Study to Assess Safety, Tolerability and Pharmacokinetics of APX001 Administered by Intravenous Infusion to Healthy Subjects

Phase 1

Completed

• Conditions: Fungal Infection
• Interventions: Drug: APX001 single dose 2; Drug: APX001 single dose 3; Drug: APX001 single dose 5; Drug: APX001 single dose 1; Drug: APX001 multiple dose 2; Drug: APX001 multiple dose 4; Drug: APX001 multiple dose 3; Drug: APX001 single dose 4; Drug: APX001 single dose 6; Drug: APX001 multiple dose 1; Drug: Matching Placebo

• Registration Number: NCT02956499
• Lead Sponsor: Basilea Pharmaceutica

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2016-05-24
• Study First Submitted: 2016-11-01
• Study First Submitted QC: 2016-11-03
• Study First Posted: 2016-11-06
• Primary Completion: 2017-07-03
• Study Completion: 2017-07-03
• Status Verified: 2024-05
• Last Update Submitted: 2024-05-14
• Last Update Posted: 2024-05-16

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 200

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Cohort 2	APX001 single dose 2	single intravenous dose
Cohort 2	Matching Placebo	single intravenous dose
Cohort 3	APX001 single dose 3	single intravenous dose
Cohort 4	Matching Placebo	single intravenous dose
Cohort 5	APX001 single dose 5	single intravenous dose
Cohort 1	APX001 single dose 1	single intravenous dose
Cohort 1	Matching Placebo	single intravenous dose
Cohort 8	APX001 multiple dose 2	multiple intravenous doses
Cohort 6	Matching Placebo	single intravenous dose
Cohort 10	APX001 multiple dose 4	multiple intravenous doses
Cohort 9	APX001 multiple dose 3	multiple intravenous doses
Cohort 3	Matching Placebo	single intravenous dose
Cohort 4	APX001 single dose 4	single intravenous dose
Cohort 5	Matching Placebo	single intravenous dose
Cohort 6	APX001 single dose 6	single intravenous dose
Cohort 7	Matching Placebo	multiple intravenous doses
Cohort 7	APX001 multiple dose 1	multiple intravenous doses
Cohort 8	Matching Placebo	multiple intravenous doses
Cohort 9	Matching Placebo	multiple intravenous doses
Cohort 10	Matching Placebo	multiple intravenous doses

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
Pharmacokinetics of single and multiple doses of APX001 as measured by terminal phase half-life (t1/2).	21 days
Pharmacokinetics of single and multiple dose of APX001 as measured by accumulation ratio.	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 volume of distribution (Vd).	21 days
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 elimination rate constant (Kel).	21 days

Trial Locations

Locations (1)

PRA Health Sciences; 🇳🇱 Groningen, Netherlands