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The study had initially anticipated dosing of the first cohort within 4-9 months of activation; however the study faced significant challenges in recruitment. In order to adhere to the previously approved PIP plan, the study could not be redesigned.
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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.
To date, there are no clinical studies evaluating rezafungin in paediatric subjects.
The primary objective of the trial was to evaluate the pharmacokinetics (PK) of a single intravenous (IV) dose of rezafungin in paediatric participants from birth to < 18 years, receiving concomitant systemic antifungals as prophylaxis for invasive fungal infection (IFI) or to treat a suspected or confirmed fungal infection.
The secondary objective was to assess the safety and tolerability of a single IV dose of rezafungin in the subjects.
This is a Phase 1, multicentre, open-label, single-dose study. The study will be conducted at approximately 10 sites across at least 3 countries in Europe.
The study will be conducted in 3 parts:
Limitations and Caveats Due to recruitment challenges, the study was terminated following enrollment of 2 participants aged between 12-17 years in Group 1 (Part 1). Group 2, 3 (Part 2) and Group 4 (Part 3) were not initiated prior to study termination. Due to the low number of participants in this study, baseline characteristics, outcome measure results and adverse events have not been reported to reduce the potential of re-identification.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Rezafungin | Experimental | It is IMP. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of treatment-emergent adverse events (TEAEs) | A TEAE was defined as any untoward medical occurrence in a clinical study participant, temporally associated with the use of rezafungin, whether or not considered related to the rezafungin that was not present prior to the administration of rezafungin or any event present that worsened in either severity or frequency following exposure to rezafungin. Clinically significant changes in laboratory evaluations (including haematology, blood chemistry and urinalysis), vital signs, 12-lead electrocardiogram (ECG) and physical examination findings were also reported as TEAEs. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Universitätsklinikum Essen Zentrum für Kinder- und Jugendmedizin | Essen | Germany | ||||
| Universitätsklinikum Frankfurt, Goethe Universität Klinik für Kinder- und Jugendmedizin |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jul 18, 2023 | Mar 13, 2025 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D000072742 | Invasive Fungal Infections |
| ID | Term |
|---|---|
| D009181 | Mycoses |
| D001423 | Bacterial Infections and Mycoses |
| D007239 | Infections |
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| ID | Term |
|---|---|
| C000629634 | Rezafungin |
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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 |
| From start of study on Day 1 to follow up Day 30 (+/- days) |
| Frankfurt |
| Germany |
| Universitätsklinikum Münster Klinik für Kinder- und Jugendmedizin | Münster | Germany |
| Hospital Universitario de Burgos | Burgos | Spain |
| Hospital Universitario 12 de Octubre. | Madrid | Spain |
| Hospital Universitario La Paz | Madrid | Spain |
| Great Ormond Street Hospital for Children NHS Foundation Trust | London | United Kingdom |
| Saint Mary's Hospital, Imperial College Healthcare NHS Trust | London | United Kingdom |
| St. George's University Hospitals, NHS Foundation Trust | London | United Kingdom |
| Southampton General Hospital, University Hospital Southampton NHS Foundation Trust | Southampton | United Kingdom |