Comparison of NovaFerrum® vs Ferrous Sulfate Treatment in Young Children With Nutritional Iron Deficiency Anemia
Condition: Iron Deficiency Anemia
MeSH terms: Anemia; Anemia, Iron-Deficiency; Iron Deficiencies; Deficiency Diseases
Study type: Interventional
Phase: Phase 4

Summary: 
      This study is a randomized, controlled, double-blinded single center trial to compare the
      efficacy of NovaFerrum® to ferrous sulfate for the treatment of nutritional iron deficiency
      anemia (IDA) in infants and young children.

      Hypothesis: NovaFerrum® has greater efficacy than ferrous sulfate in increasing hemoglobin
      concentration during a twelve week course of treatment to subjects with iron deficiency
      anemia.

      Primary Aim:

      To compare the efficacy of NovaFerrum® to ferrous sulfate for the treatment of nutritional
      IDA in infants and young children as determined by increase in hemoglobin concentration.

      Secondary Aims:

        1. To compare the adverse effects of treatment for IDA between ferrous sulfate and
           NovaFerrum®

        2. To compare normalization of iron stores as demonstrated by laboratory measures of IDA
           (ferritin, TIBC, reticulocyte hemoglobin content) between subjects treated with ferrous
           sulfate or NovaFerrum®

        3. To compare the adherence to study medication between subjects on ferrous sulfate and
           NovaFerrum®

        4. To demonstrate efficacy of a once daily dosing regimen in the treatment of nutritional
           IDA
    

Detailed description: 
      Screening/Baseline Assessment (Week 0):

        -  History and Physical Examination:

        -  Detailed history regarding medical disorders predisposing to iron deficiency and diet
           (breast feeding, iron fortified formula, cow's milk), including total daily amount.

        -  Review of existing medical records (including blood counts and iron studies) submitted
           by primary care provider or recorded on Children's electronic medical record.

        -  History of pica and/or other sequelae from iron deficiency.

        -  Details regarding prior or current iron oral therapy (dose, preparation, timing,
           response) or recent blood transfusions.

        -  Comprehensive physical exam (PE) (baseline visit) consisting of vital signs, general,
           HEENT, cardio-respiratory, abdominal, extremities and skin; Focused PE (Weeks 4 and 12)
           consisting of vital signs, general, cardio-respiratory, abdominal and skin.

        -  Laboratory (Only #3 below for research only, not standard of care):

             1. Complete blood count (CBC), reticulocyte count and reticulocyte hemoglobin content.

             2. Serum ferritin, iron and total body iron capacity (TIBC).

      Treatment Interventions:

        -  Patients who meet eligibility criteria and whose parents provide written informed
           consent will be randomized in a 1:1 ratio to receive either ferrous sulfate drops (15
           mg/ml) or NovaFerrum® drops (15 mg/ml).

        -  Subjects will be assigned to receive a single daily dose of 3 mg/kg elemental iron.
           Timing of study drug will be at bedtime.

        -  As a part of standard care, patients will be advised to reduce cow milk intake to a
           maximum 16 oz. daily and not to give any milk after medication administration. There
           will be no other dietary modification prescribed by the study.

        -  Other iron containing medications (including vitamins) will be discontinued.

        -  Families will be asked to make a daily diary entry documenting administration and
           adverse effects and return the diary at follow-up clinic visits.

      Follow-up Phone Contact (Weeks 2, 6, 10 - not standard of care)

      - Phone contact with parents will be made biweekly between scheduled visits (e.g. Weeks 2, 6,
      and 10) to assess adverse effects, promote strict adherence and remind them of the next
      scheduled visit.

      Assessment During Follow-up Visits at Weeks 4, 8, and 12 after Initiation of Therapy
      (Follow-up visits during weeks 4 and 12 are standard of care; Follow-up visit at week 8 is
      for research only):

        -  Review of interval history and diet by direct questioning of the parents and review of
           diary regarding adherence, adverse effects of iron therapy (e.g., refusal to take,
           spitting/vomiting, abdominal pain, constipation, black stools, stained teeth). The diary
           will also contain distracter items to assess background "noise".

        -  Recording of other medications or intercurrent illnesses.

        -  Laboratory studies at each follow-up visit: CBC with red blood cell (RBC) indices, (MCV,
           RDW) reticulocyte count, reticulocyte hemoglobin content, serum ferritin, serum iron and
           total iron binding capacity.

        -  Blood lead measurement at week 4 (research only). Will repeated only if abnormal.

        -  Focused physical exam at 4 and 12 week visits
    

Intervention name(s): elemental iron (NovaFerrum®); elemental iron (Ferrous Sulfate)
Intervention type(s): Drug; Drug
Intervention description(s): single daily dose (3mg/kg) of a 15 mg/ml elemental iron preparation, NovaFerrum®, for 12 weeks; single daily dose (3mg/kg) of a 15 mg/ml elemental iron preparation, Ferrous Sulfate, for 12 weeks
Other intervention name(s): Generic Name: polysaccharide iron vitamin mineral complex; Ferrous Sulfate
Intervention MeSH terms: Iron
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: randomized controlled trial; iron medication; Anemia; Hematologic Disease; Anemia, Iron deficiency; anemia, nutritional; ferrous sulfate; NovaFerrum®