Chronic Kidney Disease; Iron Deficiency Anemia Clinical Trial
Official title:
A Phase 3, Randomized, Open-Label, Multicenter Study to Evaluate the Safety (Compared to Iron Sucrose), Efficacy and Pharmacokinetics of Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects With Chronic Kidney Disease (CKD)
Primary Objectives: To evaluate the safety (compared to iron sucrose) and efficacy of ferumoxytol in pediatric CKD subjects with iron deficiency anemia (IDA) or who are at risk of development of IDA Secondary Objective: To determine the single-dose pharmacokinetics (PK) and pharmacodynamics (PD) profile of ferumoxytol in pediatric subjects.
| Status | Recruiting |
| Enrollment | 129 |
| Est. completion date | August 31, 2023 |
| Est. primary completion date | August 31, 2023 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 2 Years to 17 Years |
| Eligibility | Inclusion Criteria: 1. Male or female 2 years to <18 years of age at time of consent 2. Has IDA defined as: a) hemoglobin <12.0 g/dL and b) with either transferrin saturation (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 3. Has Chronic Kidney Disease defined as one of the following: 1. on chronic hemodialysis; 2. receiving chronic peritoneal dialysis; 3. estimated glomerular filtration rate (eGFR) of <60 mL/min/1.73 m2; 4. 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. 4. 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 5. 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 Exclusion Criteria: 1. Known hypersensitivity reaction to any component of ferumoxytol and iron sucrose 2. History of allergy to intravenous (IV) iron 3. History of multiple drug allergies (>2) 4. Low systolic blood pressure (Age 1-9 years <70 + [age in years x 2] mmHg, Age 10-17 years <90 mmHg) 5. Hemoglobin =7.0 g/dL 6. Serum ferritin level >600 ng/mL |
| Country | Name | City | State |
|---|---|---|---|
| Hungary | Semmelweis Egyetem - Altalanos Orvostudomanyi Kar (SE AOK) - I. sz. Gyermekgyogyaszati Klinika | Budapest | |
| Hungary | University Of Szeged | Szeged | |
| Lithuania | Hospital of Lithuanian University of Health Sciences Kauno klinikos | Kaunas | |
| Lithuania | Klaipeda Children's Hospital | Klaipeda | |
| Lithuania | Vsl Vilniaus Universiteto Vaiku Ligonine (VUVL) (Vilnius University Children's Hospital) | Vilnius | |
| Mexico | Instituto Mexicano De Trasplantes S.C | Cuernavaca | |
| Mexico | JM Research, SC | Cuernavaca | |
| Mexico | Hospital Civil de Guadalajara Fray Antonio Alcalde | Guadalajara | |
| Mexico | Instituto Nacional de Pediatria (INP) (National Institute of Pediatrics) | Mexico City | |
| Poland | Uniwersytecki Szpital Kliniczny Im. Zamenhofa w Bialystoku | Bialystok | |
| Poland | Specjalistyczne Gabinety Sp. z o.o | Kraków | |
| Poland | University Children Hospital | Kraków | |
| Poland | Polish Mother's Memorial Hospital Research Institute | Lódz | |
| United States | Akron Nephrology Associates, Inc. | Akron | Ohio |
| United States | Montefiore Medical Center (MMC) - The Children's Hospital at Montefiore (CHAM) | Bronx | New York |
| United States | Wayne State University | Detroit | Michigan |
| United States | Memorial Healthcare System | Hollywood | Florida |
| United States | Children's Mercy Hospital | Kansas City | Missouri |
| United States | The Feinstein Institute Medical Research Organization of Northwell Health, Inc. | Lake Success | New York |
| United States | West Virginia University | Morgantown | West Virginia |
| United States | Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH) | New York | New York |
| Lead Sponsor | Collaborator |
|---|---|
| AMAG Pharmaceuticals, Inc. |
United States, Hungary, Lithuania, Mexico, Poland,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Other | Area Under the Curve (AUC) | Pharmacokinetic parameter: Area Under the Curve (AUC) | 35 days | |
| Other | Clearance | Pharmacokinetic parameter: clearance | 35 days | |
| Other | Distribution and elimination half-lives | Pharmacokinetic parameter: distribution and elimination half-lives | 35 days | |
| Primary | Hemoglobin change of at least 0.5 g/dL from Baseline to Week 5 | Proportion of patients achieving a hemoglobin change of at least 0.5 g/dL during the period from Baseline to Week 5 | 35 days | |
| Secondary | Incidence of Treatment Emergent Adverse Events | Incidence of Treatment Emergent Adverse Events | 49 days | |
| Secondary | Incidence of adverse events of special interest (AESI) | Incidence of adverse events of special interest (AESI) (hypotension and hypersensitivity) | 49 days |