Clinical Trials

Numerous clinical trials spanning early-phase research to Phase 4 evaluations have investigated tolcapone across neurodegenerative, psychiatric, and oncological indications, including transthyretin amyloidosis, schizophrenia, obsessive-compulsive disorder, frontotemporal lobar degeneration, addiction, and neuroblastoma. Supported by academic medical centers and industrial sponsors, these studies encompass completed, active, terminated, and withdrawn statuses to evaluate central dopamine pathway modulation and transthyretin protein stabilization.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT05624528 ACTIVE_NOT_RECRUITING
Obsessive-Compulsive Disorder; OCD
University of Chicago
 2023-06-22 PHASE2
NCT03904498 COMPLETED
Alcohol Use Disorder; Attention Deficit Hyperactivity Disorder
University of Colorado, Denver
 2021-08-16 PHASE2
NCT05065671 COMPLETED
Microbial Colonization
Rutgers, The State University of New Jersey
 2022-02-01 PHASE1
NCT06387771 UNKNOWN
Schizophrenia
Clinica Universidad de Navarra, Universidad de Navarra
 2014-09-22 PHASE2
NCT04205994 COMPLETED
Decision Making; Executive Function; Dopamine
University of California, Berkeley
 2019-12-01 EARLY_PHASE1
NCT02260570 COMPLETED
Mild Traumatic Brain Injury
VA Office of Research and Development
 2015-08-01 EARLY_PHASE1
NCT02949934 COMPLETED
Alcohol Use Disorder
Medical University of South Carolina
 2016-05-01 PHASE2
NCT03348930 COMPLETED
Obsessive-Compulsive Disorder
University of Chicago
 2018-03-20 PHASE2; PHASE3
NCT02652598 UNKNOWN
Brain Injuries, Traumatic; Neurocognitive Disorders; Brain Injuries
Sheppard Pratt Health System
 2015-11 PHASE2
NCT02740582 COMPLETED
Alcohol Abuse; Impulsive Behavior
Jennifer Mitchell
 2016-10-01 PHASE2
NCT02630043 TERMINATED
Neuroblastoma
Giselle Sholler
 2015-12 PHASE1
NCT02448654 COMPLETED
Nicotine Dependence
Yale University
 2015-10-23
NCT03591757 COMPLETED
Transthyretin Amyloidosis; Amyloidosis, Leptomeningeal, Transthyretin-Related
Boston University
 2018-10-30 EARLY_PHASE1
NCT01158950 COMPLETED
Impulsive Behavior
University of California, San Francisco
 2010-03
NCT03633591 COMPLETED
Healthy Volunteers
Corino Therapeutics, Inc.
 2018-06-25 PHASE1
NCT03591757 Completed
Transthyretin Amyloidosis|Amyloidosis Leptomeningeal Transthyretin-Related
Boston University|Corino Therapeutics Inc.
 2018-10-30 Early Phase 1
NCT03273062 UNKNOWN
Brain Injuries; Brain Injuries, Traumatic; Brain Injury, Chronic
Sheppard Pratt Health System
 2017-07-20 PHASE2
NCT02929485 WITHDRAWN
Addiction
University of California, Berkeley
 2013-07 PHASE4
NCT00604591 COMPLETED
Frontotemporal Lobar Degeneration
Columbia University
 2008-01-30 PHASE2
NCT00044083 TERMINATED
Schizophrenia
National Institute of Mental Health (NIMH)
 2002-08 PHASE2
NCT02630043 Terminated
Neuroblastoma
Giselle Sholler|Milton S. Hershey Medical Center
 2015-12 Phase 1
NCT02772978 COMPLETED
Pathological Gambling
University of California, San Francisco
 2014-08
NCT02080715 COMPLETED
Healthy
University of Zurich
 2013-06 PHASE1
NCT02929485 Withdrawn
Addiction
University of California Berkeley|University of California San Francisco
 2013-07 Phase 4
NCT02080715 Completed
Healthy
University of Zurich
 2013-06 Phase 1
NCT01001520 COMPLETED
Tobacco Use Disorder
University of Pennsylvania
 2010-01 PHASE2
NCT00927563 COMPLETED
Pathological Gambling
University of Chicago
 2009-06 PHASE2
NCT00604591 Completed
Frontotemporal Lobar Degeneration
Columbia University|National Institute of Neurological Disorders and Stroke (NINDS)
 2011-07 Phase 2
NCT00906828 COMPLETED
Parkinson Disease
Uppsala University
 2008-10 PHASE4
NCT01202955 COMPLETED
Nicotine Dependence
University of Pennsylvania
 2008-07 PHASE2
NCT00033059 UNKNOWN
Cocaine-Related Disorders
National Institute on Drug Abuse (NIDA)
 2001-07 PHASE1

(data from https://clinicaltrials.gov, updated on 2026-06-10)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Tolcapone acts as a potent, selective, and reversible inhibitor of catechol-O-methyltransferase (COMT, Ki = 30 nM), effectively blocking the enzymatic methylation and degradation of catecholamines such as dopamine. By preventing catecholamine breakdown and enhancing dopaminergic signaling in the central nervous system, tolcapone provides therapeutic utility in dopamine-regulated neurological and psychiatric indications such as schizophrenia and obsessive-compulsive disorder investigated in clinical trials.

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.