Fungal InfectionPhase 1NCT02956499
Study to Assess Safety, Tolerability and Pharmacokinetics of APX001 Administered by Intravenous Infusion to Healthy Subjects
A Phase 1 study testing APX001 single dose 1, APX001 single dose 2 and APX001 single dose 3 in people with Fungal Infection, run by Basilea Pharmaceutica at 2 sites in Netherlands.
Trial summary
First In Human (FIH), randomized, double-blind, placebo-controlled single ascending dose (SAD) and multiple ascending dose (MAD) escalation study of approximately 80 subjects. The SAD portion of the study will enroll six cohorts of eight healthy subjects per cohort, for a total of approximately 48 healthy subjects. Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Fungal Infection
- What is being tested?
- APX001 single dose 1, APX001 single dose 2 and APX001 single dose 3
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 200 participants
- Where
- 2 sitesNetherlands
- Study dates
- 24 May 2016to 3 Jul 2017
- 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 18 to 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Women of childbearing potential must agree to avoid pregnancy during the study and to use contraception at least 2 weeks before the start of the study until 3 months after the last dose of study drug.
- Males with partner(s) of childbearing potential must agree to use appropriate barrier contraception from the screening period until 3 months after the last dose of study drug.
- Screening hematology, clinical chemistry, coagulation and urinalysis consistent with overall good health.
- No significantly abnormal findings on physical examination, ECG and vital signs.
- Willing and able to provide written informed consent.
Who cannot join
- Any uncontrolled or active major systemic disease including, but not limited to: cardiovascular, pulmonary, gastrointestinal, metabolic, urogenital, neurological, immunological, psychiatric, or neoplastic disorder with metastatic potential.
- History or presence of malignancy within the past year. Subjects who have been successfully treated with no recurrence of basal cell carcinoma of the skin or carcinoma in-situ of the cervix may be enrolled.
- Use of prescription medication within 14 days prior to the first dose of study drug and throughout the study.
- Use of non-prescription or over-the-counter medications within 7 days prior to the first dose of study drug and throughout the study.
- Positive results on any of the following Screening laboratory tests: serum pregnancy test, urine alcohol test, urine drugs of abuse, hepatitis B surface antigen, hepatitis C antibody, and human immunodeficiency virus (HIV) antibody.
Trial locations and contacts
2 sites in 1 country.
Netherlands2 sites
PRA Health Sciences
Groningen, 9728 NZ
PRA Health Sciences
Groningen
About this study
Official title: A First-in-Human, Randomized, Dose-Escalation, Double-Blind, Placebo-Controlled Study to Assess Safety, Tolerability and Pharmacokinetics of APX001 Administered by Intravenous Infusion to Healthy Subjects
First In Human (FIH), randomized, double-blind, placebo-controlled single ascending dose (SAD) and multiple ascending dose (MAD) escalation study of approximately 80 subjects. The SAD portion of the study will enroll six cohorts of eight healthy subjects per cohort, for a total of approximately 48 healthy subjects. The MAD portion of the study will enroll four cohorts of eight healthy subjects per cohort, for a total of approximately 32 healthy subjects.
- Conditions
- Fungal Infection
- Being tested
- APX001 single dose 1, APX001 single dose 2, APX001 single dose 3, APX001 single dose 4, APX001 single dose 5
- Medical subject headings
- Mycoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
APX001 single dose 1Drug
APX001 single dose 2Drug
APX001 single dose 3Drug
APX001 single dose 4Drug
APX001 single dose 5Drug
APX001 single dose 6Drug
APX001 multiple dose 1Drug
APX001 multiple dose 2Drug
APX001 multiple dose 3Drug
APX001 multiple dose 4Drug
Matching PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | single intravenous dose Receives: Drug: APX001 single dose 1; Drug: Matching Placebo |
| Cohort 2 | Experimental | single intravenous dose Receives: Drug: APX001 single dose 2; Drug: Matching Placebo |
| Cohort 3 | Experimental | single intravenous dose Receives: Drug: APX001 single dose 3; Drug: Matching Placebo |
| Cohort 4 | Experimental | single intravenous dose Receives: Drug: APX001 single dose 4; Drug: Matching Placebo |
| Cohort 5 | Experimental | single intravenous dose Receives: Drug: APX001 single dose 5; Drug: Matching Placebo |
| Cohort 6 | Experimental | single intravenous dose Receives: Drug: APX001 single dose 6; Drug: Matching Placebo |
| Cohort 7 | Experimental | multiple intravenous doses Receives: Drug: APX001 multiple dose 1; Drug: Matching Placebo |
| Cohort 8 | Experimental | multiple intravenous doses Receives: Drug: APX001 multiple dose 2; Drug: Matching Placebo |
| Cohort 9 | Experimental | multiple intravenous doses Receives: Drug: APX001 multiple dose 3; Drug: Matching Placebo |
| Cohort 10 | Experimental | multiple intravenous doses Receives: Drug: APX001 multiple dose 4; Drug: Matching Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of single and multiple doses of APX001 as measured by adverse events (AEs), physical examinations (PE), vital signs (VS), laboratory safety tests, urinalysis and 12-lead electrocardiograms (ECG).
Measured: 21 days
Secondary outcomes (6)
Pharmacokinetics of single and multiple doses of APX001 as measured by maximum observed concentration (Cmax).
Measured: 21 days
Pharmacokinetics of single and multiple doses of APX001 as measured by area under the curve (AUC).
Measured: 21 days
Pharmacokinetics of single and multiple doses of APX001 as measured by terminal phase half-life (t1/2).
Measured: 21 days
Pharmacokinetics of single and multiple doses of APX001 as measured by volume of distribution (Vd).
Measured: 21 days
Pharmacokinetics of single and multiple doses of APX001 as measured by elimination rate constant (Kel).
Measured: 21 days
Pharmacokinetics of single and multiple dose of APX001 as measured by accumulation ratio.
Measured: 21 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2016 (estimated)
- Study start24 May 2016
- Primary completion3 Jul 2017
- Study completion3 Jul 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Fungal InfectionStudies 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
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 200 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
- NCT02956499
- Sponsor's protocol ID
- APX001-101
- Other IDs
- C4791001
- Status last verified
- Sep 2025
- First submitted
- 1 Nov 2016
- Last updated
- 10 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Hodges MR, Ople E, Wedel P, Shaw KJ, Jakate A, Kramer WG, Marle SV, van Hoogdalem EJ, Tawadrous M. Safety and Pharmacokinetics of Intravenous and Oral Fosmanogepix, a First-in-Class Antifungal Agent, in Healthy Volunteers. Antimicrob Agents Chemother. 2023 Apr 18;67(4):e0162322. doi: 10.1128/aac.01623-22. Epub 2023 Mar 29.
PubMed 36988461
Study at a glance
From the registry record, last updated by the sponsor on 10 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A First-in-Human, Randomized, Dose-Escalation, Double-Blind, Placebo-Controlled Study to Assess Safety, Tolerability and Pharmacokinetics of APX001 Administered by Intravenous Infusion to Healthy Subjects
- ClinicalTrials.gov ID
- NCT02956499
- Acronym / sponsor ID
- APX001-101
- Conditions
- Fungal Infection
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 200 participants (actual)
- Lead sponsor
- Basilea PharmaceuticaIndustry (pharma, biotech, devices)
- Study start
- 24 May 2016 (actual)
- Primary completion
- 3 Jul 2017 (actual)
- Study completion
- 3 Jul 2017 (actual)
- First posted
- 7 Nov 2016 (estimated)
- 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.
First In Human (FIH), randomized, double-blind, placebo-controlled single ascending dose (SAD) and multiple ascending dose (MAD) escalation study of approximately 80 subjects. The SAD portion of the study will enroll six cohorts of eight healthy subjects per cohort, for a total of approximately 48 healthy subjects. The MAD portion of the study will enroll four cohorts of eight healthy subjects per cohort, for a total of approximately 32 healthy subjects.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind (participant, investigator)
- Number of arms
- 10
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Drug: APX001 single dose 1, Drug: Matching Placebosingle intravenous dose |
| Cohort 2 | Experimental | Drug: APX001 single dose 2, Drug: Matching Placebosingle intravenous dose |
| Cohort 3 | Experimental | Drug: APX001 single dose 3, Drug: Matching Placebosingle intravenous dose |
| Cohort 4 | Experimental | Drug: APX001 single dose 4, Drug: Matching Placebosingle intravenous dose |
| Cohort 5 | Experimental | Drug: APX001 single dose 5, Drug: Matching Placebosingle intravenous dose |
| Cohort 6 | Experimental | Drug: APX001 single dose 6, Drug: Matching Placebosingle intravenous dose |
| Cohort 7 | Experimental | Drug: APX001 multiple dose 1, Drug: Matching Placebomultiple intravenous doses |
| Cohort 8 | Experimental | Drug: APX001 multiple dose 2, Drug: Matching Placebomultiple intravenous doses |
| Cohort 9 | Experimental | Drug: APX001 multiple dose 3, Drug: Matching Placebomultiple intravenous doses |
| Cohort 10 | Experimental | Drug: APX001 multiple dose 4, Drug: Matching Placebomultiple intravenous doses |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- Women of childbearing potential must agree to avoid pregnancy during the study and to use contraception at least 2 weeks before the start of the study until 3 months after the last dose of study drug.
- Males with partner(s) of childbearing potential must agree to use appropriate barrier contraception from the screening period until 3 months after the last dose of study drug.
- Screening hematology, clinical chemistry, coagulation and urinalysis consistent with overall good health.
- No significantly abnormal findings on physical examination, ECG and vital signs.
- Willing and able to provide written informed consent.
Key exclusion criteria (5 listed)
- Any uncontrolled or active major systemic disease including, but not limited to: cardiovascular, pulmonary, gastrointestinal, metabolic, urogenital, neurological, immunological, psychiatric, or neoplastic disorder with metastatic potential.
- History or presence of malignancy within the past year. Subjects who have been successfully treated with no recurrence of basal cell carcinoma of the skin or carcinoma in-situ of the cervix may be enrolled.
- Use of prescription medication within 14 days prior to the first dose of study drug and throughout the study.
- Use of non-prescription or over-the-counter medications within 7 days prior to the first dose of study drug and throughout the study.
- Positive results on any of the following Screening laboratory tests: serum pregnancy test, urine alcohol test, urine drugs of abuse, hepatitis B surface antigen, hepatitis C antibody, and human immunodeficiency virus (HIV) antibody.
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 |
|---|---|---|
| APX001 single dose 1 | Drug | Cohort 1 |
| APX001 single dose 2 | Drug | Cohort 2 |
| APX001 single dose 3 | Drug | Cohort 3 |
| APX001 single dose 4 | Drug | Cohort 4 |
| APX001 single dose 5 | Drug | Cohort 5 |
| APX001 single dose 6 | Drug | Cohort 6 |
| APX001 multiple dose 1 | Drug | Cohort 7 |
| APX001 multiple dose 2 | Drug | Cohort 8 |
| APX001 multiple dose 3 | Drug | Cohort 9 |
| APX001 multiple dose 4 | Drug | Cohort 10 |
| Matching Placebo | Drug | Cohort 1, Cohort 10, Cohort 2, Cohort 3, Cohort 4, Cohort 5, Cohort 6, Cohort 7, Cohort 8, Cohort 9 |
Primary outcome
Safety and tolerability of single and multiple doses of APX001 as measured by adverse events (AEs), physical examinations (PE), vital signs (VS), laboratory safety tests, urinalysis and 12-lead electrocardiograms (ECG).
Time frame: 21 days
6 secondary outcomes
Pharmacokinetics of single and multiple doses of APX001 as measured by maximum observed concentration (Cmax).
Time frame: 21 days
Pharmacokinetics of single and multiple doses of APX001 as measured by area under the curve (AUC).
Time frame: 21 days
Pharmacokinetics of single and multiple doses of APX001 as measured by terminal phase half-life (t1/2).
Time frame: 21 days
Pharmacokinetics of single and multiple doses of APX001 as measured by volume of distribution (Vd).
Time frame: 21 days
Pharmacokinetics of single and multiple doses of APX001 as measured by elimination rate constant (Kel).
Time frame: 21 days
Pharmacokinetics of single and multiple dose of APX001 as measured by accumulation ratio.
Time frame: 21 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Sep 2025.
- Results publication: PubMed 36988461Hodges MR, Ople E, Wedel P, Shaw KJ, Jakate A, Kramer WG, Marle SV, van Hoogdalem EJ, Tawadrous M. Safety and Pharmacokinetics of Intravenous and Oral Fosmanogepix, a First-in-Class Antifungal Agent, in Healthy Volunteers. Antimicrob Agents Chemother. 2023 Apr 18;67(4):e0162322. doi: 10.1128/aac.01623-22. Epub 2023 Mar 29.
- 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.
Marc Engelhardt study director
Basilea Pharmaceutica International Ltd, Allschwil
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing APX001 single dose 1, APX001 single dose 2 and APX001 single dose 3 in people with Fungal Infection, run by Basilea Pharmaceutica at 2 sites in Netherlands.
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 5 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 10 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 2 sites in Netherlands. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Safety and tolerability of single and multiple doses of APX001 as measured by adverse events (AEs), physical examinations (PE), vital signs (VS), laboratory safety tests, urinalysis and 12-lead electrocardiograms (ECG). (time frame: 21 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 NCT02956499, 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 10 Sep 2025.