Study to Assess Safety, Tolerability and Pharmacokinetics of APX001 Administered by Intravenous Infusion to Healthy Subjects
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
2 other identifiers
interventional
200
1 country
2
Brief 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. The MAD portion of the study will enroll four cohorts of eight healthy subjects per cohort, for a total of approximately 32 healthy subjects.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_1
Started May 2016
2 active sites
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
May 24, 2016
CompletedFirst Submitted
Initial submission to the registry
November 1, 2016
CompletedFirst Posted
Study publicly available on registry
November 7, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 3, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
July 3, 2017
CompletedSeptember 10, 2025
September 1, 2025
1.1 years
November 1, 2016
September 3, 2025
Conditions
Outcome Measures
Primary Outcomes (1)
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).
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 doses of APX001 as measured by area under the curve (AUC).
21 days
Pharmacokinetics of single and multiple doses of APX001 as measured by terminal phase 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 (10)
Cohort 1
EXPERIMENTALsingle intravenous dose
Cohort 2
EXPERIMENTALsingle intravenous dose
Cohort 3
EXPERIMENTALsingle intravenous dose
Cohort 4
EXPERIMENTALsingle intravenous dose
Cohort 5
EXPERIMENTALsingle intravenous dose
Cohort 6
EXPERIMENTALsingle intravenous dose
Cohort 7
EXPERIMENTALmultiple intravenous doses
Cohort 8
EXPERIMENTALmultiple intravenous doses
Cohort 9
EXPERIMENTALmultiple intravenous doses
Cohort 10
EXPERIMENTALmultiple intravenous doses
Interventions
Eligibility Criteria
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
Groningen, 9728 NZ, Netherlands
PRA Health Sciences
Groningen, Netherlands
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
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Marc Engelhardt
Basilea Pharmaceutica International Ltd, Allschwil
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 1, 2016
First Posted
November 7, 2016
Study Start
May 24, 2016
Primary Completion
July 3, 2017
Study Completion
July 3, 2017
Last Updated
September 10, 2025
Record last verified: 2025-09
Data Sharing
- IPD Sharing
- Will not share