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

An Open-label, Multicenter Study to Determine the Pharmacokinetics, Safety, and Tolerability of AX251 Long-Acting Injectable (LAI) Administered as a Single Dose in Patients With Schizophrenia

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Schizophrenia
• Intervention / Treatment: Drug: AX251 LAI 45 mg; Drug: AX251 LAI 90 mg; Drug: AX251 LAI 135 mg; Drug: AX251 LAI 180 mg

• Study Type: Interventional
• Enrollment (Estimated): 48
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Mico Hsu; Phone Number: 886-5716223; Email: mico_hsu@anxo.com.tw

Study Locations

• India
  • Nagar, India
    • Ratandeep Multispeciality Hospital
    • Contact: Vaishal Vora; Phone Number: +91 98253 67148; Email: vaishal.vora@ratandeepmsh.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: AX251 LAI 45 mg; AX251 LAI 45mg	Drug: AX251 LAI 45 mg; Cariprazine 45 mg
Experimental: AX251 LAI 90mg	Drug: AX251 LAI 90 mg; Cariprazine 90 mg
Experimental: AX251 LAI 135mg	Drug: AX251 LAI 135 mg; Cariprazine 135 mg
Experimental: AX251 LAI 180mg	Drug: AX251 LAI 180 mg; Cariprazine 180 mg

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety: Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)	Number and proportion of participants experiencing treatment-emergent adverse events and serious adverse events during the study.	Baseline to Day 70

Collaborators and Investigators

• Sponsor: Anxo Pharmaceutical Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Estimated): July 1, 2026
• Primary Completion (Estimated): July 1, 2028
• Study Completion (Estimated): July 1, 2028

Study Registration Dates

• First Submitted: March 16, 2026
• First Submitted That Met QC Criteria: March 18, 2026
• First Posted (Actual): March 20, 2026

Study Record Updates

• Last Update Posted (Actual): March 20, 2026
• Last Update Submitted That Met QC Criteria: March 18, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: AX251
• Additional Relevant MeSH Terms: Schizophrenia Spectrum and Other Psychotic Disorders; Mental Disorders; Schizophrenia
• Other Study ID Numbers: AX251-01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No