Evaluating the safety and effects of MTX-101 in healthy adults and patients with celiac disease or type 1 diabetes

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

This study is testing a new drug called MTX-101 to see how safe it is and how it affects healthy adults and people with celiac disease or type 1 diabetes.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 96 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Mozart Therapeutics Australia Pty Ltd (Industry-sponsored)
• Locations: 4 sites (Auchenflower, Queensland and 3 other locations)
• Trial ID: NCT06324604 on ClinicalTrials.gov

What this trial studies

This clinical trial is a first-in-human study designed to assess the safety, pharmacokinetics, and pharmacodynamics of MTX-101. It involves a multi-center, randomized, double-blind, placebo-controlled approach, with participants receiving either the drug or a placebo. The study is divided into two parts: Part A includes healthy adults, while Part B focuses on patients with celiac disease and type 1 diabetes. The trial aims to understand how MTX-101 interacts with the human immune system and its duration in the body.

Who should consider this trial

Good fit: Ideal candidates include healthy adults aged 18-65 and patients diagnosed with celiac disease or type 1 diabetes.

Not a fit: Patients with significant chronic medical conditions or autoimmune diseases may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide insights into a new treatment option for patients with celiac disease and type 1 diabetes.

How similar studies have performed: Other studies evaluating similar pharmacological approaches have shown promise, but this specific study is novel in its focus on MTX-101.

Eligibility criteria

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.

Where this trial is running

Auchenflower, Queensland and 3 other locations

• Wesley Research Institute — Auchenflower, Queensland, Australia (Active_not_recruiting)
• Austin Health — Heidelberg, Victoria, Australia (Recruiting)
• The Royal Melbourne Hospital — Melbourne, Victoria, Australia (Recruiting)
• St Vincent's Hospital Melbourne (SVHM) — Melbourne, Victoria, Austria (Recruiting)

Study contacts

• Study coordinator: Heather Director, Clinical Operations

Conditions Healthy Volunteers; Type 1 Diabetes