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

NCT06324604 | Recruiting Phase 1 DMC

• 1 other identifier: MT-101-101
• Study Type: interventional
• Target: 96
• Locations: 1 country
• Sites: 3

Brief Summary

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.

Conditions

Type 1 Diabetes; Healthy Volunteers

Keywords

Healthy Volunteers; Type 1 Diabetes

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (4)

• pharmacokinetics (PK) of MTX-101

  Enrollment to 11 weeks following the last dose

• pharmacokinetics (PK) of MTX-101

  Enrollment to 11 weeks following the last dose

• pharmacokinetics (PK) of MTX-101

  Enrollment to 11 weeks following the last dose

• anti-drug antibody (ADA) formation

  Enrollment to 11 weeks following the last dose

Other Outcomes (2)

• pharmacodynamics (PD) of MTX-101

  Enrollment up to 11 weeks following the last dose

• Receptor occupancy of MTX-101

  Enrollment up to 11 weeks following the last dose

Study Arms (8)

Cohort AS1 - Healthy Volunteers

PLACEBO COMPARATOR

(n = 6): MTX-101, Dose level 1 IV or Placebo IV, Single dose

Drug: Placebo; Drug: MTX-101

Cohort AS2 - Healthy Volunteers

PLACEBO COMPARATOR

(n = 6): MTX-101, Dose Level 2 IV or Placebo IV, Single dose

Drug: Placebo; Drug: MTX-101

Cohort AS3 - Healthy Vounteers

PLACEBO COMPARATOR

(n = 6): MTX-101, Dose Level 3 IV or Placebo IV, single dose

Drug: Placebo; Drug: MTX-101

Cohort AS4 - Healthy Volunteers

PLACEBO COMPARATOR

(n =6): MTX-101, Dose Level 4 IV or Placebo IV, single dose

Drug: Placebo; Drug: MTX-101

Cohort AS5 - Healthy Volunteers

PLACEBO COMPARATOR

(n = 6): MTX-101, Dose level 6 IV or Placebo IV, Single Dose

Drug: Placebo; Drug: MTX-101

Cohort AM1 - Healthy Volunteers

PLACEBO COMPARATOR

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: Placebo; Drug: MTX-101

Cohort B8 - Type 1 Diabetes Patients

PLACEBO COMPARATOR

* Cohort B8 (n=12): * MTX-101 up to Dose Group 4 IV Day 1 and 29

Drug: MTX-101

Cohort B9 - Type 1 Diabetes Patients

EXPERIMENTAL

Optional Cohort B9 (n=12): • MTX-101 up to Dose 6 IV Day 1 and 29

Drug: MTX-101

Interventions

MTX-101 DRUG

MTX-101 (bispecific CD8 Treg modulator)

Cohort AM1 - Healthy Volunteers; Cohort AS1 - Healthy Volunteers; Cohort AS2 - Healthy Volunteers; Cohort AS3 - Healthy Vounteers; Cohort AS4 - Healthy Volunteers; Cohort AS5 - Healthy Volunteers; Cohort B8 - Type 1 Diabetes Patients; Cohort B9 - Type 1 Diabetes Patients

Placebo DRUG

MTX-101

Cohort AM1 - Healthy Volunteers; Cohort AS1 - Healthy Volunteers; Cohort AS2 - Healthy Volunteers; Cohort AS3 - Healthy Vounteers; Cohort AS4 - Healthy Volunteers; Cohort AS5 - Healthy Volunteers

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Mozart Therapeutics Australia Pty Ltd: lead

Study Sites (3)

Austin Health

Heidelberg, Victoria, 3084, Australia

RECRUITING

The Royal Melbourne Hospital

Melbourne, Victoria, 3050, Australia

RECRUITING

St Vincent's Hospital Melbourne (SVHM)

Fitzroy, 3065, Australia

RECRUITING

MeSH Terms

Conditions

Diabetes Mellitus, Type 1

Condition Hierarchy (Ancestors)

Diabetes Mellitus; Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Endocrine System Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

Heather Director, Clinical Operations

CONTACT

1-253-358-9586; hwroe@mozart-tx.com

Study Design

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

Model Details: Part A will enroll Healthy Adult Volunteers will be randomized, double-blind, placebo-controlled, single ascending dose (SAD), randomized to MTX-101 or placebo in a 1:1 ratio for the first 2 participants (sentinel dosing) and a 3:1 ratio thereafter. Participants in the multiple ascending dose (MAD) will be randomized in a 3:1 ratio and be dosed on Days 1 and 22. Part B of this study plans to enroll at least 24 (up to 44) participants with CeD or T1D, randomized in a 1:1 ratio, in 2 sequential cohorts. Approximately 12 CeD and 12 T1D patients will be enrolled in Part B, with the option to enroll up to a maximum of 44 total (T1D patients). Participants in Cohort B8 will be dosed with MTX-101 on Days 1 \& 29. Participants in Cohort B9 will be dosed with MTX-101 or placebo on Day 1 and MTX-101 on Day 29. Patients will be followed for 6 months after the first dose.

Study Record Dates

• First Submitted: February 27, 2024
• First Posted: March 22, 2024
• Study Start: June 13, 2024
• Primary Completion (Estimated): March 31, 2027
• Study Completion (Estimated): June 30, 2027
• Last Updated: May 18, 2026

Record last verified: 2026-05

Locations

AU (3)