Triferic IRIDA (Iron-Refractory Iron-Deficiency Anemia) Protocol

Inclusion criteria

• . Adult patients have provided informed consent and have signed the informed consent form. For pediatric patients, parents/legal guardians of the patient have the ability to understand the requirements of the study and have demonstrated a willingness to have their child comply with all study procedures by signing the informed consent form. Where applicable, assent of the pediatric patient has also been obtained for all study procedures prior to any study-related activities.
• . Patient has a history of congenital hypochromic microcytic anemia.
• . Patient has a mean corpuscular volume (MCV) ≤75 fluid ounce (fL) at screening.
• . Patient has a serum transferrin saturation ≤15% at screening.
• . Patient has a history of no or incomplete response to oral iron therapy.
• . Patient has a history of no or incomplete response to intravenous iron administration.
• . Patient has a history of hepcidin concentration ≥10 nanograms per milliliter (ng/mL) (3.58 nanometer (nM)) off iron supplements, adjusted for the specific hepcidin assay used to be at least 1 standard deviation (SD) greater than the mean for patients with iron-deficiency anemia.
• . Patient is documented to have homozygous or compound heterozygous pathogenic mutations in Transmembrane Serine Protease 6 (TMPRSS6) in a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory.

Exclusion criteria