Research study to evaluate the experimental drug ferumoxytol, to see how well it works for children with chronic kidney disease (CKD) and iron deficiency anemia (IDA) or who are at risk of developing IDA, and to see how safe it is compared to another marketed iron product – iron sucrose.
- Conditions
- Iron Deficiency Anemia (IDA) in Pediatric Subjects with Chronic Kidney Disease (CKD)MedDRA version: 20.0Level: LLTClassification code 10022974Term: Iron deficiency anemiaSystem Organ Class: 100000004851Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- EUCTR2017-004026-15-HU
- Lead Sponsor
- AMAG Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 129
1. Male or female 2 years to <18 years of age at time of consent
2. Has IDA defined as: a) hemoglobin <12.0 g/dL and b) with either TSAT <40% or ferritin <100 ng/mL; or considered to be at risk of development of IDA, i.e., TSAT<20% with falling hemoglobin during the preceding 2 months and a history of hemoglobin <12 g/dL
3. Has Chronic Kidney Disease defined as one of the following:
a. on chronic hemodialysis;
b. receiving chronic peritoneal dialysis;
c. eGFR of <60 mL/min/1.73 m2;
d. has evidence of structural and/or functional abnormalities e.g., persistent albuminuria, abnormal urine sediment, electrolyte and other abnormalities due to tubular disorders for > 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
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 <70 + [age in years x 2] mmHg, Age 10-17 years <90 mmHg)
5. Hemoglobin =7.0 g/dL
6. Serum ferritin level >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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method