MedPath

Gadopiclenol Pharmacokinetics, Safety and Efficacy in Children < 2 Years of Age

Phase 2
Completed
Conditions
Central Nervous System Diseases
Pediatric Disorder
Body Indication
Interventions
Registration Number
NCT05590884
Lead Sponsor
Guerbet
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.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
36
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, [...]

  4. 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 [...]

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Age Group 1: patients aged 3 to 23 monthsGadopiclenolOne Investigational Medicinal Product (IMP) dose of 0.05 mmol/kg will be injected in all patients.
Age Group 3: patients aged from birth to 27 days (term newborns)GadopiclenolOne Investigational Medicinal Product (IMP) dose of 0.05 mmol/kg will be injected in all patients.
Age Group 2: patients aged 28 days to less than 3 monthsGadopiclenolOne Investigational Medicinal Product (IMP) dose of 0.05 mmol/kg will be injected in all patients.
Primary Outcome Measures
NameTimeMethod
Simulated concentrations at 10 minutes post injectionConcentration is simulated at 10 minutes post injection

Determined from population PK

Simulated concentrations at 20 minutes post injectionConcentration is simulated at 20 minutes post injection

Determined from population PK

Simulated concentrations at 30 minutes post injectionConcentration is simulated at 30 minutes post injection

Determined from population PK

Area Under the CurveA 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).

Determined from population PK

Elimination half-lifeA 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).

Determined from population PK

Total clearanceA 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).

Determined from population PK

Volume of distributionA 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).

Determined from population PK

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (11)

Országos Idegtudományi Intézet

🇭🇺

Budapest, Hungary

Semmelweis Egyetem

🇭🇺

Budapest, Hungary

Szpital Uniwersytecki nr 1 im. dr Antoniego Jurasza

🇵🇱

Bydgoszcz, Poland

Uniwersytecki Szpital Dzieciecy

🇵🇱

Lublin, Poland

Instytut "Pomnik - Centrum Zdrowia Dziecka"

🇵🇱

Warszawa, Poland

University of Debrecen Clinical Center Pediatric Department

🇭🇺

Debrecen, Hungary

Uniwersyteckie Centrum Kliniczne Warszawskiego

🇵🇱

Warszawa, Poland

Instytut Centrum Zdrowia Matki Polki

🇵🇱

Lodz, Poland

Kliniczny Szpital Wojewodzki Nr 2 im. Sw. Jadwigi Krolowej

🇵🇱

Rzeszów, Poland

Cincinnatti Childrens Hospital

🇺🇸

Cincinnati, Ohio, United States

Medical University of South Carolina

🇺🇸

Charleston, South Carolina, United States

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