Research study to evaluate the experimental drug ferumoxytol, to see how well it works for children with 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 SubjectsMedDRA version: 20.0Level: LLTClassification code 10022974Term: Iron deficiency anemiaSystem Organ Class: 100000004851Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- EUCTR2018-004304-19-PL
- 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
- 75
1. Male or female 2 years to <18 years of age at time of consent
2. Has IDA at Screening defined as:
a) Hemoglobin (Hgb) <11.0 g/dL
AND
b) 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
4. If sexually active and of childbearing potential (for both male and female subjects), be on an effective method of birth control for at least 1 month prior to Day 1 and agree to remain on birth control until completion of the study
5. Subject and legal guardian is capable of understanding and complying with the protocol requirements and is available for the duration of the study
6. Subject and legal guardian has been informed of the investigational nature of this study; legal guardian has given voluntary written informed consent and, if appropriate, the subject has provided ‘assent’ 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: 75
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 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
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
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