NCT02957929

Brief Summary

This is a Phase l double-blind, placebo-controlled, randomized study to investigate the safety, tolerability, pharmacokinetics, bioavailability and food effect of single doses of APX001 administered intravenously and orally, followed by an evaluation of the safety, tolerability, pharmacokinetics and drug-drug interaction potential of multiple doses of APX001 administered orally.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
46

participants targeted

Target at P50-P75 for phase_1

Timeline
Completed

Started Oct 2016

Shorter than P25 for phase_1

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 31, 2016

Completed
1 day until next milestone

First Submitted

Initial submission to the registry

November 1, 2016

Completed
7 days until next milestone

First Posted

Study publicly available on registry

November 8, 2016

Completed
5 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 20, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 20, 2017

Completed
Last Updated

September 10, 2025

Status Verified

September 1, 2025

Enrollment Period

6 months

First QC Date

November 1, 2016

Last Update Submit

September 3, 2025

Conditions

Fungal Infection

Outcome Measures

Primary Outcomes (1)

  • Safety and tolerability of single and multiple oral doses of APX001 as measured by adverse events (AEs), physical examinations (PE), vital signs (VS), laboratory safety tests, urinalysis and 12-lead electrocardiograms (ECG).

    21 days

Secondary Outcomes (6)

  • Pharmacokinetics of single and multiple doses of APX001 as measured by maximum observed concentration (Cmax).

    21 days

  • Pharmacokinetics of single and multiple dose of APX001 as measured by area under the curve (AUC).

    21 days

  • Pharmacokinetics of single and multiple doses of APX001 as measured by terminal half life (t1/2).

    21 days

  • Pharmacokinetics of single and multiple doses of APX001 as measured by volume of distribution (Vd).

    21 days

  • Pharmacokinetics of single and multiple doses of APX001 as measured by elimination rate constant (Kel).

    21 days

  • +1 more secondary outcomes

Study Arms (9)

Cohort 1a, Period A

EXPERIMENTAL

single intravenous dose, crossover

Drug: APX001 single IV doseDrug: Matching placebo control

Cohort 1a, Period B

EXPERIMENTAL

single oral dose, crossover

Drug: APX001 single oral dose 1Drug: Matching placebo control

Cohort 1a, Period C

EXPERIMENTAL

single oral dose

Drug: APX001 single oral dose 2Drug: Matching placebo control

Cohort 1a, Period D

EXPERIMENTAL

single oral dose, crossover

Drug: APX001 single oral dose 3Drug: Matching placebo control

Cohort 1b, Period E

EXPERIMENTAL

Single oral dose under fasted conditions, crossover

Drug: APX001 single oral dose fastedDrug: Matching placebo control

Cohort 1b, Period F

EXPERIMENTAL

Single oral dose under fed conditions, crossover

Drug: APX001 single oral dose fedDrug: Matching placebo control

Cohort 2

EXPERIMENTAL

Multiple oral doses

Drug: APX001 multiple oral doses 1Drug: Matching placebo control

Cohort 3

EXPERIMENTAL

Multiple oral doses

Drug: APX001 multiple oral doses 2Drug: Matching placebo control

Cohort 4

EXPERIMENTAL

Multiple oral doses in presence of CYP probe substrates

Drug: APX001 single IV doseDrug: APX001 multiple oral doses 3Drug: Cytochrome P450 substrates

Interventions

APX001 single IV doseDRUG
Cohort 1a, Period ACohort 4
APX001 single oral dose 1DRUG
Cohort 1a, Period B
APX001 single oral dose 2DRUG
Cohort 1a, Period C
APX001 single oral dose 3DRUG
Cohort 1a, Period D
APX001 single oral dose fastedDRUG
Cohort 1b, Period E
APX001 single oral dose fedDRUG
Cohort 1b, Period F
APX001 multiple oral doses 1DRUG
Cohort 2
APX001 multiple oral doses 2DRUG
Cohort 3
APX001 multiple oral doses 3DRUG
Cohort 4
Cytochrome P450 substratesDRUG
Cohort 4
Matching placebo controlDRUG
Cohort 1a, Period ACohort 1a, Period BCohort 1a, Period CCohort 1a, Period DCohort 1b, Period ECohort 1b, Period FCohort 2Cohort 3

Eligibility Criteria

Age18 Years - 55 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

PRA Health Sciences (PRA) - Early Development Services (EDS)

Groningen, 9728 NZ, Netherlands

Location

PRA Health Sciences

Groningen, Netherlands

Location

Related Publications (1)

  • 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.

    PMID: 36988461RESULT

MeSH Terms

Conditions

Mycoses

Condition Hierarchy (Ancestors)

Bacterial Infections and MycosesInfections

Study Officials

  • Marc Engelhardt

    Basilea Pharmaceutica International Ltd, Allschwil

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 1, 2016

First Posted

November 8, 2016

Study Start

October 31, 2016

Primary Completion

April 20, 2017

Study Completion

April 20, 2017

Last Updated

September 10, 2025

Record last verified: 2025-09

Data Sharing

IPD Sharing
Will not share

Locations

NL(2)