Clinical Trials

Numerous clinical trials across Phase 1 through Phase 4 development have evaluated xanomeline, primarily in combination formulations such as xanomeline/trospium, for psychiatric and neurological conditions. These studies assess acute efficacy, long-term tolerability, and overall clinical potential in schizophrenia, acute psychosis, cognitive impairment, bipolar mania, schizoaffective disorder, Alzheimer's disease, and healthy volunteers. Sponsored by commercial developers like Karuna Therapeutics and Zai Lab alongside academic organizations such as the European Group for Research In Schizophrenia, trial statuses range from completed and recruiting to not yet recruiting.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07686263 NOT_YET_RECRUITING
Schizophrenia
Karuna Therapeutics, Inc., a Bristol Myers Squibb company
 2026-09-03 PHASE3
NCT07084831 NOT_YET_RECRUITING
Schizophrenia; Psychosis; Cognitive Impairment
European Group for Research In Schizophrenia
 2027-01-01 PHASE3
NCT07011732 RECRUITING
Alzheimer Disease
Bristol-Myers Squibb
 2025-07-10 PHASE3
NCT07011745 RECRUITING
Alzheimer's Disease
Bristol-Myers Squibb
 2025-07-16 PHASE3
NCT07221877 RECRUITING
Schizophrenia
Bristol-Myers Squibb
 2026-01-20 PHASE4
NCT07140913 RECRUITING
Mania; Bipolar Disorder
Bristol-Myers Squibb
 2025-10-08 PHASE3
NCT07423546 RECRUITING
SCHIZOPHRENIA 1 (Disorder); Schizoaffecitve Disorder
New York State Psychiatric Institute
 2026-07-01 PHASE1; PHASE2
NCT07063342 RECRUITING
Healthy Volunteers
Bristol-Myers Squibb
 2025-06-27 PHASE1
NCT07204418 RECRUITING
Healthy Volunteers
Karuna Therapeutics, Inc., a Bristol Myers Squibb company
 2025-10-13 PHASE1
NCT07681076 NOT_YET_RECRUITING
Healthy Volunteers
Karuna Therapeutics, Inc., a Bristol Myers Squibb company
 2026-08-03 PHASE4
NCT06923891 NOT_YET_RECRUITING
Schizophrenia
Vanguard Research Group
 2025-04-16 PHASE4
NCT07257120 COMPLETED
Healthy Volunteers
Karuna Therapeutics, Inc., a Bristol Myers Squibb company
 2026-01-09 PHASE4
NCT05304767 COMPLETED
Schizophrenia
Karuna Therapeutics, Inc., a Bristol Myers Squibb company
 2022-03-07 PHASE3
NCT06729970 COMPLETED
Healthy Volunteers
Karuna Therapeutics, Inc., a Bristol Myers Squibb company
 2024-12-26 PHASE1
NCT06924255 NOT_YET_RECRUITING
Schizophrenia Disorders
Collaborative Neuroscience Research, LLC
 2025-04 PHASE4
NCT06853171 COMPLETED
Psychiatric Disorders
Bristol-Myers Squibb
 2025-04-29 PHASE1
NCT06605950 COMPLETED
Healthy Volunteers
Karuna Therapeutics, Inc., a Bristol Myers Squibb company
 2024-10-01 PHASE1
NCT05145413 COMPLETED
Schizophrenia
Karuna Therapeutics, Inc., a Bristol Myers Squibb company
 2021-11-12 PHASE3
NCT05919823 COMPLETED
Schizophrenia
Karuna Therapeutics, Inc., a Bristol Myers Squibb company
 2023-05-29 PHASE3
NCT04820309 COMPLETED
Schizophrenia
Karuna Therapeutics, Inc., a Bristol Myers Squibb company
 2021-06-02 PHASE3
NCT04659174 COMPLETED
Schizophrenia
Karuna Therapeutics, Inc., a Bristol Myers Squibb company
 2021-02-01 PHASE3
NCT05643170 TERMINATED
Schizophrenia
Karuna Therapeutics, Inc., a Bristol Myers Squibb company
 2022-11-08 PHASE3
NCT04738123 COMPLETED
Schizophrenia; Schizophrenia; Psychosis
Karuna Therapeutics, Inc., a Bristol Myers Squibb company
 2021-04-06 PHASE3
NCT04659161 COMPLETED
Schizophrenia; Schizophrenia; Psychosis
Karuna Therapeutics, Inc., a Bristol Myers Squibb company
 2020-12-16 PHASE3
NCT03697252 COMPLETED
Schizophrenia
Karuna Therapeutics, Inc., a Bristol Myers Squibb company
 2018-09-18 PHASE2
NCT02831231 COMPLETED
Schizophrenia
Karuna Therapeutics, Inc., a Bristol Myers Squibb company
 2016-09-07 PHASE1

(data from https://clinicaltrials.gov, updated on 2026-07-07)

Check the Xanomeline product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Xanomeline acts as a selective agonist of muscarinic M1 and M4 receptors, stimulating postsynaptic cholinergic signaling pathways to enhance neuronal excitability and suppress dopamine-driven hyperactive behaviors. This dual muscarinic receptor activation modulates central neurotransmission without direct dopamine receptor blockade, offering therapeutic efficacy in mitigating symptoms of schizophrenia, psychosis, and cognitive impairment.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.