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

Basilea Pharmaceutica

Status and phase: Completed; Phase 1

Conditions: Fungal Infection

Treatments: Drug: APX001 single dose 3; Drug: APX001 multiple dose 1; Drug: APX001 multiple dose 2; Drug: APX001 multiple dose 3; Drug: APX001 single dose 2; Drug: Matching Placebo; Drug: APX001 single dose 1; Drug: APX001 multiple dose 4; Drug: APX001 single dose 6; Drug: APX001 single dose 4; Drug: APX001 single dose 5

Study type: Interventional

Funder types: Industry

Identifiers

NCT02956499

APX001-101

C4791001 (Other Identifier)

Details and patient eligibility

About

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.

Enrollment: 200 patients

Sex: All

Ages: 18 to 55 years old

Volunteers: Accepts Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Double Blind

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200 participants in 10 patient groups

Cohort 1

Experimental group

Description:

single intravenous dose

Treatment:

Drug: Matching Placebo

Drug: APX001 single dose 1

Cohort 2

Experimental group

Description:

single intravenous dose

Treatment:

Drug: Matching Placebo

Drug: APX001 single dose 2

Cohort 3

Experimental group

Description:

single intravenous dose

Treatment:

Drug: Matching Placebo

Drug: APX001 single dose 3

Cohort 4

Experimental group

Description:

single intravenous dose

Treatment:

Drug: APX001 single dose 4

Drug: Matching Placebo

Cohort 5

Experimental group

Description:

single intravenous dose

Treatment:

Drug: APX001 single dose 5

Drug: Matching Placebo

Cohort 6

Experimental group

Description:

single intravenous dose

Treatment:

Drug: APX001 single dose 6

Drug: Matching Placebo

Cohort 7

Experimental group

Description:

multiple intravenous doses

Treatment:

Drug: Matching Placebo

Drug: APX001 multiple dose 1

Cohort 8

Experimental group

Description:

multiple intravenous doses

Treatment:

Drug: Matching Placebo

Drug: APX001 multiple dose 2

Cohort 9

Experimental group

Description:

multiple intravenous doses

Treatment:

Drug: Matching Placebo

Drug: APX001 multiple dose 3

Cohort 10

Experimental group

Description:

multiple intravenous doses

Treatment:

Drug: APX001 multiple dose 4

Drug: Matching Placebo