Invasive Fungal InfectionsPhase 1NCT05534529
Rezafungin Paediatric PK Study in Paediatric Subjects From Birth to <18 Years of Age
A Phase 1 study testing Rezafungin Acetate in people with Invasive Fungal Infections, run by Mundipharma Research Limited at 10 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 17 years
- Conditions the study is for
- Invasive Fungal Infections
- What is being tested?
- Rezafungin Acetate
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 10 sitesin 3 countries
- Study dates
- 13 Sep 2023to 14 Oct 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
10 sites in 3 countries.
10 sites in 3 countries
United Kingdom4 sites
Great Ormond Street Hospital for Children NHS Foundation Trust
London
Saint Mary's Hospital, Imperial College Healthcare NHS Trust
London
St. George's University Hospitals, NHS Foundation Trust
London
Southampton General Hospital, University Hospital Southampton NHS Foundation Trust
Southampton
Germany3 sites
Universitätsklinikum Essen Zentrum für Kinder- und Jugendmedizin
Essen
Universitätsklinikum Frankfurt, Goethe Universität Klinik für Kinder- und Jugendmedizin
Frankfurt
Universitätsklinikum Münster Klinik für Kinder- und Jugendmedizin
Münster
Spain3 sites
Hospital Universitario de Burgos
Burgos
Hospital Universitario 12 de Octubre.
Madrid
Hospital Universitario La Paz
Madrid
About this study
Official title: A Phase 1, Multicentre, Open-Label Study to Evaluate the PK, Safety, and Tolerability of a Single IV Dose of Rezafungin in Paediatric Subjects, Receiving Systemic Antifungals as Prophylaxis for IFI or to Treat a Suspected or Confirmed FI
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:
Read more
- 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.
Read the detailed description
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:
- Part 1 will include subjects aged 12 to <18 years (Group 1)
- Part 2 will include subjects aged 6 to <12 years (Group 2), and subjects aged 2 to <6 years (Group 3).
- Part 3 will include subjects from birth to <2 years (Group 4) The study design for the 3 parts is similar and comprises a Screening (pre-treatment) period from Day -3 to Day -1, Dosing on Day 1 (single IV infusion of rezafungin) followed by multiple PK sampling, and a Follow up visit on Day 30 (± 5 days). PK sampling will be performed at specified timepoints for each group.
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.
- Conditions
- Invasive Fungal Infections
- Being tested
- Rezafungin Acetate
- Keywords
- Rezafungin, children and adolescents, systemic anti-fungal treatment, Pharmacokinetics
- Medical subject headings
- Invasive Fungal Infections
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Rezafungin AcetateDrug
Also called Rezafungin
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.
| Study group | Role | What they receive |
|---|---|---|
| Rezafungin | Experimental | It is IMP. Receives: Drug: Rezafungin Acetate |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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)
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.
Measured: From start of study on Day 1 to follow up Day 30 (+/- days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2022
- Study start13 Sep 2023
- Primary completion23 May 2024
- Study completion14 Oct 2024
Why it stopped: 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.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Invasive Fungal InfectionsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mundipharma Research Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05534529
- Sponsor's protocol ID
- MR907-1501
- Status last verified
- Apr 2025
- First submitted
- 1 Sep 2022
- Last updated
- 6 Apr 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Multicentre, Open-Label Study to Evaluate the PK, Safety, and Tolerability of a Single IV Dose of Rezafungin in Paediatric Subjects, Receiving Systemic Antifungals as Prophylaxis for IFI or to Treat a Suspected or Confirmed FI
- ClinicalTrials.gov ID
- NCT05534529
- Acronym / sponsor ID
- MR907-1501
- Conditions
- Invasive Fungal Infections
- Keywords
- Rezafungin, children and adolescents, systemic anti-fungal treatment, Pharmacokinetics
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: 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.
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Mundipharma Research LimitedIndustry (pharma, biotech, devices)
- Study start
- 13 Sep 2023 (actual)
- Primary completion
- 23 May 2024 (actual)
- Study completion
- 14 Oct 2024 (actual)
- First posted
- 9 Sep 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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:
- Part 1 will include subjects aged 12 to <18 years (Group 1)
- Part 2 will include subjects aged 6 to <12 years (Group 2), and subjects aged 2 to <6 years (Group 3).
- Part 3 will include subjects from birth to <2 years (Group 4) The study design for the 3 parts is similar and comprises a Screening (pre-treatment) period from Day -3 to Day -1, Dosing on Day 1 (single IV infusion of rezafungin) followed by multiple PK sampling, and a Follow up visit on Day 30 (± 5 days). PK sampling will be performed at specified timepoints for each group.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Rezafungin | Experimental | Drug: Rezafungin AcetateIt is IMP. |
Who may qualify
- Ages
- Up to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- 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.
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Rezafungin AcetateAlso: RezafunginThis 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. | Drug | Rezafungin |
Primary outcome
Maximum Observed Plasma Concentration (Cmax) of Rezafungin
Time frame: 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
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.
Time at which the Cmax of Rezafungin Was Observed (Tmax)
Time frame: End of infusion and 3-4, 6-8, 48, and 168 hours after start of infusion
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.
Area Under the Plasma Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-t) of Rezafungin
Time frame: End of infusion and 3-4, 6-8, 48, and 168 hours after start of infusion
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.
Area Under the Plasma Concentration-time Curve From Time 0 Extrapolated to Infinity (AUC0-∞) of Rezafungin
Time frame: End of infusion and 3-4, 6-8, 48, and 168 hours after start of infusion
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.
Total Clearance (CL) of Rezafungin
Time frame: End of infusion and 3-4, 6-8, 48, and 168 hours after start of infusion
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.
Volume of Distribution of Rezafungin at Steady-state (Vss)
Time frame: End of infusion and 3-4, 6-8, 48, and 168 hours after start of infusion
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.
Apparent Volume of Distribution of Rezafungin During the Terminal Phase (Vz)
Time frame: End of infusion and 3-4, 6-8, 48, and 168 hours after start of infusion
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.
Terminal Elimination Half-life of Rezafungin (t1/2)
Time frame: End of infusion and 3-4, 6-8, 48, and 168 hours after start of infusion
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.
1 secondary outcome
Incidence of treatment-emergent adverse events (TEAEs)
Time frame: From start of study on Day 1 to follow up Day 30 (+/- days)
Results and safety
The study was stopped early (reason given: 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.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Apr 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
10 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Rezafungin Acetate in people with Invasive Fungal Infections, run by Mundipharma Research Limited at 10 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 6 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United Kingdom, Germany, Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: 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.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum Observed Plasma Concentration (Cmax) of Rezafungin (time frame: 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). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Apr 2025.