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

May 14, 2026 updated by: Mozart Therapeutics Australia Pty Ltd
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

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
96

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.
  • 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

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Safety of single, ascending dose levels of MTX-101
Time Frame: Enrollment to 8 weeks post dose
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
Time Frame: Enrollment to 11 weeks following the last dose
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
pharmacokinetics (PK) of MTX-101
Time Frame: Enrollment to 11 weeks following the last dose
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
Time Frame: Enrollment to 11 weeks following the last dose
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
Time Frame: Enrollment to 11 weeks following the last dose
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
Time Frame: Enrollment to 11 weeks following the last dose
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

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
pharmacodynamics (PD) of MTX-101
Time Frame: Enrollment up to 11 weeks following the last dose
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
Time Frame: Enrollment up to 11 weeks following the last dose
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

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Mozart Therapeutics Australia Pty Ltd

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
June 13, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 31, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
June 30, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 27, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 21, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 22, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 18, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 14, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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