A Phase 1, Single-Ascending-Dose Study of BT051 in Healthy Subjects
Condition: Healthy Volunteers
Study type: Interventional
Phase: Phase 1

Summary: 
      This is a randomized, double-blind, placebo-controlled study to assess the safety and
      tolerability of single ascending doses of BT051 in healthy male or female volunteers aged 18
      to <50 years. A total of 50 subjects will be randomized to receive a single oral dose of
      BT051 or matching placebo in a ratio of 4 active:1 placebo in 5 ascending dose cohorts (10
      subjects per cohort) at active dose levels of 100mg, 300mg, 700mg, 1500mg or 3500mg. The
      study Safety Review Committee (SRC) will evaluate if any dose-limiting adverse events (AEs)
      occurred in a cohort through Day 3, as well as review cumulative safety data for all previous
      cohorts and any available pharmacokinetic (PK) data before proceeding to dosing in the next
      cohort.
    

Detailed description: 
      BT051-1-001 is a phase 1, randomized, double-blind, single center, single-ascending-dose
      study in which healthy subjects will receive a single oral dose of BT051 or placebo while
      confined to the clinical unit. Approximately 50 subjects will be enrolled in 5 sequential,
      ascending dose cohorts.

      Healthy male and female adult subjects will be enrolled and screened for participation within
      28 days before the scheduled administration of study drug. After written informed consent is
      obtained, the screening procedures will include: medical history, documentation of prior
      medications (i.e., medications taken within 30 days before the scheduled dose of study drug),
      viral serology tests, clinical laboratory testing, pregnancy testing (for women of
      childbearing potential), 12 lead electrocardiograms (ECGs), vital sign measurements, and
      physical examination.

      After confirmation of inclusion and exclusion criteria, subjects eligible for randomization
      will be admitted to the clinical unit 1 day before the scheduled administration of study drug
      (Day -1) and will be confined in the clinical unit until the morning of Day 3. All subjects
      will return to the clinical unit on Day 7 (-1 or +2 days) and for the last follow-up on Day
      30 (±3 days) for study assessments.

      A total of 10 subjects will be randomized to receive BT051 or placebo (8 active:2 placebo) in
      each of the following sequential dose escalating cohorts: 100mg, 300mg, 700mg. 1500mg and
      3500mg. Administration of a single dose of study medication on Day 1 will occur under fasted
      conditions (i.e., no food allowed overnight before dosing until at least 4 hours after
      dosing). Except for approximately 240-480 mL of water given with study drug, no fluid will be
      allowed from 1 hour before dosing until 1 hour postdose; water will be provided ad libitum at
      all other times.

      The study Safety Review Committee (SRC) will evaluate if any dose-limiting adverse events
      (AEs) through Day 3 occurred in a cohort before proceeding to dosing in the next cohort. In
      addition, cumulative safety data will be reviewed for all previous cohorts along with any
      available pharmacokinetic (PK) data. A lower dose may be explored in case dose-limiting AEs
      are observed at a higher dose level.

      For PK analyses, blood, urine, and fecal samples will be collected from each subject. Blood
      samples will be collected on Day 1 at predose and at 0.5, 1, 2, 3, 4, 6, 8, 12, 16, 24, and
      48 hours postdose. Urine samples will be collected at the following intervals (pooled for
      each collection interval): 0-4, 4-8, 8-24, and 24-48 hours postdose. One stool sample will be
      collected prior to dose (from Days -2 to Day 1). Following dosing on Day 1, all stool samples
      will be collected through Day 3 (48 hours postdose) while the subject is confined to the
      clinical unit. In addition, a stool sample will be collected on Day 7 (-1 or +2 days).

      Safety assessments will include monitoring of AEs, clinical laboratory testing, vital sign
      measurements, physical examinations, and ECGs at select time points for 30 days following the
      dose of study drug. Potential systemic pharmacologic T-cell immunosuppressive activity will
      be evaluated.
    

Intervention name(s): BT051 100mg; BT051 300mg; BT051 700mg; BT051 1500mg; BT051 3500mg; Matching Placebo
Intervention type(s): Drug; Drug; Drug; Drug; Drug; Drug
Intervention description(s): Single oral dose of 100mg BT051; Single oral dose of 300mg BT051; Single oral dose of 700mg BT051; Single oral dose of 1500mg BT051; Single oral dose of 3500mg BT051; Placebo Matching BT051
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Basic Science