IDA-301 Study and Results
IDA-301 was a double-blind, placebo-controlled trial designed to compare the safety and efficacy of a one gram intravenous (IV) course of ferumoxytol to IV saline given as placebo. In this study, 608 subjects were treated with ferumoxytol and 200 received placebo, with the demographics and all baseline parameters well balanced between the two treatment groups. The primary efficacy endpoint for U.S. regulators is the proportion of subjects who achieved a ≥ 2.0 g/dL increase in hemoglobin at any time from baseline to week 5; the primary efficacy endpoint for European Union (E.U.) regulators is the mean change in hemoglobin from baseline to week 5.
In the IDA-301 trial, ferumoxytol achieved both primary efficacy endpoints. Over 80% of study participants treated with ferumoxytol achieved an increase of ≥ 2.0 g/dL in hemoglobin compared to only 5.5% of subjects who received placebo, meeting the protocol defined measure of superiority (p<0.0001). The mean change in hemoglobin in ferumoxytol-treated subjects was 2.7 g/dL, compared to a mean 0.1 g/dL increase in subjects receiving placebo (p<0.0001).
Data from IDA-301 also showed a direct correlation between a rise in hemoglobin and improvement in subject-reported fatigue scores using the Functional Assessment of Chronic Illness Therapy (FACIT) instrument. At baseline, IDA-301 participants reported mean FACIT-Fatigue levels of 24, which are comparable to those described in the medical literature for anemic cancer patients receiving chemotherapy. 3 Following a one gram course of therapy with ferumoxytol, the subjects in this study reported a significant improvement in fatigue scores (less fatigue) with a mean 12 point increase in FACIT-Fatigue scores from baseline to week 5 (p<0.05). In published literature, the U.S. mean FACIT-Fatigue score in a randomly selected group of 1,075 subjects was 40. 4 In IDA-301, subjects treated with ferumoxytol achieved mean FACIT-Fatigue scores of 36 at week 5, close to those of the general U.S. population.“Symptoms of anemia can have a negative impact on a patient’s quality of life,” said Dr. Saroj Vadhan-Raj, a principal investigator of the IDA-301 study and Professor and Chief of the Section of Cytokines & Supportive Oncology at University of Texas MD Anderson Cancer Center. “Subjects in IDA-301 treated with ferumoxytol had significant increases in hemoglobin levels and we observed a direct correlation between a rise in hemoglobin and an improvement in these subjects’ measures of fatigue. Patients with iron deficiency anemia and an unsatisfactory history with oral iron have a real need for additional treatment options and the data from the studies presented at ASH suggest that ferumoxytol may have the potential to address that need.”