Clinical trial NCT01904864
Conditions: Iron Deficiency Anemia
Keywords: randomized controlled trial iron medication Anemia Hematologic Disease Anemia, Iron deficiency anemia, nutritional ferrous sulfate NovaFerrum®
Sponsor: University of Texas Southwestern Medical Center
Start date: 2013-07-01; Completion date: 2015-11-01
Enrollment: 80 participants; Allocation: RANDOMIZED; Intervention model: PARALLEL

Brief 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

Arms: arm 1: Subjects randomized to this arm will receive a single daily dose (3mg/kg) of a 15 mg/ml elemental iron preparation, NovaFerrum®, for 12 weeks. arm 2: Subjects randomized to this arm will receive a single daily dose (3mg/kg) of a 15 mg/ml elemental iron preparation, ferrous sulfate, for 12 weeks.
Interventions: intervention 1: elemental iron (NovaFerrum®) intervention 2: elemental iron (Ferrous Sulfate)
Intervention descriptions: intervention 1: single daily dose (3mg/kg) of a 15 mg/ml elemental iron preparation, NovaFerrum®, for 12 weeks intervention 2: single daily dose (3mg/kg) of a 15 mg/ml elemental iron preparation, Ferrous Sulfate, for 12 weeks

Protocol documents: