Evaluate Pharmacokinetics, Safety, and Tolerability of AX251 LAI in Patients With Schizophrenia

NCT07484204 | Not Yet Recruiting Phase 1 FDA Drug

• 1 other identifier: AX251-01
• Study Type: interventional
• Target: 48
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to assess the pharmacokinetics (PK), safety, and tolerability of AX251 long-acting injectable (LAI) administered as a single dose in patients with schizophrenia. The study will include sequential dose-escalation cohorts to evaluate different dose levels of AX251 LAI.

Conditions

Schizophrenia

Keywords

AX251

Outcome Measures

Primary Outcomes (1)

• Pharmacokinetic Parameter

  Maximum observed serum concentration of cariprazine and its metabolites desmethyl-cariprazine (DCAR) and didesmethyl-cariprazine (DDCAR) following a single dose of AX251 long-acting injectable (LAI).

  Day 1 pre-dose and 1, 2, 3, 4, 8, 12, and 24 hours post-dose. Days 3, 5, 7, 14, 21, 28, 42, 56, and 70

Secondary Outcomes (1)

• Safety: Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)

  Baseline to Day 70

Study Arms (4)

AX251 LAI 45 mg

EXPERIMENTAL

AX251 LAI 45mg

Drug: AX251 LAI 45 mg

AX251 LAI 90mg

EXPERIMENTAL

AX251 LAI 90mg

Drug: AX251 LAI 90 mg

AX251 LAI 135mg

EXPERIMENTAL

AX251 LAI 135mg

Drug: AX251 LAI 135 mg

AX251 LAI 180mg

EXPERIMENTAL

AX251 LAI 180mg

Drug: AX251 LAI 180 mg

Interventions

AX251 LAI 45 mg DRUG

Cariprazine 45 mg

AX251 LAI 45 mg

AX251 LAI 90 mg DRUG

Cariprazine 90 mg

AX251 LAI 90mg

AX251 LAI 135 mg DRUG

Cariprazine 135 mg

AX251 LAI 135mg

AX251 LAI 180 mg DRUG

Cariprazine 180 mg

AX251 LAI 180mg

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female subjects aged 18 to 65 years (inclusive).
• clinical diagnosis of schizophrenia per the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition, Text Revision (DSM-5-TR) (or later) criteria, at screening.
• Body Mass Index (BMI) 18.5-35.0 kg/m² at screening.
• Clinical Global Impression-Severity (CGI-S) score ≤4 at screening.
• Positive and Negative Syndrome Scale (PANSS) total score ≤75 at screening.
• Clinically stable schizophrenia on current antipsychotic medication other than cariprazine for at least 3 months prior to screening.
• Subjects must not be taking more than two antipsychotic medications.
• Able to remain at the study site for 7 days following AX251 LAI injection.
• Judged by the investigator to be physically and mentally able to participate in the study based on clinical evaluation, physical examination, electrocardiogram (ECG), and laboratory assessments.
• Willing and able to comply with study procedures.
• Agrees to use protocol-defined contraception during the study.
• Previous tolerability to oral cariprazine (1.5-6 mg) or willingness to undergo a short oral tolerability test (2-7 days) followed by washout before study drug administration.

You may not qualify if:

• Diagnosis of psychiatric disorders other than schizophrenia according to DSM-5-TR (e.g., schizoaffective disorder, major depressive disorder, bipolar disorder, generalized anxiety disorder, obsessive-compulsive disorder, post-traumatic stress disorder, dementia, mild neurocognitive disorder, or personality disorders), except caffeine- or tobacco-related disorders.
• Substance use disorder (including alcohol or benzodiazepines) within 180 days prior to screening, excluding caffeine and tobacco.
• History of neuroleptic malignant syndrome, seizure disorder, or clinically significant tardive dyskinesia, akathisia, or extrapyramidal symptoms.
• Clinically significant cardiovascular, hematologic, metabolic, hepatic, renal, immunologic, or neurological disease that may interfere with study participation.
• Severe hepatic impairment (Child-Pugh Class C) at screening.
• Uncontrolled hypertension.
• Clinically significant electrocardiogram abnormalities at screening.
• Severe renal impairment (estimated glomerular filtration rate \<30 mL/min).
• History of syncope or significant orthostatic hypotension at screening.
• Failure to complete required washout period (≥5 half-lives) for prohibited medications before administration of AX251 LAI.
• Current treatment with other antipsychotic long-acting injectable (LAI) therapies.
• Electroconvulsive therapy within 60 days prior to screening.
• Acute psychosis posing imminent risk to self or others.
• Acute relapse of schizophrenia at screening.

Sponsors & Collaborators

• Anxo Pharmaceutical Co., Ltd.: lead

Study Sites (1)

Ratandeep Multispeciality Hospital

Nagar, India

Location

MeSH Terms

Conditions

Schizophrenia

Condition Hierarchy (Ancestors)

Schizophrenia Spectrum and Other Psychotic Disorders; Mental Disorders

Central Study Contacts

Mico Hsu

CONTACT

886-5716223; mico_hsu@anxo.com.tw

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 16, 2026
• First Posted: March 20, 2026
• Study Start (Estimated): July 1, 2026
• Primary Completion (Estimated): July 1, 2028
• Study Completion (Estimated): July 1, 2028
• Last Updated: March 20, 2026

Record last verified: 2026-03

Locations

IN (1)