Back to resultsEfficacy and Safety of Intravenous Ferric Carboxymaltose in Pediatric Patients With Iron Deficiency Anemia and Unsatisfactory Response Oral Iron Under Study Protocol 1VIT17044
Primary Purpose
Iron Deficiency Anemia
Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Ferric carboxymaltose
Sponsored by
About this trial
This is an interventional treatment trial for Iron Deficiency Anemia
Eligibility Criteria
Inclusion Criteria:
- Unsatisfactory response to oral iron or those that required a concomitant intervention, (defined as, blood transfusion, use of IV or oral iron outside of protocol, increase in erythropoietin for any reason [Day 0 thru Day 35 of study protocol 1VIT17044], change in IBD treatment).
- Hgb <11 g/dL
- Ferritin ≤300 ng/mL and TSAT <30%
Exclusion Criteria:
- Known history of hypersensitivity reaction to any component of FCM.
- History of acquired iron overload, hemochromatosis, or other iron accumulation disorders.
- History of significant diseases of the liver, hematopoietic system, cardiovascular system, psychiatric disorder, or other conditions which, in the opinion of the investigator, may place a subject at added risk for participation in the study.
- Any existing non-viral infection.
- Known history of positive HBsAg or HCV with evidence of active hepatitis.
- Known history of positive HIV-1/HIV-2 antibodies (anti-HIV).
- Anemia due to reasons other than iron deficiency (e.g., hemoglobinopathy and vitamin B12 or folic acid deficiency) that has not been corrected.
- Administration and / or use of an investigational product (drug or device) within 30 days of screening.
- Alcohol or drug abuse within the past six months.
- Female participant who is pregnant or lactating, or sexually active females who are of childbearing potential not willing to use an acceptable form of contraceptive precautions during the study.
- Unable to comply with study procedures and assessments.
Sites / Locations
- South Florida Research Phase I-IV
- Miami Clinical Research
Arms of the Study
Arm 1
Arm Type
Other
Arm Label
IV Iron
Arm Description
The primary objective in this study is to allow participants who were randomized to receive oral iron in the 1VIT17044 trial and who had an unsatisfactory response to oral iron or those that required a concomitant intervention, (defined as, blood transfusion, use of IV or oral iron outside of protocol, increase in erythropoietin for any reason [Day 0 thru Day 35 of study protocol 1VIT17044], change in IBD treatment) to receive one course of FCM. This course of FCM will consist of two doses of FCM at 15 mg/kg (maximum single dose of 750 mg), separated by seven days.
Outcomes
Primary Outcome Measures
Change in Hemoglobin From Baseline to Day 35
The change in hemoglobin from Baseline to Day 35 was assessed using paired t-tests (two-sided test, α = 0.05). Baseline hemoglobin was defined as the last hemoglobin obtained before the first dose.
Secondary Outcome Measures
Full Information
NCT ID
NCT04269707
First Posted
February 12, 2020
Last Updated
March 17, 2022
Sponsor
American Regent, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT04269707
Brief Title
Efficacy and Safety of Intravenous Ferric Carboxymaltose in Pediatric Patients With Iron Deficiency Anemia and Unsatisfactory Response Oral Iron Under Study Protocol 1VIT17044
Official Title
Evaluating the Efficacy and Safety of Intravenous Ferric Carboxymaltose in Pediatric Patients With Iron Deficiency Anemia and an Unsatisfactory Response to Oral Iron Under Study Protocol 1VIT17044
Study Type
Interventional
2. Study Status
Record Verification Date
March 2022
Overall Recruitment Status
Completed
Study Start Date
November 19, 2019 (Actual)
Primary Completion Date
December 30, 2020 (Actual)
Study Completion Date
January 29, 2021 (Actual)
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
No
5. Study Description
Brief Summary
Evaluating the Efficacy and Safety of Intravenous Ferric Carboxymaltose in Pediatric Patients with Iron Deficiency Anemia and an Unsatisfactory Response to Oral Iron under Study Protocol 1VIT17044
Detailed Description
This is a single arm, open-label, multi-center, multi-national, non-randomized study that will evaluate the efficacy and safety of a one course treatment with FCM in participants who had an unsatisfactory response to oral iron in study 1VIT17044.
Subjects will have an End of Study (EOS) Visit (Day 35) from the 1VIT17044 study to qualify for entry. Subjects will then be screened, meet all inclusion criteria, no exclusion criteria and have a baseline evaluation. Based on the successful completion of the baseline evaluations, subjects will be enrolled and will receive the first dose of FCM (Day 0). The second FCM dose will occur 7 days from the first dose. Subjects will return to the clinic 14 and 28 days post their first dose for laboratory and safety assessments, and at 35 days post the first dose, participants will return to the clinic for final safety and efficacy assessments. Once all assessments are complete the participant will exit the study.
An unsatisfactory response to oral iron (i.e., an increase in hemoglobin (Hgb) of <1 g/dL from baseline) will be defined as non-responders to oral iron treatment. Non-responders who continue to meet all inclusion criteria (including Hgb <11 g/dL) and none of the exclusion criteria will receive 1 course of FCM.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Iron Deficiency Anemia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Model Description
This is a single arm, open-label, multi-center, multi-national, non-randomized study that will evaluate the efficacy and safety of a one course treatment with FCM in participants who had an unsatisfactory response to oral iron in study 1VIT17044.
Masking
None (Open Label)
Allocation
N/A
Enrollment
7 (Actual)
8. Arms, Groups, and Interventions
Arm Title
IV Iron
Arm Type
Other
Arm Description
The primary objective in this study is to allow participants who were randomized to receive oral iron in the 1VIT17044 trial and who had an unsatisfactory response to oral iron or those that required a concomitant intervention, (defined as, blood transfusion, use of IV or oral iron outside of protocol, increase in erythropoietin for any reason [Day 0 thru Day 35 of study protocol 1VIT17044], change in IBD treatment) to receive one course of FCM. This course of FCM will consist of two doses of FCM at 15 mg/kg (maximum single dose of 750 mg), separated by seven days.
Intervention Type
Drug
Intervention Name(s)
Ferric carboxymaltose
Intervention Description
IV iron
Primary Outcome Measure Information:
Title
Change in Hemoglobin From Baseline to Day 35
Description
The change in hemoglobin from Baseline to Day 35 was assessed using paired t-tests (two-sided test, α = 0.05). Baseline hemoglobin was defined as the last hemoglobin obtained before the first dose.
Time Frame
The change in Hgb from baseline to day 35
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Unsatisfactory response to oral iron or those that required a concomitant intervention, (defined as, blood transfusion, use of IV or oral iron outside of protocol, increase in erythropoietin for any reason [Day 0 thru Day 35 of study protocol 1VIT17044], change in IBD treatment).
Hgb <11 g/dL
Ferritin ≤300 ng/mL and TSAT <30%
Exclusion Criteria:
Known history of hypersensitivity reaction to any component of FCM.
History of acquired iron overload, hemochromatosis, or other iron accumulation disorders.
History of significant diseases of the liver, hematopoietic system, cardiovascular system, psychiatric disorder, or other conditions which, in the opinion of the investigator, may place a subject at added risk for participation in the study.
Any existing non-viral infection.
Known history of positive HBsAg or HCV with evidence of active hepatitis.
Known history of positive HIV-1/HIV-2 antibodies (anti-HIV).
Anemia due to reasons other than iron deficiency (e.g., hemoglobinopathy and vitamin B12 or folic acid deficiency) that has not been corrected.
Administration and / or use of an investigational product (drug or device) within 30 days of screening.
Alcohol or drug abuse within the past six months.
Female participant who is pregnant or lactating, or sexually active females who are of childbearing potential not willing to use an acceptable form of contraceptive precautions during the study.
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, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
South Florida Research Phase I-IV
City
Miami Springs
State/Province
Florida
ZIP/Postal Code
33166
Country
United States
Facility Name
Miami Clinical Research
City
Miami
State/Province
Florida
ZIP/Postal Code
33155
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Efficacy and Safety of Intravenous Ferric Carboxymaltose in Pediatric Patients With Iron Deficiency Anemia and Unsatisfactory Response Oral Iron Under Study Protocol 1VIT17044
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