Safety, Tolerability, PK and PD of Intravenous Ferric Carboxymaltose in Infants With Iron Deficiency Anemia

Phase 2

Withdrawn

• Conditions: Iron Deficiency, Anaemia
• Interventions: Drug: Ferric carboxymaltose

• Registration Number: NCT04968379
• Lead Sponsor: American Regent, Inc.

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).

Study Timeline

• Study First Submitted: 2020-11-02
• Study First Submitted QC: 2021-07-08
• Study First Posted: 2021-07-20
• Study Start: 2022-07-21
• Status Verified: 2023-01
• Last Update Submitted: 2023-01-05
• Last Update Posted: 2023-01-06
• Primary Completion: 2024-10-08
• Study Completion: 2024-12-12

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

1. 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).

2. Screening Hb ≥7 g/dL to <10 g/dL.

3. 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

1. Known history of hypersensitivity reaction to FCM.
2. Body weight <2.5 kg.
3. History of acquired iron overload, hemochromatosis, or other iron accumulation disorders.
4. Hemodialysis-dependent chronic kidney disease.
5. 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.
6. Active infection.
7. Anemia due to reasons other than iron deficiency (e.g., hemoglobinopathy vitamin B12 deficiency, or folic acid deficiency).
8. Blood transfusion in the 4 weeks prior to consent.
9. Administration of an iron-containing product within 14 days of administration of the study article.
10. Administration and / or use of an investigational product (drug or device) within 30 days of screening.
11. Current participation in another clinical trial.
12. Unable to comply with study procedures and assessments.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Ferric Carboxymaltose	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
Injectafer	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 7.5 mg/kg dose dose of FCM.

Primary Outcome Measures

Name	Time	Method
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.
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
Treatment-emergent adverse events	Baseline to Day 36	Treatment-emergent clinical laboratory test (clinical chemistry and hematology) abnormalities
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.

Trial Locations

Locations (4)

St. Christopher's Hospital for Children; 🇺🇸 Philadelphia, Pennsylvania, United States

University of Iowa; 🇺🇸 Iowa City, Iowa, United States

Cohen Children's Medical Center; 🇺🇸 New Hyde Park, New York, United States

Nationwide Children's Hospital; 🇺🇸 Columbus, Ohio, United States