Clinical trial NCT03893045: A Study to Evaluate Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Iron Deficiency Anemia
Keywords: IDA
anemia
pediatrics
ferumoxytol
iron sucrose
Lead sponsor: AMAG Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2019-03-28
Enrollment: 75
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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).

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: Change in Hemoglobin from Baseline to Week 5
timeFrame: 35 days
Secondary outcomes:
measure: Incidence of Treatment Emergent Adverse Events
timeFrame: 49 days
measure: Incidence of adverse events of special interest (AESI)
timeFrame: 49 days
Other outcomes:
measure: Area Under the Curve (AUC)
timeFrame: 35 days
measure: Clearance
timeFrame: 35 days
measure: Distribution
timeFrame: 35 days
measure: Elimination half-lives
timeFrame: 35 days
Locations:
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Miami
country: United States
facility: Optimus U Corporation
state: Florida
city: Miami
country: United States
facility: Biomedical Research LLC
state: Florida
city: Buford
country: United States
facility: Gwinnett Research Institute
state: Georgia
city: San Antonio
country: United States
facility: Sun Research Institute
state: Texas
city: Kaunas
country: Lithuania
facility: JSC Saules seimos medicinos centras
city: Klaipėda
country: Lithuania
facility: Klaipeda Children's Hospital
city: Vilnius
country: Lithuania
facility: Children's Hospital-Affiliate of Vilnius University Hospital Santariskiu Klinikos
city: Bydgoszcz
country: Poland
facility: Osrodek Badan Klinicznych In Vivo sp. z o.o.
city: Debica
country: Poland
facility: Prywatny Gabinet Lekarski Dr N. med. Jerzy Brzostek
city: Katowice
country: Poland
facility: Pro Familia Altera Sp. z o.o.
city: Rzeszów
country: Poland
facility: Korczowski Bartosz, Gabinet Lekarski
city: Warsaw
country: Poland
facility: Centrum Zdrowia MDM
city: Wrocław
country: Poland
facility: Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu
Study officials:
References: