A Study to Evaluate Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects

Официальное название

A Phase 3, Randomized, Open-Label, Multicenter Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects

Обзор

This is a Phase 3, randomized, open-label, multicenter, study in male and female pediatric subjects (2 years to \<18 years of age) with IDA, or felt by their clinician to be at risk of developing IDA. This study allows for enrollment of subjects with IDA regardless of etiology, except for CKD subjects (pediatric CKD subjects are being studied in a separate ferumoxytol protocol).

Подробное описание

Subjects will be randomized to treatment in a 2:1 ratio (ferumoxytol: iron sucrose) and stratified by age group (2 to \<6 years; 6 to \<12 years; and 12 to \<18 years). Subjects will receive one of the following treatment regimens:

• Ferumoxytol: 7 mg Fe/kg IV (maximum 510 mg/dose) x 2 doses, the first dose administered on Day 1 and the second 2 to 8 days later.

OR

• Iron sucrose (Venofer®): 4 mg Fe/kg IV (maximum 200 mg/dose) x 5 doses, the first dose on Day 1 and subsequent doses administered at least once per week and up to 3 times/week. All subjects will be monitored at the study site through at least 1 hour after the completion of each infusion of study drug. Assessment of blood Hgb concentrations, adverse events, and other safety assessments will be performed through study Week 5.

Вмешательства

• Препарат ferumoxytol
  Each 20 mL single-use vial contains 17 mL of ferumoxytol that consists of iron at a concentration of 30 mg Fe/mL, coated with polyglucose sorbitol carboxymethylether and formulated with mannitol, at a concentration of 44 mg/mL, in a black to reddish brown sterile, aqueous, colloidal, isotonic solution
• Препарат Iron sucrose
  Each mL contains 20 mg of elemental iron as iron sucrose in water for injection. The 5 mL single-use vial contains 100 mg of iron per 5 mL. The drug product contains approximately 30% sucrose (300 mg/mL).

Первичные конечные точки

• Change in Hemoglobin from Baseline to Week 5 [Срок оценки: 35 days]

Вторичные конечные точки (2)

• Incidence of Treatment Emergent Adverse Events [Срок оценки: 49 days]
• Incidence of adverse events of special interest (AESI) [Срок оценки: 49 days]

Критерии участия

Критерии включения

• Male or female 2 years to <18 years of age at time of consent
• Has IDA defined as:

• Hemoglobin (Hgb) <11.0 g/dL AND
• Any one or more of the following:

• Transferrin saturation (TSAT) <20%
• ferritin <100 ng/mL
• Documented history of unsatisfactory oral iron therapy or in whom oral iron cannot be tolerated, or for whom oral iron is considered medically inappropriate

Критерии исключения

• Known hypersensitivity reaction to any component of ferumoxytol or iron sucrose
• History of allergy to intravenous (IV) iron
• History of ≥2 clinically significant drug allergies
• Subjects with CKD (defined as eGFR of <60 mL/min/1.73 m2 or a requirement for chronic hemodialysis or peritoneal dialysis during Screening)
• Low systolic blood pressure (BP) (age 1 to 9 years <70 + \[age in years x 2\] mmHg, age 10 to 17 years <90 mmHg)
• Hgb ≤7.0 g/dL
• Serum ferritin level >600 ng/mL

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 6 центров

• Arkansas Children's Hospital — Little Rock
• University of Florida — Gainesville
• Optimus U Corporation — Miami
• Biomedical Research LLC — Miami
• Gwinnett Research Institute — Buford
• Sun Research Institute — San Antonio

Польша · 6 центров

• Osrodek Badan Klinicznych In Vivo sp. z o.o. — Bydgoszcz
• Prywatny Gabinet Lekarski Dr N. med. Jerzy Brzostek — Dębica
• Pro Familia Altera Sp. z o.o. — Katowice
• Korczowski Bartosz, Gabinet Lekarski — Rzeszów
• Centrum Zdrowia MDM — Warsaw
• Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu — Wroclaw

Литва · 3 центра

• JSC Saules seimos medicinos centras — Kaunas
• Klaipeda Children's Hospital — Klaipėda
• Children's Hospital-Affiliate of Vilnius University Hospital Santariskiu Klinikos — Vilnius

Идентификаторы

NCT: NCT03893045 · AMAG-FER-IDA-352

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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