A Study of LTX-001 In Adult Participants With Acute Exacerbation of Schizophrenia

NCT07734493 | Not Yet Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: LTX-001-103
• Study Type: interventional
• Target: 90
• Locations: 1 country
• Sites: 10

Brief Summary

The purpose of this clinical trial is to learn whether the investigational drug LTX-001 is safe and well tolerated in adults with a diagnosis of schizophrenia who are having a recent worsening of their symptoms (an acute exacerbation). Additional questions it aims to answer are:

• how does the amount of the study drug in the body change over time (pharmacokinetics)
• how does the study drug affect the symptoms of schizophrenia
• how do certain markers in the blood change after administration of LTX-001 (pharmacodynamics) Researchers will compare different dose levels of LTX-001 to an inactive product (placebo) to see what differences there are between the two treatments. After successfully screening for the study, participants will stay in a research unit for about five weeks and return one week later for a follow-up visit.

Conditions

Schizophrenia

Keywords

schizophrenia

Outcome Measures

Primary Outcomes (18)

• Safety and Tolerability - Adverse Events

  Incidence and severity of treatment-emergent adverse events (TEAEs), and treatment-emergent serious adverse events (TESAEs).

  Screening through Day 42

• Safety and Tolerability - Clinical Laboratory Tests

  Clinical laboratory tests (including serum chemistry, hematology, urinalysis)

  Screening through Day 42

• Safety and Tolerability - Electrocardiograms (ECG)

  ECGs will measure heart rate (HR), QRS complex and QT, QTcF, and PR intervals

  Screening through Day 42

• Safety and Tolerability - Physical and Neurological Exams

  Physical and neurological exams will be performed periodically to ensure participant safety.

  Screening through Day 42

• Safety and Tolerability - Suicidality

  Suicidality as assessed by the Columbia Suicide Severity Rating Scale (C-SSRS)

  Screening through Day 42

• Safety and Tolerability - Extrapyramidal Symptoms

  Extrapyramidal symptoms (EPS) as assessed by the Modified Simpson-Angus Scale (M-SAS), the Abnormal Involuntary Movement Scale (AIMS) and the Barnes Akathisia Rating scale (BARS)

  Screening through Day 42

• Vital signs - blood pressure

  Vital signs will be collected, including blood pressure (mm Hg)

  Screening through Day 42

• Vital Signs - heart rate

  Vital signs will be collected, including heart rate (beats per minute)

  Screening through Day 42

• Vital Signs - respiratory rate

  Vital signs will be collected, including respiratory rate (breaths per minute)

  Screening through Day 42

• Vital Signs - body temperature

  Vital signs will be collected, including body temperature (°C or °F)

  Screening through Day 42

• Vital Signs - total body weight

  Vital signs will be collected, including total body weight (kilograms)

  Screening through Day 42

• Vital Signs - height

  Vital signs will be collected, including height (centimeters)

  Screening through Day 42

• Vital Signs - Body Mass Index (BMI)

  Body Mass Index (BMI) will be calculated using the values of total body weight and height, with the formula BMI = weight in kg/(height in cm)\^2.

  Screening through Day 42

• Ocular evaluation - Best Corrected Visual Acuity (BCVA)

  Ocular evaluations, including Best Corrected Visual Acuity (BCVA) will be performed

  Screening through Day 42

• Ocular evaluation - dilated Color Fundus Photography (CFP)

  Ocular evaluations, including dilated Color Fundus Photography (CFP) will be performed

  Screening through Day 42

• Ocular evaluation - Fundus Autofluorescence (FAF)

  Ocular evaluations, including Fundus Autofluorescence (FAF) will be performed

  Screening through Day 42

• Ocular evaluation - dilated Optical Coherence Tomography (OCT)

  Ocular evaluations, including dilated Optical Coherence Tomography (OCT) of the macula and optic nerve will be performed

  Screening through Day 42

• Ocular evaluation - slit lamp examination

  Ocular evaluations, including slit lamp examination with measurement of intraocular pressure (IOP) will be performed

  Screening through Day 42

Secondary Outcomes (2)

• Pharmacokinetics of LTX-001 - Single Dose

  Days 1 and 8

• Pharmacokinetics of LTX-001 - Multiple Dose

  Days 8 and 35

Other Outcomes (7)

• Efficacy of LTX-001 - PANSS

  Screening through Day 37

• Efficacy of LTX-001 - Marder factors of PANSS

  Screening through Day 37

• Efficacy of LTX-001 - BACS

  Screening through Day 37

Study Arms (3)

Placebo

PLACEBO COMPARATOR

Placebo

Drug: Placebo

LTX-001 once daily

EXPERIMENTAL

LTX-001

Drug: LTX-001 once daily

LTX-001 twice daily

EXPERIMENTAL

LTX-001

Drug: LTX-001 twice daily

Interventions

LTX-001 once daily DRUG

LTX-001 40 mg and 60 mg administered as oral suspension

LTX-001 once daily

Placebo DRUG

Oral suspension without LTX-001

Placebo

LTX-001 twice daily DRUG

LTX-001 30 mg and 60 mg administered as oral suspension

LTX-001 twice daily

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• BMI \> 17.5 and \< 38.0 kg/m2 and a total body weight \> 50.0 kg (110 lbs)
• Primary diagnosis of schizophrenia per the Diagnostic and Statistical Manual of Mental Disorders, 5th Edition (DSM-5-TR)
• Experiencing an acute exacerbation or relapse of symptoms, with onset less than 2 months prior to screening for the study
• PANSS total score ≥ 80 at the time of screening for the study
• Not currently on antipsychotic medication, or is willing to discontinue all prohibited psychotropic medications prior to and during the trial period
• Sexually active adults must be willing to use an acceptable contraceptive method throughout the study

You may not qualify if:

• Evidence of organ dysfunction or any clinically significant abnormal finding at physical examination
• History of treatment resistance (e.g., failure to respond to ≥ 2 adequate antipsychotic trials) or failure to respond to use of clozapine
• Current DSM-5-TR diagnosis other than schizophrenia
• Pregnant or lactating female
• Substance use disorder within 1 year prior to screening or recreational use of drugs within 3 months prior to screening
• Moderate to severe alcohol use disorder as per DSM-5-TR within the past year or excessive use of alcohol within 12 months prior to screening
• Positive testing for human immunodeficiency virus (HIV)-1 and -2 antibody, hepatitis B surface antigen (HBsAg), or hepatitis C virus (HCV) antibody at screening

Sponsors & Collaborators

• Leal Therapeutics, Inc: lead

Study Sites (10)

Pillar Clinical Research, LLC

Little Rock, Arkansas, 72204, United States

Location

Collaborative Neuroscience Research, LLC

Los Alamitos, California, 90720, United States

Location

University Clinical Research-DeLand, LLC d/b/a Accel Research Sites - DeLand Clinical Research Unit

DeLand, Florida, 32720, United States

Location

Behavioral Clinical Research, Inc.

Hollywood, Florida, 33021, United States

Location

CenExel Hollywood FL

Hollywood, Florida, 33024, United States

Location

Innovative Clinical Research, Inc.

Miami Lakes, Florida, 33016, United States

Location

CenExel iResearch, LLC

Decatur, Georgia, 30030, United States

Location

CBH Health LLC., dba CenExel Gaithersburg MD

Gaithersburg, Maryland, 20877, United States

Location

Hassman Research Institute, LLC

Marlton, New Jersey, 08053, United States

Location

Pillar Clinical Research, LLC

Richardson, Texas, 75080, United States

Location

MeSH Terms

Conditions

Schizophrenia

Condition Hierarchy (Ancestors)

Schizophrenia Spectrum and Other Psychotic Disorders; Mental Disorders

Central Study Contacts

Clinical Trials Information Medical Monitor

CONTACT

267-503-4800; clinical.trials@lealtx.com

Study Director Medical Monitor

CONTACT

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 20, 2026
• First Posted: July 29, 2026
• Study Start: August 1, 2026
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): March 1, 2027
• Last Updated: July 29, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (10)