Multicenter Randomized Active-controlled Study to Investigate Efficacy & Safety of IV FCM in Pediatric Patients With IDA

Iron Deficiency Anemia Clinical Trial

Official title:

A Multicenter, Randomized, Active-Controlled Study to Investigate the Efficacy and Safety of Intravenous Ferric Carboxymaltose in Pediatric Patients With Iron Deficiency Anemia

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03523117
• Other study ID #: 1VIT17044
• Status: Completed
• Phase: Phase 3
• Start date: January 31, 2019
• Est. completion date: January 29, 2021

Study information

• Verified date: May 2022
• Source: American Regent, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The primary objective of this study is to demonstrate the efficacy and safety of intravenous ferric carboxymaltose (FCM), compared to oral iron, in pediatric participants who have iron deficiency anemia.

Description:

This is a Phase III, multicenter, randomized, active-controlled study that compares the efficacy and safety of FCM to oral iron in pediatric participants with IDA and a documented history of an inadequate response to oral iron therapy at least 8 weeks (56 days) prior to randomization.

Participants who satisfy the inclusion requirements and no exclusion criteria will be eligible to participate in this study and enter into a screening phase to confirm eligibility. Randomization will occur via the Interactive Response Technology (IRT) system in a 1:1 ratio to either Group A, participants receiving FCM, or Group B, participants receiving oral iron (oral solution drops, elixir or oral tablets). Randomization will be stratified by baseline Hgb (<10, ≥10 g/dL) and age (1 to <12 years and ≥12 to 17 years).

The oral ferrous sulfate formulation received will be based on the participant's age, such that infants and children (1 to <4 years of age) will receive ferrous sulfate drops, children (≥4 to <12 years of age) will receive ferrous sulfate elixir, and adolescents (≥12 to 17 years of age) will receive ferrous sulfate tablets. Participants who experience adverse clinical symptoms due to the oral iron during the treatment phase may have a weight-based dose of ferrous sulfate reduced from 6 mg/kg to 3 mg/kg. If the participant is receiving tablets, the dose will be reduced from one tablet taken twice daily to one tablet per day.

Once randomized, all participants will return for efficacy and safety evaluations, including adverse events and laboratory assessments, on Days 7, 14, 28, and 35. Additional pharmacokinetic sampling and analyses will be performed for participants receiving FCM on Days 0 and 7.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 79
• Est. completion date: January 29, 2021
• Est. primary completion date: December 22, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 17 Years

Eligibility

Inclusion Criteria:

1. Male or female participants 1 to 17 years of age with assent to participation and his/her parent or guardian is willing and able to sign the informed consent approved by the Independent Review Board / Ethics Committee.

2. Screening Hgb <11 g/dL.

3. Screening ferritin =300 ng/mL and transferrin saturation (TSAT) <30%.

4. Participants must have a documented history of an inadequate response to any oral iron therapy for at least 8 weeks (56 days) prior to randomization.

5. For participants who are receiving an erythropoietin stimulating agent (ESA): stable ESA therapy (+/- 20% of current dose) for at least 8 weeks prior to the qualifying screening visit and no ESA dosing or product changes anticipated for the length of the trial.

6. Participants undergoing treatment for inflammatory bowel disease (IBD) must be on stable therapy for at least 8 weeks prior to consent.

Exclusion Criteria:

1. Known history of hypersensitivity reaction to any component of FCM.

2. Previous randomization and treatment in this study or any other clinical study of FCM or VIT-45.

3. History of acquired iron overload, hemochromatosis, or other iron accumulation disorders.

4. Chronic kidney disease participants on hemodialysis.

5. History of significant diseases of the liver, hematopoietic system, cardiovascular system, psychiatric disorder, or other conditions which, on the opinion of the investigator, may place a subject at added risk for participation in the study.

6. Any existing non-viral infection.

7. Known history of positive hepatitis B antigen (HBsAg) or hepatitis C viral antibody (HCV) with evidence of active hepatitis.

8. Known history of positive HIV-1/HIV-2 antibodies (anti-HIV).

9. Anemia due to reasons other than iron deficiency (e.g., hemoglobinopathy and vitamin B12 or folic acid deficiency) that has not been corrected.

10. Intravenous iron and /or blood transfusion in the 4 weeks prior to consent.

11. Administration and / or use of an investigational product (drug or device) within 30 days of screening.

12. Alcohol or drug abuse within the past six months.

13. Female participant who is pregnant or lactating, or sexually active female who are of childbearing potential not willing to use an acceptable form of contraceptive precautions during the study.

14. Unable to comply with study procedures and assessments

Related Conditions & MeSH terms

• Anemia
• Anemia, Iron-Deficiency
• Iron Deficiency Anemia

Intervention

Drug:
• Ferric carboxymaltose
Intravenous iron
• Ferrous Sulfate
oral iron therapy

Locations

Country	Name	City	State
United States	Tiga Pediatrics, PC	Bronx	New York
United States	Caro Health Plaza	Caro	Michigan
United States	Galen Research	Chesterfield	Missouri
United States	Cincinnati Children's Hospital and Medical Center	Cincinnati	Ohio
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	International Research Partners, Inc.	Doral	Florida
United States	ProHealth Research Center	Doral	Florida
United States	Cook Children's Medical Center	Fort Worth	Texas
United States	Baylor College of Medicine/Texas Children Hospital	Houston	Texas
United States	Riley Hospital for Children,Room 4340	Indianapolis	Indiana
United States	Arkansas Children's Hospital	Little Rock	Arkansas
United States	Garden Medical Research, Inc.	Miami	Florida
United States	Miami Clinical Research	Miami	Florida
United States	South Florida Research Phase I-IV	Miami Springs	Florida
United States	Aspen Clinical Research	Orem	Utah
United States	Tekton Research	San Antonio	Texas

Sponsors (1)

Lead Sponsor	Collaborator
American Regent, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in Hemoglobin g/dL	Change in hemoglobin g/dL from baseline to day 35 will be analyzed using parametric analysis of covariance (ANCOVA). The model will include terms for the randomization strata (hemoglobin and age categories), baseline hemoglobin, as well as treatment group. Baseline hemoglobin will be defined as the last hemoglobin obtained before randomization.	Baseline to day 35
Secondary	Change in Ferritin µg/L From Baseline to Day 35	Change in ferritin µg/L from baseline to day 35 was analyzed using parametric analysis of covariance (ANCOVA). The model included terms for the randomization strata (hemoglobin and age categories), baseline ferritin as a covariate.	Baseline to day 35
Secondary	Change in TSAT (%) From Baseline to Day 35	Change in TSAT (%) from baseline to day 35 was analyzed using parametric analysis of covariance (ANCOVA). The model included terms for the randomization strata (hemoglobin and age categories), baseline ferritin as a covariate	Baseline to day 35
Secondary	Change in Reticulocyte Hemoglobin (Picograms) Content From Baseline to Day 35	Change in reticulocyte hemoglobin (picograms) content from baseline to day 35 was analyzed using a mixed model repeated. The model included terms for the randomization strata (hemoglobin and age categories), baseline ferritin as a reticulocyte hemoglobin content.	Baseline to day 35