Safety, Pharmacokinetics, and Pharmacodynamics of MTX-101 in Healthy Adults and Patients

First in human study to understand the potential side effects of MTX-101, how long MTX-101 lasts in the human body, and how MTX-101 affects specific human immune cells.

Study Overview

• Status: Recruiting
• Conditions: Type 1 Diabetes; Healthy Volunteers
• Intervention / Treatment: Drug: MTX-101; Drug: Placebo

Detailed Description

A Phase 1, Part A -prospective, randomized, single-blind, placebo-controlled, single ascending dose (SAD) and multiple ascending dose (MAD) study to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of MTX-101 in healthy adults (HA). Part B - A multi-center, randomized, open-label study to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of MTX-101 in participants with type 1 diabetes (T1D).

• Study Type: Interventional
• Enrollment (Estimated): 96
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Heather Director, Clinical Operations; Phone Number: 1-253-358-9586; Email: hwroe@mozart-tx.com

Study Locations

• Australia
  • Fitzroy, Australia, 3065
    • Recruiting
    • St Vincent's Hospital Melbourne (SVHM)
    • Principal Investigator: David O'Neal, MD
  • Victoria
    • Heidelberg, Victoria, Australia, 3084
      • Recruiting
      • Austin Health
      • Principal Investigator: Elif Ekinci, MD
    • Melbourne, Victoria, Australia, 3050
      • Recruiting
      • The Royal Melbourne Hospital
      • Principal Investigator: John Wentworth, MD

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

• Adults, age ≥ 18 and ≤ 65 years at the time of anticipated dosing (Day 1).
• Healthy individuals without known current or chronic medical conditions, including no history of any autoimmune diseases, in the opinion of the Investigator.
• Body mass index (BMI) ≥ 18 kg/m2 and ≤ 35 kg/m2 AND body weight ≥ 55 and ≤ 120 kg.
• Negative Coronavirus Disease 2019 (COVID-19) test within 24 hours prior to each dose.
• Persons of child-bearing potential must have a negative pregnancy test and either abstain from sex or use highly effective method(s) of birth control from Day 1 through the duration of the study.

Exclusion Criteria:

• Clinically significant findings in physical examination (PE), vital signs (blood pressure, heart rate, and body temperature), electrocardiogram (ECG), and safety laboratory parameters at Screening in the opinion of the Investigator.
• Prior or concurrent malignancies.
• Renal function calculated by the Chronic Kidney Disease-Epidemiology (CKD-EPI) equation with estimated glomerular filtration rate (eGFR) < 90 mL/min/1.73 m2 or abnormal level of proteinuria detected by dipstick at the time of Screening.
• Any disease or condition that, in the opinion of the Investigator, might significantly compromise the cardiovascular, hematological, renal, hepatic, pulmonary (including chronic asthma), endocrine (e.g., diabetes), central nervous, or gastrointestinal (including an ulcer) systems.
• Receipt of an investigational drug within 28 days or 5 half-lives (whichever is longer) of the investigational drug(s) prior to Day 1.
• Positive serology for human immunodeficiency virus (HIV) type 1 or 2, hepatitis (Hep) B surface antigen, or Hep C.
• Positive test results for drug screen, including alcohol, at the time of Screening or on Day 1 prior to randomization.
• Use of tobacco or nicotine-containing products more than the equivalent of 5 cigarettes/week within 30 days prior to (first) dosing.

Participants must abstain from nicotine use while inpatient.

• History of receiving a live vaccine within 1 month of Screening.
• History of splenectomy.
• History of COVID or influenza vaccine within 2 weeks prior to Screening.
• Planning to receive any vaccinations during the study period.
• History of recurrent infections of uncertain cause.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Basic Science
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Triple

Number of Arms

8

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Cohort AS1 - Healthy Volunteers; (n = 6): MTX-101, Dose level 1 IV or Placebo IV, Single dose	Drug: MTX-101; MTX-101 (bispecific CD8 Treg modulator); Drug: Placebo; MTX-101
Placebo Comparator: Cohort AS2 - Healthy Volunteers; (n = 6): MTX-101, Dose Level 2 IV or Placebo IV, Single dose	Drug: MTX-101; MTX-101 (bispecific CD8 Treg modulator); Drug: Placebo; MTX-101
Placebo Comparator: Cohort AS3 - Healthy Vounteers; (n = 6): MTX-101, Dose Level 3 IV or Placebo IV, single dose	Drug: MTX-101; MTX-101 (bispecific CD8 Treg modulator); Drug: Placebo; MTX-101
Placebo Comparator: Cohort AS4 - Healthy Volunteers; (n =6): MTX-101, Dose Level 4 IV or Placebo IV, single dose	Drug: MTX-101; MTX-101 (bispecific CD8 Treg modulator); Drug: Placebo; MTX-101
Placebo Comparator: Cohort AS5 - Healthy Volunteers; (n = 6): MTX-101, Dose level 6 IV or Placebo IV, Single Dose	Drug: MTX-101; MTX-101 (bispecific CD8 Treg modulator); Drug: Placebo; MTX-101
Placebo Comparator: Cohort AM1 - Healthy Volunteers; Cohort AM1 (n = 6): MTX-101, Dose Level 5 IV or Placebo IV, dosed on Days 1 and 22 for a total of 2 doses	Drug: MTX-101; MTX-101 (bispecific CD8 Treg modulator); Drug: Placebo; MTX-101
Placebo Comparator: Cohort B8 - Type 1 Diabetes Patients; Cohort B8 (n=12):; MTX-101 up to Dose Group 4 IV Day 1 and 29	Drug: MTX-101; MTX-101 (bispecific CD8 Treg modulator)
Experimental: Cohort B9 - Type 1 Diabetes Patients; Optional Cohort B9 (n=12): • MTX-101 up to Dose 6 IV Day 1 and 29	Drug: MTX-101; MTX-101 (bispecific CD8 Treg modulator)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety of single, ascending dose levels of MTX-101	Assess the safety of single, ascending dose levels of MTX-101 by evaluating the incidence, severity, and seriousness of treatment-emergent adverse events	Enrollment to 8 weeks post dose
Safety of multiple, ascending dose levels of MTX-101	Assess the safety of multiple, ascending dose levels of MTX-101by evaluating the incidence, severity, and seriousness of treatment-emergent adverse events	Enrollment to 11 weeks following the last dose

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
pharmacokinetics (PK) of MTX-101	Characterize the pharmacokinetics (PK) of MTX-101 by measuring the maximum time of occurrence for maximum plasma drug concentration (Cmax)	Enrollment to 11 weeks following the last dose
pharmacokinetics (PK) of MTX-101	Characterize the pharmacokinetics (PK) of MTX-101 by measuring the time of occurrence for maximum plasma drug concentration (Tmax).	Enrollment to 11 weeks following the last dose
pharmacokinetics (PK) of MTX-101	Characterize the pharmacokinetics (PK) of MTX-101 by measuring the maximum plasma drug concentration (Cmax), minimum plasma drug concentration (Cmin), and area under the plasma drug concentration versus time curve from time 0 to last measurable concentration (AUC(0-t))	Enrollment to 11 weeks following the last dose
anti-drug antibody (ADA) formation	Evaluate incidence of anti-drug antibody (ADA) formation by measuring the detect the presence of anti-MTX-101 antibodies in participant's blood.	Enrollment to 11 weeks following the last dose

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
pharmacodynamics (PD) of MTX-101	Evaluate how MTX-101 affect the immune system by the measuring the activity, presence and amount of signaling proteins and cells that help control inflammation.	Enrollment up to 11 weeks following the last dose
Receptor occupancy of MTX-101	To examine the binding ability of MTX-101 to targets on the cell surface.	Enrollment up to 11 weeks following the last dose

Collaborators and Investigators

• Sponsor: Mozart Therapeutics Australia Pty Ltd

Study record dates

Study Major Dates

• Study Start (Actual): June 13, 2024
• Primary Completion (Estimated): March 31, 2027
• Study Completion (Estimated): June 30, 2027

Study Registration Dates

• First Submitted: February 27, 2024
• First Submitted That Met QC Criteria: March 21, 2024
• First Posted (Actual): March 22, 2024

Study Record Updates

• Last Update Posted (Actual): May 18, 2026
• Last Update Submitted That Met QC Criteria: May 14, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Healthy Volunteers; Type 1 Diabetes
• Additional Relevant MeSH Terms: Endocrine System Diseases; Metabolic Diseases; Autoimmune Diseases; Immune System Diseases; Glucose Metabolism Disorders; Diabetes Mellitus; Nutritional and Metabolic Diseases; Diabetes Mellitus, Type 1
• Other Study ID Numbers: MT-101-101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

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