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.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Amag Pharmaceuticals Inc.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nutritional and Metabolic Diseases [C18]

Trial duration

3 Sep 2019 → ongoing

Decision date (initial)

2024-09-13

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

AMAG Pharmaceuticals, Inc.

External identifiers

EU CT number

2024-516264-28-00

EudraCT number

2018-004304-19

ClinicalTrials.gov

NCT03893045

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Efficacy, Safety, Therapy

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 objectives 1

1. To determine the single-dose pharmacokinetics (PK) profile and describe the efficacy of ferumoxytol in pediatric subjects.

Conditions and MedDRA coding

Iron Deficiency Anemia (IDA)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

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.

Exclusion criteria 16

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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 7

1. Safety Endpoints: Incidence of adverse events of special interest (hypotension and hypersensitivity).
2. Incidence of serious adverse events (SAEs).
3. Incidence of severe adverse events (AEs).
4. Incidence of cardiovascular AEs (myocardial infarction, heart failure, moderate to severe hypertension, and hospitalization due to any cardiovascular cause).
5. Incidence of AEs leading to study drug discontinuation.
6. Incidence of treatment emergent AEs (TEAEs).
7. Change in vital signs (BP, heart rate, respiration rate) and body temperature, and routine laboratory parameters (hematology, chemistry, and iron panel).

Secondary endpoints 11

1. Efficacy Endpoints: Proportion of subjects achieving a Hgb increase of at least 0.5 g/dL from Baseline to Week 5.
2. 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.
3. Proportion of subjects achieving a TSAT increase of at least 10% from Baseline to Week 5.
4. Change in Hgb from Baseline to Week 5.
5. Proportion of subjects achieving a Hgb increase of at least 1.0 g/dL from Baseline to Week 5.
6. Change in TSAT from Baseline to Week 5.
7. Proportion of subjects receiving blood transfusions during the study.
8. Change in other markers of iron stores (e.g., serum ferritin and serum iron) from Baseline to Week 5.
9. Pharmacokinetic Endpoints: Area Under the Curve (AUC).
10. Clearance.
11. Distribution and elimination half-lives. All parameters will be obtained from the population model.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Comparator 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Amag Pharmaceuticals Inc.

Sponsor organisation

Amag Pharmaceuticals Inc.

Address

1100 Winter Street Floor 2

City

Waltham

Postcode

02451-1427

Country

United States

Scientific contact point

Organisation

Amag Pharmaceuticals Inc.

Contact name

Medical Information

Public contact point

Organisation

Amag Pharmaceuticals Inc.

Contact name

Medical Information

Third parties 2

Locations

2 EU/EEA countries · 3 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 16 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications