Iron Deficiency Anemia (IDA) in Pediatric Subjects MedDRA version: 20.0 Level: LLT Classification code 10022974 Term: Iron deficiency anemia System Organ Class: 100000004851
Conditions
Sponsors
AMAG Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female 2 years to =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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 600 ng/mL 8. Parenteral iron therapy or blood transfusion within 4 weeks prior to Screening or planned for administration during the study 9. ESA therapy within 4 weeks prior to Screening, or planned for administration during the study 10. Known causes of anemia other than iron deficiency (e.g. vitamin B12 or folate deficiency, hemolytic anemia, etc.) 11. Major surgery or invasive intervention within 4 weeks prior to Screening, or planned during the course of the study 12. Active malignancy within 2 years prior to Screening (except non-melanoma skin cancer or carcinoma in situ that has been excised) 13. Active clinically significant infection (e.g., systemic bacterial infection) or acute serious medical illness requiring treatment or intervention within 2 weeks prior to Screening 14. Received another investigational agent within 4 weeks prior to Screening, or planned receipt of an investigational agent not specified by this protocol during the study 15. Female subjects who are pregnant or intend to become pregnant, are breastfeeding, are within 3 months postpartum, or have a positive pregnancy test 16. Any other clinically significant condition or subject responsibility that, in the Investigator’s opinion, may interfere with a subject’s (and/or legal guardian’s) ability to adhere to the protocol, interfere with assessment of the investigational product, or serve as a contraindication to the subject’s participation in the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety of ferumoxytol (7 mg Fe/kg [maximum 510 mg/dose] x 2 doses) in pediatric subjects with iron deficiency anemia (IDA) or who are at risk of development of IDA. ;Secondary Objective: To determine the single-dose pharmacokinetics (PK) profile and describe the efficacy of ferumoxytol in pediatric subjects. ;Primary end point(s): Safety Endpoints: • Incidence of adverse events of special interest (hypotension and hypersensitivity) • Incidence of serious adverse events (SAEs) • Incidence of severe adverse events (AEs) • Incidence of cardiovascular AEs (myocardial infarction, heart failure, moderate to severe hypertension, and hospitalization due to any cardiovascular cause) • Incidence of AEs leading to study drug discontinuation • Incidence of treatment emergent AEs (TEAEs) • Change in vital signs (BP, heart rate, respiration rate) and body temperature, and routine laboratory parameters (hematology, chemistry, and iron panel) ;Timepoint(s) of evaluation of this end point: Safety endpoints: ongoing during the study duration | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Efficacy Endpoints • Proportion of subjects achieving a Hgb increase of at least 0.5 g/dL from Baseline to Week 5 • Proportion of subjects achieving a Hgb increase of at least 0.5 g/dL or TSAT increase of at least 10% from Baseline to Week 5 • Proportion of subjects achieving a TSAT increase of at least 10% from Baseline to Week 5 • Change in Hgb from Baseline to Week 5 • Proportion of subjects achieving a Hgb increase of at least 1.0 g/dL from Baseline to Week 5 • Change in TSAT from Baseline to Week 5 • Proportion of subjects receiving blood transfusions during the study • Change in other markers of iron stores (e.g., serum ferritin and serum iron) from Baseline to Week 5 Pharmacokinetic Endpoints • Area Under the Curve (AUC) • Clearance • Distribution and elimination half-lives All parameters will be obtained from the population model. ;Timepoint(s) of evaluation of this end point: Efficacy endpoint: Week 5 PK endpoint: PK blood samples will be collected 10 minutes prior to administering the first dose; at 18 minutes (not before infusion ends and no later than 10 minutes after infusion ends), 1 hour (±15 minutes), 3 hours (±30 minutes), 5 hours (±30 minutes), 24 hours (±2 hours), and 48 hours (±4 hours) after the start of the infusion. For PK subjects, the 48-hour PK sample must be collected before administration of dose 2. PK blood samples will not be collected from subjects weighing <10 kg. | — |
Countries
Lithuania, Poland, United States
Contacts
Public ContactMedical Information
AMAG Pharmaceuticals, Inc.
Outcome results
None listed