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

NCT03523117 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: 1VIT17044
• Study Type: interventional
• Target: 79
• Locations: 1 country
• Sites: 16

Brief 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.

Conditions

Iron Deficiency Anemia

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Change in Ferritin µg/L From Baseline to Day 35

  Baseline to day 35

• Change in TSAT (%) From Baseline to Day 35

  Baseline to day 35

• Change in Reticulocyte Hemoglobin (Picograms) Content From Baseline to Day 35

  Baseline to day 35

Study Arms (2)

Ferric Caroboxymaltose

ACTIVE COMPARATOR

Ferric Carboxymaltose - 2 doses (day 0 and day 7) at 15 mg/kg to a maximum single dose of 750 mg (whichever is smaller) up to a maximum of total dose of 1500 mg administered as either an undiluted IV push at a rate of 100 mg (2mL)/minute OR in no more than 250 mL of normal saline and infused over 15 minutes.

Drug: Ferric carboxymaltose

Oral Ferrous Sulfate

ACTIVE COMPARATOR

Oral Ferrous Sulfate - will receive an age-dependent formulation of oral ferrous sulfate daily for 28 days as follows: participants \<12 years of age will receive 6 mg (elemental iron)/kg/day divided into 2 daily doses of an oral liquid formulation, either drops or elixir, and participants ≥12 will receive 2 daily doses of oral tablets. Infants and children (ages 1 to \<4 years) will receive oral ferrous sulfate drops, while children (ages ≥4 to \<12 years) will receive oral ferrous sulfate elixir. Adolescents (ages ≥12 to 17 years) will receive an oral ferrous sulfate tablet (65 mg of elemental iron/tablet/dose) twice a day (BID). The maximum daily dose for all participants is 130 mg of elemental iron.

Drug: Ferrous Sulfate

Interventions

Ferric carboxymaltose DRUG

Intravenous iron

Also known as: Injectafer, Ferinject

Ferric Caroboxymaltose

Ferrous Sulfate DRUG

oral iron therapy

Also known as: oral iron

Oral Ferrous Sulfate

Eligibility Criteria

• Age: 1 Year - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.
• Screening Hgb \<11 g/dL.
• Screening ferritin ≤300 ng/mL and transferrin saturation (TSAT) \<30%.
• 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.
• 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.
• Participants undergoing treatment for inflammatory bowel disease (IBD) must be on stable therapy for at least 8 weeks prior to consent.

You may not qualify if:

• Known history of hypersensitivity reaction to any component of FCM.
• Previous randomization and treatment in this study or any other clinical study of FCM or VIT-45.
• History of acquired iron overload, hemochromatosis, or other iron accumulation disorders.
• Chronic kidney disease participants on hemodialysis.
• 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.
• Any existing non-viral infection.
• Known history of positive hepatitis B antigen (HBsAg) or hepatitis C viral antibody (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.
• Intravenous iron and /or blood transfusion in the 4 weeks prior to consent.
• 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 female 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

Sponsors & Collaborators

• American Regent, Inc.: lead

Study Sites (16)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

International Research Partners, Inc.

Doral, Florida, 33122, United States

Location

ProHealth Research Center

Doral, Florida, 33166, United States

Location

Garden Medical Research, Inc.

Miami, Florida, 33155, United States

Location

Miami Clinical Research

Miami, Florida, 33155, United States

Location

South Florida Research Phase I-IV

Miami Springs, Florida, 33166, United States

Location

Riley Hospital for Children,Room 4340

Indianapolis, Indiana, 46202, United States

Location

Caro Health Plaza

Caro, Michigan, 48723, United States

Location

Galen Research

Chesterfield, Missouri, 63005, United States

Location

Tiga Pediatrics, PC

The Bronx, New York, 10467, United States

Location

Cincinnati Children's Hospital and Medical Center

Cincinnati, Ohio, 45229, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Cook Children's Medical Center

Fort Worth, Texas, 76101, United States

Location

Baylor College of Medicine/Texas Children Hospital

Houston, Texas, 77030, United States

Location

Tekton Research

San Antonio, Texas, 78240, United States

Location

Aspen Clinical Research

Orem, Utah, 84058, United States

Location

MeSH Terms

Conditions

Anemia, Iron-Deficiency

Interventions

ferric carboxymaltose; ferrous sulfate; Iron

Condition Hierarchy (Ancestors)

Anemia, Hypochromic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Iron Deficiencies; Iron Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Metals, Heavy; Elements; Inorganic Chemicals; Transition Elements; Metals

Results Point of Contact

• Title: Mark A. Falone, MD
• Organization: American Regent, Inc.

mfalone@americanregent.com

631.772.3544

Study Officials

• Mark Falone

  American Regent, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 17, 2018
• First Posted: May 14, 2018
• Study Start: January 31, 2019
• Primary Completion: December 22, 2020
• Study Completion: January 29, 2021
• Last Updated: June 22, 2022
• Results First Posted: June 22, 2022

Record last verified: 2022-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (16)