Safety, Tolerability, PK and PD of Intravenous Ferric Carboxymaltose in Infants With Iron Deficiency Anemia
Study Details
Study Description
Brief Summary
An Open-Label, Multi-Center Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intravenous Ferric Carboxymaltose (FCM) in Infants (0-1 year) with Iron Deficiency Anemia.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
Detailed Description
A phase II, open-label, multi-center study with 2 Cohorts to evaluate the safety, tolerance, PK, and PD profile of intravenous (IV) FCM in infants 0 to 1 year of age with IDA after receiving either a 5.0 mg/kg or 7.5 mg/kg dose of FCM.
Participants will have a screening evaluation within 14 days of the first dose of study drug. A medically supervised environment is required on Day 1 (day of dosing) and for 4 hours post dosing. Participants are allowed to be enrolled if satisfying the inclusion and exclusion criteria. Participants will return to the study site for additional evaluation and sampling on Days 8 (± 2 days), 15 (± 2 days), 22 (± 2 days), and 36 (± 2 days).
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Ferric Carboxymaltose To evaluate the safety, tolerance, PK and PD profile of intravenous (IV) FCM in infants 0 to 1 year of age with IDA after receiving a 5.0 mg/kg dose of FCM |
Drug: Ferric carboxymaltose
Intravenous
Other Names:
|
| Experimental: Injectafer To evaluate the safety, tolerance, PK and PD profile of intravenous (IV) FCM in infants 0 to 1 year of age with IDA after receiving a 7.5 mg/kg dose dose of FCM. |
Drug: Ferric carboxymaltose
Intravenous
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Treatment-emergent adverse events [Baseline to Day 36]
Treatment-emergent clinical laboratory test (clinical chemistry and hematology) abnormalities
- Change in hemoglobin (Hb): g/dL [baseline to Days 8, 15, 22, and 36]
determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters
- Change in reticulocytes count: % [baseline to Days 8, 15, 22, and 36]
determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters
- Evaluate the PD parameters - Change in serum iron: mcg/dL [baseline to Day 36]
To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.
- Evaluate the PD parameters - Change in serum ferritin: ng/mL [baseline to Day 36]
Description: To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.
- Evaluate the PD parameters - Change in total iron binding capacity [TIBC]): mcg/dL [baseline to Day 36]
Description: To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.
- Evaluate the PD parameters - Change in serum transferrin saturation [TSAT]): mg/dL [baseline to Day 36]
Description: To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Male and female participants 0 to 1 year of age, medically indicated for iron replacement, with his/her parent or legal guardian willing and able to sign the informed consent form approved by the IRB / Independent Ethics Committee (IEC).
-
Screening Hb ≥7 g/dL to <10 g/dL.
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Infants with any of the following conditions:
-
Heart failure with IDA defined as syndromes of excessive preload, excessive afterload, abnormal rhythm, or decreased contractility
-
Gastrointestinal diseases with acquired short bowel syndrome (due to volvulus, necrotizing enterocolitis from surgical resection or spontaneous intestinal perforation)
-
Gastrointestinal intolerance of oral iron or an unsatisfactory response to oral iron
-
Other conditions associated with IDA which in the opinion of the investigator might benefit from administration of FCM
Exclusion Criteria:
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Known history of hypersensitivity reaction to FCM.
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Body weight <2.5 kg.
-
History of acquired iron overload, hemochromatosis, or other iron accumulation disorders.
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Hemodialysis-dependent chronic kidney disease.
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History of significant diseases of the liver, hematopoietic system, cardiovascular system, or other conditions which, on the opinion of the investigator, may place a participant at added risk for participation in the study.
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Active infection.
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Anemia due to reasons other than iron deficiency (e.g., hemoglobinopathy vitamin B12 deficiency, or folic acid deficiency).
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Blood transfusion in the 4 weeks prior to consent.
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Administration of an iron-containing product within 14 days of administration of the study article.
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Administration and / or use of an investigational product (drug or device) within 30 days of screening.
-
Current participation in another clinical trial.
-
Unable to comply with study procedures and assessments.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | University of Iowa | Iowa City | Iowa | United States | 52242 |
| 2 | Cohen Children's Medical Center | New Hyde Park | New York | United States | 11040 |
| 3 | Nationwide Children's Hospital | Columbus | Ohio | United States | 43205 |
| 4 | St. Christopher's Hospital for Children | Philadelphia | Pennsylvania | United States | 19134 |
Sponsors and Collaborators
- American Regent, Inc.
Investigators
- Study Director: Mark Falone, MD, American Regent
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 1VIT19046