Multiple clinical trials spanning Phase 1 through Phase 4 evaluate the therapeutic profile of pimozide across indications such as Tourette syndrome, amyotrophic lateral sclerosis, schizophrenia, subjective tinnitus, and drug-induced QT prolongation. Sponsored by academic institutions and regulatory bodies—including Yale University, the Icahn School of Medicine at Mount Sinai, the University of Calgary, and the FDA—these studies encompass completed, terminated, and unknown recruitment statuses. Specific investigations range from Phase 1 electrophysiological safety studies and Phase 2 trials in amyotrophic lateral sclerosis to Phase 4 adjunct therapy evaluations in schizophrenia.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT04529226 | UNKNOWN | Psychosis; Intellectual Disability |
Fundación Pública Andaluza para la gestión de la Investigación en Sevilla |
2020-11-26 | PHASE2 |
| NCT05716854 | COMPLETED | Drug-induced QT Prolongation; Pharmacokinetics; Pharmacodynamics |
Food and Drug Administration (FDA) |
2023-03-21 | PHASE1 |
| NCT05507372 | UNKNOWN | Tinnitus, Subjective |
Applied Biology, Inc. |
2022-10-01 | |
| NCT03272503 | UNKNOWN | ALS; Amyotrophic Lateral Sclerosis |
University of Calgary |
2017-10-27 | PHASE2 |
| NCT02374567 | TERMINATED | Dementia; Depression; Schizophrenia; Psychosomatic Disorders; Anxiety Disorders |
Hannover Medical School |
2015-01 | PHASE3 |
| NCT02463825 | UNKNOWN | Amyotrophic Lateral Sclerosis (ALS) |
University of Calgary |
2015-04 | PHASE2 |
| NCT02307396 | COMPLETED | Schizophrenia; Schizophrenia and Disorders With Psychotic Features; Schizoaffective Disorders |
Technical University of Munich |
2015-02-01 | PHASE4 |
| NCT00374244 | COMPLETED | Schizophrenia |
Yale University |
2004-01 | PHASE2 |
| NCT00158223 | COMPLETED | Schizophrenia; Psychotic Disorders |
Icahn School of Medicine at Mount Sinai |
2004-10 | PHASE4 |
| NCT00004652 | COMPLETED | Tourette Syndrome |
National Center for Research Resources (NCRR) |
1993-02 | PHASE2 |
(data from https://clinicaltrials.gov, updated on 2024-03-20)
Mechanism and Biochemical Profile
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>).