Iron Deficiency Anemia
Conditions
Brief summary
Evaluating the Efficacy and Safety of Intravenous Ferric Carboxymaltose in Pediatric Patients with Iron Deficiency Anemia and an Unsatisfactory Response to Oral Iron under Study Protocol 1VIT17044
Detailed description
This is a single arm, open-label, multi-center, multi-national, non-randomized study that will evaluate the efficacy and safety of a one course treatment with FCM in participants who had an unsatisfactory response to oral iron in study 1VIT17044. Subjects will have an End of Study (EOS) Visit (Day 35) from the 1VIT17044 study to qualify for entry. Subjects will then be screened, meet all inclusion criteria, no exclusion criteria and have a baseline evaluation. Based on the successful completion of the baseline evaluations, subjects will be enrolled and will receive the first dose of FCM (Day 0). The second FCM dose will occur 7 days from the first dose. Subjects will return to the clinic 14 and 28 days post their first dose for laboratory and safety assessments, and at 35 days post the first dose, participants will return to the clinic for final safety and efficacy assessments. Once all assessments are complete the participant will exit the study. An unsatisfactory response to oral iron (i.e., an increase in hemoglobin (Hgb) of \<1 g/dL from baseline) will be defined as non-responders to oral iron treatment. Non-responders who continue to meet all inclusion criteria (including Hgb \<11 g/dL) and none of the exclusion criteria will receive 1 course of FCM.
Interventions
DRUGFerric carboxymaltose
IV iron
Sponsors
American Regent, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
This is a single arm, open-label, multi-center, multi-national, non-randomized study that will evaluate the efficacy and safety of a one course treatment with FCM in participants who had an unsatisfactory response to oral iron in study 1VIT17044.
Eligibility
Sex/Gender
ALL
Age
1 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
1. Unsatisfactory response to oral iron or those that required a concomitant intervention, (defined as, blood transfusion, use of IV or oral iron outside of protocol, increase in erythropoietin for any reason \[Day 0 thru Day 35 of study protocol 1VIT17044\], change in IBD treatment). 2. Hgb \<11 g/dL 3. Ferritin ≤300 ng/mL and TSAT \<30%
Exclusion criteria
1. Known history of hypersensitivity reaction to any component of FCM. 2. History of acquired iron overload, hemochromatosis, or other iron accumulation disorders. 3. History of significant diseases of the liver, hematopoietic system, cardiovascular system, psychiatric disorder, or other conditions which, in the opinion of the investigator, may place a subject at added risk for participation in the study. 4. Any existing non-viral infection. 5. Known history of positive HBsAg or HCV with evidence of active hepatitis. 6. Known history of positive HIV-1/HIV-2 antibodies (anti-HIV). 7. Anemia due to reasons other than iron deficiency (e.g., hemoglobinopathy and vitamin B12 or folic acid deficiency) that has not been corrected. 8. Administration and / or use of an investigational product (drug or device) within 30 days of screening. 9. Alcohol or drug abuse within the past six months. 10. Female participant who is pregnant or lactating, or sexually active females who are of childbearing potential not willing to use an acceptable form of contraceptive precautions during the study. 11. Unable to comply with study procedures and assessments.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Hemoglobin From Baseline to Day 35 | The change in Hgb from baseline to day 35 | The change in hemoglobin from Baseline to Day 35 was assessed using paired t-tests (two-sided test, α = 0.05). Baseline hemoglobin was defined as the last hemoglobin obtained before the first dose. |
Countries
United States
Participant flow
Pre-assignment details
1VIT17044, NCT03523117 can be referenced to make the information accessible.
Participants by arm
| Arm | Count |
|---|---|
| IV Iron The primary objective in this study is to allow participants who were randomized to receive oral iron in the 1VIT17044 trial and who had an unsatisfactory response to oral iron or those that required a concomitant intervention, (defined as, blood transfusion, use of IV or oral iron outside of protocol, increase in erythropoietin for any reason \[Day 0 thru Day 35 of study protocol 1VIT17044\], change in IBD treatment) to receive one course of FCM. This course of FCM will consist of two doses of FCM at 15 mg/kg (maximum single dose of 750 mg), separated by seven days.
Ferric carboxymaltose: IV iron | 7 |
| Total | 7 |
Baseline characteristics
| Characteristic | IV Iron |
|---|---|
| Age, Customized Age | 7 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 7 Participants |
| Sex: Female, Male Female | 5 Participants |
| Sex: Female, Male Male | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 7 |
| other Total, other adverse events | 0 / 7 |
| serious Total, serious adverse events | 0 / 7 |
Outcome results
Primary
Change in Hemoglobin From Baseline to Day 35
The change in hemoglobin from Baseline to Day 35 was assessed using paired t-tests (two-sided test, α = 0.05). Baseline hemoglobin was defined as the last hemoglobin obtained before the first dose.
Time frame: The change in Hgb from baseline to day 35
Population: The change in hemoglobin from Baseline to Day 35 was assessed using paired t-tests (two-sided test, α = 0.05). Baseline hemoglobin was defined as the last hemoglobin obtained before the first dose.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| IV Iron | Change in Hemoglobin From Baseline to Day 35 | 0.70 g/dL |
p-value: 0.171195% CI: [-0.4, 1.8]t-test, 2 sided