Rezafungin Paediatric PK Study in Paediatric Subjects From Birth to <18 Years of Age

NCT05534529 | Terminated Phase 1 DMC

• 1 other identifier: MR907-1501
• Study Type: interventional
• Target: 32
• Locations: 3 countries
• Sites: 10

Brief Summary

This study aimed to learn what levels of rezafungin were in the blood after dosing and how safe it was, in children and adolescents below 18 years old who were already receiving treatment for a fungal infection, a suspected fungal infection or at risk of fungal infection. The main question the researchers wanted to answer in this trial was: • What were the levels of rezafungin in the blood after the participants were dosed? The researchers also wanted to know what medical problems happened during this trial. The participants in this trial received one dose of rezafungin on day 1 through a needle into a vein, called an intravenous (IV) infusion. The dose of rezafungin was measured in milligrams (mg) and given to the participants according to their body weight in kilograms (mg/kg). The doctors checked the participants' health and asked questions about what medications they were taking and took blood samples to check the levels of rezafungin in the participants' blood. After receiving the treatment at day 30, the doctors checked the participants' health. This was an "open-label" trial. This means each participant knew what they were receiving, and the doctors and trial staff also knew.

Conditions

Invasive Fungal Infections

Keywords

Rezafungin, children and adolescents, systemic anti-fungal treatment; Pharmacokinetics

Outcome Measures

Primary Outcomes (8)

• Maximum Observed Plasma Concentration (Cmax) of Rezafungin

  Blood samples of 1 mL each were collected for the analysis of rezafungin plasma concentration. PK parameters were determined from the rezafungin concentration-time data using non-compartmental methods and actual sampling times.

  At end-of-infusion ± 15 minutes, then between 3 and 4 hours after start of infusion, between 6 and 8 hours after start of infusion, at 48 hours (± 4 hours) after start of infusion, and at 168 hours (± 12 hours) after start of infusion

• Time at which the Cmax of Rezafungin Was Observed (Tmax)

  Blood samples of 1 mL each were collected for the analysis of rezafungin plasma concentration. PK parameters were determined from the rezafungin concentration-time data using non-compartmental methods and actual sampling times.

  End of infusion and 3-4, 6-8, 48, and 168 hours after start of infusion

• Area Under the Plasma Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-t) of Rezafungin

  Blood samples of 1 mL each were collected for the analysis of rezafungin plasma concentration. PK parameters were determined from the rezafungin concentration-time data using non-compartmental methods and actual sampling times.

  End of infusion and 3-4, 6-8, 48, and 168 hours after start of infusion

• Area Under the Plasma Concentration-time Curve From Time 0 Extrapolated to Infinity (AUC0-∞) of Rezafungin

  Blood samples of 1 mL each were collected for the analysis of rezafungin plasma concentration. PK parameters were determined from the rezafungin concentration-time data using non-compartmental methods and actual sampling times.

  End of infusion and 3-4, 6-8, 48, and 168 hours after start of infusion

• Total Clearance (CL) of Rezafungin

  Blood samples of 1 mL each were collected for the analysis of rezafungin plasma concentration. PK parameters were determined from the rezafungin concentration-time data using non-compartmental methods and actual sampling times.

  End of infusion and 3-4, 6-8, 48, and 168 hours after start of infusion

• Volume of Distribution of Rezafungin at Steady-state (Vss)

  Blood samples of 1 mL each were collected for the analysis of rezafungin plasma concentration. PK parameters were determined from the rezafungin concentration-time data using non-compartmental methods and actual sampling times.

  End of infusion and 3-4, 6-8, 48, and 168 hours after start of infusion

• Apparent Volume of Distribution of Rezafungin During the Terminal Phase (Vz)

  Blood samples of 1 mL each were collected for the analysis of rezafungin plasma concentration. PK parameters were determined from the rezafungin concentration-time data using non-compartmental methods and actual sampling times.

  End of infusion and 3-4, 6-8, 48, and 168 hours after start of infusion

• Terminal Elimination Half-life of Rezafungin (t1/2)

  Blood samples of 1 mL each were collected for the analysis of rezafungin plasma concentration. PK parameters were determined from the rezafungin concentration-time data using non-compartmental methods and actual sampling times.

  End of infusion and 3-4, 6-8, 48, and 168 hours after start of infusion

Secondary Outcomes (1)

• Incidence of treatment-emergent adverse events (TEAEs)

  From start of study on Day 1 to follow up Day 30 (+/- days)

Study Arms (1)

Rezafungin

EXPERIMENTAL

It is IMP.

Drug: Rezafungin Acetate

Interventions

Rezafungin Acetate DRUG

This is a Phase 1, multicentre, open-label, single-dose study. The study will be conducted at 10 sites across at least 3 countries in Europe.

Also known as: Rezafungin

Rezafungin

Eligibility Criteria

• Age: Up to 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Male or female paediatric subjects from birth to \<18 years of age who are receiving concomitant systemic antifungals (oral or IV) as prophylaxis for invasive fungal infection (IFI) or to treat a suspected or confirmed fungal infection.

You may not qualify if:

• History of anaphylaxis, hypersensitivity, or any serious reaction to the echinocandin class of antifungals and/or excipients of this formulation
• Previous or current medical conditions of severe ataxia, persistent tremors, intracranial hemorrhage or neuropathy, or a diagnosis of epilepsy, multiple sclerosis, or a movement disorder
• Subjects with impaired renal or hepatic functions
• Subjects with intestinal hypoxia, ischemia, necrosis, or necrotizing enterocolitis
• Subject status is unstable
• Subject is unlikely to complete required study procedures
• Participation in another interventional treatment trial with an investigational agent or presence of an investigational device at the time of informed consent or within 28 days preceding the informed consent.

Sponsors & Collaborators

• Mundipharma Research Limited: lead

Study Sites (10)

Universitätsklinikum Essen Zentrum für Kinder- und Jugendmedizin

Essen, Germany

Location

Universitätsklinikum Frankfurt, Goethe Universität Klinik für Kinder- und Jugendmedizin

Frankfurt, Germany

Location

Universitätsklinikum Münster Klinik für Kinder- und Jugendmedizin

Münster, Germany

Location

Hospital Universitario de Burgos

Burgos, Spain

Location

Hospital Universitario 12 de Octubre.

Madrid, Spain

Location

Hospital Universitario La Paz

Madrid, Spain

Location

Great Ormond Street Hospital for Children NHS Foundation Trust

London, United Kingdom

Location

Saint Mary's Hospital, Imperial College Healthcare NHS Trust

London, United Kingdom

Location

St. George's University Hospitals, NHS Foundation Trust

London, United Kingdom

Location

Southampton General Hospital, University Hospital Southampton NHS Foundation Trust

Southampton, United Kingdom

Location

MeSH Terms

Conditions

Invasive Fungal Infections

Interventions

Rezafungin

Condition Hierarchy (Ancestors)

Mycoses; Bacterial Infections and Mycoses; Infections

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 1, 2022
• First Posted: September 9, 2022
• Study Start: September 13, 2023
• Primary Completion: May 23, 2024
• Study Completion: October 14, 2024
• Last Updated: April 6, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

ES (3); GB (4); DE (3)