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
Overview
- Phase
- Phase 2
- Intervention
- Ferric carboxymaltose
- Conditions
- Iron Deficiency, Anaemia
- Sponsor
- American Regent, Inc.
- Locations
- 4
- Primary Endpoint
- Evaluate the PD parameters - Change in serum transferrin saturation [TSAT]): mg/dL
- Status
- Withdrawn
- Last Updated
- 3 years ago
Overview
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.
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).
Investigators
Eligibility 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.
- •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
- •Known history of hypersensitivity reaction to FCM.
- •Body weight \<2.5 kg.
- •History of acquired iron overload, hemochromatosis, or other iron accumulation disorders.
- •Hemodialysis-dependent chronic kidney disease.
- •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.
- •Active infection.
- •Anemia due to reasons other than iron deficiency (e.g., hemoglobinopathy vitamin B12 deficiency, or folic acid deficiency).
- •Blood transfusion in the 4 weeks prior to consent.
- •Administration of an iron-containing product within 14 days of administration of the study article.
- •Administration and / or use of an investigational product (drug or device) within 30 days of screening.
Arms & Interventions
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
Intervention: Ferric carboxymaltose
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.
Intervention: Ferric carboxymaltose
Outcomes
Primary Outcomes
Evaluate the PD parameters - Change in serum transferrin saturation [TSAT]): mg/dL
Time Frame: baseline to Day 36
Description: To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.
Change in reticulocytes count: %
Time Frame: 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 hemoglobin (Hb): g/dL
Time Frame: 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
Treatment-emergent adverse events
Time Frame: Baseline to Day 36
Treatment-emergent clinical laboratory test (clinical chemistry and hematology) abnormalities
Evaluate the PD parameters - Change in serum iron: mcg/dL
Time Frame: 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
Time Frame: 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
Time Frame: baseline to Day 36
Description: To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.