CandidemiaPhase 2NCT03604705
An Efficacy and Safety Study of APX001 in Non-Neutropenic Patients With Candidemia
A Phase 2 study testing APX001 in people with Candidemia, run by Basilea Pharmaceutica at 29 sites in 5 countries.
Trial summary
This is a multicenter, open-label, non-comparative, single-arm study to evaluate the efficacy and safety of APX001 for the first-line treatment for candidemia including suspected or confirmed antifungal-resistant candidemia in non-neutropenic patients 18 yeas of age and older. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Candidemia
- What is being tested?
- APX001
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 29 sitesin 5 countries
- Study dates
- 3 Oct 2018to 2 Jul 2020
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Provision of written consent
- Adults ages 18 and above male or female
- New diagnosis of candidemia
- Able to have pre-existing intravascular catheters removed and replaced (as necessary)
Who cannot join
- neutropenia
- deep-seated Candida-related infections
- hepatosplenic candidiasis
- received more than 2 days of prior systemic antifungal treatment for current candidemia episode
- severe hepatic impairment
Trial locations and contacts
29 sites in 5 countries.
29 sites in 5 countries
United States9 sites
Augusta University (Georgia Regents University)
Augusta, Georgia, 30901
University of Alabama at Birmingham School of Medicine
Birmingham, Alabama, 35233
University of Alabama at Birmingham (UAB)
Birmingham, Alabama, 35249
University of Chicago
Chicago, Illinois, 60637
University of California, Davis
Davis, California, 95616
Duke University Hospital Medical Center
Durham, North Carolina, 27710
University of Texas- Health Science Center and Medical School at Houston
Houston, Texas, 77030
University of California-Davis Medical Center
Sacramento, California, 95817
Washington University
St Louis, Missouri, 63110
Belgium7 sites
Institut Jules Bordet,Service De Microbiologie
Brussels, 1000
Hopital Erasme
Brussels, 1070
Institut Jules Bordet
Brussels
Universite Libre de Bruxelles (ULB) - Hopital Erasme
Brussels
CHU de Charleroi - Hopital Civil Marie Curie
Lodelinsart
Mont-Godinne University Hospital
Yvoir, 5530
University Hospital Mont-Godinne
Yvoir
Israel6 sites
Infectious Diseases Unit, Rambam Medical Center
Haifa, 3109601
Rambam Medical Center
Haifa
Infectious Diseases Unit
Tel Aviv, 6423906
Sourasky Medical Center
Tel Aviv
Infectious Diseases Unit,Sheba Medical Center
Tel HaShomer, 52621
Sheba Medical Center
Tel HaShomer
Spain4 sites
Hospital General Universitario de Alicante
Alicante
Hospital Universitari Vall d'Hebron
Barcelona, 08035
Hospital VLL D Hebron
Barcelona
Hospital Universitario Mutua de Terrassa
Terrassa, Barcelona
Germany3 sites
Klinik I fur Innere Medizin- Uniklinik Koln
Cologne
University Hospital Heidelberg
Heidelberg
Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz - III. Medizinische Klinik Haematologie/Onkologie
Mainz
About this study
Official title: An Open-Label Study to Evaluate the Efficacy and Safety of APX001 in Non Neutropenic Patients With Candidemia, With or Without Invasive Candidiasis, Inclusive of Patients With Suspected Resistance to Standard of Care Antifungal Treatment
This is a multicenter, open-label, non-comparative, single-arm study to evaluate the efficacy and safety of APX001 for the first-line treatment for candidemia including suspected or confirmed antifungal-resistant candidemia in non-neutropenic patients 18 yeas of age and older.
Suspicion of antifungal-resistant candidemia is sufficient (documented resistance is not required for enrollment). The Study Drug Treatment Period of APX001 will be a maximum of 14 days. After completion of 14 days study drug therapy, if further antifungal treatment is indicated to complete treatment of candidemia in accordance with standard practice guidelines, fluconazole (unless susceptibility results warrant alternative antifungal therapy) may commence for up to a further 7 days. There will be a Follow up Period of 4 weeks (+4 days) after EOT. The total duration of participation in the study is up to approximately 7.5 weeks.
This study will be conducted at approximately 20 sites in the United States and globally.
- Conditions
- Candidemia
- Being tested
- APX001
- Medical subject headings
- Candidemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
APX001Drug
APX001
| Study group | Role | What they receive |
|---|---|---|
| APX001 Treatment | Experimental | Not described Receives: Drug: APX001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment Success at End of Study Treatment (EOST) as Determined by the Data Review Committee (DRC)
Treatment Success is defined as meeting all of the following criteria:
Two consecutive blood cultures negative for Candida spp. Alive at EOST No concomitant use of any other systemic antifungal therapies through end of study treatment
Measured: One to forty-two days
Secondary outcomes (5)
Time to First Negative Blood Culture
Time to first negative blood culture was defined as the number of days from first dose date of study drug to the date of first post-Baseline negative blood culture + 1. Patients without a negative blood culture at post-Baseline visits were censored at the last assessment date.
Measured: One to forty-nine days
Percentage of Patients With Mycological Outcomes at End of Study Treatment (EOST), End of Treatment (EOT), and 2 and 4 Weeks After End of Treatment (EOT)
Measured: End of study treatment (EOST), end of treatment (EOT), and 2 and 4 weeks after end of treatment (EOT)
Percentage of Patients With Treatment Success at End of Treatment (EOT), and 2 and 4 Weeks After End of Treatment (EOT)
Measured: 2 and 4 weeks after end of treatment (EOT)
Overall Survival at Study Day 30
Measured: Day 30
Number of Patients With Treatment Emergent Adverse Events (TEAEs)
Measured: One to forty-nine days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jul 2018
- Study start3 Oct 2018
- Primary completion31 Mar 2020
- Study completion2 Jul 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for CandidemiaStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Basilea Pharmaceutica
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Marc Engelhardt, Study director, Basilea Pharmaceutica International Ltd, Allschwil
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03604705
- Sponsor's protocol ID
- APX001-201
- Other IDs
- C4791009
- Status last verified
- Sep 2025
- First submitted
- 19 Jul 2018
- Last updated
- 16 Sep 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Pappas PG, Vazquez JA, Oren I, Rahav G, Aoun M, Bulpa P, Ben-Ami R, Ferrer R, Mccarty T, Thompson GR, Schlamm H, Bien PA, Barbat SH, Wedel P, Oborska I, Tawadrous M, Hodges MR. Clinical safety and efficacy of novel antifungal, fosmanogepix, for the treatment of candidaemia: results from a Phase 2 trial. J Antimicrob Chemother. 2023 Oct 3;78(10):2471-2480. doi: 10.1093/jac/dkad256.
PubMed 37596890
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Study to Evaluate the Efficacy and Safety of APX001 in Non Neutropenic Patients With Candidemia, With or Without Invasive Candidiasis, Inclusive of Patients With Suspected Resistance to Standard of Care Antifungal Treatment
- ClinicalTrials.gov ID
- NCT03604705
- Acronym / sponsor ID
- APX001-201
- Conditions
- Candidemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Basilea PharmaceuticaIndustry (pharma, biotech, devices)
- Study start
- 3 Oct 2018 (actual)
- Primary completion
- 31 Mar 2020 (actual)
- Study completion
- 2 Jul 2020 (actual)
- First posted
- 27 Jul 2018 (actual)
- Results
- Posted 5 Aug 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a multicenter, open-label, non-comparative, single-arm study to evaluate the efficacy and safety of APX001 for the first-line treatment for candidemia including suspected or confirmed antifungal-resistant candidemia in non-neutropenic patients 18 yeas of age and older.
Suspicion of antifungal-resistant candidemia is sufficient (documented resistance is not required for enrollment). The Study Drug Treatment Period of APX001 will be a maximum of 14 days. After completion of 14 days study drug therapy, if further antifungal treatment is indicated to complete treatment of candidemia in accordance with standard practice guidelines, fluconazole (unless susceptibility results warrant alternative antifungal therapy) may commence for up to a further 7 days. There will be a Follow up Period of 4 weeks (+4 days) after EOT. The total duration of participation in the study is up to approximately 7.5 weeks.
This study will be conducted at approximately 20 sites in the United States and globally.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| APX001 Treatment | Experimental | Drug: APX001 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Provision of written consent
- Adults ages 18 and above male or female
- New diagnosis of candidemia
- Able to have pre-existing intravascular catheters removed and replaced (as necessary)
Key exclusion criteria (5 listed)
- neutropenia
- deep-seated Candida-related infections
- hepatosplenic candidiasis
- received more than 2 days of prior systemic antifungal treatment for current candidemia episode
- severe hepatic impairment
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 |
|---|---|---|
| APX001APX001 | Drug | APX001 Treatment |
Primary outcome
Treatment Success at End of Study Treatment (EOST) as Determined by the Data Review Committee (DRC)
Time frame: One to forty-two days
Treatment Success is defined as meeting all of the following criteria: Two consecutive blood cultures negative for Candida spp. Alive at EOST No concomitant use of any other systemic antifungal therapies through end of study treatment
5 secondary outcomes
Time to First Negative Blood Culture
Time frame: One to forty-nine days
Percentage of Patients With Mycological Outcomes at End of Study Treatment (EOST), End of Treatment (EOT), and 2 and 4 Weeks After End of Treatment (EOT)
Time frame: End of study treatment (EOST), end of treatment (EOT), and 2 and 4 weeks after end of treatment (EOT)
Percentage of Patients With Treatment Success at End of Treatment (EOT), and 2 and 4 Weeks After End of Treatment (EOT)
Time frame: 2 and 4 weeks after end of treatment (EOT)
Overall Survival at Study Day 30
Time frame: Day 30
Number of Patients With Treatment Emergent Adverse Events (TEAEs)
Time frame: One to forty-nine days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 37596890Pappas PG, Vazquez JA, Oren I, Rahav G, Aoun M, Bulpa P, Ben-Ami R, Ferrer R, Mccarty T, Thompson GR, Schlamm H, Bien PA, Barbat SH, Wedel P, Oborska I, Tawadrous M, Hodges MR. Clinical safety and efficacy of novel antifungal, fosmanogepix, for the treatment of candidaemia: results from a Phase 2 trial. J Antimicrob Chemother. 2023 Oct 3;78(10):2471-2480. doi: 10.1093/jac/dkad256.
- Individual participant dataSharing planned: No
Related trials for Candidemia
RecruitingObservational study
Middle East Study of Infections in Patients With Hematological Malignancies
Hematological Malignancy, Hematopoetic Stem Cell Transplantation
Recruiting
Comparison of Uncomplicated Candidemia Therapy Duration in Children and Adults
Invasive Candidiasis
RecruitingPhase 3
A Phase 3 Efficacy and Safety Study of Fosmanogepix for the Treatment of Adult Participants With Candidemia and/or Invasive Candidiasis.
Candidemia, Candidiasis, Invasive
Not yet recruitingPhase 4
Shortening Treatment Duration in Uncomplicated Candidemia: the CanTEN Trial
Candidemia, Invasive Candidiasis
Not yet recruitingObservational study
A Study Comparing Short-course Antifungal Therapy (SCAT) 7 Day vs Standard 14 Day Antifungal Therapy for Uncomplicated Candidemia
Candidemia
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.
Marc Engelhardt study director
Basilea Pharmaceutica International Ltd, Allschwil
29 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing APX001 in people with Candidemia, run by Basilea Pharmaceutica at 29 sites in 5 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 4 inclusion and 5 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: finished, as of the sponsor's last update on 16 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 29 sites in United States, Belgium, Israel, Spain, Germany. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 5 Aug 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Treatment Success at End of Study Treatment (EOST) as Determined by the Data Review Committee (DRC) (time frame: One to forty-two days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03604705, which also lists 1 linked publication.
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 16 Sep 2025.