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

Primary Purpose

Iron Deficiency, Anaemia

Status

Withdrawn

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

Ferric carboxymaltose

About this trial

This is an interventional treatment trial for Iron Deficiency, Anaemia

Eligibility Criteria

undefined - 1 Year (Child)All SexesDoes not accept healthy volunteers

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.
Current participation in another clinical trial.
Unable to comply with study procedures and assessments.

Sites / Locations

University of Iowa
Cohen Children's Medical Center
Nationwide Children's Hospital
St. Christopher's Hospital for Children

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

Ferric Carboxymaltose

Injectafer

Arm Description

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

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.

Outcomes

Primary Outcome Measures

Treatment-emergent adverse events

Treatment-emergent clinical laboratory test (clinical chemistry and hematology) abnormalities

Change in hemoglobin (Hb): g/dL

determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters

Change in reticulocytes count: %

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

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

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

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

Description: To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.

Secondary Outcome Measures

Full Information

NCT ID

NCT04968379

First Posted

November 2, 2020

Last Updated

January 5, 2023

Sponsor

American Regent, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT04968379

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

January 2023

Overall Recruitment Status

Withdrawn

Why Stopped

Difficult to recruit and enroll patients for this study in a reasonable amount of time.

Study Start Date

July 21, 2022 (Actual)

Primary Completion Date

October 8, 2024 (Anticipated)

Study Completion Date

December 12, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

American Regent, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. 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.

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Iron Deficiency, Anaemia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

0 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Ferric Carboxymaltose

Arm Type

Experimental

Arm Description

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

Arm Title

Injectafer

Arm Type

Experimental

Arm Description

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 Type

Drug

Intervention Name(s)

Ferric carboxymaltose

Other Intervention Name(s)

Injectafer

Intervention Description

Intravenous

Primary Outcome Measure Information:

Title

Treatment-emergent adverse events

Description

Treatment-emergent clinical laboratory test (clinical chemistry and hematology) abnormalities

Time Frame

Baseline to Day 36

Title

Change in hemoglobin (Hb): g/dL

Description

determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters

Time Frame

baseline to Days 8, 15, 22, and 36

Title

Change in reticulocytes count: %

Description

determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters

Time Frame

baseline to Days 8, 15, 22, and 36

Title

Evaluate the PD parameters - Change in serum iron: mcg/dL

Description

To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.

Time Frame

baseline to Day 36

Title

Evaluate the PD parameters - Change in serum ferritin: ng/mL

Description

Description: To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.

Time Frame

baseline to Day 36

Title

Evaluate the PD parameters - Change in total iron binding capacity [TIBC]): mcg/dL

Description

Description: To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.

Time Frame

baseline to Day 36

Title

Evaluate the PD parameters - Change in serum transferrin saturation [TSAT]): mg/dL

Description

Description: To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.

Time Frame

baseline to Day 36

10. Eligibility

Sex

All

Maximum Age & Unit of Time

1 Year

Accepts Healthy Volunteers

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. Current participation in another clinical trial. Unable to comply with study procedures and assessments.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Mark Falone, MD

Organizational Affiliation

American Regent

Official's Role

Study Director

Facility Information:

Facility Name

University of Iowa

City

Iowa City

State/Province

Iowa

ZIP/Postal Code

52242

Country

United States

Facility Name

Cohen Children's Medical Center

City

New Hyde Park

State/Province

New York

ZIP/Postal Code

11040

Country

United States

Facility Name

Nationwide Children's Hospital

City

Columbus

State/Province

Ohio

ZIP/Postal Code

43205

Country

United States

Facility Name

St. Christopher's Hospital for Children

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19134

Country

United States

12. IPD Sharing Statement

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

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