A Clinical Study Aiming to Assess Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of TRX-100 in Healthy Volunteers

NCT06757738 | Completed Phase 1 DMC

• 2 other identifiers: TRX-100-0001CT-2024-CTN-03164
• Study Type: interventional
• Target: 66
• Locations: 1 country
• Sites: 1

Brief Summary

This is a clinical study aiming to assess pharmacokinetics, pharmacodynamics and preliminary efficacy of TRX-100 (and its major active metabolite TRX-101) in Healthy Volunteers

Conditions

Healthy Volunteer Study

Outcome Measures

Primary Outcomes (7)

• To assess the safety and tolerability of a single oral administration of TRX-100

  Incidence, severity and relationship of AEs/serious AEs (SAEs) (including withdrawals due to AEs)

  up to Day 28

• To assess the safety and tolerability of a single oral administration of TRX-100

  Change from baseline in body weight in kg

  up to Day 28

• To assess the safety and tolerability of a single oral administration of TRX-100

  Change from baseline in systolic and diastolic blood pressure (mm Hg)

  up to Day 28

• To assess the safety and tolerability of a single oral administration of TRX-100

  Change from baseline in QRS duration (miliseconds)

  up to Day 28

• To assess the safety and tolerability of a single oral administration of TRX-100

  Change from baseline in heart rate (beats per minute)

  up to Day 28

• To assess the safety and tolerability of a single oral administration of TRX-100

  Change from baseline in body temperature (Celsius)

  up to Day 28

• To assess the safety and tolerability of a single oral administration of TRX-100 a

  Change from baseline in QT interval (miliseconds)

  up to Day 28

Secondary Outcomes (7)

• To measure the pharmacokinetics of TRX-100 and its active metabolite TRX-101 in plasma

  up to Day 28

• To measure the pharmacokinetics of TRX-100 and its active metabolite TRX-101 in plasma

  up to Day 28

• To measure the pharmacokinetics of TRX-100 and its active metabolite TRX-101 in plasma

  up to Day 28

• To measure the pharmacokinetics of TRX-100 and its active metabolite TRX-101 in plasma

  up to Day 28

• To measure the pharmacokinetics of TRX-100 and its active metabolite TRX-101 in plasma

  up to Day 28

Other Outcomes (1)

• Exploratory (optional): To evaluate TRX100/TRX101 target engagement.

  up to Day 28

Study Arms (4)

Single dose level 1 or placebo

EXPERIMENTAL

SAD dose level 1

Drug: TRX-100; Drug: Placebo

Single dose level 2 or placebo

EXPERIMENTAL

SAD dose level 2

Drug: TRX-100; Drug: Placebo

Single dose level 3 or placebo

EXPERIMENTAL

SAD dose level 3

Drug: TRX-100; Drug: Placebo

Single dose level 4 or placebo

EXPERIMENTAL

SAD dose level 4

Drug: TRX-100; Drug: Placebo

Interventions

TRX-100 DRUG

CEN inhibitor, dosage form - capsules, dosing regimen - QD

Single dose level 1 or placebo; Single dose level 2 or placebo; Single dose level 3 or placebo; Single dose level 4 or placebo

Placebo DRUG

Placebo, dosage form - capsules, dosing regimen - QD

Single dose level 1 or placebo; Single dose level 2 or placebo; Single dose level 3 or placebo; Single dose level 4 or placebo

Eligibility Criteria

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

You may qualify if:

• Must have given written informed consent before any study-related activities are carried out and must be able to understand the full nature and purpose of the trial, including possible risks and adverse effects.
• Adult males and females, 18 to 64 years of age (inclusive) at screening.
• Body mass index (BMI) ≥ 18.5 and ≤ 32.0 kg/m2, with a body weight (to 1 decimal place) ≥ 50.0 kg at screening.
• Medically healthy without clinically significant abnormalities (in the opinion of the Investigator) at the screening visit and prior to dosing at the timepoints indicated in the SoA, including:
• Physical examination without any clinically significant findings in the opinion of the Investigator.
• Systolic blood pressure in the range of 90 mm Hg to 149 mm Hg; diastolic blood pressure in the range of 50 mm Hg to 90 mm Hg after 5 minutes in a supine or semi-supine position
• Heart rate (HR) in the range of 40 to 100 bpm
• Body temperature (tympanic or oral) in the range 35.5°C to 37.5°C (inclusive).
• No clinically significant findings in serum chemistry, hematology, coagulation and urinalysis tests as judged by the Investigator.
• Triplicate 12-lead ECG (taken after the volunteer has been supine or semi-supine for at least 5 minutes) with a QTcF ≤ 450 msec for males and ≤ 470 msec for females and no clinically significant abnormalities.
• Hemoglobin A1C (HbA1c) levels within the local laboratory standard reference range.
• Be willing to not smoke (including tobacco, nicotine replacement therapy, e-cigarettes) from 7 days prior to dose administration on Day 1 to Day 12 (inclusive) for all cohorts. For optional Cohort 4 only, participants must also be willing to not smoke (including tobacco, nicotine replacement therapy, e-cigarettes) from 7 days prior to dose administration on Day 28 to Day 39 (inclusive) .
• Female volunteers must:
• Be of nonchildbearing potential i.e., surgically sterilized (hysterectomy, bilateral salpingectomy, bilateral oophorectomy at least 6 weeks before screening) or postmenopausal (where postmenopausal is defined as no menses for 12 months without an alternative medical cause, and if under the age of 55 years a follicle-stimulating hormone (FSH) level \>40 IU/L at the screening visit), or
• If of childbearing potential, must agree not to donate ova, not to attempt to become pregnant and, if engaging in sexual intercourse with a male partner, must agree to use an acceptable method of contraception from signing the consent form until at least 30 days after the last dose of the study drug.

You may not qualify if:

• History or presence of significant cardiovascular, pulmonary, hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic or neurological disease, including any acute illness or major surgery within the past 3 months determined by the PI to be clinically significant.
• Acute infections within 4 weeks prior to screening or current infection that requires systemically absorbed antibiotic, antifungal, antiparasitic or antiviral medications.
• Presence or history of any abnormality or illness, including gastrointestinal surgery, which in the opinion of the PI may affect absorption, distribution, metabolism or elimination of the study drug.
• Any history of malignant disease in the last 5 years (excludes surgically resected skin squamous cell or basal cell carcinoma).
• Any screening laboratory result outside the normal laboratory reference range (as confirmed upon repeated testing) and deemed clinically significant by the PI.
• Presence of clinically relevant immunosuppression from, but not limited to, immunodeficiency conditions such as common variable hypogammaglobulinemia.
• Use of or plans to use systemic immunosuppressive (e.g., corticosteroids by any route, methotrexate, azathioprine, cyclosporine) or immunomodulating medications (e.g., interferon) during the study or within 5 half-lives of individual agent or within 28 days (whichever is longer) prior to Screening.
• Use of or plans to use agents (e.g., grapefruit and grapefruit products) that have clinically significant interaction with CYP3A4 or the use of any medications that could have a significantly impact on organ function (e.g., barbiturates, omeprazole, cimetidine) during the study or within 5 half-lives of individual agent or within 7 days (whichever is longer) prior to dosing.
• History of risk factors for torsade de pointes (including a family history of long QT syndrome or sudden cardiac death) or clinically significant arrythmia.
• Participant is planning to have surgery between Screening and the EoS visit.
• Positive test results for active human immunodeficiency virus (HIV-1 or HIV-2), hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibodies at the screening visit.
• Presence or having sequelae of gastrointestinal, liver, kidney, or other conditions known to interfere with the absorption, distribution, metabolism, or excretion of drugs.
• Estimated creatinine clearance \< 60 mL/min using the Cockcroft-Gault formula.
• Creatine kinase \>2.0 x ULN at Day -1.
• History of any drug or alcohol abuse in the past 2 years defined as \>21 units of alcohol per week for males and \>14 units of alcohol per week for females. Where 1 unit = 360 mL of beer, 150 mL wine, or 30 mL of spirits.

Sponsors & Collaborators

• Traws Pharma, Inc.: lead

Study Sites (1)

Scientia Clinical Research

Sydney, Greater Sydney Area, NSW 2031, Australia

Location

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: double-blind, placebo-controlled
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Participants will be randomized into 4 cohorts (8 participant per cohort) to receive study drug

Study Record Dates

• First Submitted: December 16, 2024
• First Posted: January 3, 2025
• Study Start: August 13, 2024
• Primary Completion: January 16, 2025
• Study Completion: January 24, 2025
• Last Updated: January 21, 2026

Record last verified: 2026-01

Locations

AU (1)