Multiple clinical trials spanning Phase 1 through Phase 3, early exploratory research, and expanded access protocols evaluate verdiperstat in healthy volunteers and patients with neurodegenerative conditions, including Multiple System Atrophy (cerebellar and parkinsonian variants), Amyotrophic Lateral Sclerosis (ALS), and Semantic Dementia. Featuring both active and completed recruitment statuses, these studies encompass Phase 3 trials and platform regimens for ALS. Sponsorship includes pharmaceutical companies such as Biohaven Pharmaceuticals and AstraZeneca, alongside academic and federal institutions including Brigham and Women's Hospital and the National Institutes of Health.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT04297683 | ACTIVE_NOT_RECRUITING | Amyotrophic Lateral Sclerosis |
Merit E. Cudkowicz, MD |
2020-06-14 | PHASE2; PHASE3 |
| NCT05184569 | ACTIVE_NOT_RECRUITING | Semantic Dementia |
Peter Ljubenkov, MD |
2022-04-14 | PHASE1 |
| NCT04436510 | COMPLETED | Amyotrophic Lateral Sclerosis |
Merit E. Cudkowicz, MD |
2020-07-28 | PHASE2; PHASE3 |
| NCT05184569 | Recruiting | Semantic Dementia |
Peter Ljubenkov MD|National Institutes of Health (NIH)|Alzheimer''s Association|National Institute on Aging (NIA)|University of California San Francisco |
2022-04-14 | Phase 1 |
| NCT04616456 | COMPLETED | Multiple System Atrophy; Multiple System Atrophy, Cerebellar Variant; Multiple System Atrophy, Parkinson Variant (Disorder); Multiple System Atrophy (MSA) With Orthostatic Hypotension |
Brigham and Women's Hospital |
2020-12-30 | EARLY_PHASE1 |
| NCT03952806 | COMPLETED | Multiple System Atrophy |
Biohaven Pharmaceuticals, Inc. |
2019-07-29 | PHASE3 |
| NCT00914303 | Completed | Healthy |
AstraZeneca |
2009-06 | Phase 1 |
(data from https://clinicaltrials.gov, updated on 2026-07-21)
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>).