Clinical trial NCT02956499: Study to Assess Safety, Tolerability and Pharmacokinetics of APX001 Administered by Intravenous Infusion to Healthy Subjects
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Fungal Infection
Lead sponsor: Basilea Pharmaceutica
Sponsor class: INDUSTRY
First posted: 2016-11-06
Enrollment: 200
Sex: ALL
Minimum age (years): 18
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: 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).
timeFrame: 21 days
Secondary outcomes:
measure: Pharmacokinetics of single and multiple doses of APX001 as measured by maximum observed concentration (Cmax).
timeFrame: 21 days
measure: Pharmacokinetics of single and multiple doses of APX001 as measured by area under the curve (AUC).
timeFrame: 21 days
measure: Pharmacokinetics of single and multiple doses of APX001 as measured by terminal phase half-life (t1/2).
timeFrame: 21 days
measure: Pharmacokinetics of single and multiple doses of APX001 as measured by volume of distribution (Vd).
timeFrame: 21 days
measure: Pharmacokinetics of single and multiple doses of APX001 as measured by elimination rate constant (Kel).
timeFrame: 21 days
measure: Pharmacokinetics of single and multiple dose of APX001 as measured by accumulation ratio.
timeFrame: 21 days
Other outcomes:
Locations:
city: Groningen
country: Netherlands
facility: PRA Health Sciences
city: Groningen
country: Netherlands
facility: PRA Health Sciences
Study officials:
affiliation: Basilea Pharmaceutica International Ltd, Allschwil
name: Marc Engelhardt
role: STUDY_DIRECTOR
References: