Clinical trial NCT05590884: Gadopiclenol Pharmacokinetics, Safety and Efficacy in Children < 2 Years of Age
Official title: Gadopiclenol Pharmacokinetics, Safety and Efficacy in Pediatric Patients < 2 Years of Age
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Central Nervous System Diseases
Pediatric Disorder
Body Indication
Lead sponsor: Guerbet
Sponsor class: INDUSTRY
First posted: 2022-10-21
Enrollment: 36
Sex: ALL
Maximum age (years): 23
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: DIAGNOSTIC

Brief summary: This Phase II open-label, uncontrolled, multicenter trial is designed to investigate the pharmacokinetic (PK) profile of gadopiclenol in plasma, in pediatric patients aged up to 23 months inclusive (term neonates or preterm infants after the neonatal period), using a population PK approach.

Primary objective is to evaluate the PK profile of gadopiclenol in plasma following single intravenous injection of 0.05 mmol/kg body weight (BW) in pediatric population aged up to 23 months (inclusive) scheduled for a contrast-enhanced MRI examination of any body region including central nervous system (CNS).

Detailed description: Three age groups are defined:

* Group 1: patients aged 3 to 23 months (inclusive)
* Group 2: patients aged 28 days to less than 3 months;
* Group 3: patients aged from birth to 27 days (term newborns).

At study set-up, the inclusions started with the oldest patients in group 1 and using an age-down staggered approach.

The decision to start the inclusions in group 2 was taken by the Trial Safety Review Board based on safety assessment over one-day period after injection of the first 13 patients in group 1.

According to the protocol version 4 (amendments 2 \& 3), the age-down staggered approach was discontinued to allow the inclusions in Group 3 (patients aged from birth to 27 days), simultaneously to inclusions in group 1 and group 2.

The inclusions in the three age groups will be completed in parallel. A total of 3 blood samples per patient will be taken post-injection for PK analysis.

Eligibility criteria: Main Inclusion Criteria:

1. Female or male pediatric patient aged from birth to 23 months of age inclusive (term neonates for all age groups or preterm infants after the neonatal period for groups 1 or 2). The neonatal period for preterm newborns is defined as the day of birth through the expected date of delivery plus 27 days. Term is defined as ≥37 completed weeks of amenorrhea,
2. Patient with known or highly suspected abnormalities/ lesion(s), scheduled to undergo contrast-enhanced MRI of any body region including CNS \[...\]

Main Exclusion Criteria:

1. Patient planned for treatment or procedure (e.g. surgery) that would prevent from obtaining the required blood samples or performing other trial procedures between the screening visit and up to one day after gadopiclenol administration,
2. Patient undergoing treatment or procedure (e.g., diuretics, clinically significant blood loss or blood transfusion) preceding or subsequent to gadopiclenol administration that would alter gadopiclenol pharmacokinetic parameters,
3. Patient with acute or chronic renal insufficiency defined as estimated Glomerular Filtration Rate (eGFR) out of age-adjusted normal value calculated based on bedside Schwartz equation, \[...\]

9. Patient undergoing a change in chemotherapy (product or dosage) within one day prior to or one day after gadopiclenol administration, 10. Patient who received or will receive any other contrast agent for CT and/or MRI within one week prior to or one week after gadopiclenol administration, 11. Patient with contraindication for MRI such as iron metal implants (e.g., aneurysm clips, pacemaker), 12. Patient with history of anaphylactoid or anaphylactic reaction to any allergen including drugs and contrast agents \[...\]

Primary outcomes:
measure: Simulated concentrations at 10 minutes post injection
timeFrame: Concentration is simulated at 10 minutes post injection
measure: Simulated concentrations at 20 minutes post injection
timeFrame: Concentration is simulated at 20 minutes post injection
measure: Simulated concentrations at 30 minutes post injection
timeFrame: Concentration is simulated at 30 minutes post injection
measure: Area Under the Curve
timeFrame: A total of 3 blood samples per patient will be taken post-injection for PK analysis, one within each window (10-60 minutes, 2-4 hours and 6-8 hours).
measure: Elimination half-life
timeFrame: A total of 3 blood samples per patient will be taken post-injection for PK analysis, one within each window (10-60 minutes, 2-4 hours and 6-8 hours).
measure: Total clearance
timeFrame: A total of 3 blood samples per patient will be taken post-injection for PK analysis, one within each window (10-60 minutes, 2-4 hours and 6-8 hours).
measure: Volume of distribution
timeFrame: A total of 3 blood samples per patient will be taken post-injection for PK analysis, one within each window (10-60 minutes, 2-4 hours and 6-8 hours).
Secondary outcomes:
Other outcomes:
Locations:
city: Cincinnati
country: United States
facility: Cincinnatti Childrens Hospital
state: Ohio
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Budapest
country: Hungary
facility: Semmelweis Egyetem
city: Budapest
country: Hungary
facility: Országos Idegtudományi Intézet
city: Debrecen
country: Hungary
facility: University of Debrecen Clinical Center Pediatric Department
city: Bydgoszcz
country: Poland
facility: Szpital Uniwersytecki nr 1 im. dr Antoniego Jurasza
city: Lodz
country: Poland
facility: Instytut Centrum Zdrowia Matki Polki
city: Lublin
country: Poland
facility: Uniwersytecki Szpital Dzieciecy
city: Rzeszów
country: Poland
facility: Kliniczny Szpital Wojewodzki Nr 2 im. Sw. Jadwigi Krolowej
city: Warszawa
country: Poland
facility: Uniwersyteckie Centrum Kliniczne Warszawskiego
city: Warszawa
country: Poland
facility: Instytut "Pomnik - Centrum Zdrowia Dziecka"
Study officials:
affiliation: Medical University of South Carolina, Division of Neuroradiology
name: Donna R Roberts, MD
role: PRINCIPAL_INVESTIGATOR
References: