Iron Deficiency AnemiaPhase 4BESTIRONNCT01904864
Comparison of NovaFerrum® vs Ferrous Sulfate Treatment in Young Children With Nutritional Iron Deficiency Anemia
A Phase 4 study testing elemental iron (NovaFerrum®) and elemental iron (Ferrous Sulfate) in people with Iron Deficiency Anemia, run by University of Texas Southwestern Medical Center at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 9 to 48 months
- Conditions the study is for
- Iron Deficiency Anemia
- What is being tested?
- elemental iron (NovaFerrum®) and elemental iron (Ferrous Sulfate)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 80 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2013to Nov 2015
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 9 to 48 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age ≥ 9 to < 48 months
- IDA documented by hematologic indices (hemoglobin, MCV, RDW, reticulocyte count, reticulocyte hemoglobin content), serum ferritin, serum iron and total iron binding capacity
Who cannot join
- Iron deficiency likely or definitely due to blood loss from the intestine or other sites.
- Evidence of response to recent/current oral iron therapy, as determined by increase in hemoglobin by > 1.0 gm/dL and MCV by 5 fL above measurements prior to iron therapy
- History or evidence of intestinal malabsorption
- History of prior intravenous iron therapy
- Major co-morbidity such as a serious chronic medical condition unrelated to iron deficiency apparent on history, physical examination, or laboratory tests
- Other causes of anemia (sickle cell disease, thalassemia, other hemolytic anemia, bone marrow failure, etc.) apparent by history, physical examination, and/or laboratory tests.
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- High likelihood of suboptimal adherence by parents with study requirements (previous missed clinic visits)
- Inability to tolerate oral medications
- History of birth at < 30 weeks gestation
- Other medical or social factors at discretion of treating physician
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Medical Center Dallas
Dallas, Texas, 75235
About this study
Official title: A Single-center, Double-blinded, Randomized, 12 Week, Superiority Study in Infants and Young Children to Compare the Efficacy of NovaFerrum® Versus Ferrous Sulfate in the Treatment of Nutritional Iron Deficiency Anemia.
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.
Read more
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:
- To compare the adverse effects of treatment for IDA between ferrous sulfate and NovaFerrum®
- 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®
- To compare the adherence to study medication between subjects on ferrous sulfate and NovaFerrum®
- To demonstrate efficacy of a once daily dosing regimen in the treatment of nutritional IDA
Read the 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):
- Complete blood count (CBC), reticulocyte count and reticulocyte hemoglobin content.
- 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
- Conditions
- Iron Deficiency Anemia
- Being tested
- elemental iron (NovaFerrum®), elemental iron (Ferrous Sulfate)
- Keywords
- randomized controlled trial, iron medication, Anemia, Hematologic Disease, Anemia, Iron deficiency, anemia, nutritional, ferrous sulfate, NovaFerrum®
- Medical subject headings
- Anemia, Iron-Deficiency, Anemia, Hematologic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
elemental iron (NovaFerrum®)Drug
Also called Generic Name: polysaccharide iron vitamin mineral complex
single daily dose (3mg/kg) of a 15 mg/ml elemental iron preparation, NovaFerrum®, for 12 weeks
elemental iron (Ferrous Sulfate)Drug
Also called Ferrous Sulfate
single daily dose (3mg/kg) of a 15 mg/ml elemental iron preparation, Ferrous Sulfate, for 12 weeks
| Study group | Role | What they receive |
|---|---|---|
| NovaFerrum® | Active comparator | 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. Receives: Drug: elemental iron (NovaFerrum®) |
| Ferrous Sulfate | Active comparator | 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. Receives: Drug: elemental iron (Ferrous Sulfate) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Hemoglobin Concentration Over Time
The primary outcome will be the change in the peripheral blood hemoglobin concentration in grams/deciliter upon serial measurements at 0, 4, 8, and 12 weeks post-initiation of treatment. The primary analysis consists of a linear mixed regression model, which incorporates all subsequent time points into the model and includes treatment and time as covariates and patient random effects to account for correlation among longitudinal measurements from the same patients.
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2013 (estimated)
- Study startJul 2013
- Primary completionNov 2015
- Study completionNov 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Iron Deficiency AnemiaStudies of the same condition, open and under way.
- More trials run by University of Texas Southwestern Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 80 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Texas Southwestern Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Gensavis Pharmaceuticals, LLC
- Responsible party
- Principal investigator, Patrick Leavey, University of Texas Southwestern Medical Center
- Investigators
- Patrick Leavey, MD, Principal investigator, U Texas Southwestern
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01904864
- Sponsor's protocol ID
- FE15929
- Acronym
- BESTIRON
- Status last verified
- Nov 2017
- First submitted
- 17 Jul 2013
- Last updated
- 14 Dec 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Powers JM, Buchanan GR, Adix L, Zhang S, Gao A, McCavit TL. Effect of Low-Dose Ferrous Sulfate vs Iron Polysaccharide Complex on Hemoglobin Concentration in Young Children With Nutritional Iron-Deficiency Anemia: A Randomized Clinical Trial. JAMA. 2017 Jun 13;317(22):2297-2304. doi: 10.1001/jama.2017.6846.
PubMed 28609534
Study at a glance
From the registry record, last updated by the sponsor on 14 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-center, Double-blinded, Randomized, 12 Week, Superiority Study in Infants and Young Children to Compare the Efficacy of NovaFerrum® Versus Ferrous Sulfate in the Treatment of Nutritional Iron Deficiency Anemia.
- ClinicalTrials.gov ID
- NCT01904864
- Acronym / sponsor ID
- BESTIRON / FE15929
- Conditions
- Iron Deficiency Anemia
- Keywords
- randomized controlled trial, iron medication, Anemia, Hematologic Disease, Anemia, Iron deficiency, anemia, nutritional, ferrous sulfate, NovaFerrum®
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 80 participants (actual)
- Lead sponsor
- University of Texas Southwestern Medical CenterUniversity, hospital or other
- Collaborators
- Gensavis Pharmaceuticals, LLC
- Study start
- Jul 2013
- Primary completion
- Nov 2015 (actual)
- Study completion
- Nov 2015 (actual)
- First posted
- 22 Jul 2013 (estimated)
- Results
- Posted 13 Sep 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- To compare the adverse effects of treatment for IDA between ferrous sulfate and NovaFerrum®
- 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®
- To compare the adherence to study medication between subjects on ferrous sulfate and NovaFerrum®
- 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):
- Complete blood count (CBC), reticulocyte count and reticulocyte hemoglobin content.
- 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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| NovaFerrum® | Active comparator | Drug: elemental iron (NovaFerrum®)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. |
| Ferrous Sulfate | Active comparator | Drug: elemental iron (Ferrous Sulfate)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. |
Who may qualify
- Ages
- 9 to 48 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Age ≥ 9 to < 48 months
- IDA documented by hematologic indices (hemoglobin, MCV, RDW, reticulocyte count, reticulocyte hemoglobin content), serum ferritin, serum iron and total iron binding capacity
Key exclusion criteria (10 listed)
- Iron deficiency likely or definitely due to blood loss from the intestine or other sites.
- Evidence of response to recent/current oral iron therapy, as determined by increase in hemoglobin by > 1.0 gm/dL and MCV by 5 fL above measurements prior to iron therapy
- History or evidence of intestinal malabsorption
- History of prior intravenous iron therapy
- Major co-morbidity such as a serious chronic medical condition unrelated to iron deficiency apparent on history, physical examination, or laboratory tests
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| elemental iron (NovaFerrum®)Also: Generic Name: polysaccharide iron vitamin mineral complexsingle daily dose (3mg/kg) of a 15 mg/ml elemental iron preparation, NovaFerrum®, for 12 weeks | Drug | NovaFerrum® |
| elemental iron (Ferrous Sulfate)Also: Ferrous Sulfatesingle daily dose (3mg/kg) of a 15 mg/ml elemental iron preparation, Ferrous Sulfate, for 12 weeks | Drug | Ferrous Sulfate |
Primary outcome
Hemoglobin Concentration Over Time
Time frame: 12 weeks
The primary outcome will be the change in the peripheral blood hemoglobin concentration in grams/deciliter upon serial measurements at 0, 4, 8, and 12 weeks post-initiation of treatment. The primary analysis consists of a linear mixed regression model, which incorporates all subsequent time points into the model and includes treatment and time as covariates and patient random effects to account for correlation among longitudinal measurements from the same patients.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Dec 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 28609534Powers JM, Buchanan GR, Adix L, Zhang S, Gao A, McCavit TL. Effect of Low-Dose Ferrous Sulfate vs Iron Polysaccharide Complex on Hemoglobin Concentration in Young Children With Nutritional Iron-Deficiency Anemia: A Randomized Clinical Trial. JAMA. 2017 Jun 13;317(22):2297-2304. doi: 10.1001/jama.2017.6846.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Patrick Leavey, MD principal investigator
U Texas Southwestern
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing elemental iron (NovaFerrum®) and elemental iron (Ferrous Sulfate) in people with Iron Deficiency Anemia, run by University of Texas Southwestern Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Dec 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 13 Sep 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Hemoglobin Concentration Over Time (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01904864, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Dec 2017.