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NCT Number: NCT07734493

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

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.

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Key information

Conditions

Schizophrenia Mental Disorders Schizophrenia Spectrum and Other Psychotic Disorders

Age range

18 year–55 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Pillar Clinical Research, LLC, Little Rock, Arkansas, United States

Loading trial locations.

About this study

After providing informed consent, eligible participants will be asked to visit the study site 3 times and stay overnight for 35 nights (up to 49 nights if up to 14 days of antipsychotic washout is applicable). Procedures will include:

  • A review of medical history and medications
  • Physical exams, including a neurologic (nervous system) assessment
  • Electrocardiograms (a measure of the heart's electrical activity)
  • Blood and urine collection
  • Psychiatric assessments including the Mini International Neuropsychiatric Exam (MINI), the Brief Assessment of Cognition in Schizophrenia (BACS), and the Positive and Negative Syndrome Scale (PANSS)
  • Overall assessment of status via the Clinical Global Impression Scale - Severity (CGI-S)
  • Assessment of 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)
  • Assessment of suicidality risk via the Columbia Suicidality Severity Rating Scale (C-SSRS)
  • Quantitative electroencephalography (qEEG) assessments
  • Ophthalmologic examinations
  • Administration of the study drug (or placebo) by mouth

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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

Treatment and study plan

LTX-001 once daily

Drug

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

Placebo

Drug

Oral suspension without LTX-001

LTX-001 twice daily

Drug

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

Primary outcomes

  1. Safety and Tolerability - Adverse Events

    Time frame: Screening through Day 42

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

  2. Safety and Tolerability - Clinical Laboratory Tests

    Time frame: Screening through Day 42

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

  3. Safety and Tolerability - Electrocardiograms (ECG)

    Time frame: Screening through Day 42

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

  4. Safety and Tolerability - Physical and Neurological Exams

    Time frame: Screening through Day 42

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

  5. Safety and Tolerability - Suicidality

    Time frame: Screening through Day 42

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

  6. Safety and Tolerability - Extrapyramidal Symptoms

    Time frame: Screening through Day 42

    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)

  7. Vital signs - blood pressure

    Time frame: Screening through Day 42

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

  8. Vital Signs - heart rate

    Time frame: Screening through Day 42

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

  9. Vital Signs - respiratory rate

    Time frame: Screening through Day 42

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

  10. Vital Signs - body temperature

    Time frame: Screening through Day 42

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

  11. Vital Signs - total body weight

    Time frame: Screening through Day 42

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

  12. Vital Signs - height

    Time frame: Screening through Day 42

    Vital signs will be collected, including height (centimeters)

  13. Vital Signs - Body Mass Index (BMI)

    Time frame: Screening through Day 42

    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.

  14. Ocular evaluation - Best Corrected Visual Acuity (BCVA)

    Time frame: Screening through Day 42

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

  15. Ocular evaluation - dilated Color Fundus Photography (CFP)

    Time frame: Screening through Day 42

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

  16. Ocular evaluation - Fundus Autofluorescence (FAF)

    Time frame: Screening through Day 42

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

  17. Ocular evaluation - dilated Optical Coherence Tomography (OCT)

    Time frame: Screening through Day 42

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

  18. Ocular evaluation - slit lamp examination

    Time frame: Screening through Day 42

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

Secondary outcomes

  1. Pharmacokinetics of LTX-001 - Single Dose

    Time frame: Days 1 and 8

    Single-dose plasma PK parameters (Days 1 and 8) will be measured

  2. Pharmacokinetics of LTX-001 - Multiple Dose

    Time frame: Days 8 and 35

    Multiple-dose plasma PK parameters (Day 35) will be measured.

Other outcomes

  1. Efficacy of LTX-001 - PANSS

    Time frame: Screening through Day 37

    Change from baseline in Positive and Negative Syndrome Scale (PANSS) total score and subscale scores

  2. Efficacy of LTX-001 - Marder factors of PANSS

    Time frame: Screening through Day 37

    Change from baseline in the Marder factors of PANSS.

  3. Efficacy of LTX-001 - BACS

    Time frame: Screening through Day 37

    Change from baseline in the Brief Assessment of Cognition in Schizophrenia (BACS)

  4. Efficacy of LTX-001 - CGI-S

    Time frame: Screening through Day 37

    Change from baseline in Clinical Global Impression Scale-Severity (CGI-S) score

  5. Pharmacodynamics of LTX-001 - plasma glutamate and glutamine

    Time frame: Day 1 through Day 36

    Change from baseline in plasma glutamine (Gln) and glutamate (Glu) concentrations and Gln/Glu ratio. In addition, using glutamine change from baseline data, the individual AUEC0-4, AUEC0-12 and AUEC0-24 will be presented for each patient, by treatment group on Days 1, 8, and 35

  6. Efficacy of LTX-001 - Quantitative electroencephalography (qEEG)

    Time frame: Day -1, Day 3 and Day 35

    Change from baseline in resting-state qEEG parameters (alpha, beta, gamma, delta, and theta) on Days -1, 3, and 35.

  7. Pharmacodynamics of LTX-001 - Metabolite Profiling

    Time frame: Day 1 through Day 36

    Metabolite profiling and identification in pooled plasma samples of selected days from LTX-001 once and twice daily groups

Study contacts

Contact information is provided by the study sponsor or research team.

Clinical Trials Information Medical Monitor

CONTACT

[email protected]

267-503-4800

Study Director Medical Monitor

CONTACT

Sponsors and collaborators

Lead sponsor

Leal Therapeutics, Inc

Industry

Registry information

Official study title

A Phase 1b, Multicenter, Randomized, Double-Blind, Dose-Escalation, Placebo-Controlled Study to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, And Early Signs of Efficacy of Orally Administered LTX-001 As Monotherapy in Adult Participants With Acute Exacerbation of Schizophrenia

Important dates

Study start
2026
Primary completion
2027
Study completion
2027
First posted
Jul 29, 2026
Registry last updated
Aug 4, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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