A Phase 1, Single-Ascending-Dose Study of BT051 in Healthy Subjects

NCT05103878 | Completed Phase 1 FDA Drug

• 1 other identifier: BT051-1-001
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Healthy Volunteers

Outcome Measures

Primary Outcomes (3)

• Evaluate the safety and tolerability of BT051 based on the difference of proportions between treatment groups of subjects experiencing treatment-emergent adverse events (TEAEs).

  Proportion of subjects experiencing a TEAE will be summarized using the MedDRA system organ class and preferred term.

  Baseline to Day 30

• Evaluate the safety and tolerability of BT051 based on the difference of proportions between treatment groups of subjects experiencing clinically significant changes from baseline in clinical laboratory tests.

  Proportion of subjects with a change from baseline from normal to abnormal in clinical laboratory tests (hematology with differential, serum chemistry, coagulation, and urinalysis) will be summarized.

  Baseline to Day 30

• Evaluate the safety and tolerability of BT051 based on the difference of proportions between treatment groups of subjects observed with a change from baseline in physical examinations, vital signs, and electrocardiograms (ECG).

  Proportion of subjects with a change from baseline from normal to abnormal in physical examinations, vital signs, and ECGs will be summarized.

  Baseline to Day 30

Secondary Outcomes (6)

• Area Under the Concentration-Time Curve (AUC) in whole blood

  Baseline to Day 3

• Maximum observed concentration (Cmax) in whole blood

  Baseline to Day 3

• Time to maximum observed concentration (Tmax) in whole blood

  Baseline to Day 3

• Half-life (t1/2) in whole blood

  Baseline to Day 3

• Stool concentration of BT051 and BT070

  Baseline to Day 7

Study Arms (6)

BT051 100 mg

EXPERIMENTAL

Participants will receive a single oral dose of 100mg BT051.

Drug: BT051 100mg

BT051 300 mg

EXPERIMENTAL

Participants will receive a single oral dose of 300mg BT051.

Drug: BT051 300mg

BT051 700 mg

EXPERIMENTAL

Participants will receive a single oral dose of 700mg BT051.

Drug: BT051 700mg

BT051 1500 mg

EXPERIMENTAL

Participants will receive a single oral dose of 1500mg BT051.

Drug: BT051 1500mg

BT051 3500 mg

EXPERIMENTAL

Participants will receive a single oral dose of 3500mg BT051.

Drug: BT051 3500mg

Placebo

PLACEBO COMPARATOR

Participants will receive a single oral dose of Placebo matching BT051 dose.

Drug: Matching Placebo

Interventions

BT051 100mg DRUG

Single oral dose of 100mg BT051

BT051 100 mg

BT051 300mg DRUG

Single oral dose of 300mg BT051

BT051 300 mg

BT051 700mg DRUG

Single oral dose of 700mg BT051

BT051 700 mg

BT051 1500mg DRUG

Single oral dose of 1500mg BT051

BT051 1500 mg

BT051 3500mg DRUG

Single oral dose of 3500mg BT051

BT051 3500 mg

Matching Placebo DRUG

Placebo Matching BT051

Placebo

Eligibility Criteria

• Age: 18 Years - 49 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Subjects must meet all the following criteria to be considered eligible to participate in the study:
• Male or female subjects age ≥18 years and \<50 years
• In good health with no clinically significant abnormalities as determined by medical history, physical exam, and laboratory values
• Able and willing to provide written informed consent
• Be able to understand the study procedures and agree to participate in the study
• For male subjects, be surgically sterile or agree to use an appropriate method of contraception (i.e., condom) or have a female sexual partner who is surgically sterile or using an insertable, injectable, transdermal, or combination oral contraceptive with a barrier method approved and deemed highly effective by the United States Food and Drug Administration (FDA) through 30 days after the dose of study drug
• For female subjects of childbearing potential, be using an insertable, injectable, or transdermal hormonal contraceptive, or combination oral contraceptive with a barrier method approved and deemed highly effective by the FDA through 30 days after the dose of study drug and have negative results on pregnancy tests done at Screening and on Day -1. NOTE: women who are surgically sterile or postmenopausal (i.e., no menses for at least 2 years or documented by follicle stimulating hormone) are also eligible to participate.
• Availability to volunteer for the entire study duration and willing to adhere to all protocol requirements, including confinement in the clinical unit through Day 3
• Body mass index (BMI) \>18.5 and \<32.0 kg/m\^2
• Ability to fast for at least 10 hours and consume standard meals
• Willingness to discontinue concomitant medications

You may not qualify if:

• Subjects who meet any of the following criteria will be excluded from participating in the study:
• Pregnant, breast feeding, or seeking pregnancy while on study
• Have, as determined by the investigator, a history or clinical manifestations of significant neurologic, renal, hepatic, hematologic, cardiac, pulmonary, metabolic, endocrine, psychiatric, gastrointestinal (GI) disorders (including infectious, ischemic, inflammatory bowel disease, irritable bowel syndrome, known lactose intolerance, or immunological diseases) or other condition that would preclude participation in the study
• Have a history of a malignancy (or active malignancy), with the exception of subjects with basal cell carcinoma, squamous cell carcinoma, or carcinoma in situ of the cervix who have been treated and cured
• Have had any symptoms of cold, flu, or febrile illness within 2 weeks prior to dosing
• Have received any live attenuated vaccine within 30 days of dosing
• Have received cyclosporine A within the last 1 month, including Restasis® (cyclosporine ophthalmic emulsion) for dry eye syndrome or any other cyclosporine ophthalmic preparation
• Participation in a clinical trial of an investigational drug or medical device within 30 days before Screening
• In the opinion of the Investigator, unable to comply with the study protocol
• Have a history of alcoholism or illicit drug use within 2 years before the scheduled dose of study drug
• A positive test result for any of the following: human immunodeficiency virus (HIV), hepatitis B virus (HBV) surface antigen, hepatitis C virus (HCV) antibody, drugs of abuse (marijuana, amphetamines, barbiturates, cocaine, opiates, phencyclidine and benzodiazepines), and alcohol.
• Have donated more than 500 mL of blood within 60 days before the scheduled dose of study drug
• Have taken prescription medications within 5 half-lives of the specific substance (or, if half-life is not known, within 48 hours) before the scheduled administration of study drug
• Have taken over-the-counter medication or supplements, including nutritional supplements, stool softeners (e.g., Miralax), or colon preparations within 7 days before dosing
• History of any hypersensitivity or allergic reaction to cyclosporine

Sponsors & Collaborators

• Bacainn Therapeutics, Inc.: lead

Study Sites (1)

Spaulding Clinical Research, LLC

West Bend, Wisconsin, 53095, United States

Location

Related Publications (1)

• Cheifetz AS, Allegretti JR, Quintas M, Dixit B, Farquhar R, Miller BW, Murphy CK, Hershberger E, Ghahramani P, Stevens AC. Small-Molecule Neutrophil Modulator ADS051 is Safe and Well-Tolerated in a Phase 1 Single Ascending Dose Study. Am J Gastroenterol. 2024 Nov 26;120(7):1585-1592. doi: 10.14309/ajg.0000000000003237.

  PMID: 39588987 DERIVED

Study Officials

• Chris Stevens, MD

  Bacainn Therapeutics, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: BASIC SCIENCE
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 26, 2021
• First Posted: November 2, 2021
• Study Start: June 24, 2020
• Primary Completion: November 13, 2020
• Study Completion: November 13, 2020
• Last Updated: March 28, 2022

Record last verified: 2022-03

Locations

US (1)