Clinical trial NCT04269707: Efficacy and Safety of Intravenous Ferric Carboxymaltose in Pediatric Patients With Iron Deficiency Anemia and Unsatisfactory Response Oral Iron Under Study Protocol 1VIT17044
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Iron Deficiency Anemia
Lead sponsor: American Regent, Inc.
Sponsor class: INDUSTRY
First posted: 2020-02-17
Enrollment: 7
Sex: ALL
Minimum age (years): 1
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
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.
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Change in Hemoglobin From Baseline to Day 35
timeFrame: The change in Hgb from baseline to day 35
Secondary outcomes:
Other outcomes:
Locations:
city: Miami Springs
country: United States
facility: South Florida Research Phase I-IV
state: Florida
city: Miami
country: United States
facility: Miami Clinical Research
state: Florida
Study officials:
affiliation: American Regent, Inc.
name: Mark Falone, MD
role: STUDY_DIRECTOR
References: