Iron Deficiency Anemia (IDA) in Pediatric Subjects with Chronic Kidney Disease (CKD) 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 3 months 4. For patients other than hemodialysis dependent CKD patients, documented history of unsatisfactory oral iron therapy or in whom oral iron cannot be tolerated, or for whom oral iron is considered medically inappropriate 5. All subjects (female and male) of childbearing potential who are sexually active must be on an effective method of birth control for at least 1 month prior to Day 1 Dosing and agree to remain on birth control until completion of the study 6. Subject and/or legal guardian is capable of understanding and complying with the protocol requirements and is available for the duration of the study 7. Subject and/or legal guardian has been informed of the investigational nature of this study and has given voluntary written informed consent and, if appropriate, the child/adolescent has provided ‘assent’ and Health Insurance Portability and Accountability Act (HIPAA) or patient protection authorization had been adhered to in accordance with institutional, local, and national personal health data protection guidelines Are the trial subjects under 18? yes Number of subjects for this age range: 129 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=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 and iron sucrose 2. History of allergy to intravenous (IV) iron 3. History of multiple drug allergies (>2) 4. Low systolic blood pressure (Age 1-9 years 600 ng/mL 7. Parenteral iron therapy within 4 weeks prior to Day 1 Dosing; or blood transfusion within 4 weeks prior to Day 1 Dosing or planned at the time of Screening 8. Erythropoiesis-stimulating agent (ESA) therapy initiated, stopped or dose changed by >25% within 4 weeks prior to Screening, or anticipated ESA dose change of >20% during the study 9. Known causes of anemia other than iron deficiency (eg, vitamin B12 or folate deficiency, hemolytic anemia, etc) 10. Major surgery or invasive intervention within 4 weeks prior to Screening, or any planned major surgery or intervention during the course of the study 11. Active malignancy within 2 years prior to Screening (except non-melanoma skin cancer or carcinoma in situ that has been excised) 12. Active clinically significant infection (eg, systemic bacterial infection) or acute serious medical illness requiring treatment or intervention within 2 weeks prior to Screening 13. 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 period 14. Female subjects who are pregnant or intend to become pregnant, or are breastfeeding, are within 3 months postpartum, or have a positive pregnancy test 15. Any other clinically significant medical or psychiatric disease or 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 (compared to iron sucrose) and efficacy of ferumoxytol (7.0 mg Fe/kg x 2 [max 510 mg/dose]) in pediatric CKD subjects with iron deficiency anemia (IDA) or who are at risk of development of IDA.;Secondary Objective: To determine the single-dose PK and PD profile of ferumoxytol (7.0 mg Fe/kg, max 510 mg/dose) in pediatric subjects.;Primary end point(s): Efficacy Endpoints: ? Primary endpoint: Proportion of patients achieving a hemoglobin increase of at least 0.5 g/dL during the period from Baseline to Week 5 ? Proportion of patients achieving a hemoglobin increase of at least 0.5 g/dL or TSAT increase of at least 10% during the period from Baseline to Week 5 ? Proportion of patients achieving a TSAT increase of at least 10% during the period from Baseline to Week 5 ? Change in hemoglobin from Baseline to Week 5 ?Whether or not the subject had an increase in hemoglobin =1.0 g/dL during the period from Baseline to Week 5 ? Change in TSAT from Baseline to Week 5 ? Whether or not the subject required initiation of ESA or a >20% increase in dose during the study ? Whether or not the subject received blood transfusions during the study ? Change in other markers of iron stores (e.g., serum ferritin and serum iron) from Baseline to Week 5 Safety Endpoints: ? Incidence of adverse events of special interest (AESI) (hypotension and hypersensitivity) ? Incidence of SAEs ? Incidence of Severe 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 ? Change in vital signs (blood pressure, heart rate, respiration rate) and body temperature, and routine laboratory parameters (hematology, chemistry, and iron panel);Timepoint(s) of evaluation of this end point: Efficacy endpoints: Week 5 Safety endpoints: ongoing during the study duration | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Pharmacokinetic Endpoints: • Area Under the Curve (AUC) • Clearance • Distribution and elimination half-lives ;Timepoint(s) of evaluation of this end point: Blood samples for PK 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) post the start of the infusion | — |
Countries
Argentina, Brazil, Hungary, Lithuania, Mexico, Poland, United States
Contacts
Public ContactMedical Information
AMAG Pharmaceuticals, Inc.
Outcome results
None listed