Clinical trial NCT04968379: Safety, Tolerability, PK and PD of Intravenous Ferric Carboxymaltose in Infants With Iron Deficiency Anemia
Official title: An Open-Label, Multi-Center Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intravenous Ferric Carboxymaltose (FCM) in Infants (0-1 Year) With Iron Deficiency Anemia
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
Conditions: Iron Deficiency, Anaemia
Lead sponsor: American Regent, Inc.
Sponsor class: INDUSTRY
First posted: 2021-07-20
Enrollment: 0
Sex: ALL
Maximum age (years): 1
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: An Open-Label, Multi-Center Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intravenous Ferric Carboxymaltose (FCM) in Infants (0-1 year) with Iron Deficiency Anemia.

Detailed description: A phase II, open-label, multi-center study with 2 Cohorts to evaluate the safety, tolerance, PK, and PD profile of intravenous (IV) FCM in infants 0 to 1 year of age with IDA after receiving either a 5.0 mg/kg or 7.5 mg/kg dose of FCM.

Participants will have a screening evaluation within 14 days of the first dose of study drug. A medically supervised environment is required on Day 1 (day of dosing) and for 4 hours post dosing. Participants are allowed to be enrolled if satisfying the inclusion and exclusion criteria. Participants will return to the study site for additional evaluation and sampling on Days 8 (± 2 days), 15 (± 2 days), 22 (± 2 days), and 36 (± 2 days).

Eligibility criteria: Inclusion Criteria:

1. Male and female participants 0 to 1 year of age, medically indicated for iron replacement, with his/her parent or legal guardian willing and able to sign the informed consent form approved by the IRB / Independent Ethics Committee (IEC).
2. Screening Hb ≥7 g/dL to \<10 g/dL.
3. Infants with any of the following conditions:

   * Heart failure with IDA defined as syndromes of excessive preload, excessive afterload, abnormal rhythm, or decreased contractility
   * Gastrointestinal diseases with acquired short bowel syndrome (due to volvulus, necrotizing enterocolitis from surgical resection or spontaneous intestinal perforation)
   * Gastrointestinal intolerance of oral iron or an unsatisfactory response to oral iron
   * Other conditions associated with IDA which in the opinion of the investigator might benefit from administration of FCM

Exclusion Criteria:

1. Known history of hypersensitivity reaction to FCM.
2. Body weight \<2.5 kg.
3. History of acquired iron overload, hemochromatosis, or other iron accumulation disorders.
4. Hemodialysis-dependent chronic kidney disease.
5. History of significant diseases of the liver, hematopoietic system, cardiovascular system, or other conditions which, on the opinion of the investigator, may place a participant at added risk for participation in the study.
6. Active infection.
7. Anemia due to reasons other than iron deficiency (e.g., hemoglobinopathy vitamin B12 deficiency, or folic acid deficiency).
8. Blood transfusion in the 4 weeks prior to consent.
9. Administration of an iron-containing product within 14 days of administration of the study article.
10. Administration and / or use of an investigational product (drug or device) within 30 days of screening.
11. Current participation in another clinical trial.
12. Unable to comply with study procedures and assessments.

Primary outcomes:
measure: Treatment-emergent adverse events
timeFrame: Baseline to Day 36
measure: Change in hemoglobin (Hb): g/dL
timeFrame: baseline to Days 8, 15, 22, and 36
measure: Change in reticulocytes count: %
timeFrame: baseline to Days 8, 15, 22, and 36
measure: Evaluate the PD parameters - Change in serum iron: mcg/dL
timeFrame: baseline to Day 36
measure: Evaluate the PD parameters - Change in serum ferritin: ng/mL
timeFrame: baseline to Day 36
measure: Evaluate the PD parameters - Change in total iron binding capacity [TIBC]): mcg/dL
timeFrame: baseline to Day 36
measure: Evaluate the PD parameters - Change in serum transferrin saturation [TSAT]): mg/dL
timeFrame: baseline to Day 36
Secondary outcomes:
Other outcomes:
Locations:
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: New Hyde Park
country: United States
facility: Cohen Children's Medical Center
state: New York
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Philadelphia
country: United States
facility: St. Christopher's Hospital for Children
state: Pennsylvania
Study officials:
affiliation: American Regent
name: Mark Falone, MD
role: STUDY_DIRECTOR
References: