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

Mozart Therapeutics

Status and phase: Enrolling; Phase 1

Conditions: Healthy Volunteers; Type 1 Diabetes; Celiac Disease

Treatments: Drug: Placebo; Drug: MTX-101

Study type: Interventional

Funder types: Industry

Identifiers

NCT06324604

MT-101-101

Details and patient eligibility

About

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.

Full description

This is a prospective, multi-center, randomized, double-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) and participants with celiac disease (CeD) and type 1 diabetes (T1D). This study will enroll HAs only in Part A and CeD and T1D patients only in Part B.

Enrollment: 96 estimated patients

Sex: All

Ages: 18 to 65 years old

Volunteers: Accepts Healthy Volunteers

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 ≤ 32 kg/m2.
• Body weight ≥ 45and ≤ 100 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.
• 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.

Trial design

Primary purpose: Basic Science

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Triple Blind

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96 participants in 8 patient groups, including a placebo group

Cohort AS1 - Healthy Volunteers

Placebo Comparator group

Description:

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

Treatment:

Drug: MTX-101

Drug: Placebo

Cohort AS2 - Healthy Volunteers

Placebo Comparator group

Description:

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

Treatment:

Drug: MTX-101

Drug: Placebo

Cohort AS3 - Healthy Vounteers

Placebo Comparator group

Description:

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

Treatment:

Drug: MTX-101

Drug: Placebo

Cohort AS4 - Healthy Volunteers

Placebo Comparator group

Description:

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

Treatment:

Drug: MTX-101

Drug: Placebo

Cohort AS5 - Healthy Volunteers

Placebo Comparator group

Description:

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

Treatment:

Drug: MTX-101

Drug: Placebo

Cohort AM1 - Healthy Volunteers

Placebo Comparator group

Description:

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

Treatment:

Drug: MTX-101

Drug: Placebo

Cohort B8 - Celiac Disease or Type 1 Diabetes Patients

Placebo Comparator group

Description:

* Dose Group 1 (n = 6): MTX-101 Dose Level 4 IV Day 1 and 29, placebo IV Day 57 * Dose Group 2 (n = 6): Placebo IV Day 1, MTX-101 Dose Level 3 IV Day 29 and 57

Treatment:

Drug: MTX-101

Drug: Placebo

Cohort B9 -Celiac Disease or Type 1 Diabetes Patients

Placebo Comparator group

Description:

Dose Group 1 (n = 6): MTX-101 Dose Level 5 IV Days 1 and 29 Dose Group 2 (n = 6): Placebo IV Day 1, MTX-101 Dose Level 5 IV (or the maximum tolerated dose in Part A MAD) Day 29

Treatment:

Drug: MTX-101

Drug: Placebo