ferumoxytol for Iron deficiency anemia

Inclusion criteria

• {"criterion_text":"- Male or female 2 years to <18 years of age at time of consent."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- Documented history of unsatisfactory oral iron therapy or in whom oral iron cannot be tolerated, or for whom oral iron is considered medically inappropriate."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- Subject and legal guardian is capable of understanding and complying with the protocol requirements and is available for the duration of the study"}
• {"criterion_text":"- 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

• {"criterion_text":"- Known hypersensitivity reaction to any component of ferumoxytol or iron sucrose."}
• {"criterion_text":"- Known causes of anemia other than iron deficiency (e.g. vitamin B12 or folate deficiency, hemolytic anemia, etc.)."}
• {"criterion_text":"- Major surgery or invasive intervention within 4 weeks prior to Screening or planned during the course of the study."}
• {"criterion_text":"- Active malignancy within 2 years prior to Screening (except non- melanoma skin cancer or carcinoma in situ that has been excised)."}
• {"criterion_text":"- Active clinically significant infection (e.g., systemic bacterial infection) or acute serious medical illness requiring treatment or intervention within 2 weeks prior to Screening."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- Female subjects who are pregnant or intend to become pregnant, are breastfeeding, are within 3 months postpartum, or have a positive pregnancy test."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- History of allergy to intravenous (IV) iron."}
• {"criterion_text":"- History of ≥2 clinically significant drug allergies."}
• {"criterion_text":"- Subjects with CKD (defined as eGFR of <60 mL/min/1.73 m2 or a requirement for chronic hemodialysis or peritoneal dialysis during Screening)."}
• {"criterion_text":"- Low systolic blood pressure (BP) (age 1 to 9 years <70 + [age in years x 2] mmHg, age 10 to 17 years <90 mmHg)."}
• {"criterion_text":"- Hgb ≤7.0 g/dL."}
• {"criterion_text":"- Serum ferritin level >600 ng/mL."}
• {"criterion_text":"- Parenteral iron therapy or blood transfusion within 4 weeks prior to Screening or planned for administration during the study."}
• {"criterion_text":"- ESA therapy within 4 weeks prior to Screening, or planned for administration during the study."}

Primary endpoints

• {"endpoint_text":"- Safety Endpoints: Incidence of adverse events of special interest (hypotension and hypersensitivity).","definition_or_measurement_approach":""}
• {"endpoint_text":"- Incidence of serious adverse events (SAEs).","definition_or_measurement_approach":""}
• {"endpoint_text":"- Incidence of severe adverse events (AEs).","definition_or_measurement_approach":""}
• {"endpoint_text":"- Incidence of cardiovascular AEs (myocardial infarction, heart failure, moderate to severe hypertension, and hospitalization due to any cardiovascular cause).","definition_or_measurement_approach":""}
• {"endpoint_text":"- Incidence of AEs leading to study drug discontinuation.","definition_or_measurement_approach":""}
• {"endpoint_text":"- Incidence of treatment emergent AEs (TEAEs).","definition_or_measurement_approach":""}
• {"endpoint_text":"- Change in vital signs (BP, heart rate, respiration rate) and body temperature, and routine laboratory parameters (hematology, chemistry, and iron panel).","definition_or_measurement_approach":""}

Secondary endpoints

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

Lithuania

Earliest CTIS Part Ii Submission Date

22-07-2024

Latest Decision Or Authorization Date

13-09-2024

Processing Time Days

53

Number Of Sites

2

Number Of Participants

11

Poland

Earliest CTIS Part Ii Submission Date

22-07-2024

Latest Decision Or Authorization Date

30-08-2024

Processing Time Days

39

Number Of Sites

1

Number Of Participants

40

Primary sponsor

Full Name

Amag Pharmaceuticals Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Ppd Inc.

Responsibilities

Pharmacokinetic test and storage of blood samples (for up to 3 years after the end of the study).

Name

Icon Clinical Research Limited

Responsibilities

Multiple study operational responsibilities (sponsorDuties codes: 1,2,3,4,5,6,7,8,10,11,12,13,15); includes 'PK samples storing at ICON Lab'.

Third parties

• {"country":"United States","full_name":"Ppd Inc.","duties_or_roles":"Pharmacokinetic test and storage of blood samples (for up to 3 years after the end of the study).","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"Multiple study operational duties (sponsorDuties codes listed in CTIS: 1,2,3,4,5,6,7,8,10,11,12,13,15); includes 'PK samples storing at ICON Lab'.","organisation_type":"Pharmaceutical company"}