Safety Study of Single Oral Doses of VBY-036 in Healthy Volunteers (VBY036P1A)

Study Overview
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Brief Summary
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VBY-036 may treat or prevent nerve pain. This study aims to find the highest safe and tolerable single dose of VBY-036 in healthy volunteers. Volunteers are randomly selected to receive one dose of either a placebo or VBY-036 (10, 30, 100, 300, 600 or 900 mg).

Detailed Description
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Detailed description is noted in Brief Summary.

Official Title
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A Double-Blind, Randomized, Placebo-Controlled, Sequential, Single Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral Doses of VBY-036 in Healthy Subjects

Conditions
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Healthy

Intervention / Treatment
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* Drug: VBY-036
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male or female, 18-60 years old Screening body mass index between 18-32 kg/m2 Good health, no clinically significant findings in medical history, 12-lead ECG, & vital signs; Clinical lab evaluations (Chem panel [fasted at least 8 hrs], CBC, HbA1c & UA in reference range for test lab (unless deemed not clinically significant); Negative test for drugs of abuse at Screening & at Check-in (includes alcohol); Negative hepatitis, HIV & TB screens; Females non-pregnant, non-lactating, & either postmenopausal for at least 1 year, surgically sterile for at least 90 days prior to Check-in, or agree to use from the time of consent until 90 days after Study Completion an effective form of contraception. For all females, a pregnancy test result must be negative at Screening & Check-in. Males will be sterile or agree to use from Check-in until 90 days following discharge an effective method of contraception. Able to comprehend & willing to sign Informed Consent Form Exclusion Criteria: Females pregnant or nursing, or childbearing potential but unwilling to use contraception. History of renal or hepatic impairment; stomach or intestinal surgery or resection, malabsorption syndrome, cholecystectomy, or gastro-intestinal dysfunction that would alter absorption &/or excretion of orally administered drugs (appendectomy or hernia repair allowed); Anemia (hemoglobin <11.5 g/dL for females; < 13 g/dL for males) or blood donation within 8 weeks of Check-in; Plasma donation within 4 weeks of Check-in; History of alcoholism or drug addiction within 6 months to Check-in; Use of drugs of abuse or prescription drugs for recreational use 6 months prior to Check-in; Use of any tobacco-containing or nicotine-containing products 6 months prior to Check-in & during study; Participation in another drug trial 30 days of Check-in (within 8 weeks if previous investigational drug has immunomodulary effects, other than cathepsin S inhibition); History or clinical manifestations of metabolic, hepatic, renal, hematological, pulmonary, cardiovascular, gastrointestinal, urological, neurological, or psychiatric disorders History of hypersensitivity or allergies to any drug compound History or presence of abnormal ECG Laboratory abnormality deemed clinically significant; Use of or inability to discontinue any prescription medications/products 14 days prior to Check-in & during study; Use of certain over-the-counter, non-prescription preparations are permitted up to 3 days before first dose; Use of alcohol-containing, grapefruit-containing, star fruit containing foods or beverages or energy drinks 72 hours to Check-in & during study; Poor peripheral venous access; Receipt of blood products 6 months to Check-in Subjects with history of Gilbert's Syndrome; Strenuous activities 48 hours before receiving experimental or placebo comparator Illness 5 days to drug administration Any acute or chronic condition

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 60 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: VBY-036<br>VBY-036 10 mg, 30 mg, 100 mg, 300 mg, 600 mg, or 900 mg | Drug: VBY-036<br>* Study drug VBY-036<br>* Other names: Cathepsin S inhibitor;Drug: Placebo<br>* Placebo<br>|
| Placebo Comparator: Placebo<br>Placebo | Drug: VBY-036<br>* Study drug VBY-036<br>* Other names: Cathepsin S inhibitor;Drug: Placebo<br>* Placebo<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Highest safe and tolerated VBY-036 dose | Single dose of VBY-036 given one day for each cohort in an ascending manner. Cohort A1 with 10 mg dose or placebo; Cohort A2 with 30 mg dose or placebo; Cohort A3 with 100 mg dose or placebo; Cohort A4 with 300 mg dose or placebo; Cohort A5 with 600 mg dose or placebo; Cohort A6 with 900 mg dose or placebo | 5-days assessment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Confirmation of cathepsin-S inhibition | Based on the accumulation of Iip 10 and/or cathepsin S protein levels | 2 days | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Healthy, Subjects, Safety, Tolerability, VBY-036, Maximum, Pharmacodynamic, Pharmacokinetic