submitted That Met QC Criteria: January 5, 2017
• First Posted (Estimate): January 6, 2017

Study Record Updates

• Last Update Posted (Actual): December 12, 2017
• Last Update Submitted That Met QC Criteria: December 8, 2017
• Last Verified: January 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nutrition Disorders; Anemia, Hypochromic; Malnutrition; Anemia, Iron-Deficiency; Hematologic Diseases; Anemia; Metabolic Diseases; Deficiency Diseases; Iron Metabolism Disorders; Hematinics; Iron isomaltoside 1000
• Other Study ID Numbers: P-Monofer-PK-IDA-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No