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

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 Overview

• Status: Completed
• Conditions: Fungal Infection
• Intervention / Treatment: Drug: Matching Placebo; Drug: APX001 single dose 1; Drug: APX001 single dose 2; Drug: APX001 single dose 3; Drug: APX001 single dose 4; Drug: APX001 single dose 5; Drug: APX001 single dose 6; Drug: APX001 multiple dose 1; Drug: APX001 multiple dose 2; Drug: APX001 multiple dose 3; Drug: APX001 multiple dose 4

• Study Type: Interventional
• Enrollment (Actual): 200
• Phase: Phase 1

Contacts and Locations

Study Locations

• Netherlands
  • Groningen, Netherlands, 9728 NZ
    • PRA Health Sciences
  • Groningen, Netherlands
    • PRA Health Sciences

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 55 years (Adult)
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Double

Number of Arms

10

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1; single intravenous dose	Drug: Matching Placebo; Drug: APX001 single dose 1
Experimental: Cohort 2; single intravenous dose	Drug: Matching Placebo; Drug: APX001 single dose 2
Experimental: Cohort 3; single intravenous dose	Drug: Matching Placebo; Drug: APX001 single dose 3
Experimental: Cohort 4; single intravenous dose	Drug: Matching Placebo; Drug: APX001 single dose 4
Experimental: Cohort 5; single intravenous dose	Drug: Matching Placebo; Drug: APX001 single dose 5
Experimental: Cohort 6; single intravenous dose	Drug: Matching Placebo; Drug: APX001 single dose 6
Experimental: Cohort 7; multiple intravenous doses	Drug: Matching Placebo; Drug: APX001 multiple dose 1
Experimental: Cohort 8; multiple intravenous doses	Drug: Matching Placebo; Drug: APX001 multiple dose 2
Experimental: Cohort 9; multiple intravenous doses	Drug: Matching Placebo; Drug: APX001 multiple dose 3
Experimental: Cohort 10; multiple intravenous doses	Drug: Matching Placebo; Drug: APX001 multiple dose 4

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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 Outcome Measures

Outcome Measure	Time Frame
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
Pharmacokinetics of single and multiple dose of APX001 as measured by accumulation ratio.	21 days

Collaborators and Investigators

• Sponsor: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): May 24, 2016
• Primary Completion (Actual): July 3, 2017
• Study Completion (Actual): July 3, 2017

Study Registration Dates

• First Submitted: November 1, 2016
• First Submitted That Met QC Criteria: November 3, 2016
• First Posted (Estimated): November 6, 2016

Study Record Updates

• Last Update Posted (Actual): October 30, 2023
• Last Update Submitted That Met QC Criteria: October 26, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Infections; Bacterial Infections and Mycoses; Mycoses
• Other Study ID Numbers: APX001-101; C4791001 (Other Identifier: Alias Study Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.