A Study to Evaluate Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects

Iron Deficiency Anemia Clinical Trial

Official title:

A Phase 3, Randomized, Open-Label, Multicenter Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03893045
• Other study ID #: AMAG-FER-IDA-352
• Status: Recruiting
• Phase: Phase 3
• Start date: September 18, 2019
• Est. completion date: October 2024

Study information

• Verified date: July 2023
• Source: AMAG Pharmaceuticals, Inc.
• Contact: Clinical Trial Interest
• Phone: 1-877-374 -4177
• Email: CTInterest[@]covispharma.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a Phase 3, randomized, open-label, multicenter, study in male and female pediatric subjects (2 years to <18 years of age) with IDA, or felt by their clinician to be at risk of developing IDA. This study allows for enrollment of subjects with IDA regardless of etiology, except for CKD subjects (pediatric CKD subjects are being studied in a separate ferumoxytol protocol).

Description:

Subjects will be randomized to treatment in a 2:1 ratio (ferumoxytol: iron sucrose) and stratified by age group (2 to <6 years; 6 to <12 years; and 12 to <18 years). Subjects will receive one of the following treatment regimens: • Ferumoxytol: 7 mg Fe/kg IV (maximum 510 mg/dose) x 2 doses, the first dose administered on Day 1 and the second 2 to 8 days later. OR • Iron sucrose (Venofer®): 4 mg Fe/kg IV (maximum 200 mg/dose) x 5 doses, the first dose on Day 1 and subsequent doses administered at least once per week and up to 3 times/week. All subjects will be monitored at the study site through at least 1 hour after the completion of each infusion of study drug. Assessment of blood Hgb concentrations, adverse events, and other safety assessments will be performed through study Week 5.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 75
• Est. completion date: October 2024
• Est. primary completion date: July 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 17 Years

Eligibility

Inclusion Criteria:

1. Male or female 2 years to <18 years of age at time of consent

2. Has IDA defined as:

1. Hemoglobin (Hgb) <11.0 g/dL AND

2. Any one or more of the following:

• Transferrin saturation (TSAT) <20%
• ferritin <100 ng/mL

3. Documented history of unsatisfactory oral iron therapy or in whom oral iron cannot be tolerated, or for whom oral iron is considered medically inappropriate

Exclusion Criteria:

1. Known hypersensitivity reaction to any component of ferumoxytol or iron sucrose

2. History of allergy to intravenous (IV) iron

3. History of =2 clinically significant drug allergies

4. Subjects with CKD (defined as eGFR of <60 mL/min/1.73 m2 or a requirement for chronic hemodialysis or peritoneal dialysis during Screening)

5. Low systolic blood pressure (BP) (age 1 to 9 years <70 + [age in years x 2] mmHg, age 10 to 17 years <90 mmHg)

6. Hgb =7.0 g/dL

7. Serum ferritin level >600 ng/mL

Related Conditions & MeSH terms

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

Intervention

Drug:
• ferumoxytol
Each 20 mL single-use vial contains 17 mL of ferumoxytol that consists of iron at a concentration of 30 mg Fe/mL, coated with polyglucose sorbitol carboxymethylether and formulated with mannitol, at a concentration of 44 mg/mL, in a black to reddish brown sterile, aqueous, colloidal, isotonic solution
• Iron sucrose
Each mL contains 20 mg of elemental iron as iron sucrose in water for injection. The 5 mL single-use vial contains 100 mg of iron per 5 mL. The drug product contains approximately 30% sucrose (300 mg/mL).

Locations

Country	Name	City	State
Lithuania	JSC Saules seimos medicinos centras	Kaunas
Lithuania	Klaipeda Children's Hospital	Klaipeda
Lithuania	Children's Hospital-Affiliate of Vilnius University Hospital Santariskiu Klinikos	Vilnius
Poland	Osrodek Badan Klinicznych In Vivo sp. z o.o.	Bydgoszcz
Poland	Prywatny Gabinet Lekarski Dr N. med. Jerzy Brzostek	Debica
Poland	Pro Familia Altera Sp. z o.o.	Katowice
Poland	Korczowski Bartosz, Gabinet Lekarski	Rzeszów
Poland	Centrum Zdrowia MDM	Warsaw
Poland	Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu	Wroclaw
United States	Gwinnett Research Institute	Buford	Georgia
United States	University of Florida	Gainesville	Florida
United States	Arkansas Children's Hospital	Little Rock	Arkansas
United States	Biomedical Research LLC	Miami	Florida
United States	Optimus U Corporation	Miami	Florida
United States	Sun Research Institute	San Antonio	Texas

Sponsors (1)

Lead Sponsor	Collaborator
AMAG Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Lithuania,  Poland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Area Under the Curve (AUC)	Pharmacokinetic parameter: Area Under the Curve (AUC)	35 days
Other	Clearance	Pharmacokinetic parameter: Clearance	35 days
Other	Distribution	Pharmacokinetic parameter: Distribution	35 days
Other	Elimination half-lives	Pharmacokinetic parameter: Elimination half-lives	35 days
Primary	Change in Hemoglobin from Baseline to Week 5	Proportion of subjects achieving a change in hemoglobin from Baseline to Week 5	35 days
Secondary	Incidence of Treatment Emergent Adverse Events	Incidence of Treatment Emergent Adverse Events	49 days
Secondary	Incidence of adverse events of special interest (AESI)	Incidence of adverse events of special interest (AESI) (hypotension and hypersensitivity)	49 days