A Study to Evaluate the Safety and Pharmacokinetics of BX-001N in Healthy Participants

A Randomized, Double-blind, Placebo-controlled, Single and Multiple Ascending Dose, Phase 1 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BX-001N After Intravenous Administration in Healthy Participants

This is a randomized, double-blind, placebo-controlled, single and multiple ascending dose, Phase 1 study to evaluate the safety, tolerability, and pharmacokinetics of BX-001N after intravenous administration in approximately 64 healthy participants

Study Overview

• Status: Recruiting
• Conditions: Ischemia-reperfusion Injury
• Intervention / Treatment: Drug: BX-001N Part 1; Drug: Placebo; Drug: BX-001N Part 2

Detailed Description

This study comprises of 2 parts:

• Part 1- Single Ascending Dose (SAD)- This part will enroll approximately 40 participants across 5 cohorts where each participant will receive a single intravenous (IV) bolus dose in healthy participants. On Day 1, participants in each cohort will receive investigational product (IP) (i.e., BX-001N or Placebo) as a single IV bolus following a minimum 8-hour fast.
• Part 2 -Multiple Ascending Dose (MAD)- This part will enroll approximately 24 participants across 3 cohorts where each participants will receive intravenous (IV) bolus dose for 7 sequential daily. At the same time each morning from Day 1 to Day 7 (inclusive), participants in each cohort will receive IP (i.e., BX-001N or Placebo) as a single IV bolus following a minimum 8-hour fast.

• Study Type: Interventional
• Enrollment (Estimated): 64
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Sang Ho Ma; Phone Number: +821065352408; Email: sangho.ma@bilix.com
• Study Contact Backup: Name: ChoonMo Kang; Phone Number: +827077915721; Email: choonmo.kang@bilix.com

Study Locations

• Australia
  • South Australia
    • Adelaide, South Australia, Australia, 5000
      • Recruiting
      • CMAX Clinical Research
      • Contact: Angela C Rowland, Dr; Phone Number: + 610439682089; Email: Teamrowland08@yahoo.com.au
      • Principal Investigator: Angela C Rowland, Dr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria:

• 18 to 50 years of age
• In good general health at Screening and/or before the first administration of IP
• BMI > 18.0 and < 32.0 kg/m2 at Screening
• Nonsmoker and must not have used any tobacco products within 2 months prior to screening
• Females must not be pregnant or lactating, and females and males must use acceptable, highly effective double contraception during study and follow-up period
• Person who can provide written informed consent prior to the commencement of all study procedures

Exclusion Criteria:

• Underlying physical or psychological medical condition to comply with the protocol or complete the study per protocol
• Genetic disorder with severe and abnormal bilirubin metabolism
• Blood or plasma donation or significant blood loss prior to the first administration of IP
• Viral or bacterial infection prior to the first administration of IP
• Poor venous access
• Significant scarring or tattoos at the planned site of IP administration
• History of severe allergic or anaphylactic reactions, or sensitivity to the IP or its constituents
• History or active cardiovascular, respiratory, kidney, endocrine, blood, digestive, central nervous, urinary and/or musculoskeletal disease
• History of malignancy prior to Screening
• Abnormal ECG findings
• History or presence of a condition associated with significant immunosuppression
• History of life-threatening infection
• Infections requiring parenteral antibiotics
• Vaccination prior to the first administration of IP
• Exposure to any significantly immune suppressing drug
• Abnormal vital signs findings
• Abnormal laboratory findings
• Positive results for viral testing at Screening
• Positive result at Screening and Day -1 for toxicology screening panel
• History of substance abuse or dependency or history of recreational intravenous (IV) drug use
• Excess of regular alcohol consumption
• Use of any IP or investigational medical device within 30 days prior to Screening
• Unable to adhere to the prohibited therapies
• Unwilling to adhere to the dietary restrictions
• Unwilling to refrain from strenuous exercise

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: BX-001N Part 1; Part 1 is SAD with 5 cohorts where each participant will receive single IV bolus following a 8hr fast.	Drug: BX-001N Part 1; Dosage form- IV bolus Dosage- In the five cohorts, each participant receives a single IV bolus administration in one of the five doses based on body weight and followed up for 7 days.
Experimental: BX-001N Part 2; Part 2 is MAD with 3 cohorts where each participant will receive 7 sequential daily IV bolus doses following a 8hr fast.	Drug: BX-001N Part 2; Dosage form- IV bolus Dosage- In the three cohorts, each participant receives a single IV bolus administration for 7 sequential days in one of the three doses based on body weight and followed up for 14 days.
Placebo Comparator: Placebo; Matching doses of placebo	Drug: Placebo; Participants will receive matching placebo across Part 1 and 2 of the study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with Treatment emergent Adverse events (TEAEs)	TEAE will be collected to assess participants' safety after BX-001N treatment	SAD-Screening to Day 7; MAD- Screening to Day 14
Number of participants with clinical laboratory abnormalities		SAD-Screening to Day 7; MAD- Screening to Day 14
Number of participants with changes in the 12-lead electrocardiogram (ECG)		SAD-Screening to Day 7; MAD- Screening to Day 14
Number of incidences of injection site reactions		SAD-Day 1 to Day 2; MAD- Day 1 to Day 8

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in Cmax (maximum Concentration) of BX-001N with 5 different doses of SAD and 3 different doses of MAD	SAD's samples of PK are collected at total 12 time points from pre-dose and up to Day 3 after dosing. MAD's samples of PK are collected at total 26 time points from pre-dose and up to Day 8 after dosing.	SAD- Day 1 to Day 3; MAD- Day 1 to Day 8
Changes in Tmax (Time of maximum Concentration) of BX-001N with 5 different doses of SAD and 3 different doses of MAD	SAD's samples of PK are collected at total 12 time points from pre-dose and up to Day 3 after dosing. MAD's samples of PK are collected at total 26 time points from pre-dose and up to Day 8 after dosing	SAD- Day 1 to Day 3; MAD- Day 1 to Day 8
Changes in AUC (area under curve) of BX-001N with 5 different doses of SAD and 3 different doses of MAD	SAD's samples of PK are collected at total 12 time points from pre-dose and up to Day 3 after dosing. MAD's samples of PK are collected at total 26 time points from pre-dose and up to Day 8 after dosing.	SAD- Day 1 to Day 3; MAD- Day 1 to Day 8
Change in Immunogenicity- Incidence of Anti-drug antibody (ADA) by polyethylene glycol (PEG)	Up to 3 samples will be collected in total and additional samples if positive results	SAD-Day 1 to Day 7; MAD- Day 1 to Day 14
Change in Immunogenicity- Titers of Anti-drug antibody (ADA) by polyethylene glycol (PEG)	Up to 3 samples will be collected in total and additional samples if positive results	SAD-Day 1 to Day 7; MAD- Day 1 to Day 14
Change in Immunogenicity- Duration of Anti-drug antibody (ADA) by polyethylene glycol (PEG)	Up to 3 samples will be collected in total and additional samples if positive results	SAD-Day 1 to Day 7; MAD- Day 1 to Day 14

Collaborators and Investigators

• Sponsor: Bilix Co.,Ltd.
• Investigators: Principal Investigator: Angela C Rowland, Dr, CMAX Clinical Research

Study record dates

Study Major Dates

• Study Start (Actual): November 17, 2023
• Primary Completion (Estimated): April 30, 2024
• Study Completion (Estimated): June 30, 2024

Study Registration Dates

• First Submitted: October 19, 2023
• First Submitted That Met QC Criteria: October 19, 2023
• First Posted (Actual): October 24, 2023

Study Record Updates

• Last Update Posted (Actual): January 12, 2024
• Last Update Submitted That Met QC Criteria: January 11, 2024
• Last Verified: December 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Cardiovascular Diseases; Vascular Diseases; Postoperative Complications; Ischemia; Reperfusion Injury
• Other Study ID Numbers: BX-001N-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No