FERUMOXYTOL for Iron deficiency anemia | Chronic kidney disease

Inclusion criteria

• {"criterion_text":"- Male or female 2 years to <18 years of age at time of consent."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- Subject and/or 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/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."}

Exclusion criteria

• {"criterion_text":"- Known hypersensitivity reaction to any component of ferumoxytol and iron sucrose."}
• {"criterion_text":"- History of allergy to intravenous (IV) iron."}
• {"criterion_text":"- History of multiple drug allergies (>2)."}
• {"criterion_text":"- Low systolic blood pressure (Age 1-9 years <70 + [age in years x 2] mmHg, Age 10-17 years <90 mmHg)."}
• {"criterion_text":"- Hemoglobin ≤7.0 g/dL."}
• {"criterion_text":"- Serum ferritin level >600 ng/mL."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- 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."}
• {"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 any planned major surgery or intervention 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 period."}
• {"criterion_text":"- Female subjects who are pregnant or intend to become pregnant, or are breastfeeding, are within 3 months postpartum, or have a positive pregnancy test."}
• {"criterion_text":"- 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."}

Primary endpoints

• {"endpoint_text":"- 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.","definition_or_measurement_approach":"Measured as the proportion of randomized subjects with hemoglobin increase ≥0.5 g/dL between Baseline and Week 5 (laboratory hemoglobin values)."}
• {"endpoint_text":"- 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.","definition_or_measurement_approach":"Measured as the proportion achieving either hemoglobin increase ≥0.5 g/dL or TSAT increase ≥10% between Baseline and Week 5."}
• {"endpoint_text":"- Proportion of patients achieving a TSAT increase of at least 10% during the period from Baseline to Week 5.","definition_or_measurement_approach":"Measured as the proportion with TSAT increase ≥10% from Baseline to Week 5."}
• {"endpoint_text":"- Change in hemoglobin from Baseline to Week 5.","definition_or_measurement_approach":"Measured as change in laboratory hemoglobin value between Baseline and Week 5."}
• {"endpoint_text":"- Whether or not the subject had an increase in hemoglobin ≥1.0 g/dL during the period from Baseline to Week 5.","definition_or_measurement_approach":"Dichotomous outcome indicating whether hemoglobin rose by ≥1.0 g/dL between Baseline and Week 5."}
• {"endpoint_text":"- Change in TSAT from Baseline to Week 5.","definition_or_measurement_approach":"Measured as change in transferrin saturation (TSAT) value between Baseline and Week 5."}
• {"endpoint_text":"- Whether or not the subject required initiation of ESA or a >20% increase in dose during the study.","definition_or_measurement_approach":"Recorded as incidence of starting ESA therapy or >20% ESA dose increase during study period."}
• {"endpoint_text":"- Whether or not the subject received blood transfusions during the study.","definition_or_measurement_approach":"Recorded as incidence of any blood transfusion during the study period."}
• {"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":"Measured as change in serum ferritin and serum iron between Baseline and Week 5."}
• {"endpoint_text":"- Safety Endpoints: Incidence of adverse events of special interest (AESI) (hypotension and hypersensitivity).","definition_or_measurement_approach":"Incidence rates of predefined AESIs (hypotension, hypersensitivity) captured during on-study period."}
• {"endpoint_text":"- Incidence of SAEs.","definition_or_measurement_approach":"Incidence of serious adverse events recorded during the study."}
• {"endpoint_text":"- Incidence of Severe AEs.","definition_or_measurement_approach":"Incidence of severe intensity adverse events recorded during the study."}
• {"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":"Incidence of specified cardiovascular adverse events as listed."}
• {"endpoint_text":"- Incidence of AEs leading to study drug discontinuation.","definition_or_measurement_approach":"Incidence of any adverse event that resulted in permanent discontinuation of study drug."}
• {"endpoint_text":"- Incidence of treatment emergent AEs.","definition_or_measurement_approach":"Incidence of adverse events emerging during treatment period (treatment-emergent)."}
• {"endpoint_text":"- Change in vital signs (blood pressure, heart rate, respiration rate) and body temperature, and routine laboratory parameters (hematology, chemistry, and iron panel).","definition_or_measurement_approach":"Measured as changes from Baseline in vital signs and routine laboratory parameters, including hematology, chemistry, and iron panel."}

Secondary endpoints

• {"endpoint_text":"- Pharmacokinetic Endpoints: Area Under the Curve (AUC).","definition_or_measurement_approach":"PK AUC calculated from plasma concentration-time data following single dose."}
• {"endpoint_text":"- Clearance","definition_or_measurement_approach":"Apparent clearance estimated from PK data."}
• {"endpoint_text":"- Distribution and elimination half-lives.","definition_or_measurement_approach":"PK parameters describing distribution and elimination half-lives calculated from concentration-time profiles."}

Methods

• K2_Doctor to Patient Letter — printed/clinic letter to inform patients (document versions indicate language variants, e.g., lt, ru).
• K2_Dr to Dr Referral Letter — referral letters for physician-to-physician referral (document versions indicate language variants).
• K2_Physician Poster — poster for physicians to inform about study and recruitment.
• K2_Patient Brochure — informational brochure for patients/parents (language-specific versions present).
• K2_Patient Study Guide — study guide for participants/patients (language-specific versions present).
• K2_Quick Facts Card — short informational cards for sites/patients.
• K2_Site Reference Cards — reference cards for site staff.
• K1_Recruitment arrangements / K1_Recruit-ICF Process — recruitment arrangements documents describing processes (blank statement templates included).
• K2_Informed Consent Aid — aids to support informed consent discussions (language variants present).
• Site-level recruitment materials available in language-specific versions (filenames indicate Lithuanian 'lt' and Russian 'ru'; other local language versions exist for participating countries).

Hungary

Earliest CTIS Part Ii Submission Date

16-08-2024

Latest Decision Or Authorization Date

05-09-2024

Processing Time Days

20

Number Of Sites

1

Number Of Participants

10

Poland

Earliest CTIS Part Ii Submission Date

16-08-2024

Latest Decision Or Authorization Date

05-10-2024

Processing Time Days

50

Number Of Sites

3

Number Of Participants

10

Lithuania

Earliest CTIS Part Ii Submission Date

16-08-2024

Latest Decision Or Authorization Date

30-06-2025

Processing Time Days

318

Number Of Sites

1

Number Of Participants

5

Primary sponsor

Full Name

Amag Pharmaceuticals Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Icon Clinical Research Limited

Responsibilities

Operational sponsor duties including PK sample storage at ICON Lab and other study conduct activities (contact: STUDY-ICR-2104-0027@iconplc.com).

Name

Ppd Inc.

Responsibilities

Pharmacokinetic testing and storage of blood samples (for up to 3 years after the end of the study) (contact: CTIS@CTIS.com).

Third parties

• {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"Sponsor duties including multiple operational roles; documented responsibility: \"PK samples storing at ICON Lab\" (other sponsor duty codes present). Contact email: STUDY-ICR-2104-0027@iconplc.com.","organisation_type":"Pharmaceutical company"}
• {"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). Contact email: CTIS@CTIS.com.","organisation_type":"Pharmaceutical company"}